| Primary | Mean Number of Daily Active Minutes of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Physical activity data was collected as daily active minutes measured on each valid day by wearing a fitness tracker continuously during study participation. Valid day = a day in which fitness tracker was worn for at least 10 hours. Daily active minutes were saved in two different sets of data collected by fitness tracker. For each set minutes were categorized by intensity of activity done. First set = minutes categorized according to time collected in each heart zone. Overall heart zone minutes = fat-burn minutes (heart rate: 50-69% of maximum) + cardio minutes (medium-to-high intensity exercise zone corresponds to heart rate: 70-84% of maximum) + peak minutes (high-intensity exercise zone, corresponds to heart rate > 85% of maximum). Second set =minutes categorized according to active zones. Overall active zone minutes=lightly active minutes +fairly active minutes+very active minutes. Moderate-to-Vigorous Physical Activity (MVPA) minutes=Fairly active minutes+Very active minutes. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Mean | 95% Confidence Interval | number of daily active minutes | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care: 18-30 Years | Participants who were 18-30 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG002 | Standard of Care:31-50 Years | Participants who were 31-50 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| | | Title | Denominators | Categories |
|---|
| Heart Zone Minutes | | | Title | Measurements |
|---|
| - OG00053.2(-45.2 to 151.6)
- OG00124.6(10.9 to 38.3)
- OG00239.2(13.9 to 64.6)
|
| | Active Zone Minutes |
| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
|---|
| Heart zone minutes: Comparison among the age groups was performed by means of a Kruskal-Wallis test. | Kruskal-Wallis | | 0.7630 | | | | | | | | | | | | | | Other | | | | Active zone minutes: Comparison among age groups was performed using Kruskal-Wallis test. |
|
| Primary | Mean Number of Steps Taken Per Day by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | Number of steps taken by the participants on each valid day were measured by wearing a fitness tracker continuously during study participation. Valid day was defined as a day in which fitness tracker was worn for at least 10 hours. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Mean | 95% Confidence Interval | number of steps per day | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care: 18-30 Years | Participants who were 18-30 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | |
|
| Primary | Mean Metabolic Equivalent of Tasks (METs) Per Day Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | The MET is an objective measure of the ratio of the rate at which a person expends energy, relative to the mass of that person, while performing some specific physical activity compared to a reference. The METs were derived as: (amount of kilo calories (kcal) collected in the fat-burn, cardio, and peak minutes) / [(sum of fat-burn, cardio and peak hours) * weight of the participant in kilograms (kg)]. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Mean | 95% Confidence Interval | MET per day | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care: 18-30 Years | Participants who were 18-30 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Mean Rate of the Number of Times Engaging in Physical Activity Per Week by Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | The activity weekly rate was derived on a per participant basis as ratio between the number of times that the type of activity is reported on the fitness tracker (numerator) and the length of the observational period (i.e. end of study date - baseline date + 1) (denominator) and then multiplied by 7. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis. | Posted | | Mean | 95% Confidence Interval | number of times per week | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care: 18-30 Years |
|
| Secondary | Mean Active Minutes by Intensity (Lightly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. In this analysis, the means and 95% confidence intervals were calculated using the median lightly active minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis. | Posted | | Mean | 95% Confidence Interval | lightly active minutes per activity | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Mean Active Minutes by Intensity (Fairly Active Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. In this analysis, the means and 95% confidence intervals were calculated using the median fairly active minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis. | Posted | | Mean | 95% Confidence Interval | fairly active minutes per activity | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Mean Active Minutes by Intensity (MVPA Minutes) for Each Type of Physical Activity Performed by Participants (by Age Categories: 12-17, 18-30, and 31-50 Years) Over the Course of the Study | The number of active minutes by intensity (lightly, fairly, and very active minutes) were obtained at the participant level on a per activity basis as the median of the values collected by the fitness tracker for each activity recorded during the observational period. The MVPA minutes is the sum of fairly active and very active minutes. In this analysis, the means and 95% confidence intervals were calculated using the median MVPA minutes by type of physical activity performed by participants with available data. The physical activities were categorized in the following groups: exercises at intervals, gym weights, running, swimming, tapis roulant, walk, general exercises: other types of physical activity identified by the fitness tracker that are not already included in the above-mentioned categories. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis. | Posted | | Mean | 95% Confidence Interval | MVPA minutes per activity | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Percentage of Participants Who Were Adherent to World Health Organization (WHO) Global Recommendations on Physical Activity for Health Over the Course of the Study | A participant was defined "adherent" to WHO guidelines according to the following algorithm: 12-17 years old: MVPA minutes per day >= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week >= 150 minutes or very active (vigorous) minutes per week >= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). A participant who was adherent to the WHO guidelines according to the above definition was defined as an "active participant", otherwise as a "sedentary participant". | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Number | | percentage of participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care: 18-30 Years | Participants who were 18-30 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study, Reported Via the ePRO Application | Bleeds that occurred the same day but in different body site were counted separately. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care:18-30 Years | Participants who were 18-30 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG002 | Standard of Care: 31-50 Years |
|
| Secondary | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Different Types of Bleeds (Spontaneous, Traumatic and Procedure/Surgery Related), Reported Via the ePRO Application | The assessment of bleeds was divided into spontaneous bleeds, traumatic bleeds, and bleeds related to procedure/surgery. Spontaneous bleeds = the occurrence of hemorrhage where neither the participant nor a caregiver could identify a reason. Traumatic bleeds = hemorrhage occurring secondary to an event such as trauma, "strenuous" activity, or "overuse". Bleeds related to procedure/surgery = such as hematomas resulting from any surgeries or invasive procedures. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months). | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care: 18-30 Years | Participants who were 18-30 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Number of Participants by the Presence of Bleeds (0 vs. ≥1 Bleeds) Over the Course of the Study According to the Bleed Sites (Urine, Feces, and Vomit, Joint, Muscle, and Other Bleeds), Reported Via the ePRO Application | Sites of bleeds were defined as urine, faeces, and vomit (three sites grouped together), joint, muscle, and other bleeds. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | Baseline and whenever bleeding occured from study initiation to completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care: 12-17 Years | Participants who were 12-17 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Standard of Care: 18-30 Years | Participants who were 18-30 years of age with severe or moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG002 |
|
| Secondary | Number of Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | From baseline to the time of switch to a new therapy (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Treated Bleeds Over the Course of the Study | An event was considered a treated bleed if coagulation factors were administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.). | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint. | Posted | | Number | | number of treated bleeds | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Bleeds | Bleeds | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Bleeds Treated With Each Type of Treatment for Bleeding (Haemostatic Treatment, Treatment for Pain) | An event was considered a treated bleed if coagulation factors were administered to treat signs or symptoms of bleeding (e.g., pain, swelling, etc.). | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint. | Posted | | Number | | number of treated bleeds | | From baseline to whenever bleeding occurred (up to 18 months) | Treated bleeds | Treated bleeds | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Duration of Treatment for Bleeding Over the Course of the Study | | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Median | Full Range | days | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Treated bleeds | Treated bleeds | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Bleeds Treated With Each Route of Administration Over the Course of the Study | Number of bleeds treated by intravenous, subcutaneous, intramuscular, nasal, ophthalmic, oral, rectal, and other routes of administration are reported here. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint. | Posted | | Number | | number of treated bleeds | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Treated bleeds | Treated bleeds | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Bleeds Which Needed to Receive a Transfusion | | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint | Posted | | Number | | number of bleeds | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Bleeds | Bleeds | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Bleeds Which Needed to Administer an Additional Hemostatic Therapy | | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint. | Posted | | Number | | number of bleeds | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Bleeds | Bleeds | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Bleeds With Adherence to Treatment Regimen for Haemophilia A in the Week Preceding the Bleeding | The adherence to the treatment regimen for haemophilia A was analyzed as the number of bleeds with an answer equal to "Yes" and "My therapeutic plan does not include any treatment in the last week" in the week preceding the bleeding. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of bleeds analyzed=number of bleeds during analysis at given timepoint. | Posted | | Number | | Number of bleeds | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | Bleeds | Bleeds | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Participants Who Were Adherent to the Treatment Regimen for Haemophilia A Over the Course of the Study by Adherence Category (<80% vs. ≥80% of Study Weeks) | The adherence to the treatment regimen for haemophilia A was analyzed as the number of participants with <80% or ≥80% of weeks on study with "Yes" and "My therapeutic plan does not include any treatment in the last week" over the total number of weeks reported via the ePRO. Missing data from some weeks was treated as "No". The total number of weeks on study for each participant was derived as (End of study date - Baseline date + 1) / 7. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. A total of 4 participants had missing data and were excluded from this analysis | Posted | | Count of Participants | | Participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | Baseline and every 3 months until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Pain Intensity, as Reported by Participants Using a Visual Analogue Scale | Participants reported the intensity of pain in their main joints (elbow, knee, ankle) by marking on a visual analogue scale (VAS), a horizontal line 100 millimeters (mm) in length, anchored by word descriptors at each end, where the left extreme means "No pain = 0" while the right extreme means "Worst Pain Imaginable = 100". The VAS score is the distance (in mm) from the left end of the line to the point where the participant marks crossed the line. VAS scale was submitted to the participants every month during the study. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Health-Related Quality of Life (HRQoL): European Quality of Life-5 Dimensions (EQ-5D-5L) Health State Utility Index Score Over Time | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The EQ-5D-5L includes five levels of severity in each of the five EuroQol-5D dimensions and the EQ VAS. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state and lower score indicate worse health state. EQ-5D-5L questionnaire was submitted to the participants every 3 month from baseline during the study. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 centimeter (cm) vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Haemophilia Joint Health Score (HJHS) Total Score Over Time | The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 6 and 12 | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Mean Body Mass Index (BMI) Over Time | | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | kilogram per square meter (kg/m2) | | Baseline, Months 3, 6, 9, 12, 15 and 18 | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Yearly Rate of Days Away From School or Work for Participants | The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Yearly Rate of Days Away From Work for Parent/Caregiver | The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents and caregivers) basis as the proportion between the total number of days away from work and duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Yearly Rate of Hospitalization Days | The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Age of Possible Early Retirement of Caregiver, as Reported by Caregivers in a Questionnaire | | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. | Posted | | Mean | 95% Confidence Interval | years | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Standard of Care for Haemophilia A | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Active Versus Sedentary Participants by the Severity (Moderate or Severe) of Haemophilia A | Participants who were adherent to the WHO guidelines for physical activity according to the below definition were defined as an "active participant", otherwise as a "sedentary participant". A participant was defined "adherent" to WHO guidelines according to the following algorithm: 12-17 years old: MVPA minutes per day >= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week >= 150 minutes or Very active (vigorous) minutes per week >= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). The participants were classified as 'Active' or 'Sedentary' based on data collected from baseline till the end of the study. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | From baseline up to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
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| Secondary | Annualized Bleeding Rates (ABR) in Active Versus Sedentary Participants | The annualized bleeding rate was derived as follows: total number of bleeds/total follow-up (person-year), where the individual's study duration was given by ([end of study date-baseline date +1] divided by 365.25). ABR was reported in active and sedentary participants. A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an "active participant", otherwise as a "sedentary participant". WHO guidelines for adherence: 12-17 years old: MVPA minutes per day >= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week >= 150 minutes or Very active (vigorous) minutes per week >= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Mean | 95% Confidence Interval | rate of bleeds per person-year | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 |
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| Secondary | Pain Intensity in Active Versus Sedentary Participants, as Reported by Participants Using a Visual Analogue Scale | Participants reported intensity of pain in their main joints (elbow, knee, ankle) by marking on a VAS, a horizontal line 100 mm in length, anchored by word descriptors at each end, where the left extreme means "No pain = 0" while right extreme means "Worst Pain Imaginable = 100". VAS score is the distance (in mm) from left end of the line to the point where participant marks crossed the line. A participant who was adherent to WHO guidelines for physical activity according to the below definition was defined as an "active participant", otherwise as a "sedentary participant". WHO guidelines for adherence: 12-17 years old: MVPA minutes per day ≥60 minutes. 18-50 years old: Fairly active (moderate) minutes per week ≥150 minutes or Very active (vigorous) minutes per week ≥75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). VAS scale was submitted to the participants every month during the study. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Number of Active Versus Sedentary Participants by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an "active participant", otherwise as a "sedentary participant". WHO guidelines for adherence: 12-17 years old: MVPA minutes per day >= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week >= 150 minutes or Very active (vigorous) minutes per week >= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). Participants can have received more than one medication. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Count of Participants | | Participants | | Baseline and every 3 months until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activities guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Active Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Active' per WHO physical activities guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Number of Active Versus Sedentary Participants by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an "active participant", otherwise as a "sedentary participant". WHO guidelines for adherence: 12-17 years old: MVPA minutes per day >= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week >= 150 minutes or Very active (vigorous) minutes per week >= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). Participants can have received more than one medication. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Count of Participants | | Participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 |
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| Secondary | Number of Active Versus Sedentary Participants by the Product Used for Treatment of Haemophilia A Over the Course of the Study | A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an "active participant", otherwise as a "sedentary participant". WHO guidelines for adherence: 12-17 years old: MVPA minutes per day >= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week >= 150 minutes or Very active (vigorous) minutes per week >= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Count of Participants | | Participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Active Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Active' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Number of Active Versus Sedentary Participants Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | A participant who was adherent to the WHO guidelines for physical activity according to the below definition was defined as an "active participant", otherwise as a "sedentary participant". WHO guidelines for adherence: 12-17 years old: MVPA minutes per day >= 60 minutes. 18-50 years old: Fairly active (moderate) minutes per week >= 150 minutes or Very active (vigorous) minutes per week >= 75 minutes or an equivalent combination of fairly and very active minutes (i.e., considering 1 fairly active minute as 0.5 very active minutes). | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Count of Participants | | Participants | | From baseline to the time of switch to a new therapy (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Active Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Active' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time for Active Versus Sedentary Participants | EQ-5D-5L is a standardized measure of health status developed by EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. EQ-5D-5L includes five levels of severity in each of five EuroQol-5D dimensions and EQ VAS. Descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. 5D-5L systems are converted into a single index utility score between 0-1, where higher score indicates a better health state and lower score indicate worse health state. A participant who was adherent to WHO guidelines for physical activity was defined as an "active participant", otherwise as a "sedentary participant". EQ-5D-5L questionnaire was submitted to participants every 3 month from baseline during the study. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Active Versus Sedentary Participants | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 centimeter (cm) vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable. A participant who was adherent to the WHO guidelines for physical activity was defined as an "active participant", otherwise as a "sedentary participant". | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
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| Secondary | HJHS Total Score for Active Versus Sedentary Participants Over Time | The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. Number analyzed is the number of participants with data available for analysis at given timepoint. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 6 and 12 | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Active Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Active' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Yearly Rate of Number of Days Away From School/Work for Active Versus Sedentary Participants | The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Active Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Active' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Yearly Rate of Number of Days Away From Work for Active Versus Sedentary Participant's Parents or Caregivers | The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents/caregivers) basis as the proportion between the total number of days away from work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Active Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Active' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Yearly Rate of Number of Hospitalization Days for Active Versus Sedentary Participants | The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | Evaluable population included all enrolled participants with at least 6 valid months of physical activity evaluations during the study. | Posted | | Median | Full Range | days | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months | | OG001 | Active Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Active' per WHO physical activities guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Annualized Bleeding Rates in Active Versus Sedentary Participants by the Severity (Moderate Versus Severe) of Haemophilia A | The annualized bleeding rate was derived as follows: total number of bleeds/total follow-up (person-year), where the individual's study duration was given by ([end of study date-baseline date +1] divided by 365.25). A participant who was adherent to the WHO guidelines for physical activity was defined as an "active participant", otherwise as a "sedentary participant". Participants were classified for disease severity at baseline: severe (Factor VIII activity <1%) or moderate (Factor VIII activity between ≥1% and ≤2%) haemophilia A. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number of participants analyzed is the number of participants with available physical activities data in ITT population for sedentary and active participants. | Posted | | Mean | 95% Confidence Interval | rate of bleeds per person-year | | Baseline and whenever bleeding occurred from study initiation to completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Sedentary Participants | Participants with severe or moderate Haemophilia A without inhibitors against Factor VIII, who classified as 'Sedentary' per WHO physical activity guideline, receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Active Participants |
|
| Secondary | Pain Intensity in Participants With Moderate Versus Severe Haemophilia A, as Reported by Participants Using a Visual Analogue Scale | Participants reported the intensity of pain in their main joints (elbow, knee, ankle) by marking on a VAS, a horizontal line 100 mm in length, anchored by word descriptors at each end, where the left extreme means "No pain = 0" while the right extreme means "Worst Pain Imaginable = 100". The VAS score is the distance (in mm) from the left end of the line to the point where the participant marks crossed the line. VAS scale was submitted to the participants every month during the study. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoint. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17 and 18 | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Number of Participants With Moderate Versus Severe Haemophilia A by the Product of Concomitant Medications Taken to Control Pain Over the Course of the Study | Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | Baseline and every 3 months until study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Participants With Moderate Versus Severe Haemophilia A by the Type of Regimen Used for Treatment of Haemophilia A Over the Course of the Study | The following types of regimens used to treat haemophilia A are reported here: on-demand (to stop bleeding) and prophylaxis (regular infusions of clotting factor to prevent bleeds). Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Participants With Moderate Versus Severe Haemophilia A by the Product Used for Treatment of Haemophilia A | Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | From baseline to study completion (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | Number of Participants With Moderate Versus Severe Haemophilia A Who Switched to a New Approved Drug for Treatment of Haemophilia A by the Product Used | Participants could have received more than one medication. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. | Posted | | Count of Participants | | Participants | | From baseline to the time of switch to a new therapy (up to 18 months) | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia A | Participants who were 12-17 years of Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
| |
| Secondary | HRQoL: EQ-5D-5L Health State Utility Index Score Over Time Moderate Versus Severe Haemophilia A | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The EQ-5D-5L includes five levels of severity in each of the five EuroQol-5D dimensions and the EQ VAS. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each dimension having 5 levels (1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems). Response options are measured with a 5-point Likert scale. The 5D-5L systems are converted into a single index utility score between 0 to 1, where higher score indicates a better health state and lower score indicate worse health state. EQ-5D-5L questionnaire was submitted to the participants every 3 month from baseline during the study. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoint. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
|
| Secondary | Health Status Measured Over Time, as Reported by Participants Using EQ-5D VAS Score in Participants With Moderate Versus Severe Haemophilia A | The EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group in order to provide a simple, generic measure of health for clinical and economic appraisal. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and the other section is an evaluation of the overall health status using VAS. The VAS records the respondent's self-rated health on a 20 cm vertical VAS from 0 to 100 on how good or bad their health is at assessment; 0 means the worst health imaginable, and 100 means the best health imaginable. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoint. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, and 18 | | | | ID | Title | Description |
|---|
| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A |
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| Secondary | HJHS Total Score for Participants With Moderate Versus Severe Haemophilia A | The HJHS is an instrument developed to measure joint health status and to identify early signs of joint disease in children. This tool assesses joints most commonly affected by bleeding (knees, ankles, and elbows). Each of the 6 joints is assessed across 8 domains (Swelling, Duration of Swelling, Atrophy, Crepitus, Flexion Loss, Extension Loss, Pain, and Strength), and a Total Score is calculated as the sum of all joint domain scores. The total score range is from 0 to 124 points (a score of 0 indicates no joint damage). A higher score indicates higher joint damage. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at given timepoints. | Posted | | Mean | 95% Confidence Interval | scores on a scale | | Baseline, Months 6 and 12 | | | | ID | Title | Description |
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| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
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| Secondary | Yearly Rate of Days Away From School for Participants With Moderate Versus Severe Haemophilia A | The number of days away from school or work (participant) was analyzed every month as a continuous variable. The yearly rate was derived on a per participant basis as the proportion between the total number of days away from school and/or work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
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| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
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| Secondary | Yearly Rate of Days Away From Work for Parents/Caregivers of Participants With Moderate Versus Severe Haemophilia A | The number of days away from work was analyzed every month as a continuous variable. The yearly rate was derived on a per participant (for parents/caregivers) basis as the proportion between the total number of days away from work and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
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| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
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| Secondary | Yearly Rate of Hospitalization Days for Participants With Moderate Versus Severe Haemophilia A | The yearly rate was derived on a per participant basis as the proportion between the total number of hospitalization days and the duration of the study (derived as end of study date - baseline date + 1) multiplied by 365.25. | ITT population included all enrolled participants who received the ePRO application and the fitness tracker. Overall number analyzed is the number of participants with data available for analysis. | Posted | | Median | Full Range | days/year | | Baseline and every month until study completion (up to 18 months) | | | | ID | Title | Description |
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| OG000 | Moderate Haemophilia A | Participants with moderate Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. | | OG001 | Severe Haemophilia A | Participants with severe Haemophilia A without inhibitors against Factor VIII receiving standard of care treatment, independently of participation in the current study, according to clinician's independent therapeutic decision were enrolled for a maximum observation period of 18 months. |
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