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| ID | Type | Description | Link |
|---|---|---|---|
| NCT02740413 | Registry Identifier | ClinicalTrials.gov |
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The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, as well as the related direct treatment costs in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF, and in subgroups (e.g. level of severity) at the MHC in Sweden.
The study population will consist of all patients diagnosed with haemophilia (D66.9 (haemophilia A) D67.9 (haemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the MHR since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product (Benefix August 27 1997, ReFacto April 13 1999, ReFacto AF July 1 2009). Diseased individuals are included. Information on drugs picked up at the pharmacy is available in the Prescribed Drug Register from 2005.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with Haemophilia A | Patients in The MHR diagnosed with Haemophilia A | ||
| Patients with Haemophilia B | Patients in The MHR diagnosed with Haemophilia B |
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| Measure | Description | Time Frame |
|---|---|---|
| Age of Participants at Disease Diagnosis | At disease diagnosis within 11 years before the study start date (Day 1) | |
| Age of Participants at Start of Treatment With Benefix or Refacto | At start of treatment with Benefix/Refacto within 11 years before the study start date (Day 1) | |
| Age of Participants at Start of Replacement Treatment | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor. Replacement treatment could be given prophylactically or to stop the bleed and/or to stop it from becoming more severe. | At start of replacement treatment within 11 years before the study start date (Day 1) |
| Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Data was measured in international units (IU) for factor VIII and factor IX concentrates. | For the duration of 11 years before the study start date (Day 1) |
| Average Prescribed Dose Per Kilogram Bodyweight for Factor VIII and Factor IX Concentrates | The average dose per kilogram body weight of factor VIII and IX was defined as a set of two derived variables from the MHR based on information on prescribed replacement treatment (Benefix or Refacto/Refacto AF): prescribed dose per infusion divided by registered body weight (all participants) and prescribed dose per week (prescribed dose per infusion multiplied by registered number of infusions per week) divided by registered body weight (only participants on prophylaxis). | For the duration of 11 years before the study start date (Day 1) |
| Percentage of Participants on Prophylactic Treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment | For the duration of 11 years before the study start date (Day 1) | |
| Average Cost of Replacement Treatment Related to Invasive Procedures | For the duration of 11 years before the study start date (Day 1) |
| Measure | Description | Time Frame |
|---|---|---|
| Average Annual Cost of Replacement Treatment Related to Invasive Procedures | Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011) | |
| Average Cost for Replacement Treatment Related to Invasive Procedures | The average cost for replacement treatment related to invasive procedures was derived from the percentage of total costs for dispensed replacement therapy and calculated as: cost of factor concentrate used for invasive procedures/ total cost of factor concentrate. |
Inclusion Criteria:
Exclusion Criteria:
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The study population will consist of all patients diagnosed with hemophilia (D66.9 (hemophilia A) D67.9 (hemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the Malmö Hemophilia Register since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product (Benefix August 27 1997, ReFacto April 13 1999, ReFacto AF July 1 2009). Diseased individuals are included.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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The study was a retrospective population-based register study. Detailed data on each participant diagnosed with haemophilia was retrieved from the Malmö Haemophilia Register (MHR) in January 2016 covering the period of January 2005 up to December 2015.
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| ID | Title | Description |
|---|---|---|
| FG000 | Benefix/Refacto/Refacto AF | Participants diagnosed with haemophilia A and B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in International Statistical Classification of Diseases and Related Health Problems (ICD-10), registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product
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| ID | Title | Description |
|---|---|---|
| BG000 | Benefix/Refacto/Refacto AF | Participants diagnosed with haemophilia A and B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Age of Participants at Disease Diagnosis | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | years | At disease diagnosis within 11 years before the study start date (Day 1) |
|
Within 11 years (from 2005 to 2015) before the study start date (Day 1)
The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non serious in another participant, or one participant may have experienced both a serious and non serious event during the study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Benefix/Refacto/Refacto AF | Participants diagnosed with haemophilia A and B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 16, 2015 | Jun 22, 2018 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 3, 2015 | Jun 22, 2018 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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Prophylactic treatment was defined as administration of drug regularly to reduce the insufficiency of coagulation factor to prevent bleeding to occur. |
| For the duration of 11 years before the study start date (Day 1) |
| Average Prescribed Frequency of Infusions Per Week Dispensed for Haemophilia A or B Participants With Prophylaxis | For the duration of 11 years before the study start date (Day 1) |
| Average Prescribed Annual Dose of Factor VIII and IX Concentrates for Participants on Prophylaxis | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate was derived from the MHR registration of prescribed annual dose of factor concentrate. | For the duration of 11 years before the study start date (Day 1) |
| Average Prescribed Annual Dose of Factor VIII and IX Concentrates Per Kilogram Bodyweight for Participants on Prophylaxis | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate per kilogram body weight was derived from the MHR registration of prescribed annual dose of factor concentrate and the registered body weight. | For the duration of 11 years before the study start date (Day 1) |
| Average Annual Registered Consumption of Factor VIII and IX Concentrates | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Average annual registered consumption of factor VIII and IX concentrates was defined as MHR registered participant's reports on factor concentrate consumption during calendar year. | For the duration of 11 years before the study start date (Day 1) |
| Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate | Participants who develop inhibitors to factor VIII or IX concentrates were treated with bypassing agents in the form of activated prothrombin complex concentrate (aPCC) which was measured in units (U) and/or recombinant factor VIIa (rFVIIa) measured in micrograms (mcg). | For the duration of 11 years before the study start date (Day 1) |
| Percentage of Time on Refacto or Benefix | Percentage of time (in days) on Refacto/Refacto AF or Benefix was calculated as total number of days when participant was prescribed Refacto/Refacto AF or Benefix over the total number of days on any replacement treatment. Total number of days on any replacement treatment was derived from date of start of replacement treatment according to MHR and August 31, 2015 or date of death, whichever was earliest. | For the duration of 11 years before the study start date (Day 1) |
| Average Use of Factor Concentrate Per Surgery Event at Hospital for Invasive Procedures | For the duration of 11 years before the study start date (Day 1) |
| Average Factor Concentrate Use at Hospital for Invasive Procedures | Average factor concentrate use was calculated as percentage of total annual use of factor concentrates (in IU) at hospital for invasive procedures. | For the duration of 11 years before the study start date (Day 1) |
| Average Annual Number of Filled Prescriptions of Factor Concentrate | Data at end of every year was based on the number of filled prescriptions during the last 3 years. | For a duration of 3 years (for up to 11 years before the study start Day 1) |
| Average Annual Number of Dispensed Units of Factor Concentrate | To assess annual consumption of factor concentrates, calculations were based on prescription date as start of use and the day before the next prescription as the last date of use of the factor concentrates retrieved. The annual number of dispensed units of factor concentrate were then summed up of all dispensed units of factor concentrate with periods within the calendar year plus estimates of average daily use periods extending over two years. Data at end of every year was based on the number of dispensed units of factor concentrate during the last 3 years. | For a duration of 3 years (for up to 11 years before the study start Day 1) |
| Average Number of Units of Benefix or Refacto | Data from the National Board of Health and Welfare (NBHW) had information on all filled prescriptions from July 1, 2005-last date of observation (August 31, 2015). Derived variable calculated as total number of units of filled prescriptions of Refacto/Refacto AF and Benefix over the total units of all factor VIII concentrates and factor IX concentrates, respectively. Total number of units were derived from the date of start of NBHW to last available observation or date of death, whichever was earliest. | For the duration of 11 years before the study start date (Day 1) |
| Total Number of Bleeds | For the duration of 11 years before the study start date (Day 1) |
| Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants | Joint bleeds included traumatic and spontaneous joint bleeds, muscle bleeds included traumatic and spontaneous soft tissue bleeds and other bleed events included intracranial bleed, gastrointestinal bleed and urinary tract bleed. | For the duration of 11 years before the study start date (Day 1) |
| Gilbert Joint Score | Gilbert joint score was an instrument to measure joint health in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in haemophilia - knees, ankles, elbows. Total score ranged from 0-100, evaluating ankle, knee and elbow, where "0" indicated normal joint function, "100" indicated worst joint function, where higher values indicated more impairment in joints. | Data analyzed on study start Day 1 for the duration of 4 years (2005 to 2009) |
| Haemophilia Joint Health Score | Haemophilia Joint Health Score (HJHS) was used to assess joint damage in participants with haemophilia. Total score ranges from 0 to 124, where, 0 indicates normal function, and 124 indicates worst joint function, higher values indicated more damage in joints. | Data analyzed on study start Day 1 for the duration of 6 years (2009 to 2015) |
| Percentage of Participants With Surgeries | Percentage of participants who had any type of surgery (arthrodesis, surgery on foot, nose, elbow, hand or shoulder, surgery on hip, surgery on knee, tooth extraction, venous port or any other) during the data observation period were reported. | For the duration of 11 years before the study start date (Day 1) |
| Average Relative Dose Intensity of Factor Concentrate Dispensed for Haemophilia A or B Participants on Prophylaxis Based on Dispensed Volume of Units | Relative dose intensity based on dispensed volume of units was calculated using annual dispensed volume of factor concentrate per participant divided by annual prescribed dose per participant. Data was analysed for 2 categories separately: children/adolescents and adults. | For the duration of 11 years before the study start date (Day 1) |
| Average Cost of Replacement Treatment (Benefix or Refacto/Refacto AF) Related to Bleed Event | For the duration of 11 years before the study start date (Day 1) |
| Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011) |
| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Year of Birth for Participants | Median | Full Range | years |
|
| Percentage of Participants Who Had Died During the Prior 11 Year Period | Number | percentage of participants |
|
| Percentage of Participants With Severe, Moderate and Mild Haemophilia A or B | Criteria for severity was defined as MHR standard categories Mild: factor (VIII and IX) level 5-40 percent (%); Moderate: factor level 1-5% and Severe: factor level less than 1%. | Number | percentage of participants |
|
| Percentage of Participants with Inhibitor Status | Participants who developed inhibitory antibodies against factor VIII or IX were analyzed and their inhibitor status were categorized as: current, ever or never, based on last available observation in the MHR. Criteria for inhibitor development was as: if noted yes in the MHR at the date of data extraction: current; if noted yes at an earlier date: ever; if never noted yes in the MHR: never. | Number | percentage of participants |
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| Percentage of Participants Infected With Hepatitis C | Participants categorized as Hepatitis-C positive based on last available observation in the MHR. | Number | percentage of participants |
|
| Percentage of Participants With Positive Human Immunodeficiency Virus (HIV) status | Participants categorized as HIV positive based on last available observation in the MHR. | Number | percentage of participants |
|
| Benefix/Refacto/Refacto AF: Haemophilia B |
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed. |
|
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| Primary | Age of Participants at Start of Treatment With Benefix or Refacto | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | years | At start of treatment with Benefix/Refacto within 11 years before the study start date (Day 1) |
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| Primary | Age of Participants at Start of Replacement Treatment | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor. Replacement treatment could be given prophylactically or to stop the bleed and/or to stop it from becoming more severe. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. | Posted | Mean | Standard Deviation | years | At start of replacement treatment within 11 years before the study start date (Day 1) |
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| Primary | Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Data was measured in international units (IU) for factor VIII and factor IX concentrates. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | IU | For the duration of 11 years before the study start date (Day 1) |
|
|
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| Primary | Average Prescribed Dose Per Kilogram Bodyweight for Factor VIII and Factor IX Concentrates | The average dose per kilogram body weight of factor VIII and IX was defined as a set of two derived variables from the MHR based on information on prescribed replacement treatment (Benefix or Refacto/Refacto AF): prescribed dose per infusion divided by registered body weight (all participants) and prescribed dose per week (prescribed dose per infusion multiplied by registered number of infusions per week) divided by registered body weight (only participants on prophylaxis). | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | IU per kilogram | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Percentage of Participants on Prophylactic Treatment | Prophylactic treatment was defined as administration of drug regularly to reduce the insufficiency of coagulation factor to prevent bleeding to occur. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. | Posted | Number | percentage of participants | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Prescribed Frequency of Infusions Per Week Dispensed for Haemophilia A or B Participants With Prophylaxis | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants with prophylaxis. | Posted | Mean | Standard Deviation | infusions per week | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Prescribed Annual Dose of Factor VIII and IX Concentrates for Participants on Prophylaxis | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate was derived from the MHR registration of prescribed annual dose of factor concentrate. | Analysis was performed on MHR Benefix or Refacto/Refacto AF analysis set. Here, "overall number of participants analysed" signifies participants who were evaluable for this outcome measure and "number analysed" signifies number of participants with available data for each category. | Posted | Mean | Standard Deviation | IU/year | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Prescribed Annual Dose of Factor VIII and IX Concentrates Per Kilogram Bodyweight for Participants on Prophylaxis | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate per kilogram body weight was derived from the MHR registration of prescribed annual dose of factor concentrate and the registered body weight. | Analysis was performed on MHR Benefix or Refacto/Refacto AF analysis set. Here, "overall number of participants analysed" signifies participants who were evaluable for this outcome measure and "number analysed" signifies number of participants with available data for each category. | Posted | Mean | Standard Deviation | IU/kg/year | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Annual Registered Consumption of Factor VIII and IX Concentrates | Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Average annual registered consumption of factor VIII and IX concentrates was defined as MHR registered participant's reports on factor concentrate consumption during calendar year. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | IU/year | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate | Participants who develop inhibitors to factor VIII or IX concentrates were treated with bypassing agents in the form of activated prothrombin complex concentrate (aPCC) which was measured in units (U) and/or recombinant factor VIIa (rFVIIa) measured in micrograms (mcg). | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. | Posted | Count of Participants | Participants | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Percentage of Time on Refacto or Benefix | Percentage of time (in days) on Refacto/Refacto AF or Benefix was calculated as total number of days when participant was prescribed Refacto/Refacto AF or Benefix over the total number of days on any replacement treatment. Total number of days on any replacement treatment was derived from date of start of replacement treatment according to MHR and August 31, 2015 or date of death, whichever was earliest. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Median | Full Range | days | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Use of Factor Concentrate Per Surgery Event at Hospital for Invasive Procedures | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. | Posted | Mean | Standard Deviation | IU per surgery event | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Factor Concentrate Use at Hospital for Invasive Procedures | Average factor concentrate use was calculated as percentage of total annual use of factor concentrates (in IU) at hospital for invasive procedures. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. | Posted | Mean | Standard Deviation | percentage of factor concentrate | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Annual Number of Filled Prescriptions of Factor Concentrate | Data at end of every year was based on the number of filled prescriptions during the last 3 years. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | prescriptions | For a duration of 3 years (for up to 11 years before the study start Day 1) |
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| Primary | Average Annual Number of Dispensed Units of Factor Concentrate | To assess annual consumption of factor concentrates, calculations were based on prescription date as start of use and the day before the next prescription as the last date of use of the factor concentrates retrieved. The annual number of dispensed units of factor concentrate were then summed up of all dispensed units of factor concentrate with periods within the calendar year plus estimates of average daily use periods extending over two years. Data at end of every year was based on the number of dispensed units of factor concentrate during the last 3 years. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | dispensed units | For a duration of 3 years (for up to 11 years before the study start Day 1) |
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| Primary | Average Number of Units of Benefix or Refacto | Data from the National Board of Health and Welfare (NBHW) had information on all filled prescriptions from July 1, 2005-last date of observation (August 31, 2015). Derived variable calculated as total number of units of filled prescriptions of Refacto/Refacto AF and Benefix over the total units of all factor VIII concentrates and factor IX concentrates, respectively. Total number of units were derived from the date of start of NBHW to last available observation or date of death, whichever was earliest. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | units | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Total Number of Bleeds | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | bleeds | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants | Joint bleeds included traumatic and spontaneous joint bleeds, muscle bleeds included traumatic and spontaneous soft tissue bleeds and other bleed events included intracranial bleed, gastrointestinal bleed and urinary tract bleed. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. | Posted | Number | bleeds | For the duration of 11 years before the study start date (Day 1) | bleeds | bleeds |
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| Primary | Gilbert Joint Score | Gilbert joint score was an instrument to measure joint health in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in haemophilia - knees, ankles, elbows. Total score ranged from 0-100, evaluating ankle, knee and elbow, where "0" indicated normal joint function, "100" indicated worst joint function, where higher values indicated more impairment in joints. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | scores on a scale | Data analyzed on study start Day 1 for the duration of 4 years (2005 to 2009) |
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| Primary | Haemophilia Joint Health Score | Haemophilia Joint Health Score (HJHS) was used to assess joint damage in participants with haemophilia. Total score ranges from 0 to 124, where, 0 indicates normal function, and 124 indicates worst joint function, higher values indicated more damage in joints. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | scores on a scale | Data analyzed on study start Day 1 for the duration of 6 years (2009 to 2015) |
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| Primary | Percentage of Participants With Surgeries | Percentage of participants who had any type of surgery (arthrodesis, surgery on foot, nose, elbow, hand or shoulder, surgery on hip, surgery on knee, tooth extraction, venous port or any other) during the data observation period were reported. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. | Posted | Number | percentage of participants | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Relative Dose Intensity of Factor Concentrate Dispensed for Haemophilia A or B Participants on Prophylaxis Based on Dispensed Volume of Units | Relative dose intensity based on dispensed volume of units was calculated using annual dispensed volume of factor concentrate per participant divided by annual prescribed dose per participant. Data was analysed for 2 categories separately: children/adolescents and adults. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | percentage of factor concentrate | For the duration of 11 years before the study start date (Day 1) |
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| Secondary | Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | SEK | For the duration of 11 years before the study start date (Day 1) |
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| Primary | Average Cost of Replacement Treatment (Benefix or Refacto/Refacto AF) Related to Bleed Event | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Swedish Krona (SEK) | For the duration of 11 years before the study start date (Day 1) |
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| Secondary | Average Cost of Replacement Treatment Related to Invasive Procedures | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | SEK | For the duration of 11 years before the study start date (Day 1) |
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| Other Pre-specified | Average Annual Cost of Replacement Treatment Related to Invasive Procedures | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | SEK | Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011) |
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| Other Pre-specified | Average Cost for Replacement Treatment Related to Invasive Procedures | The average cost for replacement treatment related to invasive procedures was derived from the percentage of total costs for dispensed replacement therapy and calculated as: cost of factor concentrate used for invasive procedures/ total cost of factor concentrate. | MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category. | Posted | Mean | Standard Deviation | percentage of cost | Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011) |
|
|
|
| 0 |
| 86 |
| 0 |
| 86 |
| 0 |
| 86 |
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
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| Title | Measurements |
|---|---|
|
| Factor aPCC |
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| 2011 |
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| 2014 |
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| Factor VIII (2011) |
|
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| Factor VIII (2014) |
|
|
| Factor IX (2005) |
|
|
| Factor IX (2008) |
|
|
| Factor IX (2011) |
|
|
| Factor IX (2014) |
|
|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Surgery on knee |
|
| Tooth extraction |
|
| Venous port |
|
| Other surgeries |
|
| Adults |
|
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|