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| Name | Class |
|---|---|
| Patient-Centered Outcomes Research Institute | OTHER |
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The purpose of this research study is to learn about ways to help children and adults with sickle cell disease who are taking the medication, hydroxyurea.
Sickle cell disease (SCD) is an inherited chronic multi-organ system disorder that affects approximately 100,000 individuals in the United States, mostly belonging to minority, under-served populations. SCD is associated with substantial morbidity, premature mortality, individual suffering, health care costs and loss of productivity. Hydroxyurea (HU) the only disease modifying therapy for SCD is efficacious in reducing complications such as pain crisis and acute chest syndrome and improving survival. It is however, vastly underutilized and poorly adhered to because of barriers at the health care system, provider, treatment, socioeconomic, and patient levels. The investigator's overarching hypothesis is that barriers to acceptance and adherence to HU are multi-factorial and that a structured set of interventions can lead to improved adherence to medication and patient centered outcomes.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adults - Mobile DOT | Experimental | Subjects with SCD that are older than 21 years old will receive comprehensive medication adherence management (Mobile DOT) after 1 month assessment period. The subjects will receive the Mobile DOT intervention for 24 months. |
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| Adults - standard of care then Mobile DOT | Active Comparator | Subjects with SCD that are older than 21 years old will receive standard of care for the first 12 months. They will then crossover to the comprehensive medication adherence management plan (Mobile DOT) after 1 month assessment period for the remaining 12 months. |
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| Children - Mobile DOT | Experimental | Subjects with SCD that are younger than 21 years old will receive comprehensive medication adherence management (Mobile DOT) after 1 month assessment period. The subjects will receive the Mobile DOT intervention for 24 months. |
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| Children - standard of care then Mobile DOT | Active Comparator | Subjects with SCD that are younger than 21 years old will receive standard of care for the first 12 months. They will then crossover to the comprehensive medication adherence management plan (Mobile DOT) after 1 month assessment period for the remaining 12 months. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mobile DOT | Behavioral | Daily reminders via text or email to send a video of themselves taking their Hydroxyurea, positive feedback, and be encouraged to contact the research coordinator with any questions, concerns, etc. |
| Measure | Description | Time Frame |
|---|---|---|
| Medication Possession Ratio (MPR) | Proportion of days the patient is in possession of the medication in the study period | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Hemoglobin (Hb) levels | Change in hemoglobin levels from baseline to 24 months will be measured using the HemoCue® rapid test. | Baseline, 24 months |
| Change in mean cell volume (MCV) | Change from baseline in MCV will be calculated as the value at 24 months minus the value at baseline. MCV is the average size of the red blood cells expressed in femtoliters. MCV is calculated by dividing the hematocrit (as percent) by the red blood cell (RBC) count in millions per microliter of blood, then multiplying by 10. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Lakshmanan Krishnamurti, MD | Emory University/Children's Healthcare of Atlanta | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States | ||
| Children's Healthcare of Atlanta |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| Baseline, 24 months |
| Change in fetal hemoglobin (HbF) levels | Change from baseline in HbF will be calculated as the value at 24 months minus the value at baseline. HbF is expressed as a percentage. | Baseline, 24 months |
| Impact of adherence on clinical outcomes and healthcare utilization | Health care utilization in the emergency department and hospitalization due to sickle cell related complications such as vaso-occlusive crisis (VOC) or acute chest syndrome (ACS). Retrospective chart review at baseline will be conducted to determine healthcare utilization. | Baseline, 24 months |
| Impact of adherence on patients' lives | Impact of adherence on patients' lives measured using patient reported outcomes (PROMIS), surveys of school attendance, work absenteeism, out-of-pocket costs incurred by patients and their caregivers | Baseline, 24 months |
| Change in adherence with using Mobile-DOT | Retrospective chart review at baseline will be conducted to determine medication possession rate (MPR) and then compared to the MPR at 24 months. | Baseline, 24 months |
| Acceptability of intervention and of Hydroxyurea | Acceptability will be measured by Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) The TSQM is a 14-item subject-assessed evaluation of treatment medication including 4 factors, Effectiveness, Side Effects, Convenience, and Global Satisfaction, and it utilizes the following responses on a 7-point Likert scale: 1=Extremely Dissatisfied, 2=Very Dissatisfied, 3=Somewhat Dissatisfied, 4=Neither Satisfied Nor Dissatisfied, 5=Somewhat Satisfied, 6=Very Satisfied, 7=Extremely Satisfied. Scores range from 0-100, with 0 as extremely dissatisfied and 100 as extremely satisfied. | Baseline, 24 months |
| Atlanta |
| Georgia |
| 30322 |
| United States |
| University of Illinois at Chicago | Chicago | Illinois | 60607 | United States |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |