Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Patient Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy With Hydroxyurea in Patients With Sickle Cell Disease

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Behavioral: Mobile DOT


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: be >2 years of age up to 65 years of age, inclusive have a diagnosis of SCD, with either βS/βS, βS/βC, βS/βD, βS/β0, βS/βO-Arab, or βS/β+ genotype prescribed Hydroxyurea for at least the 6 months prior to study entry have daily access to a smart phone, tablet, personal computer or other device capable of producing and transmitting videos over the internet be willing and able to record and transmit videos Exclusion Criteria: patient or caregiver refuses to take Hydroxyurea as treatment for SCD diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study an assessment by the investigator that the subject will not comply with the study procedures outlined in the study protocol patients receiving automatic home delivery of medications since medication possession ratio is reflective of the patient initiation the refill when they have exhausted the home supply of HU

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Health Services Research
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Adults - Mobile DOT<br>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. | Behavioral: Mobile DOT<br>* 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.<br>* Other names: Comprehensive medication adherence management;|
| Active Comparator: Adults - standard of care then Mobile DOT<br>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. | Behavioral: Mobile DOT<br>* 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.<br>* Other names: Comprehensive medication adherence management;|
| Experimental: Children - Mobile DOT<br>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. | Behavioral: Mobile DOT<br>* 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.<br>* Other names: Comprehensive medication adherence management;|
| Active Comparator: Children - standard of care then Mobile DOT<br>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. | Behavioral: Mobile DOT<br>* 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.<br>* Other names: Comprehensive medication adherence management;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

Secondary Outcome Measures

| Outcome Measure | 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. | 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 |