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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
Investigator (PI): Krishnamurti, Lakshmanan
Performing Organization (PO): (Current): Emory University, School of Medicine, Department of Pediatrics, Division of Hematology/Oncology / (404) 785-1200
(Past): University of Pittsburgh Medical Center, Children's Hospital of Pittsburgh, Department of Pediatrics, Division of Pediatric Hematology/Oncology / (412) 692-5055
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2013
Final Year: 2019
Record Source/Award ID: PCORI/CE-1304-6859-EM
Funding: Total Award Amount: $2,148,601
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: 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, underserved 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 system, provider, treatment, socioeconomic, and patient levels. Our 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. To test our hypothesis we propose the following specific aims. Aim 1 is to determine in a randomized clinical trial if individualized structured interventions will improve adherence with HU and will be acceptable to patients with SCD. Children, adolescents, and adult subjects with SCD will be randomized in a multi-center study to adherence intervention arm or standard of clinical care. Interventions will be individualized to patient barriers and preferences and will consist of improved access to care through virtual clinic visits and in-home testing, adherence management through a novel cell phone video-based directly observed therapy (Mobile-DOT) and patient support using text and telephone follow-up. The standard arm will consist of the usual clinical care. We will measure adherence through review of pharmacy records and in patients on the intervention arm through direct video record of adherence as well as by validated measures of self-reported medication adherence. We will administer qualitative interviews to patients to explore attitudes towards acceptability and utility to patients of structured interventions to improve HU adherence. Aim 2 is to determine the impact of adherence with HU on clinical and patient-reported outcomes, and patient burden of care. Randomized clinical trial will generate detailed measures of the level of adherence among patients in the intervention and standard-of-care arms. Using standardized survey instruments we will determine patient-reported outcomes of physical functioning, fatigue, pain and sleep, pediatric and adult quality of life, clinical and laboratory parameters, health care utilization, functioning at school and work, individual and system financial savings, and burden of implementing the interventions. This will help determine the impact of adherence with HU on patient-reported, clinical, laboratory, and health services outcomes. The completion of these aims will help define factors which predict adherence to HU in SCD, and determine the effectiveness of a set of structured interventions in improving both adherence and patient-related outcomes.
MeSH Terms:
  • Adolescent
  • Adult
  • Anemia, Sickle Cell /*drug therapy
  • /*psychology
  • Cell Phone
  • Child
  • Health Services Accessibility
  • Humans
  • Hydroxyurea /*therapeutic use
  • * Medication Adherence
  • Multicenter Studies as Topic
  • Outcome Assessment, Health Care
  • Patient Education as Topic /*methods
  • Patient Participation
  • * Patient-Centered Care
  • Quality of Life
  • Randomized Controlled Trials as Topic
  • Reminder Systems
  • Telephone
  • Text Messaging
  • United States
Country: United States || United States
State: Georgia || Pennsylvania
Zip Code: 15224 / 30322
UI: 20143530
CTgovId: NCT02371720
Project Status: Completed
Record History: ('2019: Project extended to 2019. 2018: Start date corrected from 2013 to 2014. 2017: Project extended to 2018. Project extended to 2017, and PO changed, 8/3/15. ',)