Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators

Study Overview
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Brief Summary
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Multi-phase, patient navigator-based program in the Richmond and Tidewater regions of Virginia to demonstrate: the feasibility of using patient navigators to improve the percentage of children and adult (age 15 and older) patients with sickle cell disease (SCD) in SCD specialty care the efficacy of using patient navigators to improve hydroxyurea (HU) (re-)initiation and adherence among adult patients with SCD eligible for HU (Patient navigators may also be known as public health workers.)

Detailed Description
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The state of Virginia, including the Virginia Department of Health and three academic medical centers and one federally qualified health center, plans a two-phase demonstration, first of improvement in the percentage of adults with SCD who are in SCD specialty care (Phase I), then of improvement in adherence to HU of eligible SCD adults (Phase II). Both phases will use existing academic SCD providers, and an innovative, multimodal strategy, featuring specially trained SCD patient navigators (PNs), that addresses barriers to care and to HU use. In Phase I we will demonstrate the feasibility of utilizing PNs to overcome patient access barriers to SCD care. In Phase II we will test the efficacy of PNs for overcoming barriers to acceptance of and adherence with HU therapy. Patients will be randomized to a PN arm or to a usual care arm. Providers will implement NIH guidelines for HU eligibility and prescribing in both arms. All HU eligible patients will be offered HU at each clinical visit. Among patients prescribed HU, if a maximum tolerated dose (MTD, defined in end points) has not been reached, providers will dose escalate every 8-12 weeks to MTD, rather than to clinical effect, before declaring treatment success or failure. This project will be critically important and impactful by demonstrating the feasibility of a statewide community-based strategy to assist vulnerable SCD adults in obtaining SCD specialty care and likely prolonging life, a model that other states could adopt.

Official Title
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Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Behavioral: Patient Navigator
* Behavioral: Recruitment into Specialty Care


Participation Criteria
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Eligibility Criteria
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PHASE I: Inclusion Criteria: Patient Self Report of Sickle Cell Disease (Genotypes: Hb SS, SC, SBoThal, SB+Thal) 15 years or older Virginia resident Exclusion Criteria: -Visited one of a pre-selected list of sickle cell specialists in Virginia within the last 6 months PHASE II: Inclusion Criteria: Sickle Cell Disease (SCD) patient (Genotypes: SS or SBoThal) Eligible for Hydroxyurea (according to NIH guidelines) 15 years or older Virginia resident Exclusion Criteria: Pregnancy Enrollment in scheduled chronic transfusion program SCD Genotype: Hb SC and SB+Thal)

Ages Eligible for Study
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Minimum Age: 15 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: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Phase I: Recruitment into Specialty Care<br>Participants in the Phase I Experimental Arm are enrolled into SCD specialty care. PN's will contact patient up to 3 times to assure patients have had an initial visit by 3 months time. | Behavioral: Recruitment into Specialty Care<br>* A specially trained (SCD knowledgeable) Patient Navigator will act as a liaison to participants in increasing adherence to Hydroxyurea. Interventions utilized by a PN include teaching disease management skills, addressing barriers to care, disease education, HU management, motivational interviewing, care coordination and social support.<br>* Other names: Sickle Cell Disease Specialty Care;|
| Experimental: Phase II: Patient Navigator Arm<br>Participants in the Phase II Experimental Arm follow routine clinical care and are assigned a Patient Navigator. A specially trained (SCD specefic)PN will work with participants for one year. Participants will be contacted by their Navigator weekly for the first 6 months, then biweekly for the second 6 months. | Behavioral: Patient Navigator<br>* A specially trained (SCD knowledgable) Patient Navigator will act as a liaison who assists participants in increasing adherence to HU through disease education, addressing barriers to care and improving adherence to HU. PN utilize various techniques geared toward the individual patients needs. Interventions utilized by a PN include improving disease management skills, educational materials about disease, HU adherence, motivational interviewing, care coordination and social support.<br>* Other names: Community Health Worker;|
| No Intervention: Phase II: Passenger Arm<br>No Intervention. Participants in the Phase II Passenger Arm follow routine clinical care. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Phase I: Percent of enrolled Phase I subjects who complete a provider visit by 3 months post enrollment |  | 3 months | 
| Phase II: Increase in fetal hemoglobin (HbF) as measured by hemoglobin electrophoresis |  | Baseline, 6 months, 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Phase II: Measures of adherence to HU | For patients prescribed HU, clinical research coordinators will assess HU prescription refills from pill counts, pharmacy records, and self-report at baseline, 6 months, and 1 year. Patient navigators will regularly assess their patients' HU adherence by conducting pill counts at home visits. | Baseline, 6 months, 1 year | 
| Phase II: Percent of patients achieving either maximum tolerated dose (MTD) or maximum dose | Maximum tolerated dose (MTD) is the daily single oral dose that can be maintained for at least 16 weeks without toxicity (< 3 x l09 neutrophils/L, <100 x l09 platelets/L, < 125 x l09 reticulocytes/L, 20% drop in [Hb] or an absolute value of <4.5 g/dL, 50% rise in creatinine or absolute increase of >0.4 mg/dL, 100% rise in ALT,GI disturbance, or rash or hair loss not attributable to other causes). Maximum dose is 35 mg/kg. | Baseline, 6 months, 1 year | 
| Phase II: Number of emergency department and hospital visits |  | Baseline, 6 months, 1 year | 
| Phase II: Mean corpuscular volume |  | Baseline, 6 months, 1 year | 
| Phase II: Total hemoglobin |  | Baseline, 6 months, 1 year | 
| Phase II: White blood cell count |  | Baseline, 6 months, 1 year | 
| Phase II: Reticulocyte count |  | Baseline, 6 months, 1 year | 
| Phase II: Quality of life measures | Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me), Patient Reported Outcomes Measurement Information System (PROMIS) | Baseline, 6 months, 1 year | 
| Phase II: Patient activation measures | Patient Activation Measure | Baseline, 6 months, 1 year | 
| Phase II: Patient knowledge measures | Assessment of Sickle Cell Knowledge - University of Florida (UF-ASCK) (unpublished) | 1 year | 
| Phase II: Health care knowledge and skills, self-efficacy, sickle cell stress measures | Sickle Cell Transition intervention Program (TIP) Survey (non-validated) | Baseline, 6 months, 1 year | 
| Phase II: Social support measures | Sickle Cell Transition intervention Program (TIP) Survey (non-validated) and Multidimensional Scale of Perceived Social Support | Baseline, 6 months, 1 year | 
| Phase II: Coping strategies | Coping Strategies Questionnaire for SCD | Baseline, 6 months, 1 year | 
| Phase II: Associated pain conditions and comorbidities | Chart Review, Self Report and Surveys: Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me), Patient Reported Outcomes Measurement Information System (PROMIS) | Baseline, 6 months, 1 year | 
| Phase II: Blood transfusion measures (if applicable) | Chart review | 6 months, 1 year | 
| Phase II: Patient Navigator Satisfaction (if applicable) | Patient Navigator Satisfaction Surveys, 12 month follow up, Patient Study Experience Review for Patient Navigators | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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sickle cell disease, hydroxyurea, patient navigator, community health worker, public health worker, fetal hemoglobin, health services research