Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators (SHiP HU)

Multi-phase, patient navigator-based program in the Richmond and Tidewater regions of Virginia to demonstrate:

1. 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
2. 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.)

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Behavioral: Recruitment into Specialty Care; Behavioral: Patient Navigator

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 353
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 15 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Health Services Research
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase I: Recruitment into Specialty Care; 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; 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.; Other Names: Sickle Cell Disease Specialty Care
Experimental: Phase II: Patient Navigator Arm; 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; 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.; Other Names: Community Health Worker; Public Health Worker
No Intervention: Phase II: Passenger Arm; No Intervention. Participants in the Phase II Passenger Arm follow routine clinical care.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Virginia Commonwealth University
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI); Eastern Virginia Medical School; Children's Hospital of The King's Daughters; Virginia Department of Health; James Madison University
• Investigators: Principal Investigator: Wally R Smith, MD, Virginia Commonwealth University

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2012
• Primary Completion (Actual): July 7, 2018
• Study Completion (Actual): December 31, 2018

Study Registration Dates

• First Submitted: July 21, 2014
• First Submitted That Met QC Criteria: July 22, 2014
• First Posted (Estimated): July 23, 2014

Study Record Updates

• Last Update Posted (Estimated): June 2, 2023
• Last Update Submitted That Met QC Criteria: May 31, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: hydroxyurea; sickle cell disease; community health worker; health services research; patient navigator; fetal hemoglobin; public health worker
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: HM14641; 1R18HL112737-01 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO