Clinical trial NCT02197845: Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators
Official title: Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Sickle Cell Disease
Keywords: sickle cell disease
hydroxyurea
patient navigator
community health worker
public health worker
fetal hemoglobin
health services research
Lead sponsor: Virginia Commonwealth University
Sponsor class: OTHER
Collaborators: name: Eastern Virginia Medical School
name: Children's Hospital of The King's Daughters
name: Virginia Department of Health
name: James Madison University
name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2014-07-23
Enrollment: 353
Sex: ALL
Minimum age (years): 15
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: HEALTH_SERVICES_RESEARCH

Brief summary: 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.)

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Phase I: Percent of enrolled Phase I subjects who complete a provider visit by 3 months post enrollment
timeFrame: 3 months
measure: Phase II: Increase in fetal hemoglobin (HbF) as measured by hemoglobin electrophoresis
timeFrame: Baseline, 6 months, 1 year
Secondary outcomes:
measure: Phase II: Measures of adherence to HU
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Percent of patients achieving either maximum tolerated dose (MTD) or maximum dose
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Number of emergency department and hospital visits
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Mean corpuscular volume
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Total hemoglobin
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: White blood cell count
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Reticulocyte count
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Quality of life measures
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Patient activation measures
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Patient knowledge measures
timeFrame: 1 year
measure: Phase II: Health care knowledge and skills, self-efficacy, sickle cell stress measures
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Social support measures
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Coping strategies
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Associated pain conditions and comorbidities
timeFrame: Baseline, 6 months, 1 year
measure: Phase II: Blood transfusion measures (if applicable)
timeFrame: 6 months, 1 year
measure: Phase II: Patient Navigator Satisfaction (if applicable)
timeFrame: 1 year
Other outcomes:
Locations:
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
Study officials:
affiliation: Virginia Commonwealth University
name: Wally R Smith, MD
role: PRINCIPAL_INVESTIGATOR
References: