Clinical trial NCT03442114: Shared-Decision Making for Hydroxyurea
Official title: Engaging Parents of Children With Sickle Cell Anemia and Their Providers in Shared-Decision Making for Hydroxyurea (ENGAGE HU)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Sickle Cell Anemia
Children, Only
Keywords: shared decision making
parent-provider communication
hydroxyurea
sickle cell disease
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2018-02-22
Enrollment: 176
Sex: ALL
Minimum age (years): 1
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Stepped Wedge Randomized Clinical Trial
Primary purpose: HEALTH_SERVICES_RESEARCH

Brief summary: The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent values and preferences). The study will compare two methods to help clinicians facilitate this-a clinician pocket guide and a clinician hydroxyurea shared decision making toolkit-in a group of parents of children ages 0-5 with sickle cell disease. The investigators hope that both methods lead to parents reaching a high-quality, well-informed decision. In addition, the team hopes to demonstrate that parents who experience a shared decision will have lower anxiety and decisional uncertainty. The researchers also expect these parents to be more likely to choose hydroxyurea and that their children will have less pain, fewer hospitalizations, better developmental outcomes, and higher quality of life. The project team hopes to show that the toolkit method is easy for clinicians to use and gives parents the support needed to make an informed decision.

Detailed description: Sickle cell disease (SCD) is a genetic blood disorder that places children at risk for serious medical complications, early morbidity and mortality, and high healthcare utilization. In the U.S., SCD primarily affects African-American and Latino children. Hydroxyurea is one of the only disease-modifying treatment for this devastating and life-threatening disease. National Evidence-Based Guidelines recommend the use of a shared decision making approach to offer hydroxyurea to all children with SCD as early as nine months of age. Hydroxyurea uptake remains low because parents lack information about hydroxyurea and have concerns about its safety and potential long-term side effects (e.g. cancer, infertility, birth defects). Clinicians do not have the training or tools to facilitate a shared discussion with parents that provides medical evidence and considers parent preferences and values. The current study compares two methods for disseminating hydroxyurea guidelines and facilitating shared decision-making: the American Society of Hematology's hydroxyurea clinician pocket guide (usual care method) and a clinician hydroxyurea shared decision-making toolkit (H-SDM toolkit). The specific aims of the study are to evaluate the effectiveness of the usual care dissemination method (clinician pocket guide) and the H-SDM clinician toolkit dissemination method on: parent report of decisional uncertainty (primary outcome chosen by parents of children with SCD), parent perception of experiencing shared decision-making, parent knowledge of hydroxyurea, the number of children offered hydroxyurea, hydroxyurea uptake (those with active prescriptions), and child health outcomes (pain, neurocognitive functioning, sickle cell related quality of life and healthcare utilization). Eligible children must be between the ages of 0 and 5 and a candidate for hydroxyurea to participate. The trial will use a stepped-wedge design (clinic is the unit of randomization). The long-term objective of the research team is to improve the quality of care for children with SCD. The investigators propose that suboptimal care for patients with SCD is preventable with the use of multicomponent dissemination methods if developed with key stakeholders and designed to address barriers to high quality care at multiple levels (patient, clinician, healthcare system, and community).

Eligibility criteria: Inclusion Criteria:

1. Diagnosis: sickle cell disease
2. Age: birth-5 years, inclusive
3. Eligible for hydroxyurea (genotype SS, Sβ0Thal or other genotype + clinical complications)
4. Child's parent, legal guardian, or designated decision maker (caregiver) must participate in both study visits
5. Child's parent, legal guardian, or designated decision maker (caregiver) must able to read, understand, and speak English

Exclusion Criteria:

1. Parent/legal guardian has previously been approached OR made a decision about whether to initiate hydroxyurea.
2. Any and all other diagnoses or conditions which, in the opinion of the site investigator or hematologist, would prevent the patient from being a suitable candidate for the study.

Primary outcomes:
measure: Decisional Conflict
timeFrame: Baseline - after shared discussion with clinician
measure: Dyadic OPTION
timeFrame: Baseline visit - after shared discussion with clinician
Secondary outcomes:
measure: Hydroxyurea Offered
timeFrame: From date of randomization until the date of first documented offering or prescription, whichever came first, assessed up to 7 months
measure: Satisfaction With Decision-Making
timeFrame: Baseline after the shared discussion
measure: Ages & Stages Questionnaire - Gross Motor Subscale
timeFrame: After discussion with clinician
measure: Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score
timeFrame: After shared discussion with clinician
measure: Hydroxyurea Knowledge
timeFrame: After shared discussion with clinician
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Beinoff Children's Hospital and Research Center at Oakland
state: California
city: Wilmington
country: United States
facility: Nemours Children's Health
state: Delaware
city: Washington
country: United States
facility: Howard University
state: District of Columbia
city: Chicago
country: United States
facility: Ann & Robert H Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia & Thrombosis Center
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: The Washington University
state: Missouri
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Lori E Crosby, PsyD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Hood AM, Strong H, Nwankwo C, Johnson Y, Peugh J, Mara CA, Shook LM, Brinkman WB, Real FJ, Klein MD, Hackworth R, Badawy SM, Thompson AA, Raphael JL, Yates AM, Smith-Whitley K, King AA, Calhoun C, Creary SE, Piccone CM, Hildenbrand AK, Reader SK, Neumayr L, Meier ER, Sobota AE, Rana S, Britto M, Saving KL, Treadwell M, Quinn CT, Ware RE, Crosby LE. Engaging Caregivers and Providers of Children With Sickle Cell Anemia in Shared Decision Making for Hydroxyurea: Protocol for a Multicenter Randomized Controlled Trial. JMIR Res Protoc. 2021 May 21;10(5):e27650. doi: 10.2196/27650.