Shared-Decision Making for Hydroxyurea

Not Applicable

Completed

Conditions: Children, Only
Sickle Cell Anemia

Interventions: Behavioral: Clinician Pocket Guide
Behavioral: Hydroxyurea SDM Toolkit

Registration Number: NCT03442114

Lead Sponsor: Children's Hospital Medical Center, Cincinnati

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).

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 176

Inclusion Criteria

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

Exclusion Criteria

Parent/legal guardian has previously been approached OR made a decision about whether to initiate hydroxyurea.
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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Clinician Pocket Guide	Clinician Pocket Guide	In this condition, sites will provide current guidelines for offering hydroxyurea and use the American Society of Hematology (ASH) pocket guide as a reference. ASH developed 'The Hydroxyurea and Transfusion Therapy for the Treatment of Sickle Cell Disease' clinician pocket guide based on the National Heart, Lung, and Blood Institute's Evidence Based Management of Sickle Cell Disease: Expert Panel Report, 2014.'
Hydroxyurea SDM Toolkit (H-SDM)	Hydroxyurea SDM Toolkit	During the H-SDM toolkit condition, sites will develop methods for identifying Eligible Patients \& Monitoring Progress, have the opportunity to use Implementation Tools, and will use the Visit Decision Aids. The H-SDM toolkit has four visit decision aids to support parents in their decision about hydroxyurea: pre-visit brochure, in-visit issue card, after-visit booklet and video narratives {videos of parents telling their story about how they made a decision about hydroxyurea).

Primary Outcome Measures

Name	Time	Method
Decisional Conflict	Baseline - after shared discussion with clinician	Decisional Conflict Scale (DCS) is a 16-item parent-completed survey that measures uncertainty experienced when feeling uninformed about options, unclear about personal values, or unsupported in making a choice. Parents report their level of agreement with each item using a 5 point likert scale (0=strongly agree to 4=strongly disagree). For the total score, items are summed, divided by 16, and multiplied by 25. All subscores consist of 3 items except the Effective Decisions subcore (4 items) that are summed, divided by the number of items (3 or4) and multiplied by 25. Scores range from 0 (feels extremely certain about best choice) to 100 (feels extremely uncertain about best choice) on the total score and all subscores. Thus, a higher score indicates a high decisional conflict.
Dyadic OPTION	Baseline visit - after shared discussion with clinician	Dyadic OPTION describes clinician behaviors to involve a patient/parent in decision-making. A total score is calculated which ranges from 0 (no involvement) to 100 (maximal involvement). Dyadic OPTION scores correlate well with OPTION scale (Melbourne et al., 2011); 1 item "My doctor and I made the decision together"(Légaré et al., 2010). Higher scores indicate that the patient/parent has higher shared decision making competencies.

Secondary Outcome Measures

Name	Time	Method
Hydroxyurea Offered	From date of randomization until the date of first documented offering or prescription, whichever came first, assessed up to 7 months	1 of 3 responses - completed by the research coordinator based on review of electronic medical record (EMR) data: hydroxyurea was not offered, offered, or previously prescribed.
Satisfaction With Decision-Making	Baseline after the shared discussion	Eight-item survey adapted from the Satisfaction With Decision scale 41 (4 items) and the Agency for Healthcare Research and Quality's Consumer Assessment of Healthcare Providers and Systems survey related to patient experience of care (4 items). 42 Items are summed to obtain a total score ranging from 0 to 28, with higher scores indicating higher satisfaction.
Ages & Stages Questionnaire - Gross Motor Subscale	After discussion with clinician	This questionnaire is a reliable, accurate developmental and social-emotional screener for children between birth and 6 years of age, with a Cronbach α = .60 to .85. Scores range from 0 to 60, with higher scores indicating that the child's development is on schedule.
Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score	After shared discussion with clinician	Parent report of sickle cell disease (SCD)-specific quality of life (QOL) and pain, as measured by the Pediatric Quality of Life (PedsQL) SCD Module, which assesses several domains of health-related quality of life (HRQOL), including pain impact, fatigue, pain management, emotions, communication, and treatment adherence. Scores range from 0 to 100, with higher scores indicating higher HRQOL or higher functioning.
Hydroxyurea Knowledge	After shared discussion with clinician	Eight-item survey developed based on the existing literature, the Ottawa Knowledge User Manual, and parent and clinician stakeholders and used in our pilot work. Items are summed to obtain a total score ranging from 0 to 9, with higher scores indicating more knowledge.

Trial Locations

Locations (11): UCSF Beinoff Children's Hospital and Research Center at Oakland
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Oakland, California, United States
Nemours Children's Health
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Wilmington, Delaware, United States
Howard University
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Washington, District of Columbia, United States
Ann & Robert H Lurie Children's Hospital of Chicago
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Chicago, Illinois, United States
Indiana Hemophilia & Thrombosis Center
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Indianapolis, Indiana, United States
Boston Children's Hospital
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Boston, Massachusetts, United States
The Washington University
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Saint Louis, Missouri, United States
Nationwide Children's Hospital
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Columbus, Ohio, United States
Children's Hospital of Philadelphia
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Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
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Nashville, Tennessee, United States
Baylor College of Medicine
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Houston, Texas, United States