Shared-Decision Making for Hydroxyurea (ENGAGE-HU)

Engaging Parents of Children With Sickle Cell Anemia and Their Providers in Shared-Decision Making for Hydroxyurea (ENGAGE HU)

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.

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Anemia; Children, Only
• Intervention / Treatment: Behavioral: Hydroxyurea SDM Toolkit; Behavioral: Clinician Pocket Guide

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).

• Study Type: Interventional
• Enrollment (Anticipated): 260
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Yolanda Johnson, MLS; Phone Number: 5138030918; Email: engagehu@cchmc.org
• Study Contact Backup: Name: Naima Griffin; Phone Number: 5136360000; Email: engagehu@cchmc.org

Study Locations

• United States
  • California
    • Oakland, California, United States, 94609
      • Recruiting
      • UCSF Beinoff Children's Hospital and Research Center at Oakland
      • Contact: Lynne Neumayr, MD
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Recruiting
      • Nemours Children's Health
      • Contact: Steven Reader, PhD
      • Principal Investigator: Steven Reader, PhD
  • District of Columbia
    • Washington, District of Columbia, United States, 20060
      • Terminated
      • Howard University
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Recruiting
      • Ann & Robert H Lurie Children's Hospital of Chicago
      • Contact: Sherif Badawy, MD
      • Sub-Investigator: Sherif Badawy, MD
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Recruiting
      • Indiana Hemophilia & Thrombosis center
      • Contact: Angeli Rampersad, MD
      • Principal Investigator: Angeli Rampersad, MD
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
      • Completed
      • Boston Children's Hospital
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Recruiting
      • The Washington University
      • Contact: Allison King, MD
      • Sub-Investigator: Cecelia Calhoun, MD
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Recruiting
      • Nationwide Children's Hospital
      • Contact: Susan Creary, MD
      • Principal Investigator: Susan Creary, MD
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
      • Principal Investigator: Alexis Thompson, MD
      • Contact: Alexis Thompson, MD
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Terminated
      • Vanderbilt University Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Baylor College of Medicine
      • Contact: Jean Raphael, MD
      • Sub-Investigator: Amber Yates, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 5 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Health Services Research
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Hydroxyurea SDM Toolkit (H-SDM); 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).	Behavioral: Hydroxyurea SDM Toolkit; Implementation tools and visit decision aids
Active Comparator: 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.'	Behavioral: Clinician Pocket Guide; current hydroxyurea protocol and ASH pocket guide

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Decisional conflict	Decisional Conflict Scale (DCS)	up to 4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Shared decision making	Dyadic OPTION	up to 4 weeks
Hydroxyurea offered	electronic medical record (EMR) data	From date of randomization until the date of first documented offering or prescription, whichever came first, assessed up to 7 months

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Investigators: Principal Investigator: Lori E Crosby, PsyD, Children's Hospital Medical Center, Cincinnati

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): July 12, 2018
• Primary Completion (Anticipated): February 28, 2022
• Study Completion (Anticipated): February 28, 2022

Study Registration Dates

• First Submitted: February 9, 2018
• First Submitted That Met QC Criteria: February 20, 2018
• First Posted (Actual): February 22, 2018

Study Record Updates

• Last Update Posted (Actual): February 25, 2022
• Last Update Submitted That Met QC Criteria: February 9, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Keywords: hydroxyurea; shared decision making; sickle cell disease; parent-provider communication
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia; Anemia, Sickle Cell; Molecular Mechanisms of Pharmacological Action; Nucleic Acid Synthesis Inhibitors; Enzyme Inhibitors; Antineoplastic Agents; Antisickling Agents; Hydroxyurea
• Other Study ID Numbers: CDR_1609_36055

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: This study will comply with the Patient Centered Outcomes Research Institute (PCORI) Public Access Policy, which ensures that the public has access to the published results of PCORI funded research.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No