Shared-Decision Making for Hydroxyurea

NCT03442114 | Completed Results DMC

• 1 other identifier: CDR_1609_36055
• Study Type: interventional
• Target: 176
• Locations: 1 country
• Sites: 11

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.

Conditions

Sickle Cell Anemia; Children, Only

Keywords

shared decision making; parent-provider communication; hydroxyurea; sickle cell disease

Outcome Measures

Primary Outcomes (2)

• Decisional Conflict

  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.

  Baseline - after shared discussion with clinician

• Dyadic OPTION

  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.

  Baseline visit - after shared discussion with clinician

Secondary Outcomes (5)

• Hydroxyurea Offered

  From date of randomization until the date of first documented offering or prescription, whichever came first, assessed up to 7 months

• Satisfaction With Decision-Making

  Baseline after the shared discussion

• Ages & Stages Questionnaire - Gross Motor Subscale

  After discussion with clinician

• Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score

  After shared discussion with clinician

• Hydroxyurea Knowledge

  After shared discussion with clinician

Study Arms (2)

Hydroxyurea SDM Toolkit (H-SDM)

EXPERIMENTAL

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

Clinician Pocket Guide

ACTIVE COMPARATOR

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

Interventions

Hydroxyurea SDM Toolkit BEHAVIORAL

Implementation tools and visit decision aids

Hydroxyurea SDM Toolkit (H-SDM)

Clinician Pocket Guide BEHAVIORAL

current hydroxyurea protocol and ASH pocket guide

Clinician Pocket Guide

Eligibility Criteria

• Age: 1 Month - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (11)

UCSF Beinoff Children's Hospital and Research Center at Oakland

Oakland, California, 94609, United States

Location

Nemours Children's Health

Wilmington, Delaware, 19803, United States

Location

Howard University

Washington D.C., District of Columbia, 20060, United States

Location

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, 46260, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02118, United States

Location

The Washington University

St Louis, Missouri, 63110, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Publications (3)

• Williford DN, McTate EA, Hood AM, Reader SK, Hildenbrand AK, Smith-Whitley K, Creary SE, Thompson AA, Hackworth R, Raphael JL, Crosby LE. Psychologists as leaders in equitable science: Applications of antiracism and community participatory strategies in a pediatric behavioral medicine clinical trial. Am Psychol. 2023 Feb-Mar;78(2):107-118. doi: 10.1037/amp0001086.

  PMID: 37011163 DERIVED

• Strong H, Hood AM, Johnson Y, Hackworth R, Reed-Shackelford M, Ramaswamy R, Varughese T, Quinn CT, Crosby LE. Using the consolidated framework for implementation research to identify recruitment barriers and targeted strategies for a shared decision-making randomized clinical trial in pediatric sickle cell disease. Clin Trials. 2023 Jun;20(3):211-222. doi: 10.1177/17407745231154199. Epub 2023 Feb 16.

  PMID: 36794731 DERIVED

• 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.

  PMID: 34018965 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

Due to challenges with study implementation, low accrual, and COVID-19, the study design was changed from a stepped wedge to a quasi-experimental observational study. We were able to maintain some of the advantages of the stepped wedge including that all sites received the Toolkit dissemination method. We also considered site and cluster effects in our analyses.

Results Point of Contact

• Title: Dr. Lori Crosby
• Organization: Cincinnati Children's Medical Center

Lori.Crosby@cchmc.org

5136365380

Study Officials

• Lori E Crosby, PsyD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Stepped Wedge Randomized Clinical Trial

Study Record Dates

• First Submitted: February 9, 2018
• First Posted: February 22, 2018
• Study Start: July 12, 2018
• Primary Completion: February 28, 2022
• Study Completion: February 28, 2022
• Last Updated: June 13, 2025
• Results First Posted: May 25, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (11)