N/A
Completed N=174
Shared-Decision Making for Hydroxyurea
Sickle-Cell Anemia · Children, Only
Source: ClinicalTrials.gov NCT03442114 ↗
Enrolled (actual)
174
Serious AEs
3.5%
Results posted
May 2025
Primary outcomePrimary: Decisional Conflict — 22.9; 19.8; 26.9; 23.5 score on a scale
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Decisional Conflict |
22.9; 19.8; 26.9; 23.5; 20.8; 16.8 | — |
| PRIMARY Dyadic OPTION |
39.8; 40.3 | — |
| SECONDARY Hydroxyurea Offered |
61; 32; 19; 25; 3; 3 | — |
| SECONDARY Satisfaction With Decision-Making |
13.5; 13.2 | — |
| SECONDARY Ages & Stages Questionnaire - Gross Motor Subscale |
50.9; 51.9 | — |
| SECONDARY Pediatric Quality of Life Inventory - Sickle Cell Disease Module (Peds QL - SCD Module) - Total Score |
82.5; 82.0 | — |
| SECONDARY Hydroxyurea Knowledge |
6.3; 6.1 | — |
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT03442114). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.