Shared-Decision Making for Hydroxyurea

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.

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.