Background and Significance: Sickle cell disease (SCD) is a serious inherited disorder of red blood cells, affecting approximately 35,000 Black and Latino children and adolescents in the United States. In SCD, red blood cells can become rigid and, thereby, occlude small blood vessels. The resulting disruption of blood flood causes pain, acute and chronic injury to important organs—including the brain—and early death. In the United States, children with SCD have benefitted from treatment advances that have reduced their risk for complications and that have nearly eliminated their risk of dying before reaching adulthood. However, SCD continues to exact an enormous toll during adulthood, through chronic complications, including the loss of cognitive ability, and early death.
Because of this, some pediatric patients with SCD and their families opt for bone marrow transplantation from a matched brother or sister, a treatment that cures SCD. While bone marrow transplantation is highly effective, it carries risk for serious and sometimes fatal complications, and patients with the lived experience perceive the transplant process as physically, emotionally and socially demanding.
The decision to undergo a matched bone marrow transplant is a high-stakes decision, and the challenges of this decision making are compounded by critical knowledge gaps. This study is designed to facilitate patient and family decision making by addressing these gaps. The study will compare the effectiveness of matched related bone marrow transplantation to nontransplant treatment with supportive care and medicines in children and adolescents with SCD.
The study aims to:
- Compare the effects of bone marrow transplantation from a matched sibling and of nontransplant treatment on patient-reported health-related quality of life (HRQoL) and on SCD-associated loss of cognitive ability in children and adolescents
- Comprehensively describe risks and benefits of bone marrow transplantation from a sibling donor in children and adolescents with SCD
- Identify factors that can be used distinguish those children and adolescents who are most likely to benefit from bone marrow transplantation from a sibling donor
Study Description: WeDecide will be a study led by researchers, physicians, and patients and families through a partnership encompassing the Globin Regional Network for Data and Discovery registry (GRNDaD), the Sickle Cell Community Consortium, and the Sickle Cell Transplant Alliance for Advocacy and Research (STAR). The study will compare a group of pediatric SCD patients who have chosen to receive nontransplant treatment and a group of pediatric SCD patients who have chosen to receive a sibling bone marrow transplant. Patients with SCD who are between 3 and 20.9 years will be eligible. These patients will be recruited at North American medical centers that are part of GRNDaD and STAR.
WeDecide will compare HRQoL and cognitive function in patients after bone marrow transplant to patients who receive nontransplant treatment. These assessments will be performed using several batteries, administered at yearly intervals via iPad. Researchers will also compare the frequency of hospitalizations, emergency room visits and other acute care events in the two groups. The study will also carefully describe important risks and benefits of sibling bone marrow transplantation. Study participants will be followed for three years.
Stakeholders (patients, families, clinicians, payers and educators) will be integral to this study, engaging in its design and development, in its conduct, in the analysis and interpretation of its results and, ultimately, in the dissemination of study findings.
*All proposed projects, including requested budgets and project periods, are approved subject to a programmatic and budget review by PCORI staff and the negotiation of a formal award contract.