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Bone marrow transplantation for sickle cell disease
M C Walters1, M Patience, W Leisenring
1Division of Clinical Research, Fred Hutchinson Cancer Research Center, Seattle, WA 98104, USA.
Insights
Allogeneic stem-cell transplantation offers a curative option for children with severe sickle cell disease, significantly improving survival and stabilizing complications like stroke and chest syndrome.
Area of Science:
- Hematology
- Pediatric Medicine
- Transplantation Immunology
Background:
- Sickle cell disease (SCD) presents significant complications in children, necessitating advanced treatment strategies.
- Allogeneic bone marrow transplantation is explored as a potential curative therapy for symptomatic SCD.
- Evaluating the risks and benefits of this procedure in pediatric SCD patients is crucial.
Purpose of the Study:
- To assess the efficacy and safety of allogeneic bone marrow transplantation in children with severe sickle cell disease.
- To determine the outcomes of transplantation in pediatric patients with SCD-related complications such as stroke, acute chest syndrome, and painful crises.
Main Methods:
- A cohort of 22 children under 16 with symptomatic SCD received HLA-identical sibling marrow allografts.
- Conditioning regimen included busulfan, cyclophosphamide, and antithymocyte globulin.
- Follow-up assessed engraftment, graft rejection, survival, and disease-specific complications.
Main Results:
- Twenty of 22 patients survived, with a median follow-up of 23.9 months.
- Sixteen patients achieved stable engraftment; three experienced graft rejection with SCD recurrence.
- Survival and event-free survival at four years were 91% and 73%, respectively, with stabilization of lung function and cerebrovascular disease observed.
Conclusions:
- Allogeneic stem-cell transplantation demonstrates curative potential in young patients with symptomatic sickle cell disease.
- The procedure offers significant long-term survival benefits and can halt disease progression.
- Transplantation should be considered for pediatric SCD patients with severe complications.
Background:
We investigated the risks and benefits of allogeneic bone marrow transplantation in children with complications of sickle cell disease.
Methods:
Twenty-two children less than 16 years of age who had symptomatic sickle cell disease received marrow allografts from HLA-identical siblings between September 1991 and April 1995. The indications for transplantation included a history of stroke (n = 12), recurrent acute chest syndrome (n = 5), and recurrent painful crises (n = 5). Patients were prepared for transplantation with busulfan, cyclophosphamide, and antithymocyte globulin.
Results:
Twenty of the 22 patients survived, with a median follow-up of 23.9 months (range, 10.1 to 51.0), and 16 patients had stable engraftment of donor hematopoietic cells. In three patients the graft was rejected and sickle cell disease recurred; in a fourth patient graft rejection was accompanied by marrow aplasia. In 1 of the 16 patients with engraftment, there was stable mixed chimerism. Two patients died of central nervous system hemorrhage or graft-versus-host disease. Kaplan-Meier estimates of survival and event-free survival at four years were 91 percent and 73 percent, respectively. Among patients with a history of acute chest syndrome, lung function stabilized; among patients with prior central nervous system vasculopathy who had engraftment, stabilization of cerebrovascular disease was documented by magnetic resonance imaging.
Conclusions:
Allogeneic stem-cell transplantation can be curative in young patients with symptomatic sickle cell disease.