Hematopoietic cell transplantation in sickle cell disease
- Shakila Khan, MD
Shakila Khan, MD
- Associate Professor of Pediatrics
- Mayo Clinic College of Medicine
- Griffin P Rodgers, MD
Griffin P Rodgers, MD
- Chief, Molecular and Clinical Hematology Branch
- National Institutes of Health
Hematopoietic cell transplantation (HCT) is an accepted form of treatment for some non-malignant hematologic disorders, such as aplastic anemia and beta thalassemia major. The use of HCT in sickle cell disease (SCD) is evolving. This topic review discusses the use of HCT in SCD. Other therapies for SCD including hydroxyurea and chronic blood transfusion therapy, and an overview of SCD management, are presented separately. (See "Hydroxyurea and other disease-modifying therapies in sickle cell disease" and "Red blood cell transfusion in sickle cell disease" and "Overview of the management and prognosis of sickle cell disease".)
The term HCT will be used throughout this review to refer to transplantation of stem and progenitor cells from any source (eg, bone marrow, peripheral blood, cord blood). In specific discussions related to the source of stem cells, the source will be specified (eg, peripheral blood stem cells).
LIMITATIONS AND BARRIERS TO HCT
HCT is a potentially curative treatment for hemoglobinopathies [1,2]. The most extensive experience showing the durable nature of cure has been established in patients with beta thalassemia major for more than a decade. In beta thalassemia major, the course of diminished life expectancy is fairly uniform; criteria for patient selection have, therefore, centered on the availability of a HLA-matched family donor and transplanting the patient early, before severe organ damage has occurred, in order to achieve a better outcome. (See "Efficacy of hematopoietic cell transplantation in beta thalassemia major".)
In the case of SCD, data concerning the efficacy of HCT are not as extensive, due in part to the following factors:
●The clinical course of the disease is quite variable, and prognostic factors that might indicate which patients are most likely to develop advanced stage disease, and thus be candidates for HCT, are lacking.
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- LIMITATIONS AND BARRIERS TO HCT
- Siblings as donors
- CLINICAL EXPERIENCE WITH TRANSPLANTATION
- Survival, engraftment, and graft-versus-host disease
- Other outcomes
- Experience in adults
- Indications for HCT
- OTHER APPROACHES
- Early transplantation
- Alternative donors
- Non-myeloablative conditioning regimens
- Donor lymphocyte infusion
- SUMMARY AND RECOMMENDATIONS