The DOI matches its Crossref record; 31 of the 31 references it cites were found in a registry or an index.
Allogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease
Open accessAbstract
BACKGROUND: Myeloablative allogeneic hematopoietic stem-cell transplantation is curative in children with sickle cell disease, but in adults the procedure is unduly toxic. Graft rejection and graft-versus-host disease (GVHD) are additional barriers to its success. We performed nonmyeloablative stem-cell transplantation in adults with sickle cell disease. METHODS: Ten adults (age range, 16 to 45 years) with severe sickle cell disease underwent nonmyeloablative transplantation with CD34+ peripheral-blood stem cells, mobilized by granulocyte colony-stimulating factor (G-CSF), which were obtained from HLA-matched siblings. The patients received 300 cGy of total-body irradiation plus alemtuzumab before transplantation, and sirolimus was administered afterward. RESULTS: All 10 patients were alive at a median follow-up of 30 months after transplantation (range, 15 to 54). Nine patients had long-term, stable donor lymphohematopoietic engraftment at levels that sufficed to reverse the sickle cell disease phenotype. Mean (+/-SE) donor-recipient chimerism for T cells (CD3+) and myeloid cells (CD14+15+) was 53.3+/-8.6% and 83.3+/-10.3%, respectively, in the nine patients whose grafts were successful. Hemoglobin values before transplantation and at the last follow-up assessment were 9.0+/-0.3 and 12.6+/-0.5 g per deciliter, respectively. Serious adverse events included the narcotic-withdrawal syndrome and sirolimus-associated pneumonitis and arthralgia. Neither acute nor chronic GVHD developed in any patient. CONCLUSIONS: A protocol for nonmyeloablative allogeneic hematopoietic stem-cell transplantation that includes total-body irradiation and treatment with alemtuzumab and sirolimus can achieve stable, mixed donor-recipient chimerism and reverse the sickle cell phenotype. (ClinicalTrials.gov number, NCT00061568.)
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Metadata
- Authors
- Hsieh, M. M., Kang, E. M., Fitzhugh, C. D., Link, M. B., Bolan, C. D., Kurlander, R., Childs, R. W., Rodgers, G. P., Powell, J. D., & Tisdale, J. F.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2009-12-10
- Volume
- 361
- Issue
- 24
- Pages
- 2309-2317
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W2065697585
- Cited by
- 455
- References
- 31
- FWCI
- 16.84
- Type
- journal article
- Language
- en
- Open access
- bronze
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- Hsieh, M. M., Kang, E. M., Fitzhugh, C. D., Link, M. B., Bolan, C. D., Kurlander, R., Childs, R. W., Rodgers, G. P., Powell, J. D., & Tisdale, J. F. (2009). Allogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease. New England Journal of Medicine. https://doi.org/10.1056/nejmoa0904971
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