The DOI matches its Crossref record; 16 of the 16 references it cites were found in a registry or an index.
Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and β-thalassemia
Open accessAbstract
We describe previously transfused patients with sickle cell disease (n = 6) and thalassemia (n = 1) who received nonmyeloablative hematopoietic stem cell transplantation (HCT) to induce stable (full or partial) donor engraftment. Patients were 3 to 20 years (median, 9 years) old. All 7 received pretransplantation fludarabine and 200 cGy of total body irradiation; 2 patients also received horse antithymocyte globulin. Patients received bone marrow (n = 6) or peripheral blood stem cells (n = 1) from HLA-identical siblings, followed by a combination of mycophenolate mofetil and cyclosporine or tacrolimus for postgrafting immunosuppression. After nonmyeloablative HCT, absolute neutrophil counts were <0.5 x 10(9)/L and <0.2 x 10(9)/L for a median of 5 days (range, 0-13 days) and 0 days (range 0-13 days), respectively. A median of 0 (range, 0-9) platelet transfusions were administered. No grade IV nonhematologic toxicities were observed. One patient experienced grade II acute graft-versus-host disease. Two months after transplantation, 6 of 7 patients had evidence of donor chimerism (range, 25%-85%). Independent of red blood cell transfusions, these 6 patients initially had increased total hemoglobin and hemoglobin A concentrations and a reduction of reticulocytosis and transfusion requirements. There were no complications attributable to sickle cell disease during the interval of transient mixed chimerism. However, after posttransplantation immunosuppression was tapered, there was loss of the donor graft, and all patients experienced autologous hematopoietic recovery and disease recurrence. One patient did not engraft. The duration of transient mixed chimerism ranged from 97 to 441 days after transplantation in patients 4 and 6, respectively, and persisted until immunosuppressive drugs were discontinued after transplantation. In summary, the nonmyeloablative HCT regimens described here produced minimal toxicity and resulted in transient donor engraftment in 6 of 7 patients with hemoglobinopathies. Although complications from the underlying hemoglobinopathies did not occur during the period of mixed chimerism, these results suggest that stable (full or partial) donor engraftment after nonmyeloablative HCT is more difficult to achieve among immunocompetent pediatric patients with hemoglobinopathies than among adults with hematologic malignancies, perhaps in part because recipients may have been sensitized to minor histocompatibility antigens of their donor by preceding blood transfusions.
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Bone Marrow Transplantation for Sickle Cell Disease
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Self-citationStable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia
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Self-citationPersistence of mixed chimerism in patients transplanted for the treatment of thalassemia
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Cited by
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Self-citationAllogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease
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Self-citationGenome editing of HBG1 and HBG2 to induce fetal hemoglobin
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Metadata
- Authors
- Iannone, R., Casella, J. F., Fuchs, E. J., Chen, A. R., Jones, R. J., Woolfrey, A., Amylon, M., Sullivan, K. M., Storb, R. F., & Walters, M. C.
- Venue
- Biology of Blood and Marrow Transplantation
- Publisher
- Elsevier BV
- Published
- 2003-08
- Volume
- 9
- Issue
- 8
- Pages
- 519-528
- ISSN
- 1083-8791 (ISSN-L)
- OpenAlex
- W2063625476
- Cited by
- 269
- References
- 16
- FWCI
- 6.78
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- CoChat. (2026). Citation check: Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and β-thalassemia. https://app.cochat.ai/papers/10.1016/s1083-8791(03)00192-7
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- Iannone, R., Casella, J. F., Fuchs, E. J., Chen, A. R., Jones, R. J., Woolfrey, A., Amylon, M., Sullivan, K. M., Storb, R. F., & Walters, M. C. (2003). Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and β-thalassemia. Biology of Blood and Marrow Transplantation. https://doi.org/10.1016/s1083-8791(03)00192-7
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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