The DOI matches its Crossref record; 6 of the 6 references it cites were found in a registry or an index.
Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease
Open accessAbstract
Allogeneic hematopoietic stem-cell transplantation (HSCT) is the only curative treatment for sickle cell disease (SCD); nevertheless, its use has been limited by the risk of transplantation-related mortality (TRM). Between November 1988 and December 2004, 87 consecutive patients with severe SCD ranging from 2 to 22 years of age received transplants in France. Cerebral vasculopathy was the principal indication for transplantation (55 patients). All the patients received grafts from a sibling donor after a myeloablative conditioning regimen (CR). The only change in the CR during the study period was the introduction of antithymocyte globulin (ATG) in March 1992. The rejection rate was 22.6% before the use of ATG but 3% thereafter. With a median follow-up of 6 years (range, 2.0 to 17.9 years), the overall and event-free survival (EFS) rates were 93.1% and 86.1%, respectively. Graft versus host disease (GVHD) was the main cause of TRM. Importantly, cord blood transplant recipients did not develop GVHD. No new ischemic lesions were detected after engraftment, and cerebral velocities were significantly reduced. The outcome improved significantly with time: the EFS rate among the 44 patients receiving transplants after January 2000 was 95.3%. These results indicate that HLA-identical sibling HSCT after myeloablative conditioning with ATG should be considered as a standard of care for SCD children who are at high risk for stroke.
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Cites
Mortality in sickle cell disease. Life expectancy and risk factors for early death
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Bone-Marrow Transplantation in a Patient with Sickle-Cell Anemia
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Cited by
Meschia, J. F., Bushnell, C., Boden-Albala, B., Braun, L. T., Bravata, D. M., Chaturvedi, S., Creager, M. A., Eckel, R. H., Elkind, M. S. V., Fornage, M., et al. (2014) · Stroke · doi:10.1161/str.0000000000000046 · Cited by 2,650
Rees, D. C., Williams, T. N., & Gladwin, M. T. (2010) · The Lancet · doi:10.1016/s0140-6736(10)61029-x · Cited by 2,504
Goldstein, L. B., Bushnell, C. D., Adams, R. J., Appel, L. J., Braun, L. T., Chaturvedi, S., Creager, M. A., Culebras, A., Eckel, R. H., Hart, R. G., et al. (2011) · Stroke · doi:10.1161/str.0b013e3181fcb238 · Cited by 1,306
Gene therapy in a patient with sickle cell disease
Ribeil, J.-A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1609677 · Cited by 696
DeWitt, M. A., Magis, W., Bray, N. L., Wang, T., Berman, J. R., Urbinati, F., Heo, S.-J., Mitros, T., Muñoz, D. P., Boffelli, D., et al. (2016) · Science Translational Medicine · doi:10.1126/scitranslmed.aaf9336 · Cited by 477
Metadata
- Authors
- Bernaudin, F., Socie, G., Kuentz, M., Chevret, S., Duval, M., Bertrand, Y., Vannier, J.-P., Yakouben, K., Thuret, I., Bordigoni, P., Fischer, A., Lutz, P., Stephan, J.-L., Dhedin, N., Plouvier, E., Margueritte, G., Bories, D., Verlhac, S., Esperou, H., Coic, L., Vernant, J.-P., & Gluckman, E.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2007-10-01
- Volume
- 110
- Issue
- 7
- Pages
- 2749-2756
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2060745772
- Cited by
- 515
- References
- 6
- FWCI
- 8.04
- Type
- journal article
- Language
- en
- Open access
- bronze
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- Bernaudin, F., Socie, G., Kuentz, M., Chevret, S., Duval, M., Bertrand, Y., Vannier, J.-P., Yakouben, K., Thuret, I., Bordigoni, P., Fischer, A., Lutz, P., Stephan, J.-L., Dhedin, N., Plouvier, E., Margueritte, G., Bories, D., Verlhac, S., Esperou, H., . . . Gluckman, E. (2007). Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. https://doi.org/10.1182/blood-2007-03-079665
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
