The DOI matches its Crossref record; 26 of the 26 references it cites were found in a registry or an index.
Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease
Abstract
Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with decided risk of both transplant-related mortality (TRM) and chronic graft-versus-host disease (GVHD). We analyzed 44 patients (median age, 5 years; range, 1-20 years) given an allogeneic related cord blood transplant for either thalassemia (n = 33) or SCD (n = 11). Thirty children were given cyclosporin A (CsA) alone as GVHD prophylaxis, 10 received CsA and methotrexate (MTX), and 4 patients received other combinations of immunosuppressive drugs. The median number of nucleated cells infused was 4.0 x 10(7)/kg (range, 1.2-10 x 10(7)/kg). No patient died and 36 of 44 children remain free of disease, with a median follow-up of 24 months (range, 4-76 months). Only one patient with SCD did not have sustained donor engraftment as compared with 7 of the 33 patients with thalassemia. Three of these 8 patients had sustained donor engraftment after BMT from the same donor. Four patients experienced grade 2 acute GVHD; only 2 of the 36 patients at risk developed limited chronic GVHD. The 2-year probability of event-free survival is 79% and 90% for patients with thalassemia and SCD, respectively. Use of MTX for GVHD prophylaxis was associated with a greater risk of treatment failure. Related CBT for hemoglobinopathies offers a good probability of success and is associated with a low risk of GVHD. Optimization of transplantation strategies could further improve these results.
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Cites
Chronic graft-versus-host syndrome in man
Shulman, H. M., Sullivan, K. M., Weiden, P. L., McDonald, G. B., Striker, G. E., Sale, G. E., Hackman, R., Tsoi, M.-S., Storb, R., & Donnall Thomas, E. (1980) · The American Journal of Medicine · doi:10.1016/0002-9343(80)90380-0 · Cited by 2,406
Bone-Marrow Transplantation: (First of Two Parts)
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Outcome of Cord-Blood Transplantation from Related and Unrelated Donors
Gluckman, E., Rocha, V., Boyer-Chammard, A., Locatelli, F., Arcese, W., Pasquini, R., Ortega, J., Souillet, G., Ferreira, E., Laporte, J.-P., et al. (1997) · New England Journal of Medicine · doi:10.1056/nejm199708073370602 · Cited by 1,249
Self-citationRocha, V., Wagner, J. E., Sobocinski, K. A., Klein, J. P., Zhang, M.-J., Horowitz, M. M., & Gluckman, E. (2000) · New England Journal of Medicine · doi:10.1056/nejm200006223422501 · Cited by 868
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Bone Marrow Transplantation in Patients with Thalassemia
Lucarelli, G., Galimberti, M., Polchi, P., Angelucci, E., Baronciani, D., Giardini, C., Politi, P., Durazzi, S. M. T., Muretto, P., & Albertini, F. (1990) · New England Journal of Medicine · doi:10.1056/nejm199002153220701 · Cited by 636
Bone Marrow Transplantation for Sickle Cell Disease
Walters, M. C., Patience, M., Leisenring, W., Eckman, J. R., Scott, J. P., Mentzer, W. C., Davies, S. C., Ohene-Frempong, K., Bernaudin, F., Matthews, D. C., et al. (1996) · New England Journal of Medicine · doi:10.1056/nejm199608083350601 · Cited by 627
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MARROW TRANSPLANTATION FOR THALASSAEMIA
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Bone-Marrow Transplantation in a Patient with Sickle-Cell Anemia
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Cited by
Rund, D., & Rachmilewitz, E. (2005) · New England Journal of Medicine · doi:10.1056/nejmra050436 · Cited by 939
Cao, A., & Galanello, R. (2010) · Genetics in Medicine · doi:10.1097/gim.0b013e3181cd68ed · Cited by 722
Sickle cell disease: old discoveries, new concepts, and future promise
Frenette, P. S., & Atweh, G. F. (2007) · Journal of Clinical Investigation · doi:10.1172/jci30920 · Cited by 400
Pathophysiology and treatment of stroke in sickle-cell disease: present and future
Switzer, J. A., Hess, D. C., Nichols, F. T., & Adams, R. J. (2006) · The Lancet Neurology · doi:10.1016/s1474-4422(06)70469-0 · Cited by 309
Locatelli, F., Kabbara, N., Ruggeri, A., Ghavamzadeh, A., Roberts, I., Li, C. K., Bernaudin, F., Vermylen, C., Dalle, J.-H., Stein, J., et al. (2013) · Blood · doi:10.1182/blood-2013-03-489112 · Cited by 244
Self-citation
Metadata
- Authors
- Locatelli, F.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2002-11-07
- Volume
- 101
- Issue
- 6
- Pages
- 2137-2143
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2133971325
- Cited by
- 386
- References
- 26
- FWCI
- 8.38
- Type
- journal article
- Language
- en
- Open access
- closed
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