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Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML.
Bone Marrow Transplant. 2006 Feb; 37(3):263-9.BM

Abstract

The major problems with busulfan/cyclophosphamide (Bu/Cy)-containing conditioning regimens are acute toxicities and graft failure. To decrease acute toxicities, we have prospectively evaluated a reduced intensity conditioning (RIC) regimen using targeted dosing of i.v. busulfan, fludarabine, and rabbit ATG (Bu/Flu/rATG) in children with diagnoses that historically would have been conditioned with Bu/Cy regimens. Nineteen pediatric patients were enrolled in the study. The donors included HLA-matched and one antigen-mismatched unrelated volunteers (n = 11), unrelated cord blood (n = 1), and related donors (n = 7). Four patients developed graft failure, which occurred between 1 and 8.5 months post transplant. All four of them underwent a second transplantation and 3/4 are alive without evidence of disease. The mean follow-up of living patients is 29.5 +/- s.d. 11 months. Despite excellent 2-year post-transplant overall survival (89 +/- s.d.7%) and event-free survival (74 +/- s.d.10%), the study was closed prematurely due to high graft failure rate (21%). Receiving a transplant from a mismatched unrelated donor was identified as a risk factor for graft failure. The Bu/Flu/rATG RIC regimen was very well tolerated, resulted in excellent overall survival, and provided sustained engraftment in patients undergoing transplant from matched sibling and unrelated donors. However, it did not provide sustained engraftment in the majority of children with nonmalignancies undergoing mismatched unrelated donor transplants.

Authors+Show Affiliations

Department of Pediatrics, University of California San Francisco, San Francisco, CA 94143-1278, USA. hornb@peds.ucsf.eduNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial, Phase II
Journal Article
Research Support, N.I.H., Extramural

Language

eng

PubMed ID

16327813

Citation

Horn, B, et al. "Reduced Intensity Conditioning Using Intravenous Busulfan, Fludarabine and Rabbit ATG for Children With Nonmalignant Disorders and CML." Bone Marrow Transplantation, vol. 37, no. 3, 2006, pp. 263-9.
Horn B, Baxter-Lowe LA, Englert L, et al. Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML. Bone Marrow Transplant. 2006;37(3):263-9.
Horn, B., Baxter-Lowe, L. A., Englert, L., McMillan, A., Quinn, M., Desantes, K., & Cowan, M. (2006). Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML. Bone Marrow Transplantation, 37(3), 263-9.
Horn B, et al. Reduced Intensity Conditioning Using Intravenous Busulfan, Fludarabine and Rabbit ATG for Children With Nonmalignant Disorders and CML. Bone Marrow Transplant. 2006;37(3):263-9. PubMed PMID: 16327813.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML. AU - Horn,B, AU - Baxter-Lowe,L-A, AU - Englert,L, AU - McMillan,A, AU - Quinn,M, AU - Desantes,K, AU - Cowan,M, PY - 2005/12/6/pubmed PY - 2006/5/9/medline PY - 2005/12/6/entrez SP - 263 EP - 9 JF - Bone marrow transplantation JO - Bone Marrow Transplant VL - 37 IS - 3 N2 - The major problems with busulfan/cyclophosphamide (Bu/Cy)-containing conditioning regimens are acute toxicities and graft failure. To decrease acute toxicities, we have prospectively evaluated a reduced intensity conditioning (RIC) regimen using targeted dosing of i.v. busulfan, fludarabine, and rabbit ATG (Bu/Flu/rATG) in children with diagnoses that historically would have been conditioned with Bu/Cy regimens. Nineteen pediatric patients were enrolled in the study. The donors included HLA-matched and one antigen-mismatched unrelated volunteers (n = 11), unrelated cord blood (n = 1), and related donors (n = 7). Four patients developed graft failure, which occurred between 1 and 8.5 months post transplant. All four of them underwent a second transplantation and 3/4 are alive without evidence of disease. The mean follow-up of living patients is 29.5 +/- s.d. 11 months. Despite excellent 2-year post-transplant overall survival (89 +/- s.d.7%) and event-free survival (74 +/- s.d.10%), the study was closed prematurely due to high graft failure rate (21%). Receiving a transplant from a mismatched unrelated donor was identified as a risk factor for graft failure. The Bu/Flu/rATG RIC regimen was very well tolerated, resulted in excellent overall survival, and provided sustained engraftment in patients undergoing transplant from matched sibling and unrelated donors. However, it did not provide sustained engraftment in the majority of children with nonmalignancies undergoing mismatched unrelated donor transplants. SN - 0268-3369 UR - https://www.unboundmedicine.com/medline/citation/16327813/Reduced_intensity_conditioning_using_intravenous_busulfan_fludarabine_and_rabbit_ATG_for_children_with_nonmalignant_disorders_and_CML_ L2 - https://doi.org/10.1038/sj.bmt.1705240 DB - PRIME DP - Unbound Medicine ER -