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Blood, 13 August 2009, Vol. 114, No. 7, pp. 1429-1436. Prepublished online as a Blood First Edition Paper on June 15, 2009; DOI 10.1182/blood-2009-01-196303.
TRANSPLANTATION Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC03131 Primary Children's Medical Center and 2 Department of Oncological Sciences, Huntsman Cancer Institute, University of Utah School of Medicine, Salt Lake City; 3 Paediatric Hematology/Oncology, CancerCare Manitoba, Winnepeg, MB; 4 Vanderbilt Children's Hospital, Nashville, TN; 5 Hopital Sainte-Justine, Université of Montreal, Montreal, QC; 6 Children's Hospital of Pittsburgh, PA; 7 Children's Hospital at Westmead, Westmead, Australia; 8 Children's Healthcare of Atlanta, Emory University School of Medicine, GA; 9 Methodist Children's Hospital of South Texas, San Antonio; 10 Children's Hospital of Philadelphia, PA; 11 Children's Memorial Medical Center at Chicago, IL; and 12 Rady Children's Hospital San Diego, CA The role of reduced-intensity conditioning (RIC) regimens in pediatric cancer treatment is unclear. To define the efficacy of a busulfan/fludarabine/antithymocyte globulin RIC regimen in pediatric patients ineligible for myeloablative transplantation, we completed a trial at 23 institutions in the Pediatric Blood and Marrow Transplant Consortium. Forty-seven patients with hematologic malignancies were enrolled. Sustained engraftment occurred in 98%, 89%, and 90%, and full donor chimerism was achieved in 88%, 76%, and 78% of evaluable related bone marrow/peripheral blood stem cells (BM/PBSCs), unrelated BM/PBSCs, and unrelated cord blood recipients. With a median follow-up of 24 months (range, 11-53 months), 2-year event-free survival, overall survival (OS), transplantation-related mortality, and relapse were 40%, 45%, 11%, and 43%, respectively. Univariate analysis revealed an inferior outcome when patients had undergone previous total body irradiation (TBI)–containing myeloablative transplantation (2-year OS, 23% vs 63% vs 52%, previous TBI transplantation vs no TBI transplantation vs no transplantation, P = .02) and when patients not previously treated with TBI had detectable disease at the time of the RIC procedure (2-year OS, 0% vs 63%, detectable vs nondetectable disease, P = .01). Favorable outcomes can be achieved with RIC approaches in pediatric patients in remission who are ineligible for myeloablative transplantation. This study was registered at www.clinicaltrials.gov as #NCT00795132 [ClinicalTrials.gov] .
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