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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.
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Submitted January 12, 2009
Accepted May 27, 2009
Reduced intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium (PBMTC) study ONC0313
Michael A. Pulsipher*, Kenneth M. Boucher, Donna Wall, Haydar Frangoul, Michel Duval, Rakesh K. Goyal, Peter J. Shaw, Ann E. Haight, Michael Grimley, Stephan A. Grupp, Morris Kletzel, and Richard Kadota
Primary Children's Medical Center, University of Utah School of Medicine, Salt Lake City, UT, United States
Department of Oncological Sciences, Huntsman Cancer Institute, University of Utah School of Medicine, Salt Lake City, UT, United States
Paediatric Hematology/Oncology, CancerCare Manitoba, Winnepeg, MB, Canada
Vanderbilt Children's Hospital, Nashville, TN, United States
Hopital Sainte-Justine, Universite of Montreal, Montreal, QC, Canada
Children's Hospital of Pittsburgh, Pittsburgh, PA, United States
The Children's Hospital at Westmead, NSW, Australia
Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA, United States
Methodist Children's Hospital of South Texas, San Antonio, TX, United States
Children's Hospital of Philadelphia, Philadelphia, PA, United States
Children's Memorial Medical Center at Chicago, Chicago, IL, United States
Rady Children's Hospital San Diego, San Diego, CA, United States
* Corresponding author; email: michael.pulsipher{at}hsc.utah.edu.
The role of reduced intensity conditioning (RIC) regimens in pediatric cancer treatment is unclear. To define the efficacy of a busulfan/fludarabine/ATG RIC regimen in pediatric patients ineligible for myeloablative transplantation, we completed a trial at 23 institutions in the Pediatric Blood and Marrow Transplant Consortium (PBMTC). Forty seven pediatric patients with hematologic malignancies were enrolled. Sustained engraftment occurred in 98, 89, and 90% and full donor chimerism by day +100 was achieved in 88, 76, and 78% of evaluable related BM/PBSC, unrelated BM/PBSC, and unrelated CB recipients. With a median follow up of 24 months (range 11-53m), 2 year event free survival (EFS), overall survival (OS), transplant related mortality (TRM), and relapse were 40, 45, 11, and 43%, respectively. Univariate analysis revealed a less favorable outcome when patients had undergone previous TBI-containing myeloablative transplantation (2yr OS 23 vs. 63 vs 52%, previous TBI BMT vs. no-TBI BMT vs. no BMT, p=0.02) and when patients not previously treated with TBI had any detectable disease at the time of the RIC procedure (2yr OS 0 vs 63%, detectable vs. non-detectable disease, p=0.01). Favorable outcomes can be achieved with RIC approaches in pediatric patients in remission who are ineligible for myeloablative transplantation. This study is registered at www.clinicaltrials.gov as NCT00795132.

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