| Title | Reduced intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium (PBMTC) study ONC0313. | | Author(s) | Pulsipher MA, Boucher KM, Wall D, Frangoul H, Duval M, Goyal RK, Shaw PJ, Haight AE, Grimley M, Grupp SA, Kletzel M, Kadota R | | Institution | Primary Children's Medical Center, University of Utah School of Medicine, Salt Lake City, UT, United States. | | Source | Blood 2009 Jun 15. | | Abstract | 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. | | Language | ENG | | Pub Type(s) | JOURNAL ARTICLE
| | PubMed ID | 19528536 |
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