Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313

Author:

Pulsipher Michael A.1,Boucher Kenneth M.2,Wall Donna3,Frangoul Haydar4,Duval Michel5,Goyal Rakesh K.6,Shaw Peter J.7,Haight Ann E.8,Grimley Michael9,Grupp Stephan A.10,Kletzel Morris11,Kadota Richard12

Affiliation:

1. 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

Abstract

AbstractThe 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.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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