Biol Blood Marrow Transplant. 2016 Sep 22. pii: S1083-8791(16)30360-3. doi: 10.1016/j.bbmt.2016.09.020. [Epub ahead of print]

Comparison of Donor Sources in Hematopoietic Stem Cell Transplantation for Childhood Acute Leukemia: a Nationwide Retrospective Study.

Sakaguchi H¹, Watanabe N², Matsumoto K³, Yabe H⁴, Kato S⁴, Ogawa A⁵, Inagaki J⁶, Goto H⁷, Koh K⁸, Yoshida N⁹, Kato K¹⁰, Cho Y¹¹, Kosaka Y¹², Takahashi Y¹³, Inoue M¹⁴, Kato K⁹, Atsuta Y¹⁵, Miyamura K¹⁶; Donor/Source Working Group of Japan Society of Hematopoietic Cell Transplantation.

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the best therapeutic option for childhood high-risk acute leukemia. However, which donor source is optimal for children lacking an identical sibling remains unclear. To evaluate the clinical impact of donor source on allo-HSCT in childhood acute leukemia, we analyzed data from 577 children who underwent allo-HSCT following the myeloablative regimen during the first or second complete remission in 2005-2012, using registry data of the Japan Society for Hematopoietic Cell Transplantation, and compared outcomes of 7-8/8 human leukocyte antigen (HLA) allelic-matched unrelated bone marrow transplantation (UR-BMT, n = 218) and 4-6/6 HLA allelic-matched unrelated cord blood transplantation (UR-CBT, n = 200) to those of HLA identical related bone marrow transplantation (ID-BMT, n = 159). Median follow-up of survivors was 40.0 months. Three-year overall survival and leukemia-free survival rates for ID-BMT, UR-BMT, and UR-CBT were 74.8/69.0%, 75.0/69.6%, and 71.8/63.8%, respectively. The multivariate analysis demonstrated that OS and LFS for the 3 groups are comparable, although UR-CBT carries a greater risk of non-relapse mortality (hazard ratio, 2.20; P = 0.03, as compared to ID-BMT) in the myeloablative setting for childhood high-risk acute leukemia.