Outcome of non-T-cell-depleted HLA-haploidentical hematopoietic stem cell transplantation from family donors in children and adolescents

Takao Yoshihara, Keiko Okada, Michihiro Kobayashi, Atsushi Kikuta, Koji Kato, Naoto Adachi, Akira Kikuchi, Hiroyuki Ishida, Yasuzou Hirota, Hiroshi Kuroda, Yoshihisa Nagatoshi, Takeshi Inukai, Kazutoshi Koike, Hisato Kigasawa, Hiroshi Yagasaki, Kiriko Tokuda, Tomoko Kishimoto, Takahide Nakano, Naoto Fujita, Hiroaki Goto, Yozo Nakazawa, Hirokazu Kanegane, Akinobu Matsuzaki, Yuko Osugi, Daiichiro Hasegawa, Nobuhiko Uoshima, Kazuhiro Nakamura, Masahiro Tsuchida, Ryuhei Tanaka, Arata Watanabe, Hiromasa Yabe
International Journal of Hematology 2007, 85 (3): 246-55
Non-T-cell-depleted HLA-haploidentical hematopoietic stem cell transplantation (SCT) from family members has been reported, but its effectiveness and safety are not fully known. In this study, we examined the outcomes of 83 children and adolescents with nonmalignant (n = 11) or malignant (n = 72) disorders who underwent SCT mismatched at 2 or 3 HLA loci, either from the mother (n = 56), a noninherited maternal antigen (NIMA)-mismatched sibling (n = 14), or the father/a noninherited paternal antigen (NIPA)-mismatched sibling (n = 13). Engraftment was satisfactory. Severe (grade III-IV) acute graft-versushost disease (GVHD) was noted only in malignant disease, with an incidence of 21 of 64 evaluable patients. GVHD prophylaxis with a combination of tacrolimus and methotrexate was significantly associated with a lower risk of severe acute GVHD, compared with other types of prophylaxis (P = .04). Nine of 11 patients with nonmalignant disease and 29 of 72 patients with malignant disease were alive at a median follow-up of 26 months (range, 4-57 months). Outcomes were not significantly different among the 3 donor groups (mother versus NIMA-mismatched sibling versus father/NIPA-mismatched sibling) for the malignancy disorders. Our results indicate that non-T-cell-depleted HLA-haploidentical SCT may be feasible, with appropriate GVHD prophylaxis, for young recipients who lack immediate access to a conventional stem cell source.

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