Prospective Phase 2 Study of Umbilical Cord Blood Transplantation in Adult Acute Leukemia and Myelodysplastic Syndrome

Seitaro Terakura, Tetsuya Nishida, Masashi Sawa, Tomonori Kato, Kotaro Miyao, Yukiyasu Ozawa, Akio Kohno, Yasushi Onishi, Noriko Fukuhara, Masanobu Kasai, Nobuharu Fujii, Hisayuki Yokoyama, Hiroatsu Iida, Nobuhiro Kanemura, Atsushi Fujieda, Hiroatsu Ago, Yutaka Tsutsumi, Fumihiko Nakamura, Kazuhiro Yago, Yukiyoshi Moriuchi, Shuichi Ota, Haruhiko Ohashi, Atsumi Yanagisawa, Ritsuro Suzuki, Yachiyo Kuwatsuka, Yoshiko Atsuta, Koichi Miyamura, Makoto Murata
Biology of Blood and Marrow Transplantation 2019 September 20
Almost comparable transplant outcomes were reported between HLA-matched unrelated donor transplantation (UDT) and cord blood transplantation (CBT). Herein, we conducted a prospective phase 2 study to assess efficacy and safety of single-unit myeloablative CBT in adult leukemia and myelodysplastic syndrome. Because day 180 survival of UDT was approximately 80%, we determined the alternative hypothesis of expected day 180 survival with successful engraftment rate as 80% and set the null hypothesis of threshold rate as 65%. Sixty-two patients (median age, 37 yrs) were registered (AML, n=28; ALL, n=25; myelodysplastic syndrome, n=9). Of 61 eligible patients, 52 were successfully engrafted and survived at day 180 (85% [95% confidence interval, 74-93%]). Single-unit CBT was judged to be effective because the null hypothesis was rejected (p<0.001). Furthermore, neutrophil engraftment was observed in 57 (92%) patients; incidences of grades II-IV acute and chronic graft-versus-host disease were 30% and 32%, respectively; and cumulative incidences of non-relapse mortality and relapse at 2 yr were 18% and 13%, respectively. The present study showed favorable survival outcomes with single-unit CBT. Therefore, this method may be considered if HLA well-matched UDT cannot be obtained. This trial was registered at UMIN-CTR as #UMIN000000600 (

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