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Biol Blood Marrow Transplant. 2019 Sep 20. pii: S1083-8791(19)30632-9. doi: 10.1016/j.bbmt.2019.09.021. [Epub ahead of print]

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

Author information

1
Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan. Electronic address: tseit@med.nagoya-u.ac.jp.
2
Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan.
3
Department of Hematology and Oncology, Anjo Kosei Hospital, Anjo, Japan.
4
Department of Hematology, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
5
Department of Hematology and Oncology, JA Aichi Konan Kosei Hospital, Konan, Japan.
6
Department of Hematology and Rheumatology, Tohoku University Hospital, Sendai, Japan.
7
Department of Hematology and Oncology, Japanese Red Cross Nagoya Daini Hospital, Nagoya, Japan.
8
Department of Hematology and Oncology, Okayama University Graduate School of Medicine, Okayama, Japan.
9
Department of Hematology, National Hospital Organization, Sendai Medical Center, Sendai, Japan.
10
Department of Hematology, Meitetsu Hospital, Nagoya, Japan; Department of Hematology, National Hospital Organization Nagoya Medical Center, Nagoya, Japan.
11
Department of Hematology, Gifu University Hospital, Gifu, Japan.
12
Department of Hematology, Mie University Hospital, Tsu, Japan.
13
Department of Hematology and Oncology, Shimane Prefectural Central Hospital, Izumo, Japan.
14
Department of Hematology, Hakodate Municipal Hospital, Hakodate, Japan.
15
Department of Hematology, National Center for Global Health and Medicine, Tokyo, Japan.
16
Department of Hematology, Shizuoka General Hospital, Shizuoka, Japan.
17
Department of Hematology, Sasebo City General Hospital, Sasebo, Japan.
18
Department of Hematology, Sapporo Hokuyu Hospital, Sapporo, Japan.
19
Department of Hematology, National Hospital Organization Nagoya Medical Center, Nagoya, Japan.
20
Japanese Data Center for Hematopoietic Cell Transplantation, Nagoya, Japan.
21
Department of Oncology and Hematology, Shimane University Hospital, Izumo, Japan.
22
Japanese Data Center for Hematopoietic Cell Transplantation, Nagoya, Japan; Department of Advanced Medicine, Nagoya University Hospital, Nagoya, Japan.

Abstract

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 (https://upload.umin.ac.jp/cgi-bin/ctr_e/ctr_view.cgi?recptno=R000000710).

KEYWORDS:

Adult; Cord blood transplantation; Phase 2; Prospective clinical study; Single-unit

PMID:
31546004
DOI:
10.1016/j.bbmt.2019.09.021

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