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

Nagoya Blood and Marrow Transplantation Group

Research output: Contribution to journalArticle

Abstract

Almost comparable transplantation outcomes have been reported with HLA-matched unrelated donor transplantation (UDT) and cord blood transplantation (CBT). We conducted a prospective phase 2 study to assess the efficacy and safety of single-unit myeloablative CBT in adult leukemia and myelodysplastic syndrome. Because the day 180 survival of UDT was approximately 80%, we determined the alternative hypothesis of expected day 180 survival with a successful engraftment rate of 80% and set the null hypothesis of threshold rate at 65%. Sixty-two patients (median age, 37 years) were registered, including 28 with acute myelogenous leukemia, 25 with acute lymphoblastic leukemia, and 9 with myelodysplastic syndrome. Of 61 eligible patients, 52 were successfully engrafted and survived at day 180 (85%; 95% confidence interval, 74% to 93%). Single-unit CBT was judged to be effective because the null hypothesis was rejected (P <.001). Furthermore, neutrophil engraftment was observed in 57 patients (92%); the incidences of grade II-IV acute and chronic graft-versus-host disease were 30% and 32%, respectively; and the cumulative incidences of nonrelapse mortality and relapse at 2 years were 18% and 13%, respectively. The present study showed favorable survival outcomes with single-unit CBT. Therefore, this method may be considered if a well-HLA-matched UDT cannot be obtained.

Original languageEnglish
JournalBiology of Blood and Marrow Transplantation
DOIs
Publication statusAccepted/In press - Jan 1 2019

Fingerprint

Myelodysplastic Syndromes
Fetal Blood
Leukemia
Transplantation
Unrelated Donors
Survival
Incidence
Graft vs Host Disease
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Acute Myeloid Leukemia
Neutrophils
Confidence Intervals
Safety
Recurrence
Mortality

Keywords

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

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

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

In: Biology of Blood and Marrow Transplantation, 01.01.2019.

Research output: Contribution to journalArticle

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abstract = "Almost comparable transplantation outcomes have been reported with HLA-matched unrelated donor transplantation (UDT) and cord blood transplantation (CBT). We conducted a prospective phase 2 study to assess the efficacy and safety of single-unit myeloablative CBT in adult leukemia and myelodysplastic syndrome. Because the day 180 survival of UDT was approximately 80{\%}, we determined the alternative hypothesis of expected day 180 survival with a successful engraftment rate of 80{\%} and set the null hypothesis of threshold rate at 65{\%}. Sixty-two patients (median age, 37 years) were registered, including 28 with acute myelogenous leukemia, 25 with acute lymphoblastic leukemia, and 9 with myelodysplastic syndrome. Of 61 eligible patients, 52 were successfully engrafted and survived at day 180 (85{\%}; 95{\%} confidence interval, 74{\%} to 93{\%}). Single-unit CBT was judged to be effective because the null hypothesis was rejected (P <.001). Furthermore, neutrophil engraftment was observed in 57 patients (92{\%}); the incidences of grade II-IV acute and chronic graft-versus-host disease were 30{\%} and 32{\%}, respectively; and the cumulative incidences of nonrelapse mortality and relapse at 2 years were 18{\%} and 13{\%}, respectively. The present study showed favorable survival outcomes with single-unit CBT. Therefore, this method may be considered if a well-HLA-matched UDT cannot be obtained.",
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AU - Nagoya Blood and Marrow Transplantation Group

AU - Terakura, Seitaro

AU - Nishida, Tetsuya

AU - Sawa, Masashi

AU - Kato, Tomonori

AU - Miyao, Kotaro

AU - Ozawa, Yukiyasu

AU - Kohno, Akio

AU - Onishi, Yasushi

AU - Fukuhara, Noriko

AU - Kasai, Masanobu

AU - Fujii, Nobuharu

AU - Yokoyama, Hisayuki

AU - Iida, Hiroatsu

AU - Kanemura, Nobuhiro

AU - Fujieda, Atsushi

AU - Ago, Hiroatsu

AU - Tsutsumi, Yutaka

AU - Nakamura, Fumihiko

AU - Yago, Kazuhiro

AU - Moriuchi, Yukiyoshi

AU - Ota, Shuichi

AU - Ohashi, Haruhiko

AU - Yanagisawa, Atsumi

AU - Suzuki, Ritsuro

AU - Kuwatsuka, Yachiyo

AU - Atsuta, Yoshiko

AU - Miyamura, Koichi

AU - Murata, Makoto

PY - 2019/1/1

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AB - Almost comparable transplantation outcomes have been reported with HLA-matched unrelated donor transplantation (UDT) and cord blood transplantation (CBT). We conducted a prospective phase 2 study to assess the efficacy and safety of single-unit myeloablative CBT in adult leukemia and myelodysplastic syndrome. Because the day 180 survival of UDT was approximately 80%, we determined the alternative hypothesis of expected day 180 survival with a successful engraftment rate of 80% and set the null hypothesis of threshold rate at 65%. Sixty-two patients (median age, 37 years) were registered, including 28 with acute myelogenous leukemia, 25 with acute lymphoblastic leukemia, and 9 with myelodysplastic syndrome. Of 61 eligible patients, 52 were successfully engrafted and survived at day 180 (85%; 95% confidence interval, 74% to 93%). Single-unit CBT was judged to be effective because the null hypothesis was rejected (P <.001). Furthermore, neutrophil engraftment was observed in 57 patients (92%); the incidences of grade II-IV acute and chronic graft-versus-host disease were 30% and 32%, respectively; and the cumulative incidences of nonrelapse mortality and relapse at 2 years were 18% and 13%, respectively. The present study showed favorable survival outcomes with single-unit CBT. Therefore, this method may be considered if a well-HLA-matched UDT cannot be obtained.

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KW - Prospective clinical study

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