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

• Published in: Biology of blood and marrow transplantation Vol. 26; no. 1; pp. 139 - 144
• Main Authors: Terakura, Seitaro, Nishida, Tetsuya, Sawa, Masashi, Kato, Tomonori, Miyao, Kotaro, Ozawa, Yukiyasu, Kohno, Akio, Onishi, Yasushi, Fukuhara, Noriko, Kasai, Masanobu, Fujii, Nobuharu, Yokoyama, Hisayuki, Iida, Hiroatsu, Kanemura, Nobuhiro, Fujieda, Atsushi, Ago, Hiroatsu, Tsutsumi, Yutaka, Nakamura, Fumihiko, Yago, Kazuhiro, Moriuchi, Yukiyoshi, Ota, Shuichi, Ohashi, Haruhiko, Yanagisawa, Atsumi, Suzuki, Ritsuro, Kuwatsuka, Yachiyo, Atsuta, Yoshiko, Miyamura, Koichi, Murata, Makoto
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.01.2020
• Subjects: Acute Disease; Adolescent; Adult; Allografts; Chronic Disease; Cord Blood Stem Cell Transplantation; Cord blood transplantation; Disease-Free Survival; Female; Graft vs Host Disease - etiology; Graft vs Host Disease - mortality; Graft vs Host Disease - prevention & control; Humans; Leukemia - mortality; Leukemia - therapy; Male; Middle Aged; Myelodysplastic Syndromes - mortality; Myelodysplastic Syndromes - therapy; Phase 2; Prospective clinical study; Prospective Studies; Single-unit; Survival Rate; Time Factors; Single-unit; Adult; Phase 2; Prospective clinical study; Cord blood transplantation
• ISSN: 1083-8791; 1523-6536
• DOI: 10.1016/j.bbmt.2019.09.021

•We conducted a phase 2 single-arm prospective study of cord blood transplantation (CBT).•We assessed the efficacy of CBT for treating patients with adult acute leukemia and myelodysplastic syndrome.•CBT showed significantly better day 180 overall survival than threshold survival.•CBT outcomes are comparable to historical unrelated donor transplantation in adults with low comorbidity.•Patients with high-risk features in complete response status may benefit from early CBT. 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.