Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents

Hiroyuki Ishida, Souichi Adachi, Daiichiro Hasegawa, Yasuhiro Okamoto, Hiroaki Goto, Jiro Inagaki, Masami Inoue, Katsuyoshi Koh, Hiromasa Yabe, Keisei Kawa, Koji Kato, Yoshiko Atsuta, Kazuko Kudo

    研究成果: ジャーナルへの寄稿記事

    7 引用 (Scopus)

    抄録

    The relative efficacy of allogeneic hematopoietic cell transplantation (allo-HCT) after reduced toxicity conditioning (RTC) compared with standard myeloablative conditioning (MAC) in pediatric patients with acute myeloid leukemia (AML) has not been studied extensively. To address whether RTC is a feasible approach for pediatric patients with AML in remission, we performed a retrospective investigation of the outcomes of the first transplant in patients who had received an allo-HCT after RTC or standard MAC, using nationwide registration data collected between 2000 and 2011 in Japan. Procedure: We compared a fludarabine (Flu) and melphalan (Mel)-based regimen (RTC; n=34) with total body irradiation (TBI) and/or busulfan (Bu)-based conditioning (MAC; n=102) in demographic- and disease-criteria-matched childhood and adolescent patients with AML in first or second complete remission (CR1/CR2). Results: The incidence of engraftment, early complications, grade II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were similar in each conditioning group. The risk of relapse (25% vs. 26%) and non-relapse mortality (13% vs. 11%) after 3 years did not differ between these groups, and univariate and multivariate analyses demonstrated that the 3-year overall survival (OS) rates after Flu/Mel-RTC and MAC were comparable (mean, 72% [range, 51-85%] and 68% [range, 58-77%], respectively). Conclusions: The results suggest that the Flu/Mel-RTC regimen is a clinically acceptable conditioning strategy for childhood and adolescent patients with AML in remission. Although this retrospective, registry-based analysis has several limitations, RTC deserves to be further investigated in prospective trials. Pediatr Blood Cancer 2015;62:883-889.

    元の言語英語
    ページ(範囲)883-889
    ページ数7
    ジャーナルPediatric Blood and Cancer
    62
    発行部数5
    DOI
    出版物ステータス出版済み - 5 1 2015

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    Busulfan
    Melphalan
    Acute Myeloid Leukemia
    Radiation
    Cell Transplantation
    Graft vs Host Disease
    Pediatrics
    Whole-Body Irradiation
    Registries
    Japan
    Multivariate Analysis
    Survival Rate
    Demography
    fludarabine
    Transplants
    Recurrence
    Mortality
    Incidence
    Neoplasms

    All Science Journal Classification (ASJC) codes

    • Pediatrics, Perinatology, and Child Health
    • Hematology
    • Oncology

    これを引用

    Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents. / Ishida, Hiroyuki; Adachi, Souichi; Hasegawa, Daiichiro; Okamoto, Yasuhiro; Goto, Hiroaki; Inagaki, Jiro; Inoue, Masami; Koh, Katsuyoshi; Yabe, Hiromasa; Kawa, Keisei; Kato, Koji; Atsuta, Yoshiko; Kudo, Kazuko.

    :: Pediatric Blood and Cancer, 巻 62, 番号 5, 01.05.2015, p. 883-889.

    研究成果: ジャーナルへの寄稿記事

    Ishida, Hiroyuki ; Adachi, Souichi ; Hasegawa, Daiichiro ; Okamoto, Yasuhiro ; Goto, Hiroaki ; Inagaki, Jiro ; Inoue, Masami ; Koh, Katsuyoshi ; Yabe, Hiromasa ; Kawa, Keisei ; Kato, Koji ; Atsuta, Yoshiko ; Kudo, Kazuko. / Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents. :: Pediatric Blood and Cancer. 2015 ; 巻 62, 番号 5. pp. 883-889.
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    title = "Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents",
    abstract = "The relative efficacy of allogeneic hematopoietic cell transplantation (allo-HCT) after reduced toxicity conditioning (RTC) compared with standard myeloablative conditioning (MAC) in pediatric patients with acute myeloid leukemia (AML) has not been studied extensively. To address whether RTC is a feasible approach for pediatric patients with AML in remission, we performed a retrospective investigation of the outcomes of the first transplant in patients who had received an allo-HCT after RTC or standard MAC, using nationwide registration data collected between 2000 and 2011 in Japan. Procedure: We compared a fludarabine (Flu) and melphalan (Mel)-based regimen (RTC; n=34) with total body irradiation (TBI) and/or busulfan (Bu)-based conditioning (MAC; n=102) in demographic- and disease-criteria-matched childhood and adolescent patients with AML in first or second complete remission (CR1/CR2). Results: The incidence of engraftment, early complications, grade II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were similar in each conditioning group. The risk of relapse (25{\%} vs. 26{\%}) and non-relapse mortality (13{\%} vs. 11{\%}) after 3 years did not differ between these groups, and univariate and multivariate analyses demonstrated that the 3-year overall survival (OS) rates after Flu/Mel-RTC and MAC were comparable (mean, 72{\%} [range, 51-85{\%}] and 68{\%} [range, 58-77{\%}], respectively). Conclusions: The results suggest that the Flu/Mel-RTC regimen is a clinically acceptable conditioning strategy for childhood and adolescent patients with AML in remission. Although this retrospective, registry-based analysis has several limitations, RTC deserves to be further investigated in prospective trials. Pediatr Blood Cancer 2015;62:883-889.",
    author = "Hiroyuki Ishida and Souichi Adachi and Daiichiro Hasegawa and Yasuhiro Okamoto and Hiroaki Goto and Jiro Inagaki and Masami Inoue and Katsuyoshi Koh and Hiromasa Yabe and Keisei Kawa and Koji Kato and Yoshiko Atsuta and Kazuko Kudo",
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    T1 - Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents

    AU - Ishida, Hiroyuki

    AU - Adachi, Souichi

    AU - Hasegawa, Daiichiro

    AU - Okamoto, Yasuhiro

    AU - Goto, Hiroaki

    AU - Inagaki, Jiro

    AU - Inoue, Masami

    AU - Koh, Katsuyoshi

    AU - Yabe, Hiromasa

    AU - Kawa, Keisei

    AU - Kato, Koji

    AU - Atsuta, Yoshiko

    AU - Kudo, Kazuko

    PY - 2015/5/1

    Y1 - 2015/5/1

    N2 - The relative efficacy of allogeneic hematopoietic cell transplantation (allo-HCT) after reduced toxicity conditioning (RTC) compared with standard myeloablative conditioning (MAC) in pediatric patients with acute myeloid leukemia (AML) has not been studied extensively. To address whether RTC is a feasible approach for pediatric patients with AML in remission, we performed a retrospective investigation of the outcomes of the first transplant in patients who had received an allo-HCT after RTC or standard MAC, using nationwide registration data collected between 2000 and 2011 in Japan. Procedure: We compared a fludarabine (Flu) and melphalan (Mel)-based regimen (RTC; n=34) with total body irradiation (TBI) and/or busulfan (Bu)-based conditioning (MAC; n=102) in demographic- and disease-criteria-matched childhood and adolescent patients with AML in first or second complete remission (CR1/CR2). Results: The incidence of engraftment, early complications, grade II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were similar in each conditioning group. The risk of relapse (25% vs. 26%) and non-relapse mortality (13% vs. 11%) after 3 years did not differ between these groups, and univariate and multivariate analyses demonstrated that the 3-year overall survival (OS) rates after Flu/Mel-RTC and MAC were comparable (mean, 72% [range, 51-85%] and 68% [range, 58-77%], respectively). Conclusions: The results suggest that the Flu/Mel-RTC regimen is a clinically acceptable conditioning strategy for childhood and adolescent patients with AML in remission. Although this retrospective, registry-based analysis has several limitations, RTC deserves to be further investigated in prospective trials. Pediatr Blood Cancer 2015;62:883-889.

    AB - The relative efficacy of allogeneic hematopoietic cell transplantation (allo-HCT) after reduced toxicity conditioning (RTC) compared with standard myeloablative conditioning (MAC) in pediatric patients with acute myeloid leukemia (AML) has not been studied extensively. To address whether RTC is a feasible approach for pediatric patients with AML in remission, we performed a retrospective investigation of the outcomes of the first transplant in patients who had received an allo-HCT after RTC or standard MAC, using nationwide registration data collected between 2000 and 2011 in Japan. Procedure: We compared a fludarabine (Flu) and melphalan (Mel)-based regimen (RTC; n=34) with total body irradiation (TBI) and/or busulfan (Bu)-based conditioning (MAC; n=102) in demographic- and disease-criteria-matched childhood and adolescent patients with AML in first or second complete remission (CR1/CR2). Results: The incidence of engraftment, early complications, grade II-IV acute graft-versus-host disease (GVHD), and chronic GVHD were similar in each conditioning group. The risk of relapse (25% vs. 26%) and non-relapse mortality (13% vs. 11%) after 3 years did not differ between these groups, and univariate and multivariate analyses demonstrated that the 3-year overall survival (OS) rates after Flu/Mel-RTC and MAC were comparable (mean, 72% [range, 51-85%] and 68% [range, 58-77%], respectively). Conclusions: The results suggest that the Flu/Mel-RTC regimen is a clinically acceptable conditioning strategy for childhood and adolescent patients with AML in remission. Although this retrospective, registry-based analysis has several limitations, RTC deserves to be further investigated in prospective trials. Pediatr Blood Cancer 2015;62:883-889.

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