Hematopoietic stem cell transplantation for pediatric acute myeloid leukemia patients with KMT2A rearrangement; A nationwide retrospective analysis in Japan

Takako Miyamura, Kazuko Kudo, Ken Tabuchi, Hiroyuki Ishida, Daisuke Tomizawa, Souichi Adachi, Hiroaki Goto, Nao Yoshida, Masami Inoue, Katsuyoshi Koh, Yoji Sasahara, Naoto Fujita, Harumi Kakuda, Maiko Noguchi, Mitsuteru Hiwatari, Yoshiko Hashii, Koji Kato, Yoshiko Atsuta, Yasuhiro Okamoto

Research output: Contribution to journalArticle

Abstract

Objective: Pediatric acute myeloid leukemia (AML) with KMT2A rearrangement is detected in 15–20% of all pediatric AML patients and is associated with adverse outcomes even after allogeneic hematopoietic stem cell transplantation (HSCT). To investigate outcomes and prognostic factors, we investigated 90 pediatric AML patients with KMT2A rearrangement after allogeneic HSCT. Methods: We retrospectively analyzed Japanese registration data for patients who had received allogeneic HSCT between 1988 and 2011. Median age was 3 years (range, 0–15 years), and no gender difference was evident. Median observation period was 119 months. Results: The 3-year overall survival (OS) rate of KMT2A-rearranged AML was 52.1% (95% confidence interval (CI), 42.4–64%, n = 90), and the 3-year disease-free survival (DFS) rate was 46.7% (95%CI, 36.8–58.2%). The 3-year DFS of KMT2A-rearranged AML was not significantly poorer than that of other AML (P = 0.09), and no significant difference was also seen in 3-year OS rate (P = 0.21). Multivariate analysis showed disease status (complete remission) at HSCT was associated with better outcomes. A significant difference in treatment-related mortality (TRM) was apparent between HSCT from a HLA full-matched related donor and that from a haploidentical donor (P = 0.001). Discussion: HSCT is a curative option for pediatric AML with KMT2A rearrangement. Pretransplant status was the most significant prognostic indicator for relapse and survival. Enhancing supportive therapy to reduce TRM will further improve treatment outcomes of KMT2A-rearranged pediatric AML.

Original languageEnglish
Article number106263
JournalLeukemia Research
Volume87
DOIs
Publication statusPublished - Dec 2019

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Hematopoietic Stem Cell Transplantation
Acute Myeloid Leukemia
Japan
Pediatrics
Survival Rate
Disease-Free Survival
Tissue Donors
Confidence Intervals
Mortality
Therapeutics
Multivariate Analysis
Observation
Recurrence
Survival

All Science Journal Classification (ASJC) codes

  • Hematology
  • Oncology
  • Cancer Research

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Hematopoietic stem cell transplantation for pediatric acute myeloid leukemia patients with KMT2A rearrangement; A nationwide retrospective analysis in Japan. / Miyamura, Takako; Kudo, Kazuko; Tabuchi, Ken; Ishida, Hiroyuki; Tomizawa, Daisuke; Adachi, Souichi; Goto, Hiroaki; Yoshida, Nao; Inoue, Masami; Koh, Katsuyoshi; Sasahara, Yoji; Fujita, Naoto; Kakuda, Harumi; Noguchi, Maiko; Hiwatari, Mitsuteru; Hashii, Yoshiko; Kato, Koji; Atsuta, Yoshiko; Okamoto, Yasuhiro.

In: Leukemia Research, Vol. 87, 106263, 12.2019.

Research output: Contribution to journalArticle

Miyamura, T, Kudo, K, Tabuchi, K, Ishida, H, Tomizawa, D, Adachi, S, Goto, H, Yoshida, N, Inoue, M, Koh, K, Sasahara, Y, Fujita, N, Kakuda, H, Noguchi, M, Hiwatari, M, Hashii, Y, Kato, K, Atsuta, Y & Okamoto, Y 2019, 'Hematopoietic stem cell transplantation for pediatric acute myeloid leukemia patients with KMT2A rearrangement; A nationwide retrospective analysis in Japan', Leukemia Research, vol. 87, 106263. https://doi.org/10.1016/j.leukres.2019.106263
Miyamura, Takako ; Kudo, Kazuko ; Tabuchi, Ken ; Ishida, Hiroyuki ; Tomizawa, Daisuke ; Adachi, Souichi ; Goto, Hiroaki ; Yoshida, Nao ; Inoue, Masami ; Koh, Katsuyoshi ; Sasahara, Yoji ; Fujita, Naoto ; Kakuda, Harumi ; Noguchi, Maiko ; Hiwatari, Mitsuteru ; Hashii, Yoshiko ; Kato, Koji ; Atsuta, Yoshiko ; Okamoto, Yasuhiro. / Hematopoietic stem cell transplantation for pediatric acute myeloid leukemia patients with KMT2A rearrangement; A nationwide retrospective analysis in Japan. In: Leukemia Research. 2019 ; Vol. 87.
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abstract = "Objective: Pediatric acute myeloid leukemia (AML) with KMT2A rearrangement is detected in 15–20{\%} of all pediatric AML patients and is associated with adverse outcomes even after allogeneic hematopoietic stem cell transplantation (HSCT). To investigate outcomes and prognostic factors, we investigated 90 pediatric AML patients with KMT2A rearrangement after allogeneic HSCT. Methods: We retrospectively analyzed Japanese registration data for patients who had received allogeneic HSCT between 1988 and 2011. Median age was 3 years (range, 0–15 years), and no gender difference was evident. Median observation period was 119 months. Results: The 3-year overall survival (OS) rate of KMT2A-rearranged AML was 52.1{\%} (95{\%} confidence interval (CI), 42.4–64{\%}, n = 90), and the 3-year disease-free survival (DFS) rate was 46.7{\%} (95{\%}CI, 36.8–58.2{\%}). The 3-year DFS of KMT2A-rearranged AML was not significantly poorer than that of other AML (P = 0.09), and no significant difference was also seen in 3-year OS rate (P = 0.21). Multivariate analysis showed disease status (complete remission) at HSCT was associated with better outcomes. A significant difference in treatment-related mortality (TRM) was apparent between HSCT from a HLA full-matched related donor and that from a haploidentical donor (P = 0.001). Discussion: HSCT is a curative option for pediatric AML with KMT2A rearrangement. Pretransplant status was the most significant prognostic indicator for relapse and survival. Enhancing supportive therapy to reduce TRM will further improve treatment outcomes of KMT2A-rearranged pediatric AML.",
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T1 - Hematopoietic stem cell transplantation for pediatric acute myeloid leukemia patients with KMT2A rearrangement; A nationwide retrospective analysis in Japan

AU - Miyamura, Takako

AU - Kudo, Kazuko

AU - Tabuchi, Ken

AU - Ishida, Hiroyuki

AU - Tomizawa, Daisuke

AU - Adachi, Souichi

AU - Goto, Hiroaki

AU - Yoshida, Nao

AU - Inoue, Masami

AU - Koh, Katsuyoshi

AU - Sasahara, Yoji

AU - Fujita, Naoto

AU - Kakuda, Harumi

AU - Noguchi, Maiko

AU - Hiwatari, Mitsuteru

AU - Hashii, Yoshiko

AU - Kato, Koji

AU - Atsuta, Yoshiko

AU - Okamoto, Yasuhiro

PY - 2019/12

Y1 - 2019/12

N2 - Objective: Pediatric acute myeloid leukemia (AML) with KMT2A rearrangement is detected in 15–20% of all pediatric AML patients and is associated with adverse outcomes even after allogeneic hematopoietic stem cell transplantation (HSCT). To investigate outcomes and prognostic factors, we investigated 90 pediatric AML patients with KMT2A rearrangement after allogeneic HSCT. Methods: We retrospectively analyzed Japanese registration data for patients who had received allogeneic HSCT between 1988 and 2011. Median age was 3 years (range, 0–15 years), and no gender difference was evident. Median observation period was 119 months. Results: The 3-year overall survival (OS) rate of KMT2A-rearranged AML was 52.1% (95% confidence interval (CI), 42.4–64%, n = 90), and the 3-year disease-free survival (DFS) rate was 46.7% (95%CI, 36.8–58.2%). The 3-year DFS of KMT2A-rearranged AML was not significantly poorer than that of other AML (P = 0.09), and no significant difference was also seen in 3-year OS rate (P = 0.21). Multivariate analysis showed disease status (complete remission) at HSCT was associated with better outcomes. A significant difference in treatment-related mortality (TRM) was apparent between HSCT from a HLA full-matched related donor and that from a haploidentical donor (P = 0.001). Discussion: HSCT is a curative option for pediatric AML with KMT2A rearrangement. Pretransplant status was the most significant prognostic indicator for relapse and survival. Enhancing supportive therapy to reduce TRM will further improve treatment outcomes of KMT2A-rearranged pediatric AML.

AB - Objective: Pediatric acute myeloid leukemia (AML) with KMT2A rearrangement is detected in 15–20% of all pediatric AML patients and is associated with adverse outcomes even after allogeneic hematopoietic stem cell transplantation (HSCT). To investigate outcomes and prognostic factors, we investigated 90 pediatric AML patients with KMT2A rearrangement after allogeneic HSCT. Methods: We retrospectively analyzed Japanese registration data for patients who had received allogeneic HSCT between 1988 and 2011. Median age was 3 years (range, 0–15 years), and no gender difference was evident. Median observation period was 119 months. Results: The 3-year overall survival (OS) rate of KMT2A-rearranged AML was 52.1% (95% confidence interval (CI), 42.4–64%, n = 90), and the 3-year disease-free survival (DFS) rate was 46.7% (95%CI, 36.8–58.2%). The 3-year DFS of KMT2A-rearranged AML was not significantly poorer than that of other AML (P = 0.09), and no significant difference was also seen in 3-year OS rate (P = 0.21). Multivariate analysis showed disease status (complete remission) at HSCT was associated with better outcomes. A significant difference in treatment-related mortality (TRM) was apparent between HSCT from a HLA full-matched related donor and that from a haploidentical donor (P = 0.001). Discussion: HSCT is a curative option for pediatric AML with KMT2A rearrangement. Pretransplant status was the most significant prognostic indicator for relapse and survival. Enhancing supportive therapy to reduce TRM will further improve treatment outcomes of KMT2A-rearranged pediatric AML.

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