Preserved High Probability of Overall Survival with Significant Reduction of Chemotherapy for Myeloid Leukemia in Down Syndrome

A Nationwide Prospective Study in Japan

Takashi Taga, Tomoyuki Watanabe, Daisuke Tomizawa, Kazuko Kudo, Kiminori Terui, Hiroshi Moritake, Akitoshi Kinoshita, Shotaro Iwamoto, Hideki Nakayama, Hiroyuki Takahashi, Akira Shimada, Tomohiko Taki, Tsutomu Toki, Etsuro Ito, Hiroaki Goto, Katsuyoshi Koh, Akiko M. Saito, Keizo Horibe, Tatsutoshi Nakahata, Akio Tawa & 1 others Souichi Adachi

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

Background: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. Procedure: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. Results: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3% ± 4.4% and 87.5% ± 3.9 %, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). Conclusions: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.

Original languageEnglish
Pages (from-to)248-254
Number of pages7
JournalPediatric Blood and Cancer
Volume63
Issue number2
DOIs
Publication statusPublished - Feb 1 2016

Fingerprint

Myeloid Leukemia
Down Syndrome
Japan
Prospective Studies
Drug Therapy
Survival
Cytarabine
Therapeutics
Induction Chemotherapy
Etoposide
Disease-Free Survival
Lymphoma
Sepsis
Leukemia
Survival Rate
Pediatrics
Recurrence

Keywords

  • acute myeloid leukemia
  • clinical trial
  • Down syndrome

ASJC Scopus subject areas

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

Cite this

Preserved High Probability of Overall Survival with Significant Reduction of Chemotherapy for Myeloid Leukemia in Down Syndrome : A Nationwide Prospective Study in Japan. / Taga, Takashi; Watanabe, Tomoyuki; Tomizawa, Daisuke; Kudo, Kazuko; Terui, Kiminori; Moritake, Hiroshi; Kinoshita, Akitoshi; Iwamoto, Shotaro; Nakayama, Hideki; Takahashi, Hiroyuki; Shimada, Akira; Taki, Tomohiko; Toki, Tsutomu; Ito, Etsuro; Goto, Hiroaki; Koh, Katsuyoshi; Saito, Akiko M.; Horibe, Keizo; Nakahata, Tatsutoshi; Tawa, Akio; Adachi, Souichi.

In: Pediatric Blood and Cancer, Vol. 63, No. 2, 01.02.2016, p. 248-254.

Research output: Contribution to journalArticle

Taga, T, Watanabe, T, Tomizawa, D, Kudo, K, Terui, K, Moritake, H, Kinoshita, A, Iwamoto, S, Nakayama, H, Takahashi, H, Shimada, A, Taki, T, Toki, T, Ito, E, Goto, H, Koh, K, Saito, AM, Horibe, K, Nakahata, T, Tawa, A & Adachi, S 2016, 'Preserved High Probability of Overall Survival with Significant Reduction of Chemotherapy for Myeloid Leukemia in Down Syndrome: A Nationwide Prospective Study in Japan', Pediatric Blood and Cancer, vol. 63, no. 2, pp. 248-254. https://doi.org/10.1002/pbc.25789
Taga, Takashi ; Watanabe, Tomoyuki ; Tomizawa, Daisuke ; Kudo, Kazuko ; Terui, Kiminori ; Moritake, Hiroshi ; Kinoshita, Akitoshi ; Iwamoto, Shotaro ; Nakayama, Hideki ; Takahashi, Hiroyuki ; Shimada, Akira ; Taki, Tomohiko ; Toki, Tsutomu ; Ito, Etsuro ; Goto, Hiroaki ; Koh, Katsuyoshi ; Saito, Akiko M. ; Horibe, Keizo ; Nakahata, Tatsutoshi ; Tawa, Akio ; Adachi, Souichi. / Preserved High Probability of Overall Survival with Significant Reduction of Chemotherapy for Myeloid Leukemia in Down Syndrome : A Nationwide Prospective Study in Japan. In: Pediatric Blood and Cancer. 2016 ; Vol. 63, No. 2. pp. 248-254.
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abstract = "Background: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. Procedure: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. Results: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3{\%} ± 4.4{\%} and 87.5{\%} ± 3.9 {\%}, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). Conclusions: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.",
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T2 - A Nationwide Prospective Study in Japan

AU - Taga, Takashi

AU - Watanabe, Tomoyuki

AU - Tomizawa, Daisuke

AU - Kudo, Kazuko

AU - Terui, Kiminori

AU - Moritake, Hiroshi

AU - Kinoshita, Akitoshi

AU - Iwamoto, Shotaro

AU - Nakayama, Hideki

AU - Takahashi, Hiroyuki

AU - Shimada, Akira

AU - Taki, Tomohiko

AU - Toki, Tsutomu

AU - Ito, Etsuro

AU - Goto, Hiroaki

AU - Koh, Katsuyoshi

AU - Saito, Akiko M.

AU - Horibe, Keizo

AU - Nakahata, Tatsutoshi

AU - Tawa, Akio

AU - Adachi, Souichi

PY - 2016/2/1

Y1 - 2016/2/1

N2 - Background: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. Procedure: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. Results: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3% ± 4.4% and 87.5% ± 3.9 %, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). Conclusions: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.

AB - Background: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. Procedure: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. Results: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3% ± 4.4% and 87.5% ± 3.9 %, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). Conclusions: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.

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