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Hematopoietic Stem Cell Transplantation Based on Minimal Residual Disease with a Unified Conditioning Regimen Comprising Total Body Irradiation, Etoposide and Cyclophosphamide: Results from the JPLSG ALL-R08-II Trial, the First Nationwide Prospective Study for Children with Intermediate-Risk Relapsed Acute Lymphoblastic Leukemia in Japan
  • +17
  • Hidemi Toyoda,
  • Hideaki Ueki,
  • Chitose Ogawa,
  • Hiroaki Goto,
  • Masanori Nishi,
  • Junko Yamanaka,
  • Shinji Mochizuki,
  • Takuro Nishikawa,
  • Tadashi Kumamoto,
  • Ritsuo Nishiuchi,
  • Atsushi Kikuta,
  • Shohei Yamamoto,
  • Shunji Igarashi,
  • Atsushi Sato,
  • Toshinori Hori,
  • Akiko Saito,
  • Tomoyuki Watanabe,
  • Takao Deguchi,
  • Atsushi Manabe,
  • Keizo Horibe
Hidemi Toyoda
Mie Daigaku

Corresponding Author:htoyoda@med.mie-u.ac.jp

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Hideaki Ueki
Narita Sekijuji Byoin
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Chitose Ogawa
Kokuritsu Gan Kenkyu Center
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Hiroaki Goto
Kanagawa Kenritsu Kodomo Iryo Center
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Masanori Nishi
Saga Daigaku Igakubu Daigakuin Igakukei Kenkyuka Shonika
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Junko Yamanaka
Kokuritsu Kokusai Iryo Kenkyu Center Byoin
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Shinji Mochizuki
Kokuritsu Kokusai Iryo Kenkyu Center Byoin
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Takuro Nishikawa
Kagoshima Daigaku Igakubu
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Tadashi Kumamoto
Kokuritsu Gan Kenkyu Center
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Ritsuo Nishiuchi
Kochi Health Sciences Centre
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Atsushi Kikuta
Fukushima Kenritsu Ika Daigaku
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Shohei Yamamoto
Tokai Daigaku - Isehara Campus
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Shunji Igarashi
Narita Sekijuji Byoin
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Atsushi Sato
Miyagi Kenritsu Kodomo Byoin
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Toshinori Hori
Aichi Ika Daigaku
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Akiko Saito
Kokuritsu Byoin Kiko Nagoya Iryo Center
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Tomoyuki Watanabe
Aichi Gakuin Daigaku
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Takao Deguchi
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center
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Atsushi Manabe
Hokkaido Daigaku
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Keizo Horibe
Kokuritsu Byoin Kiko Nagoya Iryo Center
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Abstract

Background: In children with intermediate-risk relapsed acute lymphoblastic leukemia (ALL), allogeneic hematopoietic stem cell transplantation (allo-HSCT) has markedly improved the outcome of patients with poor minimal residual disease (MRD) response. However, there is no consensus on the optimal conditioning regimen for allo-HSCT. Procedure: We prospectively analyzed the efficacy and safety of allo-HSCT with a unified conditioning regimen for children with intermediate-risk relapsed ALL, based on MRD in the bone marrow after induction, in the Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG) ALL-R08-II nationwide cohort. The conditioning regimen for allo-HSCT comprised total body irradiation (TBI), etoposide (ETP) and cyclophosphamide (CY) (UMIN000002025). Results: Twenty patients with post-induction MRD ≥ 10 −3 and two with MRD that could not be evaluated underwent allo-HSCT. Engraftment was confirmed in all patients. No transplantation-related mortality was observed. The 3-year event-free survival and overall survival after transplantation were 86.4% ± 7.3% and 95.5% ± 4.4%, respectively. Conclusion: Allo-HSCT based on post-induction MRD with TBI + ETP + CY conditioning was highly effective and feasible for Japanese children with intermediate-risk relapsed ALL.