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Successful treatment of young childhood standard-risk hepatoblastoma with cisplatin monotherapy using central review system
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  • Isamu Saeki,
  • Kohmei Ida,
  • Sho Kurihara,
  • Kenichiro Watanabe,
  • Makiko Mori,
  • Tomoro Hishiki,
  • Akiko Yokoi,
  • Junya Fujimura,
  • Shohei Honda,
  • Yuki Aoki,
  • Tomoko Iehara,
  • Takuro Kazama,
  • Masahiro Sekiguchi,
  • Norihiko Kitagawa,
  • Risa Matsumura,
  • Motonari Nomura,
  • Yohei Yamada,
  • Ryo Hanaki,
  • Hide Kaneda,
  • Yuichi Takama,
  • Takeshi Inouse,
  • Yukichi Tanaka,
  • Osamu Miyazaki,
  • Hiroki Nagase,
  • Tetsuya Takimoto,
  • Kenichi Yoshimura,
  • Eiso Hiyama
Isamu Saeki
Hiroshima Daigaku
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Kohmei Ida
Teikyo Daigaku Igakubu Fuzoku Mizonokuchi Byoin
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Sho Kurihara
Hiroshima Daigaku
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Kenichiro Watanabe
Shizuoka Children’s Hospital
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Makiko Mori
Saitama Children’s Medical Center
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Tomoro Hishiki
Chiba Daigaku Daigakuin Igaku Kenkyuin Igakubu
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Akiko Yokoi
Kobe Children’s Hospital
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Junya Fujimura
Juntendo University School of Medicine
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Shohei Honda
Hokkaido Daigaku Daigakuin Igaku Kenkyuin Shokaki Gekagaku Kyoshitsu I
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Yuki Aoki
Kokuritsu Gan Center Higashi Byoin
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Tomoko Iehara
Kyoto Daigaku Daigakuin Igaku Kenkyuka Igakubu Hattatsu Shoni Kagaku
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Takuro Kazama
Tohoku Daigaku Byoin
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Masahiro Sekiguchi
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Kenkyujo
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Norihiko Kitagawa
Kanagawa Kenritsu Gan Center Rinsho Kenkyujo
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Risa Matsumura
Hiroshima Daigaku Daigakuin Ikei Kagaku Kenkyuka Shoni Kagaku
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Motonari Nomura
Osaka University
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Yohei Yamada
Keio University School of Medicine
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Ryo Hanaki
Mie University Hospital
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Hide Kaneda
Nihon University School of Medicine
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Yuichi Takama
Osaka Shiritsu Sogo Iryo Center
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Takeshi Inouse
Osaka Shiritsu Sogo Iryo Center
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Yukichi Tanaka
Kanagawa Children’s Medical Center
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Osamu Miyazaki
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Kenkyujo
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Hiroki Nagase
Juntendo University
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Tetsuya Takimoto
Kokuritsu Kenkyu Kaihatsu Hojin Kokuritsu Seiiku Iryo Kenkyu Center Kenkyujo
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Kenichi Yoshimura
Hiroshima Daigaku Byoin
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Eiso Hiyama
Hiroshima Daigaku

Corresponding Author:eiso@hiroshima-u.ac.jp

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Abstract

Background: The JPLT3-S (Japanese study group for Pediatric Liver Tumors) 3 study, conducted cisplatin (CDDP) monotherapy for young children (< 3 years old) with standard risk hepatoblastoma (HB) evaluated central review system in Japan. In the previous JPLT2 study, cases with resectable tumors without any annotation factors in the PRETEXT classification (standard risk HB) showed favorable outcomes by the therapies consisted of CDDP and pirarubicin, but showed toxicities and late complications. In this JPLT3-S trial, less intensity regimen consisted of CDDP alone were evaluated in the young children (< 3 years old) with standard risk HB. Methods: Patients who were less than three years of age, who had PRETEXT I, II, or III HB without any annotation factors (e.g., E1, E1a, E2, E2a, H1, N1, P2, P2a, V3, and V3a) were eligible for inclusion in this study. In this trial, we introduced central radiological and pathological reviews of all patients. The primary outcome was 3-year progression-free survival (PFS). Results: A total of 38 patients (23 female) were included. The median patient age was 12 (range, 2-34) months. Two patients discontinued treatment because of progressive disease, and five patients discontinued treatment for other reasons. The 3- year PFS rate was 93.9% (95% confidence interval [CI], 86.4 to 100). All 38 patients survived (follow-up period 38-98 months), and the OS rate was 100% (confidence interval, 100). There were no cases with late complication without ototoxicity. Conclusion: CDDP monotherapy regimen is feasible in young patients with localized HB classified by central review.
Submitted to Pediatric Blood & Cancer
28 May 2024Assigned to Editor
28 May 2024Submission Checks Completed
16 Jun 2024Review(s) Completed, Editorial Evaluation Pending
29 Jun 2024Assigned to Editor
29 Jun 2024Submission Checks Completed
29 Jun 20241st Revision Received
02 Jul 2024Review(s) Completed, Editorial Evaluation Pending
06 Jul 2024Reviewer(s) Assigned
28 Jul 2024Editorial Decision: Accept