Clinical characteristics, treatment, and survival outcome in pediatric patients with atypical teratoid/rhabdoid tumors: a retrospective study by the Japan Children’s Cancer Group

Kai Yamasaki MD1, Chikako Kiyotani MD, PhD2, Keita Terashima MD, PhD2, Yuko Watanabe MD, PhD3, Masayuki Kanamori MD, PhD4, Yuhki Koga MD, PhD5, Nobuhiro Hata MD, PhD6, Fuminori Iwasaki MD, PhD7, Hiroaki Goto MD, PhD7, Katsuyoshi Koh MD, PhD8, Jun Kurihara MD, PhD9, Shinya Tokunaga MD10, Yoshiki Arakawa MD, PhD10, Daiichiro Hasegawa MD, PhD11, Yoshiyuki Kosaka MD, PhD11, and Junichi Hara MD, PhD1
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  • 1 Department of Pediatric Hematology and Oncology, Osaka City General Hospital, Osaka;
  • | 2 Division of Pediatric Oncology, National Center for Child Health and Development, Tokyo;
  • | 3 Departments of Pediatrics and
  • | 4 Neurosurgery, Tohoku University School of Medicine, Sendai;
  • | 5 Departments of Pediatrics and
  • | 6 Neurosurgery, Kyushu University, Fukuoka;
  • | 7 Department of Hematology and Oncology, Kanagawa Children’s Medical Center, Kanagawa;
  • | 8 Departments of Hematology/Oncology and
  • | 9 Neurosurgery, Saitama Children’s Medical Center, Saitama;
  • | 10 Department of Neurosurgery, Kyoto University Graduate School of Medicine, Kyoto; and
  • | 11 Department of Hematology and Oncology, Kobe Children’s Hospital, Kobe, Japan
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The prognosis of atypical teratoid/rhabdoid tumors (ATRTs) has improved in recent years with the use of multimodal therapy, mainly in cases not involving metastatic disease. The authors wanted to obtain historical control data and evaluate the suitable treatments in Japanese children with ATRTs that were proven negative for INI-1 immunostaining.


The authors retrospectively collected clinical information on 38 pediatric patients with ATRTs treated from 2005 to 2016 and analyzed the data for this series.


The median age of the patient population was 1.3 years, and the male/female ratio was approximately 2:1. Twenty-three patients (60.5%) had metastases. The effects of treatment on prognosis were analyzed for 34 patients after exclusion of 4 patients who could not receive curative treatment. At a median follow-up of 40.9 months, the mean (± SD) progression-free survival (PFS) and overall survival (OS) were 66.6% ± 8.3% and 45.9% ± 8.7% at 2 years and 44.2% ± 9.9% and 34.2% ± 8.9% at 5 years, respectively. The metastasis stage at diagnosis (M0–1 vs M2–4) (HR 2.68, 95% CI 1.08–6.65; p = 0.0338) and gross tumor resection (yes vs no) (HR 3.49, 95% CI 1.01–12.1; p = 0.0481) were prognostic factors for PFS but not for OS. Postoperative chemotherapy was performed in all 34 cases. High-dose chemotherapy was performed in 19 (55.8%) of 34 patients and showed a positive impact on OS (HR 0.31, 95% CI 0.11–0.86; p = 0.0254); the most commonly used regimen was a double-conditioning regimen of thiotepa plus melphalan. Local radiotherapy had a positive impact on both PFS and OS; however, craniospinal irradiation (CSI) performed in 12 patients as the primary therapy was associated with a poor outcome. Disseminated recurrence within 12 months from diagnosis was the most common pattern of treatment failure regardless of CSI.


There has been an improvement in outcomes for pediatric ATRT patients since the introduction of multimodal therapy in Japan, mainly in patients without metastases. Even if selection bias is taken into consideration, CSI did not contribute to an improved prognosis. Novel treatment approaches are required for pediatric ATRT patients with metastases.


ATRT = atypical teratoid/rhabdoid tumor; CSI = craniospinal irradiation; HDC = high-dose chemotherapy; JPBTC = Japanese Brain Tumor Consortium; JCCG = Japan Children’s Cancer Group; PFS = progression-free survival; OS = overall survival.

Supplementary Materials

    • Tables S1–S3 and Figs. S1–S6 (PDF 744 KB)
Images from Fomchenko et al. (pp 121–130).

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Contributor Notes

Correspondence Kai Yamasaki: Osaka City General Hospital, Osaka, Japan.

INCLUDE WHEN CITING Published online November 15, 2019; DOI: 10.3171/2019.9.PEDS19367.

Disclosures Dr. Arakawa reports receiving grant or research support unrelated to the current paper from the following: Siemens, Philips, Sanofi, Nihon Medi-Physics Co., Ltd., Brainlab, Zeiss, Tanabe Mitsubishi, Chugai, Eisai, Merck, Meiji Seika, Daiichi Sankyo, CLS, Takeda, and Pfizer; he also reports being on the speaker’s bureau of the following: Nippon Kayaku, Chugai, Eisai, Merck, Meiji Seika Pharma Co., Ltd., Otsuka, Brainlab, Daiichi Sankyo, UCB, Novocure, Abbvie, and CSL Behring.


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