TY - JOUR
T1 - Clinical characteristics, treatment, and survival outcome in pediatric patients with atypical teratoid/rhabdoid tumors
T2 - A retrospective study by the Japan Children’s Cancer Group
AU - Yamasaki, Kai
AU - Kiyotani, Chikako
AU - Terashima, Keita
AU - Watanabe, Yuko
AU - Kanamori, Masayuki
AU - Koga, Yuhki
AU - Hata, Nobuhiro
AU - Iwasaki, Fuminori
AU - Goto, Hiroaki
AU - Koh, Katsuyoshi
AU - Kurihara, Jun
AU - Tokunaga, Shinya
AU - Arakawa, Yoshiki
AU - Hasegawa, Daiichiro
AU - Kosaka, Yoshiyuki
AU - Hara, Junichi
N1 - Funding Information:
This work was supported by the Brain Tumor Committee of Japan Children’s Cancer Group and a grant from the Japan Agency for Medical Research and Development (AMED) under grant number JP19ck0106330h0003. We are grateful to the cooperative members of this committee and all physicians who treated the children with ATRTs at the participating institutions described in Table S3. We are especially thankful to Drs. Sayaka Nakamura and Kiyotaka Isobe for help with data collection.
Publisher Copyright:
©AANS 2020,
PY - 2020
Y1 - 2020
N2 - OBJECTIVE 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. METHODS The authors retrospectively collected clinical information on 38 pediatric patients with ATRTs treated from 2005 to 2016 and analyzed the data for this series. RESULTS 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. CONCLUSIONS 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.
AB - OBJECTIVE 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. METHODS The authors retrospectively collected clinical information on 38 pediatric patients with ATRTs treated from 2005 to 2016 and analyzed the data for this series. RESULTS 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. CONCLUSIONS 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.
KW - ATRT
KW - Atypical teratoid/rhabdoid tumor
KW - Brain tumor
KW - Central nervous system tumors
KW - Children
KW - Oncology
KW - Prognosis
KW - Treatment
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U2 - 10.3171/2019.9.PEDS19367
DO - 10.3171/2019.9.PEDS19367
M3 - Article
C2 - 31731266
AN - SCOPUS:85079008595
SN - 1933-0707
VL - 25
SP - 111
EP - 120
JO - Journal of Neurosurgery: Pediatrics
JF - Journal of Neurosurgery: Pediatrics
IS - 2
ER -
