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Strategy to minimize radiation burden in infants and high‐risk medulloblastoma using intrathecal methotrexate and high‐dose chemotherapy: A prospective registry study in Japan
Author(s) -
Yamasaki Kai,
Okada Keiko,
Soejima Toshinori,
Sakamoto Hiroaki,
Hara Junichi
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28012
Subject(s) - medicine , thiotepa , medulloblastoma , radiation therapy , chemotherapy , adverse effect , prospective cohort study , concomitant , methotrexate , surgery , oncology , cyclophosphamide , cancer research
Background Most childhood medulloblastoma (MB) cases are curable using multimodal treatment, including craniospinal irradiation (CSI). However, late effects are a serious problem for survivors. This prospective registry study evaluated Japanese patients to determine whether a reduced radiation dose was feasible. Patients and Methods Patients with MB were classified as an infant group (<3 years old) and a high‐risk (HR) group (≥3 years old with metastasis). The HR group received intrathecal methotrexate (IT‐MTX) and high‐dose chemotherapy (HDC) using thiotepa and melphalan, as well as concomitant radiotherapy with a recommended CSI dose of 18 Gy and a total local dose of 50 Gy. Radiotherapy was only considered for infants if residual tumors were present after the HDC. Results Between 1997 and 2006, we identified 28 HR patients (M1: 9, M2/3: 19) and 17 infant patients (M0: 11, M1: 3, M2/3: 3). During the median follow‐up of 9.4 years for the entire HR group, the 5‐year progression‐free survival (PFS) rate was 82.1 ± 7.2% and the 5‐year overall survival (OS) rate was 85.7 ± 6.6%. Subanalyses of the patients who received the recommended treatment revealed that the 5‐year PFS and OS rates were both 90.5 ± 6.4%. In the infant group, the 5‐year PFS rate was 52.9 ± 12.1% and the 5‐year OS rate was 51.8 ± 12.4%. There were no serious adverse events associated with the IT‐MTX and HDC treatments. Conclusion Intensified chemotherapy using HDC and IT‐MTX might allow for a reduced prophylactic radiation dose in patients with MB with metastases. Further studies are needed to validate these findings.