Successful treatment of metastatic alveolar rhabdomyosarcoma with MGMT gene promoter methylation by temozolomide‐based combination chemotherapy |
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| Authors: | Mariko Kinoshita Ai Yamada Daisuke Sawa Sachiyo Kamimura Mitsuru Miyachi Hiroshi Moritake |
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| Affiliation: | 1. Division of Pediatrics, Faculty of Medicine, University of Miyazaki, Miyazaki, Japan;2. Department of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kyoto, Japan |
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| Abstract: | A 3‐year‐old male presented with a large retroperitoneal mass and multiple metastases. Biopsy results suggested alveolar rhabdomyosarcoma bearing a methylated O6‐methylguanine‐DNA methyltransferase (MGMT) gene promoter. Serum microRNA‐206 levels were elevated and remained high after three cycles of vincristine, dactinomycin, and cyclophosphamide (VAC). Replacement of vincristine, irinotecan, and temozolomide (VIT) for VAC induced a marked tumor reduction and normalization of the miR‐206 levels. The patient completed 14 cycles of VIT with local radiotherapy and has been in remission for 31 months. Temozolomide could be effective for tumors with a methylated MGMT gene promoter. Individualized therapy is warranted for such patients. |
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| Keywords: | methylation MGMT gene rhabdomyosarcoma temozolomide |
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