Temozolomide is an active agent in children with recurrent medulloblastoma/primitive neuroectodermal tumor: an Italian multi-institutional phase II trial

作者：Graziella Cefalo, Maura Massimino, Antonio Ruggiero, Giovanni Barone, Vita Ridola, Filippo Spreafico, Paolo Potepan, Massimo Eraldo Abate, Maurizio Mascarin, Maria Luisa Garrè, Giorgio Perilongo, E Madon, Cesare Colosimo, Riccardo Riccardi · 发表于：Neuro-Oncology · 年份：2014 · DOI：10.1093/neuonc/not320 · 被引用次数：60 · 研究领域：Glioma Diagnosis and Treatment、Hedgehog Signaling Pathway Studies、Neuroblastoma Research and Treatments

BACKGROUND: The aim of this study was to assess the objective response rate (ORR) of children and young adults with recurrent medulloblastoma/primitive neuroectodermal tumor (MB/PNET) treated with temozolomide (TMZ). The secondary purpose was to analyze the toxicity profile of TMZ when administered orally for 5 days in 3 divided daily doses every 28 days. METHODS: Forty-two patients with recurrent MB/PNET, aged 21 years and younger, were recruited. Patients were treated with oral TMZ. Starting doses ranged from 120 to 200 mg/m(2)/day based on previous treatments. A craniospinal MRI was performed prior to the first cycle of TMZ and following every 2 cycles of treatment. RESULTS: Median age was 10 years (range, 2-21 years). Forty of 42 patients were assessed for response and toxicity. The objective response rate was 42.5%: 6 patients achieved a complete response, 11 had a partial response, and 10 had stable disease. Progression-free survival rates for all patients at 6 and 12 months were 30% and 7.5%, respectively. Their median overall survival rates at 6 and 12 months were 42.5% and 17.5%, respectively. No major extrahematological effects or life-threatening events were reported. The most common grade 3/4 toxicity included thrombocytopenia (17.5%), neutropenia (7.5%), and anemia (2.5%). CONCLUSIONS: TMZ proved to be an effective agent in children and young adults with MB/PNET, heavily pre-treated, with a tolerable toxicity profile.