Temozolomide is an active agent in children with recurrent medulloblastoma/primitive neuroectodermal tumor: an Italian multi-institutional phase II trial
Temozolomide is an active agent in children with recurrent medulloblastoma/primitive neuroectodermal tumor: an Italian multi-institutional phase II trial
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DOI：
10.1093/neuonc/not320
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发表时间：
2014-05-01
期刊：
影响因子：
15.9
通讯作者：
Riccardi, Riccardo
中科院分区：
文献类型：
--
作者：
Cefalo, Graziella;Massimino, Maura;Riccardi, Riccardo
本研究的目的是评估替莫唑胺（TMZ）治疗复发性髓母细胞瘤/原始神经外胚层肿瘤（MB/PNET）的儿童和年轻人的客观缓解率（ORR）。次要目的是分析TMZ每28天分3次口服5天的毒性。42例复发性MB/PNET患者，年龄在21岁及以下。患者口服TMZ治疗。根据以前的治疗，起始剂量为120至200 mg/m(2)/天。在TMZ第一周期治疗前和每2周期治疗后进行颅脊髓MRI检查。中位年龄为10岁（范围221岁）。对42例患者中的40例进行了反应和毒性评估。客观缓解率为42.5，完全缓解6例，部分缓解11例，病情稳定10例。所有患者在6个月和12个月的无进展生存率分别为30%和7.5%。6个月和12个月的中位总生存率分别为42.5和17.5。没有重大的血液学外影响或危及生命的事件报道。最常见的3/4级毒性包括血小板减少症（17.5）、中性粒细胞减少症（7.5）和贫血（2.5）。经证实，TMZ对患有MB/PNET的儿童和年轻人有效，经过大量预处理，毒性谱可耐受。
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.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.Median age was 10 years (range, 221 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).TMZ proved to be an effective agent in children and young adults with MB/PNET, heavily pre-treated, with a tolerable toxicity profile.