Historical benchmarks for medical therapy trials in surgery- and radiation-refractory meningioma: a RANO review
Historical benchmarks for medical therapy trials in surgery- and radiation-refractory meningioma: a RANO review
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DOI：
10.1093/neuonc/not330
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发表时间：
2014-06-01
期刊：
影响因子：
15.9
通讯作者：
Wen, Patrick
中科院分区：
文献类型：
--
作者：
Kaley, Thomas;Barani, Igor;Wen, Patrick
手术和放射难治性脑膜瘤患者接受药物治疗的结局尚不明确。已发表的报告受到患者人数少、选择偏倚、纳入混合组织学分级和疾病分期以及世界卫生组织（WHO）标准变化的限制。本分析旨在为未来的临床试验设计确定结果基准。对所有关于脑膜瘤药物治疗的英文出版物进行PubMed文献检索。将报告制成表格并分析患者数量、组织学分级、既往治疗、总生存期、无进展生存期（PFS）和放射学反应，确定了47篇出版物，并按组织学和既往治疗进行分类，仅包括那些治疗手术和放射难治性患者的出版物，以供进一步分析。这包括来自回顾性、先导性和II期研究、其他研究的探索性组和一项III期研究的各种药物（羟基脲、替莫唑胺、伊立替康、干扰素-α、米非司酮、奥曲肽类似物、醋酸甲地孕酮、贝伐珠单抗、伊马替尼、厄洛替尼和吉非替尼）。从所有研究中提取的唯一结果是PFS 6个月率，分别计算WHO I级脑膜瘤和合并的WHO II/III级脑膜瘤的加权平均值。对于WHO I脑膜瘤，加权平均PFS-6为29%（95%置信区间[CI]：20.3%-37.7%）。对于WHO II/III级脑膜瘤，加权平均PFS-6为26%（95%CI：19.3%-32.7%）。这项全面的综述证实了手术和放射难治性脑膜瘤的药物治疗效果较差。我们建议将上述PFS-6基准用于未来的试验设计。
The outcomes of patients with surgery- and radiation-refractory meningiomas treated with medical therapies are poorly defined. Published reports are limited by small patient numbers, selection bias, inclusion of mixed histologic grades and stages of illness, and World Health Organization (WHO) criteria changes. This analysis seeks to define outcome benchmarks for future clinical trial design.A PubMed literature search was performed for all English language publications on medical therapy for meningioma. Reports were tabulated and analyzed for number of patients, histologic grade, prior therapy, overall survival, progression-free survival (PFS), and radiographic response.Forty-seven publications were identified and divided by histology and prior therapies, including only those that treated patients who were surgery and radiation refractory for further analysis. This included a variety of agents (hydroxyurea, temozolomide, irinotecan, interferon-alpha, mifepristone, octreotide analogues, megestrol acetate, bevacizumab, imatinib, erlotinib, and gefitinib) from retrospective, pilot, and phase II studies, exploratory arms of other studies, and a single phase III study. The only outcome extractable from all studies was the PFS 6-month rate, and a weighted average was calculated separately for WHO grade I meningioma and combined WHO grade II/III meningioma. For WHO I meningioma, the weighted average PFS-6 was 29% (95% confidence interval [CI]: 20.3%-37.7%). For WHO II/III meningioma, the weighted average PFS-6 was 26% (95% CI: 19.3%-32.7%).This comprehensive review confirms the poor outcomes of medical therapy for surgery- and radiation-refractory meningioma. We recommend the above PFS-6 benchmarks for future trial design.