REGOSARC: Regorafenib versus placebo in doxorubicin-refractory soft-tissue sarcoma-A quality-adjusted time without symptoms of progression or toxicity analysis.
REGOSARC: Regorafenib versus placebo in doxorubicin-refractory soft-tissue sarcoma-A quality-adjusted time without symptoms of progression or toxicity analysis.
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作者：
Berry V;Basson L;Bogart E;Mir O;Blay JY;Italiano A;Bertucci F;Chevreau C;Clisant-Delaine S;Liegl-Antzager B;Tresch-Bruneel E;Wallet J;Taieb S;Decoupigny E;Le Cesne A;Brodowicz T;Penel N
在一项安慰剂对照、随机 2 期试验（ClinicalTrials.gov 标识符 NCT01900743）中，瑞戈非尼改善了阿霉素预处理的晚期非脂肪细胞肉瘤患者的无进展生存期 (PFS)。应用无进展或毒性症状的质量调整时间（Q-TWiST）事后探索性分析来提供其临床益处的综合衡量。在基本案例分析中，每位患者的总生存期 (OS) 被分为 3 个相互排斥的健康状态：发生 3 或 4 级不良事件 (TOX) 的时间、没有疾病症状或治疗后 3 或 4 级毒性的时间，以及肿瘤进展或复发后的时间。每个状态所花费的时间均使用与该状态相关的健康状态效用进行加权，并进行求和以计算 Q-TWiST。通过多项敏感性分析探讨了基本案例分析的稳定性。在非脂肪细胞肉瘤中，瑞戈非尼组的 PFS 为（4.0 个月 [2.6-5.5 个月]，安慰剂组为 1.0 个月 [0.9-1.8 个月]；风险比，0.36 [0.25-0.53]；P < .0001）；瑞戈非尼组的 OS 为 13.4 个月（8.6‐17.3 个月），安慰剂组为 9.0 个月（6.8‐12.5 个月）（风险比为 0.67 [0.44‐1.02]）。根据 TOX 的经典定义（包括所有 3 级和 4 级临床不良事件），瑞戈非尼组的 Q-TWiST 为 8.0 个月（7.0-9.0 个月），安慰剂组为 5.7 个月（4.9-6.4 个月）（P < .001）。对于经阿霉素预处理的软组织肉瘤患者，与安慰剂相比，瑞戈非尼显着改善了质量调整生存期。癌症 2017；123：2294–2302。 © 2017 作者。 《癌症》由 Wiley periodicals, Inc. 代表美国癌症协会出版。这是根据知识共享署名非商业许可条款的开放获取文章，允许在任何媒体中使用、分发和复制，前提是正确引用原始作品并且不将其用于商业目的。在阿霉素预处理的非脂肪细胞性软组织肉瘤患者中，与安慰剂相比，瑞戈非尼显着改善了质量调整生存期（8.0 个月 vs 5.7 个月；P < .001）。另请参见第 2200-2 页。
In a placebo‐controlled, randomized phase 2 trial (ClinicalTrials.gov identifier NCT01900743), regorafenib improved progression‐free survival (PFS) for patients with doxorubicin‐pretreated advanced nonadipocytic sarcoma. A quality‐adjusted time without symptoms of progression or toxicity (Q‐TWiST) post hoc exploratory analysis was applied to provide an integrated measure of its clinical benefit. In the base‐case analysis, each patient's overall survival (OS) was partitioned into 3 mutually exclusive health states: the time with a grade 3 or 4 adverse event (TOX), the time without symptoms of disease or grade 3 or 4 toxicity from treatment, and the time after tumor progression or relapse. The time spent in each state was weighted with a health‐state utility associated with that state and was summed to calculate the Q‐TWiST. The stability of the base‐case analysis was explored with several sensitivity analyses. In nonadipocytic sarcoma, the PFS was (4.0 months [2.6‐5.5 months] with regorafenib vs 1.0 month [0.9‐1.8 months] with a placebo; hazard ratio, 0.36 [0.25‐0.53]; P < .0001); the OS was 13.4 months (8.6‐17.3 months) with regorafenib and 9.0 months (6.8‐12.5 months) with a placebo (hazard ratio, 0.67 [0.44‐1.02]). With the classic definition of TOX (including all grade 3 and 4 clinical adverse events), the Q‐TWiSTs were 8.0 months (7.0‐9.0 months) with regorafenib and 5.7 months (4.9‐6.4 months) with a placebo (P < .001). For patients with doxorubicin‐pretreated soft‐tissue sarcoma, regorafenib significantly improved quality‐adjusted survival in comparison with a placebo. Cancer 2017;123:2294–2302. © 2017 The Authors. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society. This is an open access article under the terms of the Creative Commons Attribution NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. In patients with doxorubicin‐pretreated nonadipocytic soft‐tissue sarcoma, regorafenib significantly improves quality‐adjusted survival in comparison with a placebo (8.0 vs 5.7 mo; P < .001). See also pages 2200‐2.