Le-Rademacher Jennifer G, Crawford Jeffrey, Evans William J, Jatoi Aminah
Department of Health Sciences Research, Mayo Clinic, Rochester, MN, United States; Department of Oncology, Mayo Clinic, Rochester, Minnesota, United States.
Duke Cancer Institute, Duke University, Durham, NC, United States.
Crit Rev Oncol Hematol. 2017 Sep;117:30-37. doi: 10.1016/j.critrevonc.2017.06.008. Epub 2017 Jun 24.
Most advanced cancer patients suffer loss of appetite (anorexia) and loss of weight. Despite the fact that cancer anorexia and weight loss are associated with a poor prognosis and detract from quality of life, no interventions have been demonstrated to palliate this syndrome in its entirety, particularly in patients with treatment-refractory malignancies. Recently, two registration trials - one with anamorelin and another with enobosarm - failed to reach their primary endpoints, thus raising questions. Were both these agents ineffective? Alternatively, did study design issues compromise the ability of these trials to identify effective agents? Thus, this review is timely insofar it serves as an introduction to study design, offers guidance on how to test promising agents for cancer anorexia/weight loss, and provides advice for overcoming trial design obstacles.
大多数晚期癌症患者会出现食欲不振（厌食）和体重减轻的症状。尽管癌症厌食和体重减轻与预后不良相关且会降低生活质量，但尚无干预措施能完全缓解这一综合征，尤其是对于治疗难治性恶性肿瘤患者。最近，两项注册试验——一项使用阿那莫林，另一项使用恩博萨莫——均未达到其主要终点，因此引发了质疑。是这两种药物均无效吗？还是研究设计问题影响了这些试验识别有效药物的能力？因此，本综述很及时，因为它介绍了研究设计，就如何测试有前景的癌症厌食/体重减轻药物提供了指导，并为克服试验设计障碍提供了建议。
