Department of Symptom Research, The University of Texas MD Anderson Cancer Center, Houston, Texas 77030, USA.
J Pain Symptom Manage. 2010 Jun;39(6):1077-85. doi: 10.1016/j.jpainsymman.2009.05.025.
The U.S. Food and Drug Administration (FDA) 2006 draft guidance on "Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims" has engendered wide discussion about patient-reported outcome (PRO) domains that should be endpoints in clinical trials. Reducing the severity and impact of symptoms is a natural intervention endpoint for cancer, a condition associated with considerable symptom burden. Because symptoms are best described by patients who have them, including PROs as measures of treatment effectiveness or the differences among treatments provides essential information about the efficacy and toxicity of a treatment and its effects on function. The FDA guidance provides a framework for addressing such issues as clinical significance, study design, and statistical methods as they relate to applications for labeling claims; however, no set of recommended approaches for assessing specific symptoms by patient report in clinical trials exists, other than for pain. Accordingly, an interdisciplinary workgroup, Assessing the Symptoms of Cancer using Patient-Reported Outcomes (ASCPRO), has been formed to generate evidence-based recommendations for the assessment of patient-reported cancer-related symptoms and the use of that information to facilitate clinical research and decision making. ASCPRO is among the first working groups to focus primarily on nonpain symptoms, including fatigue, sleep disturbance, appetite loss, depression, cognitive impairment, and shortness of breath. ASCPRO members are stakeholders in optimal symptom assessment, including patient advocates, academics, clinicians, those who pay for symptom control and monitor quality of care, and those who produce products that palliate cancer-related symptoms but that may also engender treatment-related symptoms.
美国食品和药物管理局（FDA）2006 年发布的关于“患者报告结局测量指标：在医疗产品开发中的应用以支持标签声明”的草案指南，引发了广泛讨论，即哪些患者报告结局（PRO）领域应作为临床试验的终点。减轻症状的严重程度和影响是癌症的自然干预终点，这种疾病与相当大的症状负担有关。由于症状是患者自己最能描述的，包括 PRO 作为治疗效果的衡量标准或治疗之间的差异，为治疗的疗效和毒性及其对功能的影响提供了重要信息。FDA 指南为解决与标签声明申请相关的临床意义、研究设计和统计方法等问题提供了框架；然而，除了疼痛之外，目前尚无一套用于在临床试验中通过患者报告评估特定症状的推荐方法。因此，成立了一个跨学科工作组，即使用患者报告结局评估癌症症状（ASCPRO），以生成评估患者报告的癌症相关症状的循证建议，并利用这些信息促进临床研究和决策制定。ASCPRO 是首批主要关注非疼痛症状的工作组之一，包括疲劳、睡眠障碍、食欲减退、抑郁、认知障碍和呼吸急促。ASCPRO 成员是最佳症状评估的利益相关者，包括患者权益倡导者、学者、临床医生、那些支付症状控制费用并监测护理质量的人，以及那些生产减轻癌症相关症状但也可能引起治疗相关症状的产品的人。
