Affiliations of authors: Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC (BBR, EB); Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, MD (SAM); Division of Cancer Prevention, National Cancer Institute, Bethesda, MD (LMM and AMO); Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, MD (AMD); Division of Health Sciences Research, Mayo Clinic, Scottsdale, AZ (ACD); Feinberg School of Medicine, Northwestern University, Chicago, IL (DC); Nell Hodgson Woodruff School of Nursing, Emory University, Atlanta, GA (CMR, DWB); Department of General Oncology, MD Anderson Cancer Center, Houston, TX (MJF); Mayo Clinic Breast SPORE, Rochester, MN (CCh); Department of Psychological Research, The Netherlands Cancer Institute, Amsterdam, The Netherlands (NKA); Quality of Life Department, European Organization for the Research and Treatment of Cancer, Brussels, Belgium (CCo).
J Natl Cancer Inst. 2014 Jul 8;106(7). doi: 10.1093/jnci/dju129. Print 2014 Jul.
The National Cancer Institute's Symptom Management and Health-Related Quality of Life Steering Committee held a clinical trials planning meeting (September 2011) to identify a core symptom set to be assessed across oncology trials for the purposes of better understanding treatment efficacy and toxicity and to facilitate cross-study comparisons. We report the results of an evidence-synthesis and consensus-building effort that culminated in recommendations for core symptoms to be measured in adult cancer clinical trials that include a patient-reported outcome (PRO).
We used a data-driven, consensus-building process. A panel of experts, including patient representatives, conducted a systematic review of the literature (2001-2011) and analyzed six large datasets. Results were reviewed at a multistakeholder meeting, and a final set was derived emphasizing symptom prevalence across diverse cancer populations, impact on health outcomes and quality of life, and attribution to either disease or anticancer treatment.
We recommend that a core set of 12 symptoms--specifically fatigue, insomnia, pain, anorexia (appetite loss), dyspnea, cognitive problems, anxiety (includes worry), nausea, depression (includes sadness), sensory neuropathy, constipation, and diarrhea--be considered for inclusion in clinical trials where a PRO is measured. Inclusion of symptoms and other patient-reported endpoints should be well justified, hypothesis driven, and meaningful to patients.
This core set will promote consistent assessment of common and clinically relevant disease- and treatment-related symptoms across cancer trials. As such, it provides a foundation to support data harmonization and continued efforts to enhance measurement of patient-centered outcomes in cancer clinical trials and observational studies.
美国国家癌症研究所的症状管理和健康相关生活质量指导委员会召开了一次临床试验规划会议（2011 年 9 月），以确定一组核心症状，用于评估肿瘤学试验，以便更好地了解治疗效果和毒性，并促进跨研究比较。我们报告了一项证据综合和共识建立工作的结果，最终提出了在包括患者报告结局（PRO）在内的成人癌症临床试验中测量核心症状的建议。
我们使用了数据驱动、共识建立的过程。一个由专家组成的小组，包括患者代表，对文献（2001-2011 年）进行了系统回顾，并分析了六个大型数据集。结果在一个多利益攸关方会议上进行了审查，并最终确定了一个强调不同癌症人群中症状的普遍性、对健康结果和生活质量的影响以及归因于疾病或抗癌治疗的核心症状集。
我们建议将 12 种症状（即疲劳、失眠、疼痛、厌食（食欲减退）、呼吸困难、认知问题、焦虑（包括担忧）、恶心、抑郁（包括悲伤）、感觉神经病变、便秘和腹泻）作为核心症状集，纳入到测量患者报告结局的临床试验中。纳入症状和其他患者报告结局应具有充分的理由、假设驱动，并对患者有意义。
这个核心症状集将促进癌症临床试验中对常见和临床相关的疾病和治疗相关症状的一致评估。因此，它为支持癌症临床试验和观察性研究中患者为中心结局的测量数据协调和持续努力提供了基础。
