对照组乳腺癌女性是否获得标准治疗？
癌症临床研究的『标准治疗』：
乳腺癌临床研究对照组女性治疗分析
为了对新的治疗方法进行有效评估，很有必要与现有可选治疗方法进行比较，于是必然产生研究组（接受新的治疗方法）和对照组（接受现有可选治疗方法）。那么，在乳腺癌临床研究中，对照组女性所获现有可选治疗方法是否符合标准？
2017年9月，美国《国家癌症综合网络杂志》正式发表澳大利亚悉尼大学医学院和公共卫生学院的研究报告，回顾了乳腺癌临床研究女性对照组的治疗情况，对符合临床指南治疗标准的比例进行了估算。
作者对2004～2014年招募患者入组比较新药治疗与『标准治疗』并在美国政府临床研究网站（ClinicalTrials.gov）登记的乳腺癌III期随机对照研究进行了分析，主要结局指标为对照组治疗符合当时NCCN指南的研究比例，次要结局分析对美国境外招募入组研究对照组治疗不符合NCCN指南对比德国妇科肿瘤学组（AGO）指南进行评定，并对主要结局与预先筛选研究特征之间的相关性进行评定。
结果，共检索出210项符合条件的研究，共招募入组患者22万9182例，其中：
127项研究（60%）在美国境外招募患者
83项研究（40%）在美国境内招募患者
60项研究（29%）未提供符合NCCN指南的对照组治疗，其中美国境外49项（39%）、美国境内11项（13%）
21%的研究对照组治疗既不符合AGO指南又不符合NCCN指南
对照组治疗不符合指南的显著相关因素：
时期（近期研究较少符合）
乳腺癌分期和类型（早期和雌激素受体阴性乳腺癌研究较少符合）
患者入组国家≥4个
在美国境外招募入组
因此，为了确保临床研究获得足以指导患者治疗的最佳信息，有必要对研究者选择和描述对照组患者『标准治疗』问题展开深入调查。
J Natl Compr Canc Netw. 2017 Sep;15(9):1131-1139.
"Standard Care" in Cancer Clinical Trials: An Analysis of Care Provided to Women in the Control Arms of Breast Cancer Clinical Trials.
Dear RF, McGeechan K, Barnet MB, Barratt AL, Tattersall MHN.
Sydney Medical School, Sydney School of Public Health, The University of Sydney, Sydney, Australia.
BACKGROUND: For trials to validly evaluate new treatments, comparison against the best existing alternative treatment is essential. We reviewed the care provided to women in control arms of breast cancer clinical trials to estimate the proportion consistent with the standard of care as defined in clinical guidelines.
METHODS: We analyzed phase III randomized controlled breast cancer trials comparing drug treatments with "standard care" enrolling between 2004 and 2014, and registered on ClinicalTrials.gov. Our primary outcome was the proportion of trials in which treatment in the control arm was consistent with concurrent NCCN Guidelines. A secondary analysis assessed trials recruiting outside the United States that provided control group therapy not consistent with NCCN Guidelines, comparing them with the German Gynecological Oncology Group (AGO) guidelines. We assessed associations between the primary outcome and a priori selected trial characteristics.
RESULTS: This study included 210 trials that recruited 229,182 women worldwide; 29% of trials (60/210) did not provide control group treatment that was consistent with NCCN Guidelines. For trials not recruiting in the United States, results were similar; in 21% of trials, control arm treatment was inconsistent with both AGO and NCCN Guidelines. Factors significantly associated with offering control arm treatment that were inconsistent with guidelines were time period (later trials were less likely to be consistent), breast cancer stage and type (trials in early-stage breast cancer and estrogen receptor-negative disease were less likely consistent), and recruitment in ≥4 countries and recruitment outside the United States.
CONCLUSIONS: To ensure that clinical trials achieve their goal of obtaining the best information to guide patient treatment, the question of how investigators chose and describe "standard care" for control arm participants warrants further investigation.
PMID: 28874598
DOI: 10.6004/jnccn.2017.7012