Cardinal Health Specialty Solutions, Dublin, Ohio.
JAMA Netw Open. 2021 Feb 1;4(2):e2036741. doi: 10.1001/jamanetworkopen.2020.36741.
In clinical trials supporting the regulatory approval of oncology drugs, solid tumor response is assessed using Response Evaluation Criteria in Solid Tumors (RECIST). Calculation of RECIST-based responses requires sequential, timed imaging data, which presents challenges to the method's application in real-world evidence research.
To evaluate the feasibility and validity of a novel real-world RECIST method in assessing tumor burden associated with therapy for a large heterogeneous patient population undergoing treatment in routine clinical practice.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study used physician-abstracted data pooled from retrospective, multisite electronic health record (EHR) review studies of patients treated with anticancer drugs at US oncology practices from 2014 through 2017. Included patients were receiving first-line treatment for thyroid cancer, breast cancer, or metastatic melanoma. Data were analyzed from March through August 2020.
Undergoing treatment with immunotherapy or targeted therapy.
Tumor response was classified according to RECIST guidelines (ie, change in sum diameter of target lesions) post hoc with measurements derived from imaging scans and reports.
Among 1308 completed electronic case report forms, 956 forms (73.1%) had adequate data to classify real-world RECIST response. The greatest difference between physician-recorded responses and real-world RECIST-based responses was found in the proportion of complete responses: 118 responses (12.3%) vs 46 responses (4.8%) (P < .001). Among 609 patients in the metastatic melanoma population, complete responses were reported in 112 physician-recorded responses (18.4%) vs 44 real-world RECIST-based responses (7.2%) (P < .001), compared with 11 of 247 responses (4.5%) to 31 of 192 responses (16.1%) across pivotal trials of the same melanoma therapies.
These findings suggest that comparing tumor lesion sizes and categorizing treatment response according to RECIST guidelines may be feasible using real-world data. This study found that physician-recorded assessments were associated with overestimation of treatment response, with the largest overestimation among complete responses. Real-world RECIST-based assessments were associated with better approximations of tumor response reported in clinical trials compared with those reported in EHRs.
在支持肿瘤药物监管批准的临床试验中，使用实体瘤反应评估标准（RECIST）评估实体肿瘤的反应。计算基于 RECIST 的反应需要连续的、定时的成像数据，这给该方法在真实世界证据研究中的应用带来了挑战。
评估一种新的真实世界 RECIST 方法在评估接受常规临床实践中治疗的大型异质患者群体的治疗相关肿瘤负担方面的可行性和有效性。
设计、地点和参与者：这项队列研究使用了从 2014 年至 2017 年在美国肿瘤学实践中接受抗癌药物治疗的患者的回顾性、多地点电子健康记录（EHR）回顾研究中医生摘录的数据进行汇总。纳入的患者正在接受甲状腺癌、乳腺癌或转移性黑色素瘤的一线治疗。数据于 2020 年 3 月至 8 月进行分析。
接受免疫治疗或靶向治疗。
根据 RECIST 指南（即靶病变总和直径的变化）对肿瘤反应进行分类，方法是从成像扫描和报告中得出测量值。
在 1308 份完成的电子病例报告表中，有 956 份（73.1%）有足够的数据来分类真实世界的 RECIST 反应。医生记录的反应与真实世界基于 RECIST 的反应之间最大的差异在于完全缓解的比例：118 个反应（12.3%）与 46 个反应（4.8%）（P<.001）。在转移性黑色素瘤患者人群中，112 名医生记录的反应中有完全缓解（18.4%），而 44 名真实世界基于 RECIST 的反应中（7.2%）（P<.001），而在同一黑色素瘤治疗的关键试验中，11 例（4.5%）与 31 例（16.1%）。
这些发现表明，使用真实世界数据根据 RECIST 指南比较肿瘤病变大小和分类治疗反应可能是可行的。本研究发现，医生记录的评估与治疗反应的高估有关，完全缓解的高估最大。与 EHR 中报告的反应相比，真实世界基于 RECIST 的评估与临床试验中报告的更接近肿瘤反应。
