Epidemiology Group, Aberdeen Centre for Arthritis and Musculoskeletal Health, University of Aberdeen, Aberdeen, UK
Epidemiology Group, Aberdeen Centre for Arthritis and Musculoskeletal Health, University of Aberdeen, Aberdeen, UK.
Ann Rheum Dis. 2020 Jul;79(7):914-919. doi: 10.1136/annrheumdis-2019-216841. Epub 2020 Apr 23.
Management guidelines assume that results from clinical trials can be generalised, although seldom is data available to test this assumption. We aimed to determine the proportion of patients commencing tumour necrosis factor inhibition (TNFi) who would have been eligible for relevant clinical trials, and whether treatment response differs between these groups and the trials themselves. The British Society for Rheumatology Biologics Register for Ankylosing Spondylitis (BSRBR-AS) recruited a real-world cohort of TNFi-naïve spondyloarthritis patients with data collection from clinical records and patient questionnaires. Participant characteristics were extracted from trials identified from a recent Health Technology Assessment of TNFi for ankylosing spondylitis/non-radiographic axial spondyloarthritis. Descriptive statistics were used to determine the differences, including treatment response, between BSRBR-AS participants who would/would not have been eligible for the clinical trials and with trial participants. Among 2420 BSRBR-AS participants, those commencing TNFi (34%) had shorter symptom duration (15 vs 22 years) but more active disease (Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) 6.4 vs 4.0; Bath Ankylosing Spondylitis Disease Functional Index (BASFI) 6.2 vs 3.8). Of those commencing TNFi, 41% met eligibility criteria for ≥1 of fourteen relevant trials; they reported higher disease activity (BASDAI 6.9 vs 6.1) and poorer function (BASFI 6.6 vs 6.0). 61.7% of trial participants reported a positive treatment response, vs 51.3% of BSRBR-AS patients (difference: 10.4%; 95% CI 4.4% to 16.5%). Potential eligibility for trials did not influence treatment response (difference 2.0%; -9.4% to 13.4%). Fewer patients in the real world respond to TNFi than is reported in the trial literature. This has important implications for the generalisability of trial results, and the cost-effectiveness of TNFi agents.
管理指南假设临床试验的结果可以推广，但很少有数据可用于检验这一假设。我们旨在确定开始使用肿瘤坏死因子抑制剂 (TNFi) 的患者中有多少人符合相关临床试验的条件，以及这些患者与试验本身之间的治疗反应是否存在差异。英国风湿病学会生物制剂登记处（BSRBR-AS）招募了一组未经 TNFi 治疗的强直性脊柱炎患者，数据来自临床记录和患者问卷。参与者特征从最近的 TNFi 治疗强直性脊柱炎/非放射性轴性脊柱关节炎的健康技术评估中确定的临床试验中提取。使用描述性统计数据来确定 BSRBR-AS 参与者与临床试验参与者之间的差异，包括治疗反应，这些差异包括那些符合/不符合临床试验条件的患者。在 2420 名 BSRBR-AS 参与者中，开始使用 TNFi 的患者（34%）的症状持续时间较短（15 年与 22 年），但疾病更为活跃（Bath 强直性脊柱炎疾病活动指数（BASDAI）6.4 分与 4.0 分；Bath 强直性脊柱炎疾病功能指数（BASFI）6.2 分与 3.8 分）。在开始使用 TNFi 的患者中，41%符合 14 项相关试验中的至少一项的入选标准；他们报告了更高的疾病活动度（BASDAI 6.9 分与 6.1 分）和较差的功能（BASFI 6.6 分与 6.0 分）。61.7%的试验参与者报告治疗反应阳性，而 BSRBR-AS 患者的这一比例为 51.3%（差异：10.4%；95%CI 4.4%至 16.5%）。在现实世界中，潜在的试验入选标准并不影响治疗反应（差异 2.0%；-9.4%至 13.4%）。现实世界中接受 TNFi 治疗的患者比试验文献中报告的要少。这对试验结果的推广性和 TNFi 药物的成本效益有重要影响。
