Beukelman Timothy, Long Millie D, Rhee Rennie L, Kappelman Michael D, Merkel Peter A, Nowell William Benjamin, Clinton Cassie, Ringold Sarah, Del Gaizo Vincent, Price Brian, Shaw Dianne G, Venkatachalam Shilpa, Cuthbertson David, Xie Fenglong, Zhang Xian, Curtis Jeffrey R
Department of Pediatrics, Division of Rheumatology, University of Alabama at Birmingham, Birmingham, AL, USA.
Department of Medicine, Division of Gastroenterology and Hepatology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Patient Relat Outcome Meas. 2023 Jun 13;14:171-180. doi: 10.2147/PROM.S392174. eCollection 2023.
The most reliable and meaningful approach for inclusion of patient-reported outcomes (PROs) in the evaluation of real-world clinical effectiveness of biologics in the treatment of autoimmune diseases is u ncertain. This study aimed to assess and compare the proportions of patients who had abnormalities in PROs measuring important general health domains at the initiation of treatment with biologics, as well as the effects of baseline abnormalities on subsequent improvement.
PROs were collected for patient participants with inflammatory arthritis, inflammatory bowel disease, and vasculitis using Patient-Reported Outcomes Measurement Information System instruments. Scores were reported as -scores normalized to the general population in the United States. Baseline PROs scores were collected near the time of biologic initiation, and follow-up scores were collected 3 to 8 months later. In addition to summary statistics, the proportion of patients with PROs abnormalities (scores ≥5 units worse than the population norm) was determined. Baseline and follow-up scores were compared, and an improvement of ≥5 units was considered significant.
There was wide variation across autoimmune diseases in baseline PROs scores for all domains. For example, the proportion of participants with abnormal baseline pain interference scores ranged from 52% to 93%. When restricted to participants with baseline PROs abnormalities, the proportion of participants experiencing an improvement of ≥5 units was substantially higher.
As expected, many patients experienced improvement in PROs following initiation of treatment with biologics for autoimmune diseases. Nevertheless, a substantial proportion of participants did not exhibit abnormalities in all PROs domains at baseline, and these participants appear less likely to experience improvement. For PROs to be reliably and meaningfully included in the evaluation of real-world medication effectiveness, more knowledge and careful consideration are needed to select the most appropriate patient populations and subgroups for inclusion and evaluation in studies measuring change in PROs.
将患者报告的结局（PROs）纳入生物制剂治疗自身免疫性疾病真实世界临床疗效评估的最可靠且有意义的方法尚不确定。本研究旨在评估和比较在开始使用生物制剂治疗时，在测量重要总体健康领域的PROs方面存在异常的患者比例，以及基线异常对后续改善的影响。
使用患者报告结局测量信息系统工具收集炎性关节炎、炎性肠病和血管炎患者参与者的PROs。分数报告为相对于美国普通人群标准化的z分数。在开始使用生物制剂时附近收集基线PROs分数，并在3至8个月后收集随访分数。除了汇总统计数据外，还确定了PROs异常（分数比人群正常水平差≥5个单位）的患者比例。比较基线和随访分数，改善≥5个单位被认为是显著的。
所有领域的基线PROs分数在自身免疫性疾病之间存在广泛差异。例如，基线疼痛干扰分数异常的参与者比例在52%至93%之间。当仅限于基线PROs异常的参与者时，改善≥5个单位的参与者比例显著更高。
正如预期的那样，许多患者在开始使用生物制剂治疗自身免疫性疾病后PROs有所改善。然而，相当一部分参与者在基线时并非在所有PROs领域都表现出异常，并且这些参与者似乎不太可能经历改善。为了将PROs可靠且有意义地纳入真实世界药物疗效评估，需要更多知识并仔细考虑，以选择最合适的患者群体和亚组纳入并评估测量PROs变化的研究。
