Centre for Big Data Research in Health, University of New South Wales, Australia; Sydney Musculoskeletal Health, Kolling Institute, The University of Sydney, Australia.
Clinical Research Centre, Zhujiang Hospital, Southern Medical University, China; Menzies Institute for Medical Research, University of Tasmania, Australia.
Osteoarthritis Cartilage. 2022 Nov;30(11):1495-1505. doi: 10.1016/j.joca.2022.06.005. Epub 2022 Jun 25.
To assess the real-world effectiveness of vitamin D supplementation in patients with knee osteoarthritis (KOA) by replicating a randomized controlled trial (RCT) design in an observational study.
This study emulated a target trial using data from the Osteoarthritis Initiative (OAI). Eligible participants were ≥45 years, had symptomatic KOA and did not take vitamin D supplements in the past 30 days. A participant can enter the trial more than once. Participants were included in vitamin D group if they took ≥1,000 IU/day for ≥4 days/week in the past 30 days at the first follow-up visit after baseline. The control group did not use vitamin D in the past 30 days. Optimal propensity score matching at 1:1 ratio was performed. The primary outcome was change in knee pain 2 years after baseline measured by the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC). Secondary outcomes included WOMAC physical function and quantitative joint space width (JSW). Standardized mean difference (SMD) was used to compare the findings with previous RCTs.
A total of 236 person-trials in the vitamin D group were pair-matched with a control. Compared to the control group, vitamin D supplementation did not reach significant changes in WOMAC pain (SMD = -0.04, 95%CI [-0.21, 0.13]), physical function and radiographic JSW over 2 years. The SMDs were consistent with the effect sizes reported in previous RCTs.
Target trial emulation in the OAI cohort demonstrated findings close to published RCTs. This supports the future use of target trial emulation in evaluating other systemic therapies for KOA.
通过在观察性研究中复制随机对照试验(RCT)设计,评估维生素 D 补充剂对膝骨关节炎(KOA)患者的真实疗效。
本研究使用 Osteoarthritis Initiative(OAI)的数据模拟目标试验。合格的参与者年龄≥45 岁,患有症状性 KOA,且在过去 30 天内未服用维生素 D 补充剂。参与者可以多次进入试验。如果参与者在基线后的第一次随访中过去 30 天内每周至少 4 天每天服用≥1000IU 的维生素 D,则将其纳入维生素 D 组。在过去 30 天内,对照组未使用维生素 D。采用 1:1 比例的最优倾向评分匹配。主要结局是用 Western Ontario and McMaster Universities Osteoarthritis Index(WOMAC)在基线后 2 年测量的膝关节疼痛变化。次要结局包括 WOMAC 身体功能和定量关节间隙宽度(JSW)。采用标准化均数差值(SMD)与之前的 RCT 进行比较。
维生素 D 组共有 236 个人试验进行了配对匹配。与对照组相比,维生素 D 补充剂在 WOMAC 疼痛(SMD=-0.04,95%CI[-0.21,0.13])、身体功能和影像学 JSW 方面未达到 2 年的显著变化。SMD 与之前 RCT 报告的效应大小一致。
在 OAI 队列中进行的目标试验模拟得出的结果与已发表的 RCT 非常接近。这支持未来在评估 KOA 的其他系统性治疗方法时使用目标试验模拟。