背景:治疗目标(T2T)的概念,一种治疗策略,其中治疗旨在达到并维持确定的目标,例如缓解或低疾病活动(LDA),已经探索了几种疾病,包括风湿性疾病,如类风湿性关节炎(RA)。然而,最近尚未对所有风湿性疾病中的T2T进行全面审查。
目的:对T2T治疗成人炎症性风湿性疾病的疗效和安全性进行系统评价和荟萃分析。
方法:PUBMED,从1990年1月至2023年12月,使用与T2T策略和风湿性疾病相关的关键字搜索EMBASE和CINAHL;包括T2T策略临床试验或观察性研究。临床,身体功能和放射学结果,成本效益,我们对T2T策略的不良事件(AE)进行了调查,并对RA研究中最常用的结局进行了随机效应荟萃分析.
结果:搜索确定了7896项研究,其中66项符合纳入标准,包括RA中的50个,3在银屑病关节炎(PsA)中,脊柱关节炎(SpA)1例,痛风12例。对于将T2T策略与RA中的常规护理(UC)进行比较的研究,83.3%(20/24)的患者采用T2T策略可取得较好的临床疗效,和荟萃分析显示,使用T2T策略治疗的患者更有可能缓解(合并RR:1.68(1.47-1.92),p<0.001]并达到DAS-28响应(合并标准化平均差(SMD):0.47(0.26-0.69),P<0.001]在1年时比UC患者医治。敏感性分析表明,采用预定义治疗方案的T2T策略比不采用方案的T2T策略具有更好的临床疗效。在改善身体机能和健康相关生活质量(HRQoL)方面,11/19(57.9%)研究发现,T2T策略比UC更有可能实现这些目标,HAQ评分平均变化的荟萃分析支持这一结论(合并SMD:1.48(0.46-2.51),p=0.004)。9项研究中有5项(55.6%)显示出T2T策略对放射学进展的更大益处。在成本效益和AE方面,2/2研究发现T2T策略比UC更具成本效益,8/8研究表明T2T策略没有更频繁发生AE的趋势。对于PsA和SpA的研究,在临床和功能益处方面,T2T策略也被证明比UC更有效,但不是放射学结果。所有痛风研究表明,通过T2T策略可以更有效地控制sUA水平,2项研究表明,T2T策略可以抑制侵蚀发展或晶体沉积。
结论:对于活动性RA患者,多项研究表明,T2T策略可增加实现临床应答和改善HRQoL的可能性,而不增加经济成本和不良事件。有限的研究表明,在活性PsA和SpA中,T2T策略具有临床和功能优势。还发现T2T策略可改善痛风的临床和放射学结果。其他风湿性疾病的T2T试验缺乏。
BACKGROUND: The concept of treat-to-target (T2T), a treatment strategy in which treatment is directed to reach and maintain a defined goal such as remission or low disease activity (LDA), has been explored for several diseases including rheumatic diseases such as rheumatoid arthritis (RA). However, a comprehensive
review of T2T in all rheumatic diseases has not recently been undertaken.
OBJECTIVE: To perform a systematic
review and meta-analysis of the efficacy and safety of a T2T strategy in the management of adult patients with inflammatory rheumatic diseases.
METHODS: PUBMED, EMBASE and CINAHL were searched from January 1990 to December 2023 using key words related to a T2T strategy and rheumatic diseases; T2T strategy clinical trials or observational studies were included. Clinical, physical function and radiologic outcomes, cost-effectiveness, and adverse events (AEs) of the T2T strategies were investigated and a random-effect meta-analysis was conducted for the most commonly used outcomes in RA studies.
RESULTS: The search identified 7896 studies, of which 66 fit inclusion criteria, including 50 in RA, 3 in psoriatic arthritis (PsA), 1 in spondyloarthritis (SpA) and 12 in
gout. For the studies comparing a T2T strategy with usual care (UC) in RA, 83.3% (20/24) showed a T2T strategy could achieve significantly better clinical outcomes, and the meta-analysis showed that patients treated with a T2T strategy were more likely to be in remission (pooled RR: 1.68 (1.47-1.92), p<0.001] and achieve DAS-28 response (pooled standardised mean difference (SMD): 0.47 (0.26-0.69), P<0.001] at 1 year than patients treated with UC. Sensitivity analyses showed that a T2T strategy with a predefined treatment protocol had better clinical efficacy than that without protocol. In terms of improving physical function and health-related quality of life (HRQoL), 11/19 (57.9%) studies found a T2T strategy was significantly more likely to achieve these than UC, with the meta-analysis for the mean change of HAQ score supporting this conclusion (pooled SMD: 1.48 (0.46-2.51), p=0.004). Five out of 9 studies (55.6%) demonstrated greater benefit regarding radiographic progression from a T2T strategy. In terms of cost-effectiveness and AEs, 2/2 studies found a T2T strategy was more cost-effective than UC and 8/8 studies showed no tendency for AEs to occur more often with a T2T strategy. For the studies in PsA and SpA, a T2T strategy was also demonstrated to be more effective than UC in clinical and functional benefits, but not in radiologic outcomes. All
gout studies showed that sUA level could be controlled more effectively with a T2T strategy, and 2 studies revealed that the T2T strategy could inhibit erosion development or crystal deposition.
CONCLUSIONS: For patients with active RA, a T2T strategy has been shown in mulitple studies to increase the likelihood of achieving clinical response and improving HRQoL without increasing economic costs and AEs. Limited studies have shown clinical and functional benefits from T2T strategies in active PsA and SpA. A T2T strategy has also been found to improve clinical and radiologic outcomes in
gout. T2T trials in other rheumatic diseases are lacking.