Center for Health Measurement, Department of Population Health Sciences, Duke University School of Medicine, Durham, NC, USA.
Center for Communication Science, RTI International, Research Triangle Park, USA.
Pediatr Rheumatol Online J. 2024 Aug 21;22(1):77. doi: 10.1186/s12969-024-01014-x.
Current treatment for localized scleroderma (LS) has been shown to halt disease activity, but little is still known about patient experiences with these treatments, nor is there consensus about optimal measurement strategies for future clinical trials.
Conduct a scoping review of the literature for the types of outcomes and measures (i.e. clinician-, patient-, and caregiver-reported) utilized in published treatment studies of LS.
Online databases were searched for articles related to the evaluation of treatment efficacy in LS with a special focus on pediatrics.
Of the 168 studies, the most common outcomes used were cutaneous disease activity and damage measured via clinician-reported assessments. The most frequently cited measure was the Localized Scleroderma Cutaneous Assessment Tool (LoSCAT). Few patient-reported outcome measures (PROMs) were used.
Some studies only vaguely reported the measures utilized, and the review yielded a low number of clinical trials.
In addition to evaluating disease activity with clinician-reported measures, the field could obtain critical knowledge on the patient experience by including high-quality PROMs of symptoms and functioning. More clinical trials using a variety of outcomes and measures are necessary to determine the most suitable course of treatment for LS patients.
目前针对局限性硬皮病 (LS) 的治疗方法已被证明可以阻止疾病活动，但对于这些治疗方法的患者体验，我们知之甚少，也没有关于未来临床试验最佳测量策略的共识。
对 LS 治疗研究中使用的各种结局和测量方法（即临床医生、患者和护理人员报告）进行文献范围综述。
在线数据库中搜索与 LS 治疗疗效评估相关的文章，特别关注儿科。
在 168 项研究中，最常用的结局是通过临床医生报告评估测量的皮肤疾病活动和损伤。最常引用的测量工具是局限性硬皮病皮肤评估工具 (LoSCAT)。很少使用患者报告的结局测量 (PROM)。
一些研究仅含糊地报告了所使用的措施，且综述得出的临床试验数量较少。
除了使用临床医生报告的措施评估疾病活动外，该领域还可以通过纳入症状和功能的高质量 PROM 来获得有关患者体验的重要知识。需要更多使用各种结局和措施的临床试验来确定最适合 LS 患者的治疗方案。
