背景:目前对局部硬皮病(LS)的治疗已被证明可以阻止疾病活动,但是对这些治疗的患者体验知之甚少,对于未来临床试验的最佳测量策略也没有达成共识.
目的:对结果和措施类型的文献进行范围审查(即临床医生-,患者-,和护理人员报告的)用于已发表的LS治疗研究。
方法:在在线数据库中搜索与评估LS治疗效果相关的文章,特别关注儿科。
结果:在168项研究中,最常用的结局是通过临床医生报告的评估测量的皮肤疾病活动度和损伤.最常引用的测量是局部硬皮病皮肤评估工具(LoSCAT)。很少使用患者报告的结果测量(PROM)。
结论:一些研究只是模糊地报告了所使用的措施,并且该审查产生的临床试验数量很少。
结论:除了用临床医生报告的措施评估疾病活动外,该领域可以通过包括症状和功能的高质量PROM来获得关于患者体验的关键知识。需要使用各种结果和措施进行更多的临床试验,以确定最适合LS患者的治疗过程。
BACKGROUND: 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.
OBJECTIVE: 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.
METHODS: Online databases were searched for articles related to the evaluation of treatment efficacy in LS with a special focus on pediatrics.
RESULTS: 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.
CONCLUSIONS: Some studies only vaguely reported the measures utilized, and the
review yielded a low number of clinical trials.
CONCLUSIONS: 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.