Importance Prospective studies of the disease course in patients with morphea are lacking, particularly those comparing adults and children.

Objective To investigate the disease course in patients with morphea treated with standard-of-care therapy using validated clinical outcome measures.

Design, Setting, and Participants Prospective cohort study of 130 adults and children from the Morphea in Adults and Children cohort with at least 2 years of clinical follow-up and Localized Scleroderma Cutaneous Assessment Tool scores recorded at each study visit. Study patients were seen at a tertiary referral center (UT Southwestern Medical Center, Dallas, Texas) from November 1, 2008, through April 1, 2016. The dates of analysis were May 2016 through July 2019.

Exposures All patients received standard-of-care therapy.

Main Outcomes and Measures Patterns in disease activity and recurrence were examined. The time to recurrence of morphea disease activity from the first visit with inactive disease was assessed using survival analysis with the log-rank test to compare differences between morphea subtypes.

Results In total, 130 adults and children (663 study visits) were included in this study. The mean (SD) age of patients was 34.4 (23.8) years, and 101 of 130 (78%) were female. The mean (SD) follow-up was 4.3 (1.7) years. Fifty patients had at least 5 years of follow-up. Most patients were white individuals (96 of 130 [74%]) and had linear subtype (72 of 130 [55%]) or generalized subtype (40 of 130 [31%]). Overall, 13 of 30 (43%) with generalized subtype had recurrence of disease activity compared with 14 of 66 (21%) with linear subtype (hazard ratio, 3.28; 95% CI, 1.38-7.79). The median (interquartile range) time to first recurrence of disease activity after initial resolution of disease activity was 1.1 (0.8-1.9) years for generalized subtype and 2.3 (1.0-3.3) years for linear subtype. Of the 50 patients followed up for at least 5 years, 18 (36%) had recurrence of disease activity.

Conclusions and Relevance Disease activity appeared to improve in most patients with morphea over 6 to 12 months using previously published treatment plans, underscoring their effectiveness. Sclerosis improved more slowly (over 2-5 years), often after discontinuation of treatment, but atrophy increased slightly as sclerosis subsided. Standard-of-care therapy appears to improve disease activity, which allows sclerosis to improve, and provides relative stability of other features of disease damage. A substantial number of patients, particularly those with generalized subtype, have a relapsing-remitting course over many years. Patients with morphea should be monitored for recurrent disease activity over extended periods.

重要性 缺乏对硬斑病患者病程的前瞻性研究，尤其是那些比较成人和儿童的研究。

目的 使用经过验证的临床结果测量，调查接受标准护理治疗的硬斑病患者的病程。

设计、设置和参与者 对来自成人和儿童硬斑病队列的 130 名成人和儿童进行前瞻性队列研究，并在每次研究访问时记录至少 2 年的临床随访和局部硬皮病皮肤评估工具评分。研究患者于 2008 年 11 月 1 日至 2016 年 4 月 1 日在三级转诊中心（UT 西南医学中心，德克萨斯州达拉斯）就诊。分析日期为 2016 年 5 月至 2019 年 7 月。

暴露 所有患者均接受标准护理治疗。

主要结果和措施 检查了疾病活动和复发的模式。使用生存分析和对数秩检验评估硬斑病活动从第一次就诊到硬斑病活动复发的时间，以比较硬皮病亚型之间的差异。

结果 总共有 130 名成人和儿童（663 次研究访问）被纳入本研究。患者的平均 (SD) 年龄为 34.4 (23.8) 岁，130 名患者中有 101 名 (78%) 为女性。平均 (SD) 随访时间为 4.3 (1.7) 年。50 名患者进行了至少 5 年的随访。大多数患者是白人个体（130 人中的 96 人 [74%]）并且具有线性亚型（130 人中的 72 人 [55%]）或全身亚型（130 人中的 40 人 [31%]）。总体而言，30 名全身亚型患者中有 13 名 (43%) 出现疾病活动性复发，而 66 名线性亚型患者中有 14 名 (21%) 出现疾病活动性复发（风险比，3.28；95% CI，1.38-7.79）。疾病活动初始消退后疾病活动首次复发的中位（四分位距）时间对于广义亚型为 1.1 (0.8-1.9) 年，对于线性亚型为 2.3 (1.0-3.3) 年。在随访至少 5 年的 50 名患者中，

结论和相关性 使用先前公布的治疗计划，大多数硬斑病患者的疾病活动似乎在 6 至 12 个月内有所改善，这凸显了它们的有效性。硬化的改善更缓慢（超过 2-5 年），通常在停止治疗后，但随着硬化消退，萎缩略有增加。标准护理疗法似乎可以改善疾病活动，从而改善硬化，并使疾病损害的其他特征相对稳定。相当多的患者，尤其是全身性亚型患者，有多年的复发-缓解过程。应长期监测硬斑病患者的复发性疾病活动。