Departments of Dermatology and Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Vasculitis Clinic, Department of Rheumatology, Mount Sinai Hospital, University of Toronto, 60 Murray Street, Toronto, ON, M5T3L9, Canada.
Trials. 2020 Apr 28;21(1):362. doi: 10.1186/s13063-020-04285-3.
Skin-limited forms of vasculitis, while lacking systemic manifestations, can persist or recur indefinitely, cause pain, itch, or ulceration, and be complicated by infection or scarring. High-quality evidence on how to treat these conditions is lacking. The aim of this comparative effectiveness study is to determine the optimal management of patients with chronic skin-limited vasculitis.
ARAMIS is a multicenter, sequential, multiple assignment randomized trial with an enrichment design (SMARTER) aimed at comparing the efficacy of three drugs-azathioprine, colchicine, and dapsone-commonly used to treat various forms of isolated skin vasculitis. ARAMIS will enroll patients with isolated cutaneous small or medium vessel vasculitis, including cutaneous small vessel vasculitis, immunoglobulin A (IgA) vasculitis (skin-limited Henoch-Schönlein purpura), and cutaneous polyarteritis nodosa. Patients not responding to the initial assigned therapy will be re-randomized to one of the remaining two study drugs (Stage 2). Those with intolerance or contraindication to a study drug can be randomized directly into Stage 2. Target enrollment is 90 participants, recruited from international centers affiliated with the Vasculitis Clinical Research Consortium. The number of patients enrolled directly into Stage 2 of the study will be capped at 10% of the total recruitment target. The primary study endpoint is the proportion of participants from the pooled study stages with a response to therapy at month 6, according to the study definition.
ARAMIS will help identify effective agents for skin-limited forms of vasculitis, an understudied group of diseases. The SMARTER design may serve as an example for future trials in rare diseases.
ClinicalTrials.gov: NCT02939573. Registered on 18 October 2016.
皮肤局限性血管炎虽然缺乏全身表现，但可能会无限期持续或复发，引起疼痛、瘙痒或溃疡，并可能并发感染或瘢痕形成。缺乏关于如何治疗这些疾病的高质量证据。本项比较疗效研究旨在确定慢性皮肤局限性血管炎患者的最佳治疗方法。
ARAMIS 是一项多中心、序贯、多次分配随机试验，采用富集设计（SMARTER），旨在比较三种药物（硫唑嘌呤、秋水仙碱和氨苯砜）治疗各种孤立性皮肤血管炎的疗效。ARAMIS 将招募孤立性皮肤小或中等血管血管炎患者，包括皮肤小血管血管炎、免疫球蛋白 A（IgA）血管炎（皮肤局限性过敏性紫癜）和皮肤多发性动脉炎。对初始治疗无反应的患者将重新随机分配至其余两种研究药物之一（第 2 阶段）。对研究药物不耐受或有禁忌证的患者可直接进入第 2 阶段随机分组。目标入组人数为 90 例，来自血管炎临床研究联盟国际合作中心。直接进入第 2 阶段研究的患者人数将占总招募目标的 10%。主要研究终点为根据研究定义，在第 6 个月时联合研究阶段的参与者中治疗有反应的比例。
ARAMIS 将有助于确定皮肤局限性血管炎的有效治疗药物，这是一组研究不足的疾病。SMARTER 设计可能为未来罕见病的试验提供范例。
ClinicalTrials.gov：NCT02939573。注册于 2016 年 10 月 18 日。
