Department of Dermatology, University of Nebraska, Omaha.
Department of Dermatology, Oregon Health & Science University, Portland.
JAMA Dermatol. 2020 May 1;156(5):521-528. doi: 10.1001/jamadermatol.2020.0035.
First-line systemic therapy for morphea includes methotrexate with or without systemic corticosteroids. When this regimen is ineffective, not tolerated, or contraindicated, a trial of mycophenolate mofetil (MMF) or mycophenolic acid (MPA)-referred to herein as mycophenolate-is recommended; however, evidence to support this recommendation remains weak.
To evaluate the effectiveness and tolerability of mycophenolate for the treatment of morphea.
DESIGN, SETTING, AND PARTICIPANTS: A retrospective cohort study was conducted from January 1, 1999, to December 31, 2018, among 77 patients with morphea from 8 institutions who were treated with mycophenolate.
The primary outcome was morphea disease activity, severity, and response at 0, 3 to 6, and 9 to 12 months of mycophenolate treatment. A secondary outcome was whether mycophenolate was a well-tolerated treatment of morphea.
There were 61 female patients (79%) and 16 male patients (21%) in the study, with a median age at disease onset of 36 years (interquartile range, 16-53 years) and median diagnostic delay of 8 months (interquartile range, 4-14 months). Generalized morphea (37 [48%]), pansclerotic morphea (12 [16%]), and linear morphea of the trunk and/or extremities (9 [12%]) were the most common subtypes of morphea identified. Forty-one patients (53%) had an associated functional impairment, and 49 patients (64%) had severe disease. Twelve patients received initial treatment with mycophenolate as monotherapy or combination therapy and 65 patients received mycophenolate after prior treatment was ineffective (50 of 65 [77%]) or poorly tolerated (21 of 65 [32%]). Treatments prior to mycophenolate included methotrexate (48 of 65 [74%]), systemic corticosteroids (42 of 65 [65%]), hydroxychloroquine (20 of 65 [31%]), and/or phototherapy (14 of 65 [22%]). After 3 to 6 months of mycophenolate treatment, 66 of 73 patients had stable (n = 22) or improved (n = 44) disease. After 9 to 12 months of treatment, 47 of 54 patients had stable (n = 14) or improved (n = 33) disease. Twenty-seven patients (35%) achieved disease remission at completion of the study. Treatments received in conjunction with mycophenolate were frequent. Mycophenolate was well tolerated. Gastrointestinal adverse effects were the most common (24 [31%]); cytopenia (3 [4%]) and infection (2 [3%]) occurred less frequently.
This study suggests that mycophenolate is a well-tolerated and beneficial treatment of recalcitrant, severe morphea.
硬斑病的一线全身治疗包括甲氨蝶呤联合或不联合全身皮质类固醇。当这种方案无效、不能耐受或禁忌时，建议试用吗替麦考酚酯（MMF）或麦考酚酸（MPA）——本文中称为麦考酚——然而，支持这一建议的证据仍然很薄弱。
评估麦考酚酯治疗硬斑病的有效性和耐受性。
设计、地点和参与者：这是一项回顾性队列研究，于 1999 年 1 月 1 日至 2018 年 12 月 31 日在 8 家机构中进行，共有 77 名硬斑病患者接受了麦考酚酯治疗。
主要结局是麦考酚酯治疗 0、3 至 6、9 至 12 个月时硬斑病的疾病活动度、严重程度和反应。次要结局是麦考酚酯是否是一种耐受良好的硬斑病治疗方法。
研究中有 61 名女性患者（79%）和 16 名男性患者（21%），发病年龄中位数为 36 岁（四分位间距，16-53 岁），诊断延迟中位数为 8 个月（四分位间距，4-14 个月）。最常见的硬斑病亚型为全身性硬斑病（37 例[48%]）、全身性硬斑病（12 例[16%]）和躯干和/或四肢的线性硬斑病（9 例[12%]）。41 例（53%）患者存在相关功能障碍，49 例（64%）患者疾病严重。12 例患者最初接受麦考酚酯单药或联合治疗，65 例患者在先前治疗无效（50/65[77%]）或不耐受（21/65[32%]）后接受麦考酚酯治疗。在接受麦考酚酯酯治疗之前，接受的治疗包括甲氨蝶呤（48/65[74%]）、全身皮质类固醇（42/65[65%]）、羟氯喹（20/65[31%]）和/或光疗（14/65[22%]）。在接受麦考酚酯酯治疗 3 至 6 个月后，73 例患者中有 66 例（22 例稳定，44 例改善）疾病稳定或改善。治疗 9 至 12 个月后，54 例患者中有 47 例（14 例稳定，33 例改善）疾病稳定或改善。27 例（35%）患者在研究结束时达到疾病缓解。同时接受的治疗很常见。麦考酚酯酯耐受性良好。胃肠道不良反应最常见（24 例[31%]）；贫血（3 例[4%]）和感染（2 例[3%]）较少见。
本研究表明，麦考酚酯是一种耐受性良好且有益的治疗难治性、严重硬斑病的药物。
