We found a match
Your institution may have rights to this item. Sign in to continue.
- Title
Evaluation of the Effectiveness and Tolerability of Mycophenolate Mofetil and Mycophenolic Acid for the Treatment of Morphea.
- Authors
Arthur, Megan; Fett, Nicole M.; Latour, Emile; Jacobe, Heidi; Kunzler, Elaine; Florez-Pollack, Stephanie; Houser, Jacob; Sharma, Shivani; Prasad, Smriti; Femia, Alisa; Stern, Marleigh J.; Pappas-Taffer, Lisa K.; Gaffney, Rebecca; Fernandez, Anthony P.; Knabel, Daniel; Cardones, Adela Rambi; Leung, Nicole; Laumann, Anne; Yu, Jeong Min; Zhao, Jeffrey
- Abstract
<bold>Importance: </bold>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.<bold>Objective: </bold>To evaluate the effectiveness and tolerability of mycophenolate for the treatment of morphea.<bold>Design, Setting, and Participants: </bold>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.<bold>Main Outcomes and Measures: </bold>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.<bold>Results: </bold>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.<bold>Conclusions and Relevance: </bold>This study suggests that mycophenolate is a well-tolerated and beneficial treatment of recalcitrant, severe morphea.
- Subjects
RESEARCH; ADRENOCORTICAL hormones; RESEARCH methodology; MYCOPHENOLIC acid; RETROSPECTIVE studies; EVALUATION research; MEDICAL cooperation; METHOTREXATE; TREATMENT effectiveness; COMPARATIVE studies; SCLERODERMA (Disease); IMMUNOSUPPRESSIVE agents; HYDROXYCHLOROQUINE; LONGITUDINAL method; DRUG administration; DRUG dosage
- Publication
JAMA Dermatology, 2020, Vol 156, Issue 5, p521
- ISSN
2168-6068
- Publication type
journal article
- DOI
10.1001/jamadermatol.2020.0035