Mycophenolate mofetil for methotrexate-resistant juvenile localized scleroderma
Autor: | Giorgia Martini, Laura Saggioro, Francesco Zulian, Alessandra Meneghel, Roberta Culpo, Fabio Vittadello |
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Jazyk: | angličtina |
Rok vydání: | 2020 |
Předmět: |
musculoskeletal diseases
Male medicine.medical_specialty Pansclerotic Morphea Kaplan-Meier Estimate Mycophenolate Gastroenterology methotrexate Scleroderma Localized Rheumatology Refractory Internal medicine Medicine Humans Pharmacology (medical) Linear Scleroderma Longitudinal Studies Treatment Failure Mychophenolate mofetil Localized Scleroderma skin and connective tissue diseases Child AcademicSubjects/MED00360 Retrospective Studies treatment business.industry juvenile localized scleroderma Clinical Science Mycophenolic Acid medicine.disease Treatment Outcome Thermography Antirheumatic Agents Juvenile Localized Scleroderma Methotrexate Female business Anaphylaxis medicine.drug |
Zdroj: | Rheumatology (Oxford, England) |
ISSN: | 1462-0332 1462-0324 |
Popis: | ObjectivesTo investigate safety and efficacy of MMF in patients with severe or MTX-refractory juvenile localized scleroderma.MethodsConsecutive juvenile localized scleroderma patients undergoing systemic treatment were included in a retrospective longitudinal study. Patients treated with MMF because they were refractory or intolerant to MTX (MMF-group) were compared with responders to MTX (MTX-group). Disease activity was assessed by Localized Scleroderma Cutaneous Assessment Tool and thermography. Disease course was established on the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan–Meier analysis.ResultsMMF and MTX groups included 22 and 47 patients, respectively. No significant difference in demographics, follow-up duration and treatment before diagnosis was observed between groups. The most represented clinical subtypes in the MMF-group were pansclerotic morphea and mixed subtype (P = 0.008 and P = 0.029, respectively), and linear scleroderma of the face in the MTX-group (P = 0.048). MMF was started because of MTX resistance (18 patients), relapse during MTX tapering/withdrawal (3 patients) and anaphylaxis to MTX (1 patient). After mean 9.4 years of follow-up, 90.9% of patients on MMF and 100% of those on MTX had inactive disease. No significant difference in relapse-free survival between the groups was found (P = 0.066, log-rank test), although MMF likely induced more persistent remission. MMF was well tolerated and combination of MMF and MTX did not increase its efficacy.ConclusionThe present study adds strong evidence on the efficacy and tolerance of MMF in severe and/or MTX-refractory juvenile localized scleroderma. Further controlled studies are needed to prove its efficacy as first line treatment. |
Databáze: | OpenAIRE |
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