title: Use of Mycophenolate Mofetil in Patients with Severe Localized Scleroderma Resistant or Intolerant to Methotrexate.
abstract: To assess the efficacy and safety of mycophenolate mofetil (MMF) in patients with localized scleroderma (LoS) resistant or intolerant to previous treatment with methotrexate (MTX). A case series of patients with LoS treated with MMF. Outcome was assessed through clinical examination. Adverse events were documented. Seven patients with LoS were treated with MMF. Median age at MMF initiation was 15 years (range 7-74 years). Three patients received MMF due to MTX ineffectiveness and 4 due to MTX intolerance. Disease remission was achieved in 4 patients and maintained in one patient. One patient showed a favourable response, but had to discontinue treatment due to elevated liver enzymes. The remaining patient experienced disease progression. MMF was shown to improve the clinical condition of patients with refractory LoS and may be a relatively safe alternative in patients who are intolerant to MTX.
authors.affiliation: Department of Dermatology, Radboud University Medical Center, 6525 GL Nijmegen, The Netherlands. Jorre.Mertens@radboudumc.nl.
authors.forename: Jorre S
authors.initials: JS
authors.lastname: Mertens
authors.forename: Diane
authors.initials: D
authors.lastname: Marsman
authors.forename: Peter C M
authors.initials: PC
authors.lastname: van de Kerkhof
authors.forename: Esther P A H
authors.initials: EP
authors.lastname: Hoppenreijs
authors.forename: Hanneke K A
authors.initials: HK
authors.lastname: Knaapen
authors.forename: Timothy R D
authors.initials: TR
authors.lastname: Radstake
authors.forename: Elke M G J
authors.initials: EM
authors.lastname: de Jong
authors.forename: Marieke M B
authors.initials: MM
authors.lastname: Seyger
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D018450:Disease Progression; D004351:Drug Resistance; D057915:Drug Substitution; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects* / Q000627:therapeutic use*; D008297:Male; D008727:Methotrexate / Q000009:adverse effects*; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000009:adverse effects / Q000627:therapeutic use*; D012074:Remission Induction; D012189:Retrospective Studies; D012594:Scleroderma, Localized / Q000188:drug therapy* / Q000473:pathology; D012720:Severity of Illness Index; D012867:Skin / Q000187:drug effects* / Q000473:pathology; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid; D008727:Methotrexate