Abstract
BackgroundCurrently, there are only few studies (mostly case reports or case series) on mycophenolate mofetil (MMF) in patients with systemic autoimmune myopathies (SAM). Therefore, the goal of the present study was to evaluate the safety and efficacy of MMF (monotherapy or coadjuvant drug) in a specific sample of patients with refractory SAM: dermatomyositis, polymyositis, anti-synthetase syndrome or clinically amyopathic dermatomyositis.MethodsA case series including 20 consecutive adult patients with refractory SAM from 2010 to 2016 was conducted. After the introduction of MMF, associated or not with other drugs, the patients were followed for 6 consecutive months.ResultsIn 17 out of 20 patients MMF was introduced without any intolerance. The clinical symptoms evaluated in these patients were muscular, cutaneous and/or pulmonary activity. During the 6-month follow-up, 11 out of 17 patients had clinical and laboratory activities response with MMF, allowing significant tapering of the prednisone median dose (15 vs. 5 mg/day, P=0.005). On the other hand, in three out of 20 patients; MMF was discontinued in less than two months, because of gastrointestinal intolerance. There were no cases of serious infection or death.ConclusionsMMF was relatively well-tolerated, safe and effective in patients with refractory SAM. Further studies are needed to confirm the data found.
Highlights
There are only few studies on mycophenolate mofetil (MMF) in patients with systemic autoimmune myopathies (SAM)
MMF treatment was defined as effective when the drug promoted over 50% improvement in the initial: cutaneous; muscular and/or laboratory parameters]; articular or pulmonary activity
Twenty consecutive patients with refractory SAM treated with MMF were initially analyzed
Summary
There are only few studies (mostly case reports or case series) on mycophenolate mofetil (MMF) in patients with systemic autoimmune myopathies (SAM). As limitations, the majority of these studies are case reports or case series [10, 12, 13, 15,16,17,18, 20] and analyzed only SAM patients with pulmonary disease activity [15, 19, 20] Those who used rituximab [10,11,12,13,14,15,16,17, 19, 20] or anti-synthetase syndrome (ASS) patients [10,11,12,13,14,15,16,17,18,19,20] have not been studied
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