title: Pilot trial of simvastatin in the treatment of sporadic inclusion-body myositis.
abstract: Sporadic inclusion-body myositis (s-IBM) is a chronic progressive inflammatory myopathy leading to severe disability. It has been suggested that statins may benefit s-IBM patients based on their pleiotropic effects on autoimmunity and possible adverse influence of increased cholesterol on muscle pathological changes. We carried out a pilot, open-label trial to evaluate safety and tolerability of oral simvastatin in s-IBM patients. Fourteen patients were treated with 40 mg of simvastatin over 12 months. Primary outcome measures included the assessment tools proposed by International Myositis Outcome Assessment Collaborative Study group and the IBM-Functional Rating Scale. As additional data, we report the results obtained from muscle MRI, biopsy and oropharyngeal scintigraphy. Ten patients completed the trial and the treatment appeared safe and well tolerated. None of the patients showed a significant clinical improvement. Outcome measures used in this study proved to be valuable tools for global assessment of s-IBM patients. At present, we cannot recommend simvastatin as a treatment for s-IBM though our data may warrant a placebo-controlled study.
authors.affiliation: Department of Neurosciences, Catholic University School of Medicine, Policlinico A Gemelli, Largo A Gemelli 8, 00168 Rome, Italy.
authors.forename: Cristina
authors.initials: C
authors.lastname: Sancricca
authors.forename: Marina
authors.initials: M
authors.lastname: Mora
authors.forename: Enzo
authors.initials: E
authors.lastname: Ricci
authors.forename: Pietro Attilio
authors.initials: PA
authors.lastname: Tonali
authors.forename: Renato
authors.initials: R
authors.lastname: Mantegazza
authors.forename: Massimiliano
authors.initials: M
authors.lastname: Mirabella
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D015551:Autoimmunity / Q000276:immunology; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000008:administration & dosage / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D018979:Myositis, Inclusion Body / Q000188:drug therapy* / Q000276:immunology; D010865:Pilot Projects; D019821:Simvastatin / Q000008:administration & dosage / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D019821:Simvastatin