Neurological Sciences

, Volume 32, Issue 5, pp 841–847 | Cite as

Pilot trial of simvastatin in the treatment of sporadic inclusion-body myositis

  • Cristina Sancricca
  • Marina Mora
  • Enzo Ricci
  • Pietro Attilio Tonali
  • Renato Mantegazza
  • Massimiliano Mirabella
Original Article
First Online:
Received:
Accepted:

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.

Keywords

Sporadic inclusion-body myositis Simvastatin Pilot clinical trial 
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Notes

Acknowledgments

This study was supported by grants from Istituto Superiore di Sanità (“Rare Diseases Italy-USA program” ISS 526D/15 grant), Don Carlo Gnocchi ONLUS Foundation, and Catholic University.

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Copyright information

© Springer-Verlag 2011

Authors and Affiliations

  • Cristina Sancricca
    • 1
    • 2
  • Marina Mora
    • 3
  • Enzo Ricci
    • 1
    • 2
  • Pietro Attilio Tonali
    • 1
    • 2
  • Renato Mantegazza
    • 3
  • Massimiliano Mirabella
    • 1
    • 2
  1. 1.Department of NeurosciencesCatholic University School of MedicineRomeItaly
  2. 2.Don Carlo Gnocchi ONLUS FoundationMilanItaly
  3. 3.Istituto Nazionale Neurologico “Carlo Besta”MilanItaly

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