Lu sur BMJOpen http://bmjopen.bmj.com/content/5/7/e007863.abstract
Pour traduire cette publication, utilisez la fonction incluse dans le message original du forum http://www.myasthenie.comEphedrine as add-on therapy for patients with myasthenia gravis: protocol for a series of randomised, placebo-controlled n-of-1 trials
Charlotte Vrinten 1,2, Alexander F Lipka 3, Erik W van Zwet 4, Kirsten J M Schimmel 5, Martina C Cornel 2, Marja R Kuijpers 6, Yechiel A Hekster 7, Stephanie S Weinreich 2, Jan J G M Verschuuren 3
Correspondence to Charlotte Vrinten; email@example.com
- Department of Epidemiology and Public Health, University College London, London, UK
- Department of Clinical Genetics, EMGO Institute for Health and Care Research, VU University Medical Center, Amsterdam, The Netherlands
- Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands
- Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands
- Department of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands
- Department of Care, Section Pharmaceutical Care, National Health Care Institute, Diemen, The Netherlands
- Department of Pharmacy, Radboud University Medical Center, Nijmegen, The Netherlands
Received 4 February 2015
Revised 8 April 2015
Accepted 12 April 2015
Published 16 July 2015
Myasthenia gravis (MG), a rare neuromuscular disease, is often initially treated using acetylcholinesterase inhibitors. Patients who do not respond adequately depend on the use of corticosteroids or other immunosuppressive medication, but these may have serious side effects. Clinical observations suggest that ephedrine can diminish, postpone or even prevent the need for immunosuppressive therapy when added to acetylcholinesterase inhibitors or low-dose prednisone. In the Netherlands, ephedrine is not licensed for MG nor is reimbursement guaranteed. MG is a rare condition, and ephedrine might be indicated only in a subset of patients. Thus, randomised controlled trials comparing large groups are difficult to conduct. We, therefore, aim to aggregate data from a small series of n-of-1 trials (also known as single patient trials) to assess the effect of ephedrine as add-on treatment for MG.
Methods and analysis
Single-centre, placebo-controlled, double-blind, randomised, multiple crossover n-of-1 studies in 4 adult patients with generalised MG who show inadequate improvement on pyridostigmine and/or immunosuppressive drugs. Each n-of-1 trial has 3 cycles of two 5-day intervention periods. Treatment: 25 mg ephedrine or placebo, twice daily. Main outcome measure: Quantitative Myasthenia Gravis (QMG) test. Statistical analysis: fixed effects linear model for QMG for all patients combined. Secondary outcome measures: Clinical: effects on MG-Composite and MG-Activities of Daily Living (MG-ADL) scales; QMG at individual level; adverse events. Acceptability of trial design: number of patients eligible and enrolled; number of treatment cycles completed; patients’ and caregivers’ experiences.
Ethics and dissemination
This study was approved by the Medical Ethics Committee of Leiden University Medical Center, No. P14.108. Results of the trial will be reported in a peer-reviewed publication. Regulatory stakeholders will comment on the suitability of the trial for market authorisation and reimbursement purposes.
Trial registration number
This study is registered under EudraCT number 2014-001355-23, protocol no. 40960, V.1.0, registration date 27 March 2014.
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