Title |
Intravenous immunoglobulin maintenance treatment in myasthenia gravis: A randomized, controlled trial sample size simulation
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Published in |
Muscle & Nerve, October 2014
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DOI | 10.1002/mus.24259 |
Pubmed ID | |
Authors |
Christian Eienbröker, Florian Seitz, Anna Spengler, Heike Kurz, Maria Seipelt, Norbert Sommer, Wolfgang H. Oertel, Nina Timmesfeld, Björn Tackenberg |
Abstract |
Introduction: In case of exacerbation or crisis myasthenia gravis (MG) patients can be treated with intravenous immunoglobulin (IVIG), plasmapheresis, or immunoadsorption. However, IVIG efficacy data in maintenance treatment are sparse. Methods: We observed n=16 index patients prospectively with chronic and insufficiently controlled MG under standard immunosuppressants and symptomatic treatment. IVIg treatment response was measured using changes in QMG and surrogates. Based on these results a sample size calculation for a future RCT was simulated. Results: There was an enduring decline in the QMG score and other parameters of about 50% under IVIg maintenance treatment. RCT sample size calculation results in n=73 or n=33 patients per arm to detect at least a 20% vs. 30% clinical difference in the QMG score. Conclusion: We recommend using the QMG score as a primary endpoint for an RCT of IVIg maintenance for chronic MG. © 2014 Wiley Periodicals, Inc. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Unknown | 1 | 100% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 1 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 47 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Researcher | 11 | 23% |
Other | 5 | 11% |
Professor > Associate Professor | 5 | 11% |
Student > Master | 4 | 9% |
Student > Ph. D. Student | 3 | 6% |
Other | 8 | 17% |
Unknown | 11 | 23% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 19 | 40% |
Neuroscience | 7 | 15% |
Agricultural and Biological Sciences | 3 | 6% |
Pharmacology, Toxicology and Pharmaceutical Science | 2 | 4% |
Engineering | 2 | 4% |
Other | 5 | 11% |
Unknown | 9 | 19% |