Phase II trial of methotrexate in myasthenia gravis

Mamatha Pasnoor; Jianghua He; Laura Herbelin; Mazen Dimachkie; Richard J Barohn; Muscle Study Group

doi:10.1111/j.1749-6632.2012.06804.x

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Phase II trial of methotrexate in myasthenia gravis

Journal article

Peer reviewed

Phase II trial of methotrexate in myasthenia gravis

Mamatha Pasnoor, Jianghua He, Laura Herbelin, Mazen Dimachkie, Richard J Barohn and Muscle Study Group

Annals of the New York Academy of Sciences, Vol.1275(1), pp.23-28

12/2012

DOI: 10.1111/j.1749-6632.2012.06804.x

PMCID: PMC3564221

PMID: 23278574

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Abstract

Prednisone is a frequently used treatment for myasthenia gravis (MG) but it has numerous side effects. Methotrexate is a selective inhibitor of dihydrofolate reductase and lymphocyte proliferation and is an effective immuosuppressive medication for autoimmune diseases. Given the negative results of the mycophenolate mofetil study, search for an effective immunosuppressant drug therapy is ongoing. The objective is to determine if oral methotrexate is safe and effective for MG patients who take prednisone. We have initiated a randomized, double-blind, placebo-controlled multicenter trial of methotrexate versus placebo in patients taking at least 10 mg/day of prednisone at enrollment. The methotrexate dose is increased to 20 mg and the prednisone dose is adjusted per protocol during the study. Clinical and laboratory evaluations are performed monthly for 12 months, with the primary efficacy measure being the nine-month prednisone area under the curve (AUC) from months 3 to 12. Secondary outcome measures include MG outcomes, quality of life measures, and a polyglutamation biomarker assay. A total of 18 U.S. sites and 2 Canadian sites are participating, with 48 screened cases, 42 enrolled, with 19 still active in the study.

Quality of Life

Double-Blind Method

Myasthenia Gravis - physiopathology

Humans

Immunosuppressive Agents - therapeutic use

Placebos

Methotrexate - therapeutic use

Myasthenia Gravis - drug therapy

Details

Title: Subtitle: Phase II trial of methotrexate in myasthenia gravis
Creators: Mamatha Pasnoor - University of Kansas Medical Center, Kansas City, Kansas, USA. mpasnoor@kumc.edu
Jianghua He
Laura Herbelin
Mazen Dimachkie
Richard J Barohn
Muscle Study Group
Contributors: Andrea Swenson (Contributor) - University of Iowa, Neurology
Resource Type: Journal article
Publication Details: Annals of the New York Academy of Sciences, Vol.1275(1), pp.23-28
DOI: 10.1111/j.1749-6632.2012.06804.x
PMID: 23278574
PMCID: PMC3564221
NLM abbreviation: Ann N Y Acad Sci
ISSN: 0077-8923
eISSN: 1749-6632
Publisher: Wiley; United States
Grant note: R01 FD003538 / FDA HHS UL1 TR 000001 / NCATS NIH HHS UL1 TR000001 / NCATS NIH HHS UL1 RR033179 / NCRR NIH HHS R01 FD 003538 / FDA HHS UL1 RR 033179 / NCRR NIH HHS
Language: English
Date published: 12/2012
Academic Unit: Neurology
Record Identifier: 9984020620702771

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