Successful treatment of a patient with severe refractory myasthenia gravis using mycophenolate mofetil

• DOI: 10.1212/wnl.51.3.912-a
• Publication Date: 2012-05-13T13:35:51Z
• Authors: Robert A. Hauser; Ali R. Malek; Raquel Rosen
• Keywords: Mycophenolate; Refractory (planetary science)

Mycophenolate mofetil is an immunosuppressive agent that inhibits the de novo pathway of guanosine nucleotide synthesis critical for B- and T-lymphocyte proliferation.1 It absorbed rapidly undergoes presystemic metabolism to mycophenolic acid-a potent, selective, noncompetitive, reversible inhibitor inosine monophosphate dehydrogenase.2 We present a patient with severe refractory myasthenia gravis (MG) who experienced excellent response mycophenolate mofetil.

A 14-year-old girl initially noticed dysarthria on sustained speech. A year later she intermittent ptosis; face, neck, limb weakness; drooling; difficulty chewing swallowing; shortness breath. Acetylcholine receptor antibody levels, Tensilon (Zeneca Pharmaceuticals; Wilmington, DE) test, repetitive nerve stimulation were all positive consistent diagnosis MG.

The was placed escalating doses pyridostigmine without benefit. She suffered cholinergic crisis pyridostigmine, 1,800 mg/d, required hospitalization mechanical ventilation. Pyridostigmine then discontinued. subsequently retried several times over ensuing years but …