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1. Muscle Nerve. 2016 May;53(5):717-25. doi: 10.1002/mus.25070. Epub 2016 Mar 3.
Amifampridine phosphate (Firdapse(®) ) is effective and safe in a phase 3 clinical trial in LEMS.
Oh SJ(1), Shcherbakova N(2), Kostera-Pruszczyk A(3), Alsharabati M(1), Dimachkie M(4), Blanco JM(5), Brannagan T(6), Lavrnić D(7), Shieh PB(8), Vial C(9), Meisel A(10), Komoly S(11), Schoser B(12), Sivakumar K(13), So Y(14); LEMS Study Group.
(1)Department of Neurology, University of Alabama at Birmingham, Birmingham, Alabama, USA. (2)Research Center of Neurology, Moscow, Russia. (3)Department of Neurology, Medical University of Warsaw, Poland. (4)University of Kansas Medical Center, Kansas City, Kansas, USA. (5)Gregorio Maranon Hospital, Madrid, Spain. (6)Columbia University Medical Center, New York, New York, USA. (7)Clinical Center of Serbia, Clinic of Neurology, Belgrade, Serbia. (8)Department of Neurology, University of California, Los Angeles, California, USA. (9)Hospital of Lyon, ENMG Service and Neuromuscular Pathology Hospital, Lyon, France. (10)Charite Universitatsmedizin Berlin-NeuroCure Clinical Research Center, Berlin, Germany. (11)University of Pécs, Department of Neurology, Pécs, Hungary. (12)Ludwig-Maximilians-University Munich Friedrich-Baur-Institute, Munich, Germany. (13)Neuromuscular Research Center, Phoenix, Arizona, USA. firstname.lastname@example.org. (14)Stanford University, Stanford, California, USA.
Muscle Nerve 53: 717-725, 2016.
We evaluated the efficacy and safety of amifampridine phosphate (Firdapse(®) ) for symptomatic treatment in Lambert-Eaton myasthenic syndrome
Phase 3, randomized, double-blind, study. Patients were treated initially with amifampridine phosphate for 7-91 days, followed by randomization
to continue amifampridine phosphate for 14 days or placebo (7-day taper, 7-day placebo). The primary efficacy endpoints were changes from aseline at day 14 in Quantitative Myasthenia Gravis and Subject Global Impression scores.
The coprimary efficacy end points and 1 of the secondary efficacy end points were met, showing a significant benefit of aminfampridine phosphate over placebo at Day 14. All 5 primary, secondary, and tertiary endpoints achieved statistical significance at Day 8. Amifampridine phosphate was well tolerated; the most common adverse events were oral and digital paresthesias, nausea, and headache.
This study provides Class I evidence of efficacy of amifampridine phosphate as a symptomatic treatment for LEMS.
© 2016 Wiley Periodicals, Inc.
PMID: 26852139 [PubMed - in process]