Neuropathic pain poses a significant epidemiological, social and financial burden, as approximately 10% of the population suffers from neuropathic pain. Neuropathic pain can be distinguished from ...mechanical or nociceptive pain by its qualities, distribution, and in some cases, diagnostic testing. There are currently over 200 known causes of peripheral neuropathy, with diabetes being the most common. However, in up to 40% of individuals no identifiable cause can be found. In this chapter, the different types, mechanisms, workup and etiology, categorization (e.g. focal or diffuse, large or small fiber), and treatment of neuropathic pain are discussed.
Objective
We evaluated the efficacy and safety of amifampridine phosphate (Firdapse®) for symptomatic treatment in Lambert‐Eaton myasthenic syndrome (LEMS).
Methods
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 baseline at day 14 in Quantitative Myasthenia Gravis and Subject Global Impression scores.
Results
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.
Conclusions
This study provides Class I evidence of efficacy of amifampridine phosphate as a symptomatic treatment for LEMS. Muscle Nerve 53: 717–725, 2016
We report the fourth case of transthyretin amyloidosis (ATTR) Ser23Asn in a 41-year-old Ecuadorian male. He has a pedigree that spans seven generations and involves 24 family members who suffered ...early cardiac death. Salient presenting symptoms were fatigue, shortness of breath, and peripheral neuropathy. The diagnosis of cardiac amyloid was confirmed by immunohistochemical staining of an endomyocardial biopsy, genotyping and by technetium pyrophosphate (
99m
Tc-PYP) scintigraphy, which remains to be established as a reliable tool to visualize myocardial amyloid involvement in patients with the Ser23Asn transthyretin (TTR) variant. The patient underwent successful combined heart and liver transplant. We add to the current ATTR literature that in patients with the rare Ser23Asn mutation, peripheral nerve in addition to cardiac involvement can occur and
99m
Tc-PYP scintigraphy can be used as an imaging modality to visualize myocardial amyloid.
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