Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension

OBJECTIVE: To evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension. METHODS: Patients who completed the REGAIN randomized controlled trial and continued into the open-label extension were included in this tertiary endpoint analysis. Patients were assessed for the MGFA post-intervention status of improved, unchanged, worse, MM, and pharmacologic remission at defined time points during REGAIN and through week 130 of the open-label study. RESULTS: A total of 117 patients completed REGAIN and continued into the open-label study (eculizumab/eculizumab: 56; placebo/eculizumab: 61). At week 26 of REGAIN, more eculizumab-treated patients than placebo-treated patients achieved a status of improved (60.7% vs 41.7%) or MM (25.0% vs 13.3%; common OR: 2.3; 95% CI: 1.1-4.5). After 130 weeks of eculizumab treatment, 88.0% of patients achieved improved status and 57.3% of patients achieved MM status. The safety profile of eculizumab was consistent with its known profile and no new safety signals were detected. CONCLUSION: Eculizumab led to rapid and sustained achievement of MM in patients with AChR+ refractory gMG. These findings support the use of eculizumab in this previously difficult-to-treat patient population. CLINICALTRIALSGOV IDENTIFIER: REGAIN, NCT01997229; REGAIN open-label extension, NCT02301624. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that, after 26 weeks of eculizumab treatment, 25.0% of adults with AChR+ refractory gMG achieved MM, compared with 13.3% who received placebo

Minimal Symptom Expression' in Patients With Acetylcholine Receptor Antibody-Positive Refractory Generalized Myasthenia Gravis Treated With Eculizumab

The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension

A case with Myoshi myopathy and retinitis pigmentosa

11th International Congress on Neuromuscular Diseases -- JUL 02-07, 2006 -- Istanbul, TURKEYWOS: 00023922940017

Pregabalin in Chronic Pelvic Pain

Objective: In women, chronic pelvic pain (CPP) is a common complaint, affecting as many as 15% of women. CPP may originate from both gynaecologic and non-gynaecologic causes, including urological, gastrointestinal, psychiatric and neuromuscular disorders. In CPP, the pain has a neuropathic component because of its chronic nature. This creates difficulty in the treatment of CPP. Thus, we examined both neuropathic pain component and effect of pregabalin in women with CPP

Diaphragm pacing stimulation system in patient with central alveolar hypo ventilation: case report and first experience in Turkey

This article aims to document the efficiency of diaphragm pacing stimulation system (DPSS) in a patient diagnosed with central alveolar hypoventilation and dependent to mechanical ventilation (MV). A 65-year-old male patient who developed respiratory failure due to sepsis secondary to pulmonary infection was monitored in intensive care with MV. Although all septic symptoms recovered, patient became dependant to MV since respiration was not triggered centrally. DPSS was implemented in a patient with laparoscopic surgery for the first time in Turkey. Patient started using DPSS as of postoperative second day and completely separated from MV dramatically. Laparoscopic DPSS can be performed safely under general anesthesia may improve similar patients' respiratory and other clinic conditions as well as health related quality of life. Therefore, DPSS may be used more commonly in patients with central alveolar hypoventilation

Vestibular impairment in chronic inflammatory demyelinating polyneuropathy

29th Meeting of the Barany-Society -- 2016 -- Seoul, SOUTH KOREAWOS: 000424711600019PubMed ID: 29260355Chronic inflammatory demyelinating polyneuropathy (CIDP) is a common, treatable, autoimmune peripheral neuropathy considered to produce imbalance by weakness and proprioceptive impairment rather than vestibular impairment. We measured semicircular canal vestibular function in 21 CIDP patients (15M/6F) by the video head impulse test and postural stability with a battery comprising the modified Clinical Test of Sensory Integration and Balance, the Berg Balance Scale, the Dynamic Gait Index, the Fall Efficiency Scale, and the International Cooperative Ataxia Rating Scale. Of the 21 patients, 16 had vestibular impairment, ranging from mild-affecting just a single semicircular canal, to severe-affecting all 6 canals. Although the severity of the vestibular impairment did not correlate either with the severity of the postural imbalance or of the peripheral neuropathy, our data show that vestibular impairment is an additional challenge to balance that some CIDP patients will face.Barany SocDokuz Eylul University Scientific Research Coordination Unit [2014.KB.SAG.42]Dokuz Eylul University Scientific Research Coordination Unit (Project 2014.KB.SAG.42)

Expert opinion on the diagnostic odyssey and management of late-onset Pompe disease: a neurologist's perspective

This consensus statement by a panel of neurology experts aimed to provide a practical and implementable guidance document to assist clinicians with the best clinical practice in terms of diagnosis, treatment, and monitoring of late-onset Pompe disease (LOPD). The participating experts consider the clinical suspicion of LOPD by the physician to be of utmost importance in the prevention of diagnostic and therapeutic delay in LOPD patients. A diagnostic algorithm is proposed to facilitate the diagnosis of LOPD in patients presenting with unexplained proximal/axial weakness (with or without respiratory symptoms) or restrictive respiratory insufficiency with hyperCKemia and/or exercise intolerance as the red flag symptoms/signs that raise the index of suspicion for LOPD diagnosis. The diagnosis is based on the subsequent use of dried blood spot (DBS) assay, and the DBS assay can be confirmed by acid alpha-glucosidase (GAA) tissue analysis in leukocytes, fibroblasts, or muscle fibers and/or genetic mutation analysis. Accordingly, experts consider increased awareness among physicians about potential presenting characteristics with a high index of suspicion for LOPD to be crucial to suspect and consider LOPD in the differential diagnosis, while strongly suggesting the use of a diagnostic algorithm combined with DBS assay and confirmatory tests in the timely diagnosis of LOPD and implementation of best practice patterns