Longterm followup of cipaglucosidase alfa/miglustat in ambulatory patients with late-onset pompe disease: an open-label phase i/ii study (atb200-02)

Barry Byrne,Benedikt Schoser,Priya Kishnani, Drago Bratkovic,Paula Clemens,Ozlem Goker-Alpan,Xue Ming,Mark Roberts, Matthias Vorgerd, Kumaraswamy Sivakumar,Ans van der Ploeg,Vipul Jain, Sheela Sitaraman, Yasmine Wasfi,Tahseen Mozaffar

Neuromuscular Disorders(2022)

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摘要
Somatosensory evoked potentials (SEPs) are widely used for the diagnosis and evaluation of neuromyelitis optica spectrum disorder (NMOSD) and multiple sclerosis (MS). However, whether the parameters of tibial nerve SEPs can help to distinguish NMOSD from MS remains unclear. Thus, the aim of this study was to investigate the utility of tibial nerve SEP parameters in differentiating patients with NMOSD and MS.The clinical data of patients with NMOSD or MS treated in our institution between 2005 and 2021 were retrospectively extracted from our electronic database. Additional inclusion criteria were presentation with sensory symptoms in the lower extremities with corresponding lesions in the magnetic resonance images as well as available data on anti-aquaporin-4 antibodies and tibial nerve SEPs. The Z-scores of the N21–P38 interval (central sensory conduction time), P38 latency, and P38 amplitude were compared between the patients with NMOSD and MS. The relationship of disease severity with the parameters of the tibial nerve SEPs was also evaluated.Twenty patients with NMOSD and 13 patients with MS were enrolled. The Z-scores of the N21–P38 interval and P38 latency were significantly higher in the MS group than in the NMOSD group (p < 0.05 and p < 0.01, respectively), whereas there was no difference in the Z-scores of the P38 amplitude between the two groups. In the MS group, only the N21–P38 interval and P38 latency were significantly correlated with disease severity (p < 0.05 and p < 0.01, respectively). In contrast, none of the tibial nerve SEP parameters were significantly correlated with disease severity in the NMOSD group.Evaluation of the N21–P38 interval and P38 latency in tibial nerve SEPs potentially helps in differentiating between NMOSD and MS.
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关键词
pompe disease,cipaglucosidase alfa/miglustat,long-term,open-label
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