16th International Congress on Neuromuscular Diseases, 21 - 22 & 28 - 29 May 2021 Virtual, Worldwide

2021 Journal of Neuromuscular Diseases  
This prospective, multicenter, single-arm, open-label phase 3 study aimed to evaluate the effi cacy and safety of IqYmune® (10% liquid human intravenous immunoglobulin (IVIg)) in patients with chronic infl ammatory demyelinating polyneuropathy (CIDP). Patients received one induction dose of 2 g/kg and then 7 maintenance doses of 1 g/kg at 3-week intervals. The primary effi cacy endpoint was the responder rate at the end of study, defi ned as an improvement of 1 point on the adjusted INCAT
more » ... ility scale. The responder rate was tested against the responder rate of a historical placebo group (33.3%) with an exact 1-sided binomial Clopper-Pearson test. Forty-three (43) patients with CIDP (21-79 years) were enrolled and treated. Among them, 42 were included in the effi cacy set. The overall response rate at the end of study was 76.2% with a 95% confi dence interval (CI) of [60.5 -87.9%] (exact 2-sided Clopper-Pearson). The superiority of IqYmune® compared to the historical placebo control was statistically signifi cant (p-value <0.0001). The median time to response was 15 weeks with a 95% CI of [8.9 -19.1%] (estimated by the Kaplan-Meier method taking into account nonresponder patients). The secondary effi cacy endpoints included the changes from baseline to the end of study of adjusted INCAT disability score, grip strength in the dominant and non-dominant hand, Rasch-built overall disability scale (R-ODS) score, Medical research council (MRC) sum score, Raschmodifi ed MRC sum score and clinical global impression of severity of illness, global improvement and effi cacy index. An improvement was shown for Abstracts S5 Serum samples were screened for immunoglobulin G(IgG), IgA and IgM antibodies against C.jejuni using the ELISA (ELISA kits, Serion, Germany) and anti-gangliosid antibodies, including IgG and IgM, were assessed for each of the antigens with the Euroimmun kit. Results: The patients were 10-85 years old and had a mean age of 48.14±19.06 years. Infection was observed in approximately 30% of the patients, and acute infl ammatory demyelinating polyneuropathy (AIDP) as the most prevalent type of GBS in 48.8%. Only 7% of the patients were positive for ganglioside antibodies and the patients positive for the campylobacter IgG antibody accounted for about 4.7% of the subjects and those positive for IgA antibody about 7%. Type of GBC was found not to be significantly related to Campilo IgG (P=0.7) and IgA (P=0.9). The relationships of gangiloside with Campylo IgG (P=0.7) and IgA (P=0.6) were also found to be insignifi cant. Conclusion: The present fi ndings suggested a low frequency of C.jejuni antibody in the GBS patients. Type of GBS was also found not to be correlated with positive serologic C.jejuni antibody and gangloside antibody.
doi:10.3233/jnd-219006 pmid:34334417 fatcat:5ixajewia5d3hhpkwj4yqyb32a