An international multicenter efficacy and safety study of IqYmune in initial and maintenance treatment of patients with chronic inflammatory demyelinating polyradiculoneuropathy: PRISM study.


Journal

Journal of the peripheral nervous system : JPNS
ISSN: 1529-8027
Titre abrégé: J Peripher Nerv Syst
Pays: United States
ID NLM: 9704532

Informations de publication

Date de publication:
12 2020
Historique:
received: 28 05 2020
revised: 07 08 2020
accepted: 10 08 2020
pubmed: 19 8 2020
medline: 28 9 2021
entrez: 19 8 2020
Statut: ppublish

Résumé

This prospective, multicenter, single-arm, open-label phase 3 study aimed to evaluate the efficacy and safety of IqYmune in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Patients received one induction dose of 2 g/kg and then seven maintenance doses of 1 g/kg at 3-week intervals. The primary endpoint was the responder rate at the end of study (EOS), defined as an improvement of ≥1 point on the adjusted inflammatory neuropathy cause and treatment (INCAT) disability scale. The responder rate was compared with the responder rate of a historical placebo group (33.3%). Secondary endpoints included changes from baseline to EOS of adjusted INCAT disability score, grip strength, Medical Research Council (MRC) sum score, Rasch-modified MRC sum score, Rasch-built overall disability scale score and the clinical global impression. Forty-two patients, including 23 Ig-naïve and 19 Ig-pre-treated, were included in the efficacy set. The overall response rate at EOS was 76.2% (95% confidence interval [60.5%-87.9%]). The superiority of IqYmune compared to the historical placebo control was demonstrated (P < .0001). The responder rate was numerically higher in Ig-pre-treated than in Ig-naïve patients but confidence intervals were overlapping (84.2% [60.4%-96.6%] vs 69.6% [47.1%-86.8%]). All secondary endpoints confirmed this conclusion. The median time to response was 15 weeks [8.9-19.1 weeks]. A total of 156 adverse events including five serious were considered related to IqYmune, 87.2% were mild. Neither hemolysis nor signs of renal or hepatic impairment were observed. These results demonstrate that IqYmune is an effective and well-tolerated treatment in patients with CIDP.

Identifiants

pubmed: 32808406
doi: 10.1111/jns.12408
pmc: PMC7754365
doi:

Substances chimiques

Immunoglobulins, Intravenous 0
Immunologic Factors 0

Types de publication

Clinical Trial, Phase III Journal Article Multicenter Study Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

356-365

Informations de copyright

© 2020 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals LLC on behalf of Peripheral Nerve Society.

Références

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Auteurs

Eduardo Nobile-Orazio (E)

Neuromuscular and Neuroimmunology Service, IRCCS Humanitas Clinical and Research Center, Milan University, Via Manzoni 56, Rozzano, Milan, 20089, Italy.

Fabrice Kasiborski (F)

LFB, Les Ulis, France.

Gilles Della Corte (GD)

Dellmed Consulting, Ornex, France.

Robert Bonek (R)

Department of Neurology, NeuroCenter, Regional Specialist Hospital, Bydgoszcz, Poland.

Dario Cocito (D)

Istituti Clinici Scientifici Maugeri, Turin, Italy.

Angelo Schenone (A)

Department of Neurosciences, Rehabilitation, Ophthalmology, Genetic and Maternal and Infantile Sciences (DINOGMI), University of Genova and IRCCS Policlinico San Martino, Genova, Italy.

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Classifications MeSH