1. Prospective open-label trial with rituximab in patients with chronic inflammatory demyelinating polyradiculoneuropathy not responding to conventional immune therapies.
- Author
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Doneddu PE, Cocito D, Fazio R, Benedetti L, Peci E, Liberatore G, Falzone YM, Germano F, Gallia F, Giannotta C, Lleixà C, Bianchi E, and Nobile-Orazio E
- Subjects
- Humans, Male, Female, Middle Aged, Prospective Studies, Adult, Aged, Treatment Outcome, Neural Conduction drug effects, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating drug therapy, Rituximab therapeutic use, Immunologic Factors therapeutic use
- Abstract
Background: To evaluate the efficacy of rituximab in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) patients not responding to conventional immune therapies., Methods: An open-label, prospective exploratory study was conducted with intravenous rituximab on 17 CIDP patients who had not responded to at least two first-line therapies. The primary endpoint was to determine the proportion of patients who showed improvement 6 months after rituximab therapy. The percentage of responders to rituximab, along with a 95% CI, was reported and compared with the 30% response rate after other immunosuppressive drugs previously documented in the literature., Results: 13 of the 17 treated patients (76.5%) showed improvement at 6 months (95% CI 50.1 to 93.2). Among the 14 patients who completed the 12-month follow-up (2 were lost to follow-up after showing improvement at months 8 and 10, and 1 deteriorated at 6 months), 13 (92.9%) demonstrated improvement at 12 months (95% CI 66.1 to 99.8). Nerve conduction parameters improved by at least 20% in two nerves in 6 out of 15 (40%) patients at 6 months and in 7 out of 13 (53.9%) at 12 months. None of the treated patients withdrew from the study due to side effects. There was a significant reduction of circulating CD19+ cells 15 days, 2, 6 and 12 months after treatment., Conclusion: Rituximab seems to be a safe therapy in most patients with CIDP not responding to conventional immune therapies. The high percentage of patients who improved in this study suggests a possible positive effect of rituximab which is worth investigating in future randomised controlled clinical trials., Trial Registration Number: NCT05877040., Competing Interests: Competing interests: PED received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. DC received honoraria for lecturing from CSL Behring—Italy, Kedrion—Italy and Shire/Takeda—Italy and received travel grants to attend scientific meetings from CSL Behring—Italy, Kedrion—Italy and Shire/Takeda. RF has served on scientific advisory boards for CSL Behring—Italy and received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. EP has received travel grants to attend scientific meetings from CSL Behring—Italy. GL has received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. EN-O reports personal fees for Advisory or Scientific Board from ArgenX—Belgium, CSL-Behring—USA, Dianthus—USA; Janssen—USA, Kedrion—Italy, LFB—France, Longboard Pharma—USA, Roche—Switzerland, Sanofi—USA and received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. The other authors declare no conflict of interest., (© Author(s) (or their employer(s)) 2024. No commercial re-use. See rights and permissions. Published by BMJ.)
- Published
- 2024
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