• Pietro Emiliano Doneddu, Dario Cocito, Raffaella Fazio, Luana Benedetti, Erdita Peci, Giuseppe Liberatore, Yuri Matteo Falzone, Francesco Germano, Francesca Gallia, Claudia Giannotta, Cinta Lleixà, Elisa Bianchi, and Eduardo Nobile-Orazio.
• Neuromuscular and Neuroimmunology Unit, IRCCS Humanitas Research Hospital, Milan, Italy.
• J. Neurol. Neurosurg. Psychiatr. 2024 Aug 16; 95 (9): 838844838-844.

BackgroundTo evaluate the efficacy of rituximab in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) patients not responding to conventional immune therapies.MethodsAn 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.Results13 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.ConclusionRituximab 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 NumberNCT05877040.© Author(s) (or their employer(s)) 2024. No commercial re-use. See rights and permissions. Published by BMJ.

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