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Original research
Prospective open-label trial with rituximab in patients with chronic inflammatory demyelinating polyradiculoneuropathy not responding to conventional immune therapies
  1. Pietro Emiliano Doneddu1,2,
  2. Dario Cocito3,
  3. Raffaella Fazio4,
  4. Luana Benedetti5,
  5. Erdita Peci6,
  6. Giuseppe Liberatore1,
  7. Yuri Matteo Falzone4,
  8. Francesco Germano5,7,
  9. Francesca Gallia1,
  10. Claudia Giannotta1,
  11. Cinta Lleixà8,
  12. Elisa Bianchi9,
  13. Eduardo Nobile-Orazio1,10
  1. 1Neuromuscular and Neuroimmunology Unit, IRCCS Humanitas Research Hospital, Milan, Italy
  2. 2Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, Milan, Italy
  3. 3Dipartimento Scienze Cliniche e Biologiche, Università di Torino, Torino, Italy
  4. 4Division of Neuroscience, Department of Neurology, Institute of Experimental Neurology (INSPE), IRCCS San Raffaele Scientific Institute, Milan, Italy
  5. 5IRCCS Ospedale Policlinico San Martino, Genova, Italy
  6. 6Presidio Sanitario Major, Istituti Clinici Scientifici Maugeri, Torino, Italy
  7. 7Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Science (DINOGMI), Genoa University, Genova, Italy
  8. 8Department of Neurology, Neuromuscular Diseases Unit, Hospital de La Santa Creu I Sant Pau, Universitat Autònoma de Barcelona, Barcelona, Spain
  9. 9Laboratorio di Malattie Neurologiche, Istituto di ricerche farmacologiche Mario Negri IRCCS, Milano, Italy
  10. 10Department of Medical Biotechnology and Translational Medicine, University of Milan, Milano, Italy
  1. Correspondence to Professor Eduardo Nobile-Orazio, Neuromuscular and Neuroimmunology Unit, Humanitas Clinical and Research Center - IRCCS, Milano 20089, Italy; eduardo.nobile{at}unimi.it

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.

  • NEUROIMMUNOLOGY
  • NEUROPATHY

Data availability statement

Data are available on reasonable request. Anonymised data used for this study are available on reasonable request from the corresponding author.

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Data availability statement

Data are available on reasonable request. Anonymised data used for this study are available on reasonable request from the corresponding author.

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Footnotes

  • X @f.germano

  • Contributors PED contributed to the conception, organisation and execution of the study and reviewed the results and contributed to the final report. DC, RF and LB contributed to the conception, organisation and execution of the study and reviewed the results and the final report. EP, GL YMF, FGermano, FGallia, CG and CL contributed to the organisation and execution of the study and reviewed the results and the final report of the study. EB performed the statistical analysis of the study and contributed to the final report of the study. EN-O contributed to the conception and organisation of the study, wrote the protocol, reviewed the results of the study and wrote the final report.EN-O accepts full responsibility for the work and/or the conduct of the study, had access to the data, and controlled the decision to publish.

  • Funding The study was supported by Italian Ministry of Health, Ricerca Finalizzata, Grant RF-2016- 02361887.

  • Disclaimer The funder had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

  • 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.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.

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