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Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy

Journal of Neurology Aims and scope Submit manuscript

Abstract

Objective

Determine toxicity and efficacy of autologous hematopoietic stem cell transplantation (HSCT) for patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) who are dependent on intravenous immunoglobulins or plasmapheresis.

Methods

Unselected peripheral blood stem cells were re-infused on day 0 after conditioning with cyclophosphamide 200 mg/kg/intravenously (IV), rATG (thymoglobulin) 5.5 mg/kg/IV, and rituximab 1000 mg/IV.

Results

Sixty-six patients underwent HSCT for CIDP. Data on sixty patients with a mean follow-up of 4.5 years (range 2–5 years) were available for analysis. There were no treatment-related deaths, and overall survival was 97%. Post-transplant immune medication-free remission was 80%, 78%, 76% 78%, and 83% at 1, 2, 3, 4, and 5 years. Ambulation without assistance improved from 33% pre-HSCT to 82% 82%, 81%, 86%, and 83% at 1, 2, 3, 4, and 5 years, respectively. Mean right/left hand grip strength (kg) improved significantly (all p values < 0.01) from 18.1/16.5 pre-HSCT to 26.3/25.4, 29.2/28.2, 28.8/28.6, 30.3/25.5, and 30.8/29.1 at 1, 2, 3, 4, and 5 years, respectively. Average nerve conduction velocity (NCV) (m/s) improved significantly (all p values ≤ 0.001) from a mean of 27.2 pre-HSCT to 33.5, 33.8, 37.7, 38.2, and 38.3 at 1, 2, 3, 4, and 5 years, respectively. Average compound motor action potential (CMAP) (mv) improved significantly (p values ≤ 0.001) from a mean of 3.6 pre-HSCT to 4.6, 4.6, 5.0, 5.1, and 4.1 at 1, 2, 3, 4, and 5 years, respectively.

Conclusion

A randomized trial is indicated to verify these results and confirm that HSCT reverses disability and offers long-term immune therapy independence.

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Acknowledgements

We wish to thank Professor Richard A. C. Hughes MD, Cochrane Neuromuscular Disease Review Group, MRC Centre of Neuromuscular Diseases, institute of Neurology, University Colleague London, London, UK, for his thoughtful discussions and comments. This study was made possible by financial support from the Danhakl family, the McNamara Purcell Foundation, and Morgan Stanley and Company. The principal investigator (RKB), statisticians (IBH, BJ), and fellow (XH) had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis.

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Not applicable.

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Authors

Contributions

All authors contributed to the study conception and design. Material preparation, data collection, and analysis were performed by RKB, XH, KQ, IA, and IH. The first draft of the manuscript was written by RKB, and all authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.

Corresponding author

Correspondence to Richard K. Burt.

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Conflicts of interest

On behalf of all authors, the corresponding author states that there is no conflict of interest except the following author. Rouman Balabanov—Scientific Advisory Boards-Biogen, Sanofi-Genzyme, Genentech, Alexion Speakers' Bureaus–Biogen Idec, Sanofi-Genzyme.

Ethics approval

Patients enrolled in this open-label prospective study were treated at Northwestern Memorial Hospital (Chicago, Illinois, USA) on an IRB and FDA approved protocol (www.clinicaltrials.gov NCT00278629) and have therefore been performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments.

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All persons gave their informed consent prior to their inclusion in the study.

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Burt, R.K., Balabanov, R., Tavee, J. et al. Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy. J Neurol 267, 3378–3391 (2020). https://doi.org/10.1007/s00415-020-10010-6

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  • DOI: https://doi.org/10.1007/s00415-020-10010-6

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