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Journal of Neurology
Published in: Journal of Neurology 11/2020

01-11-2020 | Stem Cell Transplantation | Original Communication

Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy

Authors: Richard K. Burt, Roumen Balabanov, Jinny Tavee, Xiaoqiang Han, Robert Sufit, Senda Ajroud-Driss, Borko Jovanovic, Kathleen Quigley, Indira Arnautovic, Irene Helenowski, Basil Sharrack

Published in: Journal of Neurology | Issue 11/2020

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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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Metadata
Title
Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy
Authors
Richard K. Burt
Roumen Balabanov
Jinny Tavee
Xiaoqiang Han
Robert Sufit
Senda Ajroud-Driss
Borko Jovanovic
Kathleen Quigley
Indira Arnautovic
Irene Helenowski
Basil Sharrack
Publication date
01-11-2020
Publisher
Springer Berlin Heidelberg
Published in
Journal of Neurology / Issue 11/2020
Print ISSN: 0340-5354
Electronic ISSN: 1432-1459
DOI
https://doi.org/10.1007/s00415-020-10010-6

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