Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy

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

doi:10.1007/s00415-020-10010-6

Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy

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

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

46 Scopus citations

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.

Original language	English (US)
Pages (from-to)	3378-3391
Number of pages	14
Journal	Journal of Neurology
Volume	267
Issue number	11
DOIs	https://doi.org/10.1007/s00415-020-10010-6
State	Published - Nov 1 2020

Funding

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. 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.

Keywords

Chronic inflammatory demyelinating polyradiculoneuropathy
Efficacy
Hematopoietic stem cell transplantation
Toxicity

ASJC Scopus subject areas

Neurology
Clinical Neurology

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1007/s00415-020-10010-6

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title = "Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy",

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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author = "Burt, {Richard K.} and Roumen Balabanov and Jinny Tavee and Xiaoqiang Han and Robert Sufit and Senda Ajroud-Driss and Borko Jovanovic and Kathleen Quigley and Indira Arnautovic and Irene Helenowski and Basil Sharrack",

note = "Funding Information: 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. Funding Information: 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. Publisher Copyright: {\textcopyright} 2020, Springer-Verlag GmbH Germany, part of Springer Nature.",

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doi = "10.1007/s00415-020-10010-6",

language = "English (US)",

volume = "267",

pages = "3378--3391",

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TY - JOUR

T1 - Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy

AU - Burt, Richard K.

AU - Balabanov, Roumen

AU - Tavee, Jinny

AU - Han, Xiaoqiang

AU - Sufit, Robert

AU - Ajroud-Driss, Senda

AU - Jovanovic, Borko

AU - Quigley, Kathleen

AU - Arnautovic, Indira

AU - Helenowski, Irene

AU - Sharrack, Basil

N1 - Funding Information: 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. Funding Information: 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. Publisher Copyright: © 2020, Springer-Verlag GmbH Germany, part of Springer Nature.

PY - 2020/11/1

Y1 - 2020/11/1

N2 - 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.

AB - 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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KW - Toxicity

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Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy

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Keywords

ASJC Scopus subject areas

UN SDGs

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