Richard K Burt1, Roumen Balabanov2, Jinny Tavee2, Xiaoqiang Han3, Robert Sufit2, Senda Ajroud-Driss2, Borko Jovanovic4, Kathleen Quigley3, Indira Arnautovic3, Irene Helenowski4, Basil Sharrack5. 1. Division of Immunotherapy, Department of Medicine, Northwestern University Feinberg School of Medicine, Axis Bldg, 446 East Ontario, Suite 10-1000, Chicago, IL, 60611, USA. rburt@northwestern.edu. 2. Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL, 60611, USA. 3. Division of Immunotherapy, Department of Medicine, Northwestern University Feinberg School of Medicine, Axis Bldg, 446 East Ontario, Suite 10-1000, Chicago, IL, 60611, USA. 4. Department of Preventive Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, 60611, USA. 5. Academic Department of Neuroscience and Sheffield, NIHR Translational Neuroscience BRC, Sheffield Teaching Hospitals, NHS Foundation Trust, University of Sheffield, Sheffield, UK.
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.
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.
Authors: John A Snowden; Isabel Sánchez-Ortega; Selim Corbacioglu; Grzegorz W Basak; Christian Chabannon; Rafael de la Camara; Harry Dolstra; Rafael F Duarte; Bertram Glass; Raffaella Greco; Arjan C Lankester; Mohamad Mohty; Bénédicte Neven; Régis Peffault de Latour; Paolo Pedrazzoli; Zinaida Peric; Ibrahim Yakoub-Agha; Anna Sureda; Nicolaus Kröger Journal: Bone Marrow Transplant Date: 2022-05-19 Impact factor: 5.174
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Authors: Richard K Burt; Paul Tappenden; Roumen Balabanov; Xiaoqiang Han; Kathleen Quigley; John A Snowden; Basil Sharrack Journal: Front Neurol Date: 2021-03-22 Impact factor: 4.003
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