Hematopoietic stem cell transplantation for uncommon immune-mediated neurological disorders: A literature review |
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| Institution: | 1. Instituto de Investigación Sanitario Fundación Jiménez Díaz, Madrid, Spain;2. Hospital Universitario Fundación Jiménez Díaz, Universidad Autónoma de Madrid, Madrid, Spain;1. Department of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center, Tampa, Florida, USA;2. Department of Pathology and Laboratory Medicine, Phoenix Children''s Hospital, Phoenix, Arizona, USA;3. Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA;4. Department of Pathology and Immunology, Washington University School of Medicine, St Louis, Missouri, USA;5. Department of Pathology and Laboratory Medicine, University of North Carolina, Chapel Hill, North Carolina, USA;6. Department of Pathology and Laboratory Medicine, University of Miami, Miami, Florida, USA;7. Department of Pathology, Molecular and Cell-Based Medicine, Mount Sinai Health System, Icahn School of Medicine, New York, New York, USA;8. Laboratory Medicine and Pathology and Center for Regenerative Medicine, Mayo Clinic, Jacksonville, Florida, USA;9. Department of Pediatric Hematology/Oncology/Neuro-Oncology/Stem Cell Transplant, Ann & Robert H. Lurie Children''s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA;10. Division of Transfusion Medicine and Cellular Therapy, Department of Pathology and Laboratory Medicine, Memorial Sloan Kettering Cancer Center, New York, New York, USA;11. Department of Clinical Hematology and Bone Marrow Transplant, Tata Medical Center, Kolkata, India;12. Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA;1. School of Basic Medicine, Guangdong Medical University, Zhanjiang, Guangdong Province, China;2. Orthopaedic Center, Affiliated Hospital of Guangdong Medical University, Guangdong Medical University, Zhanjiang, Guangdong Province, China;3. Guangdong Engineering Research Center for Translation of Medical 3D Printing Application, Guangdong Provincial Key Laboratory of Medical Biomechanics, School of Basic Medical Sciences, Southern Medical University, Guangzhou, Guangdong Province, China;1. The Emmes Company, LLC, Rockville, Maryland, USA;2. Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas, USA;3. Moffitt Cancer Center, Tampa, Florida, USA;4. Interdisciplinary Stem Cell Institute, Miller School of Medicine, University of Miami, Miami, Florida, USA;5. Molecular and Cellular Therapeutics, University of Minnesota, Saint Paul, Minnesota, USA;1. Medical Affairs and Innovation, Héma-Québec, Québec City, Canada;2. Cord Blood Bank, Héma-Québec, Montréal, Canada;3. Centre for Innovation, Canadian Blood Services, Ottawa, Canada;4. Biochemistry, Microbiology and Immunology Department, University of Ottawa, Ottawa, Canada |
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| Abstract: | BackgroundChronic inflammatory demyelinating polyneuropathy (CIDP), stiff-person syndrome (SPS), neuromyelitis optica spectrum disorders (NMOSD) and severe refractory myasthenia gravis (MG) are immune-mediated neurological diseases that severely affect patients’ functionality and quality of life, with a considerable percentage undergoing relapse or not responding to conventional treatment options. Autologous hematopoietic stem cell transplantation (auto-HSCT) has emerged as a potential second-line treatment alternative.MethodsWe performed a literature review in PubMed/Medline, EMBASE, Web of Science and Cochrane Library from inception to September 2021 of reported cases and studies of CIDP, SPS, NMOSD and MG that underwent HSCT as a treatment option.ResultsA total of 173 patients who underwent HSCT were found, including 32 patients described in case reports and 60 in a phase 2 clinical trial with CIDP, 29 patients with SPS, 42 patients with NMOSD and 10 patients with refractory MG. Complete remission was documented in 68/92 patients with CIDP, 13/29 with SPS and 10/10 with MG. From the NMOSD cases, 24/42 were relapse-free at last follow-up, with 13/33 having negative anti-AQ4 antibodies after HSCT. From all the included studies, only 8/173 patients received an allogeneic HSCT, 4/8 after a failed auto-HSCT. All showed clinical improvement and disease remission.ConclusionHSCT has the potential to induce long-term remission in patients with CIDP, NMOSD, SPS or MG with adequate safety and tolerability. Collaboration between centers is needed to implement larger, homogeneous prospective studies, focusing on immunological correlates of favorable long-term response. |
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