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Bone Marrow Transplant. 2019 Sep 26. doi: 10.1038/s41409-019-0684-0. [Epub ahead of print]

Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE).

Author information

1
Department of Neurology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
2
NIHR Neurosciences Biomedical Research Centre, University of Sheffield, Sheffield, UK.
3
Cell Therapy and Transfusion Medicine Unit, Careggi University Hospital, Firenze, Italy.
4
Klinik fur Rheumatologie und Klinische Immunologie, Charite-Universitatsmedizin, Berlin, Germany.
5
EBMT Paris study office, Department of Haematology, Saint Antoine Hospital, INSERM UMR 938, Sorbonne University, Paris, France.
6
Department of Neuroscience, Uppsala University, Uppsala, Sweden.
7
Unité de Médecine Interne, Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Hôpital St-Louis, AP-HP, Paris, France.
8
Centre de Référence des Maladies Auto-Immunes Systémiques Rares d'Ile-de-France, Filière, FAI2R, Paris, France.
9
EA 3518, Université Denis Diderot, Paris, France.
10
Department of Internal Medicine, McGill University, Montreal, QC, Canada.
11
Hematology and Bone Marrow Transplantation Unit, Istituto di Ricovero e Cura a Carattere Scientifico, San Raffaele Scientific Institute, Milan, Italy.
12
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
13
Kings Health Partners, Department of Haematology, Guys Hospital, London, UK.
14
N.N. Blokhin National Medical Center of Oncology, Institute of Pediatric Oncology and Hematology, Moscow, Russia.
15
Department of Neuroscience, University of Genova and Clinical Scientific Institutes Maugeri, Genoa, Italy.
16
Neuroimmunology and MS Research, Neurology Clinic, University Hospital, Zurich, Switzerland.
17
Haematology Department, St. Vincent's Health Network, Darlinghurst, NSW, Australia.
18
Department of Brain Sciences, Imperial College London, London, UK.
19
BMT Unit, Department of Hematology, IDIBAPS, Hospital Clinic, Institut Josep Carreras, Barcelona, Spain.
20
Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy.
21
IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
22
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. john.snowden1@nhs.net.

Abstract

These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development. The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies. aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified. Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.

PMID:
31558790
DOI:
10.1038/s41409-019-0684-0

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