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. 2020 Feb;55(2):283-306.
doi: 10.1038/s41409-019-0684-0. Epub 2019 Sep 26.

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)

Basil Sharrack  1   2 , Riccardo Saccardi  3 , Tobias Alexander  4 , Manuela Badoglio  5 , Joachim Burman  6 , Dominique Farge  7   8   9   10 , Raffaella Greco  11 , Helen Jessop  12 , Majid Kazmi  13 , Kirill Kirgizov  14 , Myriam Labopin  5 , Gianluigi Mancardi  15 , Roland Martin  16 , John Moore  17 , Paolo A Muraro  18 , Montserrat Rovira  19 , Maria Pia Sormani  20   21 , John A Snowden  22 ; European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and EBMT (JACIE)
Collaborators, Affiliations

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)

Basil Sharrack et al. Bone Marrow Transplant. 2020 Feb.

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.

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Conflict of interest statement

JAS declares speaker fees at educational events supported by Sanofi, Janssen, Jazz, Mallinckrodt and Gilead, is a member of a trial IDMC for Kiadis Pharma and Chairs NHS England Clinical Reference Group (CRG) for Blood and Marrow Transplantation. RM has received personal remuneration for advisory functions and presentations by Merck, Teva, Sanofi, Novartis, Roche, Biogen, Cell Protect and Neuway Pharma. He is a co-founder and co-owner of Cellerys. PAM reports personal fees and non-financial support from Bayer, Biogen, Merck and Novartis, unrelated to the manuscript. PAM, BS and JS are grateful for support from the UK NIHR EME and Biomedical Research Centre funding schemes. The other authors declare no competing interests.

Figures

Fig. 1
Fig. 1
EBMT ADWP activity—autologous HSCT for MS, other immune-mediated neurological diseases and other autoimmune diseases by year, 1994–2018 (N = 2766)
Fig. 2
Fig. 2
EBMT registry: overall national activity in autologous HSCT indicated for MS, other immune-mediated neurological diseases and other autoimmune diseases by country, 1994–2018 (N = 2766)
Fig. 3
Fig. 3
EBMT registry: relative activity according to reported multiple sclerosis type: RR-MS versus progressive MS (SPMS/PPMS) versus aggressive/malignant MS
Fig. 4
Fig. 4
Trends in transplant conditioning used for autologous HSCT in Multiple Sclerosis: BEAM-ATG versus Cy-ATG (EBMT Registry 1995–2018)
See this image and copyright information in PMC

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