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 , 12 , for the 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)
26 September 2019
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.