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Bone Marrow Transplant. 2019 Jan 31. doi: 10.1038/s41409-019-0430-7. [Epub ahead of print]

General information for patients and carers considering haematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases (ADs): A position statement from the EBMT Autoimmune Diseases Working Party (ADWP), the EBMT Nurses Group, the EBMT Patient, Family and Donor Committee and the Joint Accreditation Committee of ISCT and EBMT (JACIE).

Author information

1
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield S10 2JF, UK.
2
Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Hôpital St-Louis, AP-HP, 1 avenue Claude Vellefaux, Paris, France.
3
Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France, Filière FAI2R, Paris, France.
4
EA 3518, Université Denis Diderot, Paris 7, France.
5
Department of Internal Medicine, McGill University, Montréal, Canada.
6
Haematology Department, Careggi University Hospital, Florence, Italy.
7
Klinik fur Rheumatologie und Klinische Immunologie, Charite-Universitatsmedizin, Berlin, Germany.
8
Department of Haematology, Institute of Haematology and Oncology, IDIBAPS, Hospital Clinic, University of Barcelona, Josep Carreras Leukaemia Research Foundation, Barcelona, Spain.
9
Department of Neurology, Sheffield Neuroscience BRC, Sheffield Teaching Hospitals NHS Foundation Trust, University of Sheffield, Sheffield S10 2JF, UK.
10
Hematology and BMT Unit, San Raffaele Scientific Institute, Via Olgettina 60, 20132, Milano, Italy.
11
Divisie Kinderen, Cluster Immunologie, Reumatologie, Hematologie en Infectiologie, Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands.
12
Department of Haematology, St Vincents Hospital Sydney, Darlinghurst, NSW, Australia.
13
Kings Healthcare Partners, Department of Haematology, Guys Hospital, London SE1 9RT, UK.
14
EBMT Paris study office/CEREST-TC, Department of Haematology, Saint Antoine Hospital - INSERM, Paris, France.
15
EBMT Executive Office, Eddific Dr. Frederic Duran i Jorda, Passeig Taulat, 116. 08005, Barcelona, Spain.
16
Christie Hospital NHS Foundation Trust, Manchester, UK.
17
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield S10 2JF, UK. john.snowden@sth.nhs.uk.

Abstract

Over the last 20 years, haematopoietic stem cell transplantation (HSCT) has been used to treat patients with severe autoimmune and inflammatory diseases whose response to standard treatment options has been limited, resulting in a poor long-term prognosis in terms of survival or disability. The vast majority of patients have received autologous HSCT where an increasing evidence-base supports its use in a wide range of autoimmune diseases, particularly relapsing remitting MS, systemic sclerosis and Crohn's disease. Compared with standard treatments for autoimmune diseases, HSCT is associated with greater short-term risks, including a risk of treatment-related mortality and long-term complications. There is a need for a careful appraisal of potential benefits and risks by disease and transplant specialists working closely together with patients and carers to determine individual suitability for HSCT. HSCT should be conducted in accredited transplant centres with robust arrangements for long-term follow-up with both disease and transplant specialists. The aim of this open-access position statement is to provide plainly worded guidance for patients and non-specialist clinicians considering HSCT for an autoimmune disease, especially when treatment abroad is being considered. Recent technical publications in the field have been referenced to support the statement and provide more detail for clinicians advising patients.

PMID:
30705338
DOI:
10.1038/s41409-019-0430-7

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