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Review
. 2019 Jul;54(7):933-942.
doi: 10.1038/s41409-019-0430-7. Epub 2019 Jan 31.

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)

Helen Jessop  1 , Dominique Farge  2   3   4   5 , Riccardo Saccardi  6 , Tobias Alexander  7 , Montserrat Rovira  8 , Basil Sharrack  9 , Raffaella Greco  10 , Nico Wulffraat  11 , John Moore  12 , Majid Kazmi  13 , Manuela Badoglio  14 , Gillian Adams  15 , Bregje Verhoeven  15 , John Murray  16 , John A Snowden  17
Affiliations
Review

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)

Helen Jessop et al. Bone Marrow Transplant. 2019 Jul.

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.

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Conflict of interest statement

JAS declares honoraria for speaking at educational meetings from Sanofi, Jazz, Janssen and Mallinckrodt. The remaining authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1
Summary of autologous HSCT procedure. In addition, robust long-term follow-up arrangements should be individualised with each patient with both disease and transplant specialists
Fig. 2
Fig. 2
Mechanism of immune re-setting with autologous HSCT in autoimmune diseases
Fig. 3
Fig. 3
a–d Activity and JACIE accreditation status in EBMT reporting centres for autologous HSCT in autoimmune diseases. a Overall. b MS and neurological diseases. c Systemic sclerosis and rheumatological diseases. d Crohn’s disease and gastrointestinal diseases. Activity is for autologous procedures from 2012–2017 inclusive, i.e., reflecting practice after the publication of EBMT ADWP Guidelines [12]. Accreditation status is indicated by asterisks as active and/or with re-accreditation in progress as per December 2018 (www.JACIE.org). Centre identities according to CIC code are available on https://www.ebmt.org/ebmt-membership-list. The information is primarily intended as a resource for clinicians in transplant centres and disease specialists who wish to make contact with experienced EBMT centres for advice, support, referral or other partnership arrangements aimed at optimising patient care whilst accommodating geographical considerations. The information may also be useful to patients and other carers; however, independent self-referral is strongly discouraged. MS multiple sclerosis, SSc systemic sclerosis, CD Crohn’s disease, MG myasthenia gravis, CIDP chronic inflammatory demyelinating polyneuropathy, NMO neuromyelitis optica
Fig. 3
Fig. 3
a–d Activity and JACIE accreditation status in EBMT reporting centres for autologous HSCT in autoimmune diseases. a Overall. b MS and neurological diseases. c Systemic sclerosis and rheumatological diseases. d Crohn’s disease and gastrointestinal diseases. Activity is for autologous procedures from 2012–2017 inclusive, i.e., reflecting practice after the publication of EBMT ADWP Guidelines [12]. Accreditation status is indicated by asterisks as active and/or with re-accreditation in progress as per December 2018 (www.JACIE.org). Centre identities according to CIC code are available on https://www.ebmt.org/ebmt-membership-list. The information is primarily intended as a resource for clinicians in transplant centres and disease specialists who wish to make contact with experienced EBMT centres for advice, support, referral or other partnership arrangements aimed at optimising patient care whilst accommodating geographical considerations. The information may also be useful to patients and other carers; however, independent self-referral is strongly discouraged. MS multiple sclerosis, SSc systemic sclerosis, CD Crohn’s disease, MG myasthenia gravis, CIDP chronic inflammatory demyelinating polyneuropathy, NMO neuromyelitis optica
See this image and copyright information in PMC

References

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