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Review
. 2022 Jul;57(7):1055-1062.
doi: 10.1038/s41409-022-01702-w. Epub 2022 May 16.

Hematopoietic stem cell transplantation and cellular therapies for autoimmune diseases: overview and future considerations from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Tobias Alexander  1 , Raffaella Greco  2
Affiliations
Review

Hematopoietic stem cell transplantation and cellular therapies for autoimmune diseases: overview and future considerations from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Tobias Alexander et al. Bone Marrow Transplant. 2022 Jul.

Abstract

Autoimmune diseases (ADs) represent a heterogenous group of complex diseases with increasing incidence in Western countries and are a major cause of morbidity. Hematopoietic stem cell transplantation (HSCT) has evolved over the last 25 years as a specific treatment for patients with severe ADs, through eradication of the pathogenic immunologic memory and profound immune renewal. HSCT for ADs is recently facing a unique developmental phase across transplant centers. This review provides a comprehensive overview of the recent evidence and developments in the area, including fundamentals of preclinical research, clinical studies in neurologic, rheumatologic and gastroenterologic diseases, which represent major indications at present, along with evidence of HSCT for rarer indications. Moreover, we describe the interwoven challenges of delivering more advanced cellular therapies, exploiting mesenchymal stem cells, regulatory T cells and potentially CAR-T cell therapies, in patients affected by ADs. Overall, we discuss past and current indications, efficacy, associated risks and benefits, and future directions of HSCT and advanced cellular therapies in the treatment of severe/refractory ADs, integrating the available literature with European Society for Blood and Marrow Transplantation (EBMT) registry data.

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Conflict of interest statement

RG discloses speaking honoraria from Biotest, Pfizer, Medac, and Magenta. TA declares speaking honoraria from Amgen, Roche, Pfizer, travel grants from Neovii, and study support from Amgen, Janssen-Cilag, and Miltenyi Biotec.

Figures

Fig. 1
Fig. 1. EBMT-ADWP Registry data: autologous HSCT for ADs (1994–2021, n = 3502).
Autologous transplants for ADs reported to the EBMT-ADWP data registry, from 1994 through 2021. Major indications for autologous HSCT are multiple sclerosis, systemic sclerosis, and Crohn’s disease. AD autoimmune disease, ADWP Autoimmune Diseases Working Party, EBMT European Society for Blood and Marrow Transplantation, HSCT hematopoietic stem cell transplantation, IDD insulin-dependent diabetes mellitus, MS multiple sclerosis, SSc systemic sclerosis, SLE systemic lupus erythematosus.
Fig. 2
Fig. 2. Autologous HSCT for AD, reported to the EBMT-ADWP Registry data, per year and indication.
Number of autologous HSCT reported per year and indication, from 1994 through 2021 (n = 3502). AD autoimmune disease, ADWP Autoimmune Diseases Working Party, EBMT European Society for Blood and Marrow Transplantation, HSCT hematopoietic stem cell transplantation, IDD insulin-dependent diabetes mellitus, MS multiple sclerosis, SSc systemic sclerosis, SLE systemic lupus erythematosus.
See this image and copyright information in PMC

References

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