open access

Vol 52, No 4 (2021)
Review article
Submitted: 2021-06-26
Accepted: 2021-06-26
Get Citation

Hematopoietic stem cell transplantation in autoimmune diseases: update from EBMT Autoimmune Diseases Working Party with special reference to Poland

John A. Snowden1, Jan Styczyński2, Emilian Snarski3, Raffaella Greco4, for the European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP)
DOI: 10.5603/AHP.2021.0043
·
Acta Haematol Pol 2021;52(4):217-224.
Affiliations
  1. Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom
  2. Department of Pediatric Hematology and Oncology, Jurasz University Hospital, Collegium Medicum, Nicolaus Copernicus University in Torun, Bydgoszcz, Poland
  3. Chair and Department of Experimental and Clinical Physiology, Laboratory of Centre for Preclinical Research, Medical University of Warsaw, Warsaw, Poland
  4. Haematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Hospital, Milan, Italy

open access

Vol 52, No 4 (2021)
REVIEW ARTICLE
Submitted: 2021-06-26
Accepted: 2021-06-26

Abstract

Hematopoietic stem cell transplantation (HSCT) is now evolving into a standard treatment in some autoimmune diseases (AD) alongside other modern therapy. The main indications are multiple sclerosis and systemic sclerosis for which HSCT has become an integral and standard-of-care part of treatment algorithms. From 1994 to the beginning of 2021, data from the EBMT Registry indicates that 3,442 patients (60% females, 40% males; 91% adults, 9% pediatric) received 3,514 transplant procedures for autoimmune diseases, with over 90% receiving autologous transplant. Autoimmune diseases are currently the fastest growing indication for autologous HSCT in European Society for Blood and Marrow Transplantation (EBMT), whilst allogeneic HSCT for ADs is mainly restricted to pediatrics, especially diseases with a genetic component. Patient selection plays a key role in providing the best risk/benefit ratio of the procedure. Intensity of conditioning regimen and center experience are also important. Ultimately, the future of HSCT for ADs depends on the standard of care therapy, which influences uptake within national/international disease specialist communities. Further studies are necessary in order to establish relative benefit over current/future standard of care therapy, to establish the best HSCT regimen for each disease, to define mechanisms, develop clinical biomarkers to help select and monitor patients, and to define health economic benefits and public health delivery. We present a current perspective summarizing activity across EBMT, including centers in Poland.

Abstract

Hematopoietic stem cell transplantation (HSCT) is now evolving into a standard treatment in some autoimmune diseases (AD) alongside other modern therapy. The main indications are multiple sclerosis and systemic sclerosis for which HSCT has become an integral and standard-of-care part of treatment algorithms. From 1994 to the beginning of 2021, data from the EBMT Registry indicates that 3,442 patients (60% females, 40% males; 91% adults, 9% pediatric) received 3,514 transplant procedures for autoimmune diseases, with over 90% receiving autologous transplant. Autoimmune diseases are currently the fastest growing indication for autologous HSCT in European Society for Blood and Marrow Transplantation (EBMT), whilst allogeneic HSCT for ADs is mainly restricted to pediatrics, especially diseases with a genetic component. Patient selection plays a key role in providing the best risk/benefit ratio of the procedure. Intensity of conditioning regimen and center experience are also important. Ultimately, the future of HSCT for ADs depends on the standard of care therapy, which influences uptake within national/international disease specialist communities. Further studies are necessary in order to establish relative benefit over current/future standard of care therapy, to establish the best HSCT regimen for each disease, to define mechanisms, develop clinical biomarkers to help select and monitor patients, and to define health economic benefits and public health delivery. We present a current perspective summarizing activity across EBMT, including centers in Poland.

Get Citation

Keywords

autoimmune diseases, stem cells, transplantation, autologous, allogeneic

About this article
Title

Hematopoietic stem cell transplantation in autoimmune diseases: update from EBMT Autoimmune Diseases Working Party with special reference to Poland

Journal

Acta Haematologica Polonica

Issue

Vol 52, No 4 (2021)

Article type

Review article

Pages

217-224

DOI

10.5603/AHP.2021.0043

Bibliographic record

Acta Haematol Pol 2021;52(4):217-224.

Keywords

autoimmune diseases
stem cells
transplantation
autologous
allogeneic

Authors

John A. Snowden
Jan Styczyński
Emilian Snarski
Raffaella Greco
for the European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP)

References (33)
  1. Snowden JA, Saccardi R, Allez M, et al. EBMT Autoimmune Disease Working Party (ADWP), Paediatric Diseases Working Party (PDWP). Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 2012; 47(6): 770–790.
  2. Snowden JA, Badoglio M, Labopin M, et al. European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP), EBMT Paediatric Working Party (PWP), Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT), EBMT (JACIE). Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases. Blood Adv. 2017; 1(27): 2742–2755.
  3. Greco R, Labopin M, Badoglio M, et al. Allogeneic HSCT for autoimmune diseases: a retrospective study from the EBMT ADWP, IEWP, and PDWP Working Parties. Front Immunol. 2019; 10: 1570.
  4. Swart JF, Delemarre EM, van Wijk F, et al. Haematopoietic stem cell transplantation for autoimmune diseases. Nat Rev Rheumatol. 2017; 13(4): 244–256.
  5. Duarte RF, Labopin M, Bader P, et al. European Society for Blood and Marrow Transplantation (EBMT). Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019. Bone Marrow Transplant. 2019; 54(10): 1525–1552.
  6. Sospedra M, Martin R. Immunology of multiple sclerosis. Annu Rev Immunol. 2005; 23: 683–747.
  7. Ismail A, Sharrack B, Saccardi R, et al. Autologous haematopoietic stem cell therapy for multiple sclerosis: a review for supportive care clinicians on behalf of the Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation. Curr Opin Support Palliat Care. 2019; 13(4): 394–401.
  8. Sharrack B, Saccardi R, Alexander T, et al. 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). 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). Bone Marrow Transplant. 2020; 55(2): 283–306.
  9. Mariottini A, Filippini S, Innocenti C, et al. Impact of autologous haematopoietic stem cell transplantation on disability and brain atrophy in secondary progressive multiple sclerosis. Mult Scler. 2021; 27(1): 61–70.
  10. Boffa G, Massacesi L, Inglese M, et al. Italian BMT-MS study group. Long-term clinical outcomes of hematopoietic stem cell transplantation in multiple sclerosis. Neurology. 2021 [Epub ahead of print].
  11. Atkins HL, Bowman M, Allan D, et al. Immunoablation and autologous haemopoietic stem-cell transplantation for aggressive multiple sclerosis: a multicentre single-group phase 2 trial. Lancet. 2016; 388(10044): 576–585.
  12. Burt RK, Balabanov R, Han X, et al. Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA. 2015; 313(3): 275–284.
  13. Nash RA, Hutton GJ, Racke MK, et al. High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS. Neurology. 2017; 88(9): 842–852.
  14. Das J, Snowden JA, Burman J, et al. Autologous haematopoietic stem cell transplantation as a first-line disease-modifying therapy in patients with 'aggressive' multiple sclerosis. Mult Scler. 2021; 27(8): 1198–1204.
  15. Mariottini A, De Matteis E, Muraro PA. Haematopoietic stem cell transplantation for multiple sclerosis: current status. BioDrugs. 2020; 34(3): 307–325.
  16. Burt RK, Shah SJ, Dill K, et al. Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. Lancet. 2011; 378(9790): 498–506.
  17. van Laar JM, Farge D, Tyndall A. Cardiac assessment before stem cell transplantation for systemic sclerosis--reply. JAMA. 2014; 312(17): 1803–1804.
  18. van Laar JM, Farge D, Sont JK, et al. EBMT/EULAR Scleroderma Study Group. Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA. 2014; 311(24): 2490–2498.
  19. Sullivan KM, Goldmuntz EA, Keyes-Elstein L, et al. SCOT Study Investigators. Myeloablative autologous stem-cell transplantation for severe scleroderma. N Engl J Med. 2018; 378(1): 35–47.
  20. Elhai M, Meune C, Boubaya M, et al. EUSTAR group. Trends in mortality in patients with systemic sclerosis over 40 years: a systematic review and meta-analysis of cohort studies. Rheumatology (Oxford). 2012; 51(6): 1017–1026.
  21. Komócsi A, Vorobcsuk A, Faludi R, et al. The impact of cardiopulmonary manifestations on the mortality of SSc: a systematic review and meta-analysis of observational studies. Rheumatology (Oxford). 2012; 51(6): 1027–1036.
  22. Henes J, Oliveira MC, Labopin M, et al. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party. Haematologica. 2021; 106(2): 375–383.
  23. Farge D, Ait Abdallah N, Marjanovic Z, et al. Autologous stem cell transplantation in scleroderma. Presse Med. 2021 [Epub ahead of print]; 50(1): 104065.
  24. Farge D, Burt RK, Oliveira MC, et al. Cardiopulmonary assessment of patients with systemic sclerosis for hematopoietic stem cell transplantation: recommendations from the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party and collaborating partners. Bone Marrow Transplant. 2017; 52(11): 1495–1503.
  25. Burt RK, Han X, Quigley K, et al. Cardiac safe hematopoietic stem cell transplantation for systemic sclerosis with poor cardiac function: a pilot safety study that decreases neutropenic interval to 5 days. Bone Marrow Transplant. 2021; 56(1): 50–59.
  26. Snowden JA, Panés J, Alexander T, et al. Autologous haematopoietic stem cell transplantation (AHSCT) in severe Crohn’s disease: a review on behalf of ECCO and EBMT. J Crohns Colitis . 2018; 12(4): 476–488.
  27. Hawkey CJ, Allez M, Clark MM, et al. Autologous hematopoetic stem cell transplantation for refractory Crohn disease: a randomized clinical trial. JAMA. 2015; 314(23): 2524–2534.
  28. Brierley CK, Castilla-Llorente C, Labopin M, et al. European Society for Blood and Marrow Transplantation [EBMT] Autoimmune Diseases Working Party [ADWP]. Autologous haematopoietic stem cell transplantation for Crohn's disease: a retrospective survey of long-term outcomes from the European Society for Blood and Marrow Transplantation. J Crohns Colitis. 2018; 12(9): 1097–1103.
  29. Burt RK, Craig R, Yun L, et al. A pilot feasibility study of non-myeloablative allogeneic hematopoietic stem cell transplantation for refractory Crohn Disease. Bone Marrow Transplant. 2020; 55(12): 2343–2346.
  30. Uhlig HH, Schwerd T, Koletzko S, et al. COLORS in IBD Study Group and NEOPICS. The diagnostic approach to monogenic very early onset inflammatory bowel disease. Gastroenterology. 2014; 147(5): 990–1007.e3.
  31. Jessop H, Farge D, Saccardi R, et al. 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). Bone Marrow Transplant. 2019; 54(7): 933–942.
  32. Roberts F, Hobbs H, Jessop H, et al. Rehabilitation before and after autologous haematopoietic stem cell transplantation (AHSCT) for patients with multiple sclerosis (MS): consensus guidelines and recommendations for best clinical practice on behalf of the Autoimmune Diseases Working Party, Nurses Group, and Patient Advocacy Committee of the European Society for Blood and Marrow Transplantation (EBMT). Front Neurol. 2020; 11: 556141.
  33. Greco R, Alexander T, Burman J, et al. European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP), Infectious Diseases Working Party (IDWP), Pediatric Working Party (PWP), Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and EBMT (JACIE), EBMT Nurses Group and Patient Advocacy Committee. Hematopoietic stem cell transplantation for autoimmune diseases in the time of COVID-19: EBMT guidelines and recommendations. Bone Marrow Transplant. 2021 [Epub ahead of print].

Regulations

Important: This website uses cookies. More >>

The cookies allow us to identify your computer and find out details about your last visit. They remembering whether you've visited the site before, so that you remain logged in - or to help us work out how many new website visitors we get each month. Most internet browsers accept cookies automatically, but you can change the settings of your browser to erase cookies or prevent automatic acceptance if you prefer.

By "Via Medica sp. z o.o." sp.k., ul. Świętokrzyska 73, 80–180 Gdańsk, Poland
tel.:+48 58 320 94 94, fax:+48 58 320 94 60, e-mail: journals@viamedica.pl