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

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), Charlotte K. Brierley, Cristina Castilla-Llorente, Myriam Labopin, Manuela Badoglio, Montserrat Rovira, Elena Ricart, Daan Dierickx, Severine Vermeire, Peter Hasselblatt, Juergen Finke, Francesco Onida, Andrea Cassinotti, Jack Satsangi, Majid Kazmi, Antonio López-Sanromán, Carsten Schmidt, Dominique Farge, Simon P.L. Travis, Chris J. HawkeyJohn A. Snowden

Research output: Contribution to journalArticle

Abstract

Background and Aims Autologous haematopoietic stem cell transplantation [AHSCT] is a therapeutic option for patients with severe, treatment-refractory Crohn's disease [CD]. The evidence base for AHSCT for CD is limited, with one randomised trial [ASTIC] suggesting benefit. The aim of this study was to evaluate safety and efficacy for patients undergoing AHSCT for CD in Europe, outside the ASTIC trial. Methods We identified 99 patients in the European Society for Blood and Marrow Transplantation [EBMT] registry, who were eligible for inclusion. Transplant and clinical outcomes were obtained for 82 patients from 19 centres in seven countries. Results Median patient age was 30 years [range 20-65]. Patients had failed or been intolerant to a median of six lines of drug therapy; 61/82 [74%] had had surgery. Following AHSCT, 53/78 [68%] experienced complete remission or significant improvement in symptoms at a median follow-up of 41 months [range 6-174]; 22/82 [27%] required no medical therapy at any point post-AHSCT. In patients who had re-started medical therapy at latest follow-up, 57% [24/42] achieved remission or significant symptomatic improvement with therapies to which they had previously lost response or been non-responsive. Treatment-free survival at 1 year was 54%. On multivariate analysis, perianal disease was associated with adverse treatment-free survival (hazard ratio 2.34, 95% confidence interval [CI] 1.14-4.83, p = 0.02). One patient died due to infectious complications [cytomegalovirus disease] at Day +56. Conclusions In this multicentre retrospective analysis of European centres, AHSCT was relatively safe and appeared to be effective in controlling otherwise treatment-resistant Crohn's disease. Further prospective randomised controlled trials against standard of care are warranted.

Original languageEnglish
Pages (from-to)1097-1103
Number of pages7
JournalJournal of Crohn's and Colitis
Volume12
Issue number9
DOIs
Publication statusPublished - Aug 29 2018

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Hematopoietic Stem Cell Transplantation
Crohn Disease
Transplantation
Bone Marrow
Therapeutics
Survival
Surveys and Questionnaires
Standard of Care
Patient Safety
Cytomegalovirus
Registries
Multivariate Analysis
Randomized Controlled Trials
Confidence Intervals
Transplants
Drug Therapy

Keywords

  • Autoimmune disease; autologous haematopoietic stem cell transplant
  • Crohn's disease

ASJC Scopus subject areas

  • Gastroenterology

Cite this

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), Brierley, C. K., Castilla-Llorente, C., Labopin, M., Badoglio, M., Rovira, M., ... Snowden, J. A. (2018). 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. Journal of Crohn's and Colitis, 12(9), 1097-1103. https://doi.org/10.1093/ecco-jcc/jjy069

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. / 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); Brierley, Charlotte K.; Castilla-Llorente, Cristina; Labopin, Myriam; Badoglio, Manuela; Rovira, Montserrat; Ricart, Elena; Dierickx, Daan; Vermeire, Severine; Hasselblatt, Peter; Finke, Juergen; Onida, Francesco; Cassinotti, Andrea; Satsangi, Jack; Kazmi, Majid; López-Sanromán, Antonio; Schmidt, Carsten; Farge, Dominique; Travis, Simon P.L.; Hawkey, Chris J.; Snowden, John A.

In: Journal of Crohn's and Colitis, Vol. 12, No. 9, 29.08.2018, p. 1097-1103.

Research output: Contribution to journalArticle

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), Brierley, CK, Castilla-Llorente, C, Labopin, M, Badoglio, M, Rovira, M, Ricart, E, Dierickx, D, Vermeire, S, Hasselblatt, P, Finke, J, Onida, F, Cassinotti, A, Satsangi, J, Kazmi, M, López-Sanromán, A, Schmidt, C, Farge, D, Travis, SPL, Hawkey, CJ & Snowden, JA 2018, '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', Journal of Crohn's and Colitis, vol. 12, no. 9, pp. 1097-1103. https://doi.org/10.1093/ecco-jcc/jjy069
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), Brierley CK, Castilla-Llorente C, Labopin M, Badoglio M, Rovira M et al. 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. Journal of Crohn's and Colitis. 2018 Aug 29;12(9):1097-1103. https://doi.org/10.1093/ecco-jcc/jjy069
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) ; Brierley, Charlotte K. ; Castilla-Llorente, Cristina ; Labopin, Myriam ; Badoglio, Manuela ; Rovira, Montserrat ; Ricart, Elena ; Dierickx, Daan ; Vermeire, Severine ; Hasselblatt, Peter ; Finke, Juergen ; Onida, Francesco ; Cassinotti, Andrea ; Satsangi, Jack ; Kazmi, Majid ; López-Sanromán, Antonio ; Schmidt, Carsten ; Farge, Dominique ; Travis, Simon P.L. ; Hawkey, Chris J. ; Snowden, John A. / 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. In: Journal of Crohn's and Colitis. 2018 ; Vol. 12, No. 9. pp. 1097-1103.
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abstract = "Background and Aims Autologous haematopoietic stem cell transplantation [AHSCT] is a therapeutic option for patients with severe, treatment-refractory Crohn's disease [CD]. The evidence base for AHSCT for CD is limited, with one randomised trial [ASTIC] suggesting benefit. The aim of this study was to evaluate safety and efficacy for patients undergoing AHSCT for CD in Europe, outside the ASTIC trial. Methods We identified 99 patients in the European Society for Blood and Marrow Transplantation [EBMT] registry, who were eligible for inclusion. Transplant and clinical outcomes were obtained for 82 patients from 19 centres in seven countries. Results Median patient age was 30 years [range 20-65]. Patients had failed or been intolerant to a median of six lines of drug therapy; 61/82 [74{\%}] had had surgery. Following AHSCT, 53/78 [68{\%}] experienced complete remission or significant improvement in symptoms at a median follow-up of 41 months [range 6-174]; 22/82 [27{\%}] required no medical therapy at any point post-AHSCT. In patients who had re-started medical therapy at latest follow-up, 57{\%} [24/42] achieved remission or significant symptomatic improvement with therapies to which they had previously lost response or been non-responsive. Treatment-free survival at 1 year was 54{\%}. On multivariate analysis, perianal disease was associated with adverse treatment-free survival (hazard ratio 2.34, 95{\%} confidence interval [CI] 1.14-4.83, p = 0.02). One patient died due to infectious complications [cytomegalovirus disease] at Day +56. Conclusions In this multicentre retrospective analysis of European centres, AHSCT was relatively safe and appeared to be effective in controlling otherwise treatment-resistant Crohn's disease. Further prospective randomised controlled trials against standard of care are warranted.",
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T1 - Autologous haematopoietic stem cell transplantation for Crohn's disease

T2 - A retrospective survey of long-term outcomes from the European Society for Blood and Marrow Transplantation

AU - 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)

AU - Brierley, Charlotte K.

AU - Castilla-Llorente, Cristina

AU - Labopin, Myriam

AU - Badoglio, Manuela

AU - Rovira, Montserrat

AU - Ricart, Elena

AU - Dierickx, Daan

AU - Vermeire, Severine

AU - Hasselblatt, Peter

AU - Finke, Juergen

AU - Onida, Francesco

AU - Cassinotti, Andrea

AU - Satsangi, Jack

AU - Kazmi, Majid

AU - López-Sanromán, Antonio

AU - Schmidt, Carsten

AU - Farge, Dominique

AU - Travis, Simon P.L.

AU - Hawkey, Chris J.

AU - Snowden, John A.

PY - 2018/8/29

Y1 - 2018/8/29

N2 - Background and Aims Autologous haematopoietic stem cell transplantation [AHSCT] is a therapeutic option for patients with severe, treatment-refractory Crohn's disease [CD]. The evidence base for AHSCT for CD is limited, with one randomised trial [ASTIC] suggesting benefit. The aim of this study was to evaluate safety and efficacy for patients undergoing AHSCT for CD in Europe, outside the ASTIC trial. Methods We identified 99 patients in the European Society for Blood and Marrow Transplantation [EBMT] registry, who were eligible for inclusion. Transplant and clinical outcomes were obtained for 82 patients from 19 centres in seven countries. Results Median patient age was 30 years [range 20-65]. Patients had failed or been intolerant to a median of six lines of drug therapy; 61/82 [74%] had had surgery. Following AHSCT, 53/78 [68%] experienced complete remission or significant improvement in symptoms at a median follow-up of 41 months [range 6-174]; 22/82 [27%] required no medical therapy at any point post-AHSCT. In patients who had re-started medical therapy at latest follow-up, 57% [24/42] achieved remission or significant symptomatic improvement with therapies to which they had previously lost response or been non-responsive. Treatment-free survival at 1 year was 54%. On multivariate analysis, perianal disease was associated with adverse treatment-free survival (hazard ratio 2.34, 95% confidence interval [CI] 1.14-4.83, p = 0.02). One patient died due to infectious complications [cytomegalovirus disease] at Day +56. Conclusions In this multicentre retrospective analysis of European centres, AHSCT was relatively safe and appeared to be effective in controlling otherwise treatment-resistant Crohn's disease. Further prospective randomised controlled trials against standard of care are warranted.

AB - Background and Aims Autologous haematopoietic stem cell transplantation [AHSCT] is a therapeutic option for patients with severe, treatment-refractory Crohn's disease [CD]. The evidence base for AHSCT for CD is limited, with one randomised trial [ASTIC] suggesting benefit. The aim of this study was to evaluate safety and efficacy for patients undergoing AHSCT for CD in Europe, outside the ASTIC trial. Methods We identified 99 patients in the European Society for Blood and Marrow Transplantation [EBMT] registry, who were eligible for inclusion. Transplant and clinical outcomes were obtained for 82 patients from 19 centres in seven countries. Results Median patient age was 30 years [range 20-65]. Patients had failed or been intolerant to a median of six lines of drug therapy; 61/82 [74%] had had surgery. Following AHSCT, 53/78 [68%] experienced complete remission or significant improvement in symptoms at a median follow-up of 41 months [range 6-174]; 22/82 [27%] required no medical therapy at any point post-AHSCT. In patients who had re-started medical therapy at latest follow-up, 57% [24/42] achieved remission or significant symptomatic improvement with therapies to which they had previously lost response or been non-responsive. Treatment-free survival at 1 year was 54%. On multivariate analysis, perianal disease was associated with adverse treatment-free survival (hazard ratio 2.34, 95% confidence interval [CI] 1.14-4.83, p = 0.02). One patient died due to infectious complications [cytomegalovirus disease] at Day +56. Conclusions In this multicentre retrospective analysis of European centres, AHSCT was relatively safe and appeared to be effective in controlling otherwise treatment-resistant Crohn's disease. Further prospective randomised controlled trials against standard of care are warranted.

KW - Autoimmune disease; autologous haematopoietic stem cell transplant

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