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 language | English |
|---|---|
| Pages (from-to) | 1097-1103 |
| Number of pages | 7 |
| Journal | Journal of Crohn's and Colitis |
| Volume | 12 |
| Issue number | 9 |
| DOIs | |
| Publication status | Published - Aug 29 2018 |
Fingerprint
Keywords
- Autoimmune disease; autologous haematopoietic stem cell transplant
- Crohn's disease
ASJC Scopus subject areas
- Gastroenterology
Cite this
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 journal › Article
}
TY - JOUR
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
KW - Crohn's disease
UR - http://www.scopus.com/inward/record.url?scp=85054748384&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85054748384&partnerID=8YFLogxK
U2 - 10.1093/ecco-jcc/jjy069
DO - 10.1093/ecco-jcc/jjy069
M3 - Article
AN - SCOPUS:85054748384
VL - 12
SP - 1097
EP - 1103
JO - Journal of Crohn's and Colitis
JF - Journal of Crohn's and Colitis
SN - 1873-9946
IS - 9
ER -