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.
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., European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) None.
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.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 7 countries.Median patient age was 30 years (range 20-65). Patients had failed or been intolerant to a median of 6 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 last 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 one year was 54%. On multivariate analysis, perianal disease was associated with adverse treatment-free survival (hazard ratio 2.34, 95% CI 1.14-4.83, p=0.02). One patient died due to infectious complications (CMV disease) at day +56.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.