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

Abstract

Background and AimsAutologous 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.MethodsWe 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.ResultsMedian 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.ConclusionsIn 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.

New and Old Therapeutic Options for Luminal Crohn's Disease - An Overview of Current Literature

“…A recent Retrospective Survey of Long-term Outcomes from the European Society for Blood and Marrow Transplantation found 68% post AHSCT patients experienced complete remission/significant improvement in symptoms at a median follow-up of 41 months. It again highlighted re-sensitivity with 57% (24/42) patients responsive to therapies of which they had previously been refractory, and similar to ASTIC perianal disease was associated with adverse treatment free survival [107].…”

Inflammatory bowel diseases and primary immunodeficiency diseases

“…In this study, autologous HSCT did not significantly improve disease remission rate at 1 year, compared with conventional therapy. In contrast, a recent multicentre retrospective analysis showed that autologous HSCT is relatively safe and effectively controls treatment-resistant CD [54]. Further prospective randomized controlled trials of autologous HSCT vs. the standard of care are warranted.…”

Conventional and Future Therapies for Crohn's Disease

“…A multicentre retrospective analysis of European centres involving 99 patients showed AHSCT to be relatively safe and effective in treatment-resistant CD. [61] Patients who have been treated with AHSCT have reported significant clinical improvement. On the other hand, The AHSCT for refractory Crohn's Disease (ASTIC) trial with the objective of evaluating the effect of autologous HSCT on refractory Crohn's disease compared AHSCT (n=23) vs standard CD treatment (n=22) and found that Eight (34.8%) vs 2 (9.1%) patients were free of active disease on endoscopy and radiology at final assessment (difference, 25.7% [95% CI, 1.1% to 47.1%]; P = .054) with a Crohn's Disease Activity Index (CDAI) <150+ at ≥3 months.…”