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Multicenter Study
. 2018 Aug 29;12(9):1097-1103.
doi: 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

Charlotte K Brierley  1 , Cristina Castilla-Llorente  2 , Myriam Labopin  3 , Manuela Badoglio  3 , Montserrat Rovira  4 , Elena Ricart  5 , Daan Dierickx  6 , Severine Vermeire  7 , Peter Hasselblatt  8 , Juergen Finke  9 , Francesco Onida  10 , Andrea Cassinotti  11 , Jack Satsangi  12 , Majid Kazmi  13 , Antonio López-Sanromán  14 , Carsten Schmidt  15 , Dominique Farge  16 , Simon P L Travis  17 , Chris J Hawkey  18 , John A Snowden  19 ; European Society for Blood and Marrow Transplantation [EBMT] Autoimmune Diseases Working Party [ADWP]
Affiliations
Multicenter Study

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

Charlotte K Brierley et al. J Crohns Colitis. .

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.

Keywords: Autoimmune disease; autologous haematopoietic stem cell transplant; Crohn’s disease.

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Figures

Figure 1.
Figure 1.
Clinical disease response. Percentage of patients in each clinical disease response category [remission, improvement, stable disease, and worsening] at 100 days, 1 year and latest follow-up [median 3.4 years].
See this image and copyright information in PMC

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