- In patients with severe, treatment-refractory Crohn's disease, autologous hematopoietic stem cell transplantation (AHSCT) appears to be safe and effective over the long term.
Why this matters
- There is only 1 previous clinical trial to support AHSCT.
- This registry study suggests that AHSCT is a viable option for severe, treatment-refractory Crohn's disease.
- Retrospective cohort of 82 patients from 19 centers in 7 countries entered into the European Society for Blood and Marrow Transplantation (EBMT) registry.
- Patients were followed for a median of 41 months (range: 6-174 months).
- Funding: European Union; Medical Research Council; Wellcome Trust; AbbVie.
- Patients (median age 30 years) had failed a median of 6 lines of drug therapy.
- 74% of the cohort had surgery.
- AHSCT was followed by complete remission or significant improvement in 68% of the cohort.
- AHSCT was followed by no medical therapy in 27% of the cohort.
- In the subset of patients who restarted medical therapy, 57% achieved remission or significant symptomatic improvement with therapies they had previously failed.
- 54% of the cohort had 1-year treatment-free survival.
- Perianal disease (vs no perianal disease) was linked to poorer treatment-free survival (HR, 2.34; P=.02).
- 1 patient died from cytomegalovirus disease.
- Retrospective design.
- No reference group.