Crohn's disease: autologous hematopoietic stem cell transplants safe, effective

  • Brierley CK & al.
  • J Crohns Colitis
  • 18 May 2018

  • curated by Miriam Davis, PhD
  • Clinical Essentials
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Takeaway

  • 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.

Study design

  • 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.

Key results

  • 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.

Limitations

  • Retrospective design.
  • No reference group.

 

 

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