Could stem cell transplantation be a fruitful solution for refractory CD?

Autologous stem cell transplantation (ASCT) may be an effective and safe option for patients with refractory Crohn’s disease (CD). Performing this procedure in centres of expertise and with a conditioning regimen of cyclophosphamide and anti-thymocyte globulin delivered promising results, providing health benefits for this difficult-to-treat population with a high disease burden.

Dr Louis Cohen (Icahn School of Medicine at Mount Sinai, NY, USA) and his study group evaluated the use of ASCT among 104 participants with refractory CD [1]. The participants needed to be refractory to all licensed medications and were not amenable to surgery. The participants received a conditioning regimen of cyclophosphamide and anti-thymocyte globulin. In the current analysis, Dr Cohen shared endoscopic, clinical, and safety results from 68 participants. “These participants were young, with a median age of 33 years, and had a median duration of disease of 14.9 years,” mentioned Dr Cohen.

At 2 years of follow-up, 65% of the participants displayed clinical remission, and another 7% showed a clinical response. Also, 54% of the participants were in endoscopic remission, and another 22% had an endoscopic response (see Figure). “We also saw a notable improvement in the quality-of-life of the 10 participants we followed at Mount Sinai,” added Dr Cohen. “Next to this, participants may be able to re-initiate advanced therapies they had previously failed to possibly achieve long-term remission.” Notably, the procedure was not associated with long-term (>100 days post-ASCT) complications such as renal failure, malignancy, or death. There were also no cases of intestinal failure with the current procedure or conditioning regimen.

Figure: Summary of outcomes for patients with refractory CD receiving ASCT [1]



“Since the 5-year mortality rate is 13% in patients with refractory CD, doing nothing is doing harm,” argued Dr Cohen. “We need to track the long-term efficacy and safety of ASCT, establish standard protocols, and identify biomarkers of transplant response to see if this therapy can become a standard-of-care.”

1. Cohen LJ, et al. Efficacy and safety of autologous hematopoietic stem cell transplantation in refractory Crohn’s disease: outcomes from an international stem cell transplant consortium. OP05, 20th Congress of ECCO, 19–22 February 2025, Berlin, Germany.

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Posted on 8 April 20258 April 2025