Stem Cell Transplant Not Found to Improve Crohn's Disease

Significant improvement in sustained disease remission following transplantation not observed.

A stem cell transplant not only failed to improve Crohn’s disease remission, it may also cause other serious issues in patients, a recent study indicates.

In adults with difficult-to-treat Crohn's disease who won’t benefit from surgery, a hematopoietic stem cell transplant (HSCT) did not result in significant improvement in sustained disease remission at 1 year compared with conventional therapy. Furthermore, the treatment was associated with significant toxicity, according to a study published in JAMA.

Researchers randomized 45 patients aged 18 to 50 years with impaired quality of life from refractory Crohn’s disease not amenable to surgery to undergo autologous HSCT or HSCT deferred for 1 year. All of the patients enrolled in the trial were also administered standard treatment.

There was not a statistically significant difference found among patients who achieved sustained disease remission, with 8.7% in the HSCT arm compared with 4.5% in the control arm.

There was, however, a statistically significant difference observed in patients who discontinued active treatment in the last 3 months, with 61% of patients in the HSCT arm compared with 23% in the control arm.

There was a significantly greater number of serious adverse events in the HSCT group, with 76 patients compared with 38 patients in the control arm. One patient who underwent HSCT died.

"Because very few patients achieved sustained disease remission, we conclude that HSCT is unlikely to alter the natural history of Crohn disease, and our findings argue against extension of HSCT to a wider group of patients outside of future additional trials," the authors wrote.

The authors noted that additional analysis of HSCT for the treatment of refractory Crohn’s disease may be beneficial.

"It is possible that optimal sustained remission after HSCT may require maintenance immunosuppressive therapy. It is also possible that patients will regain responsiveness to treatments to which they were previously refractory,” the authors wrote. “Therefore, future trials should assess the benefit of maintenance therapy. Toxicity will remain the most significant barrier to HSCT in patients with Crohn disease. Therefore, identification of factors that predict either the risk of adverse effects or response to treatment will enhance the utility of this treatment in clinical practice."