Stem Cell Transplant Shows No Improvement in Crohn’s Disease

Specialty Pharmacy Times, May/June 2016, Volume 7, Issue 3

A HEMATOPOIETIC STEM CELL TRANSPLANT (HSCT) caused no significant improvement in patients with difficult-to-treat Crohn’s disease who were unresponsive to surgery.

In a study published in JAMA, 45 patients aged 18 to 50 with impaired quality of life from refractory Crohn’s disease and amenable to surgery were randomized to undergo autologous HSCT or HSCT deferred for 1 year.

The researchers found there was no statistically significant difference among patients who achieved sustained disease remission, with 8.7% achieving remission in the HSCT group versus 4.5% in the control group.

However, there was a statistically significant difference found in patients who discontinued treatment during the final 3 months of the study, with 61% of patients in the HSCT group versus 23% in the control group. In the HSCT cohort, 76 patients experienced a serious adverse event compared with 38 patients in the control group.

One patient died from HSCT. The study noted that optimal sustained remission after HSCT may require maintenance immunosuppressive therapy, and that patients could begin to show a response to treatments to which they were previously refractory.

As a result, future research is need to determine the benefit of maintenance therapy.

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