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No benefit of stem cell transplant for refractory Crohn's patients
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Results from a randomized controlled trial showed autologous hematopoietic stem cell transplantation did not significantly improve sustained remission at 1 year in patients with refractory Crohn’s disease, and was associated with significant toxicity.
“Case reports and series describe long-term treatment-free disease regression with autologous and allogeneic [hematopoietic stem cell transplantation] in some but not all patients with Crohn’s disease,” the researchers wrote. “To follow up on these promising but preliminary data, we conducted the Autologous Stem Cell Transplantation International Crohn’s Disease … trial to evaluate the effect of autologous [hematopoietic stem cell transplantation] on disease activity, mucosal healing and quality of life in patients with resistant Crohn’s disease.”
The researchers enrolled adult patients from 11 transplant units in six European countries who had Crohn’s disease refractory to treatment with three or more immunosuppressants or biologics in addition to corticosteroids, who had impaired quality of life and who were unwilling to undergo surgery. All patients underwent stem cell mobilization, and from July 2007 to September 2011, 23 patients were randomly assigned to undergo immediate hematopoietic stem cell transplantation (HSCT), while 22 were assigned to undergo conventional therapy for 1 year and then delayed HSCT. Standard care was provided as needed during follow-up to March 2013.
One-year sustained disease remission served as the primary endpoint, defined as a composite of clinical remission, no use of corticosteroids, immunosuppressants or biologics for the last 3 months or longer, and no evidence of active disease confirmed by endoscopy or radiology.
The researchers found no significant difference in the primary endpoint, with 8.7% in the HSCT group vs. 4.5% of controls achieving sustained disease remission and 60.9% of the HSCT group vs. 22.7% of controls able to discontinue active treatment in the last 3 months (median difference, 38.1; 95% CI, 9.3-59.3). In 19 HSCT patients, 76 serious adverse events occurred vs. 38 in 15 control patients; serious adverse events were more common in the HSCT group in the 100 days after HSCT (for number of events, P = .02; for number of patients, P = .01).
“These findings do not support the widespread use of HSCT for patients with refractory Crohn’s disease,” the researchers concluded. – by Adam Leitenberger
Disclosures: Hawkey reports he received an NIH Research Senior Investigator Award and funding from the University of Nottingham Medical School Dean’s Fund and the Nottingham University Hospitals NHS Trust Research and Development Fund.
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