Is stem cell transplantation an effective treatment for Crohn disease?

Updated: Jul 26, 2019
  • Author: Leyla J Ghazi, MD; Chief Editor: Praveen K Roy, MD, AGAF  more...
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Answer

The first randomized controlled trial of stem cell transplantation in treatment-resistant Crohn disease was performed in 2013, in 45 patients with moderately to severely active disease. All patients underwent stem cell mobilization with cyclophosphamide and filgrastim and were then randomly assigned to immediate stem cell transplantation (at 1 month) or delayed transplantation (at 13 months; control group). At 1-year follow-up, objective endoscopic findings were substantially better in the treatment group; on the Simple Endoscopic Score for Crohn's Disease (SES-CD), the mean lower gastrointestinal score fell from 13 to 4 in the treatment group but remained unchanged in the control group. The treatment group also showed a greater decrease than the control group in median Crohn's Disease Activity Index score (approximately 165 vs 50 points). Two thirds of the treatment group were able to discontinue immunosuppressive drugs and steroids at 1 year, compared with 15% of the control group. [86]


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