Autologous Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease
Unfortunately, some patients with Crohn's disease (CD) fail to respond to the best clinical treatments and some only experience temporary benefit. For severe Crohn's disease, there is an experimental treatment called high dose immunoablation followed by autologous hematopoietic stem cell transplantation (HSCT). This study removes over active lymphocytes (immunoablation) and replaces them using blood stem cells that have been taken from the patient's own body. The aim of the study is to reset or reprogram the patient's immune system to its state prior to diagnosis.
• Aged 13-28 years are eligible
• Confirmed diagnosis of active Crohn's disease:
‣ Diagnosis of Crohn's disease based on typical radiological appearances and / or typical histology at least 6 months prior to screening.
⁃ Active disease at the time of registration to the trial, defined as
• i) PCDAI \> 30, and ii) Two of the following:
⁃ elevated CRP
⁃ endoscopic evidence of active disease confirmed by histology
⁃ clear evidence of active small bowel Crohn's disease on CT or MR enterography.
• Unsatisfactory course despite 3 immunosuppressive agents (usually azathioprine, methotrexate and infliximab, adalimumab and/or certolizumab) in addition to corticosteroids. Patients should have relapsing disease (i.e. 1 exacerbation/year) despite thiopurines, methotrexate and/or infliximab/adalimumab/certolizumab maintenance therapy or clear demonstration of intolerance / toxicity to these drugs.
• Current problems unsuitable for surgery or patient at risk for developing short bowel syndrome.
• Accepted by a majority of the members of the combined IBD Center as an appropriate candidate (see Selection description below).
• Informed consent
‣ Prepared to undergo additional study procedures as per trial schedule
⁃ Patient has undergone intensive counseling about risks