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About
The purpose of this study is to evaluate the safety and potential clinical benefit of lymphoablation followed by autologous HSCT rescue in therapy refractory Crohn's disease
Full description
The treatment of Crohn's disease has proven to be quite efficacious in the majority of patients with the timely use of combination therapies for remission induction (corticosteroids and/or biologics) and maintenance of disease control (immunosuppressives and/or biologics). However, a proportion of patients fail to achieve complete and long term disease control and often require multiple intestinal surgeries with a risk of developing short bowel syndrome. Lymphoablation followed by hematopoietic stem cell transplantation to rescue the immune system has been proposed as an alternative strategy to induce long term disease control in this high risk population. It has been demonstrated that despite the potential toxicity and morbidity associated with the procedure, the benefit-risk ratio is favorable. Hence, we propose to offer HSCT to selected CD patients, and to study mechanisms of reducing T cell autoreactivity which will hopefully lead to more focused therapeutic approaches in the future.
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Inclusion criteria
Age between 7 and 50 years (patients aged 50 - 70 can participate up to the principal investigators discretion).
Confirmed diagnosis of active Crohn's disease:
i)Crohn's Disease Activity Index (CDAI) > 250, and ii)Two of the following:
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.
Informed consent:
Exclusion criteria
Pregnancy or unwillingness to use adequate contraception during the study, in women of childbearing age. Unwillingness of using appropriate contraceptive measures in males.
Concomitant severe disease
Infection or risk thereof:
Significant malnutrition: Body Mass Index (BMI) ≤ 18, serum albumin < 20g/l.
Previous poor compliance.
Concurrent enrollment in any other protocol using an investigational drug or hematopoietic growth factor up to four weeks before study entry.
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0 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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