Hematopoietic stem cell transplantation in Crohn's disease: a comprehensive review

Despite advances in medical therapies for the treatment of Crohn's disease (CD), 20-30% of patients fail to respond to these therapies (i.e. refractory CD). Medically refractory CD leads to significant disability increasing morbidity and mortality. To prevent the disability of refractory CD, hematopoietic stem cell transplantation (SCT) has emerged as a therapeutic strategy.

Autologous (auto-SCT) and allogeneic SCT (allo-SCT) have been explored in clinical trials for refractory CD patients. We will review the stem cell transplant process, how each part of stem cell transplantation affects clinical efficacy and safety, and how specific clinical trials advanced our understanding of the role of stem cell transplant in the treatment of refractory CD.

As multiple clinical trials using the same auto-SCT protocol demonstrated auto-SCT as clinically efficacious for refractory CD it supports that this treatment may be adopted as standard of care for select patients with refractory CD. To establish auto-SCT as a standard therapy will require the creation of international registries to track long-term SCT outcomes and translational studies to refine SCT protocols for CD patients as a cellular therapy that truly restores healthy intestinal immune cell populations from hematopoietic stem cells.