Oyama Y et al. (2005) Autologous hematopoietic stem cell transplantation in patients with refractory Crohn's disease. Gastroenterology 128: 552–563

Autologous hematopoietic stem cell transplantation (HSCT) is a safe and feasible approach for treating patients with refractory Crohn's disease, according to data from a small, phase I study published in Gastroenterology.

Crohn's disease is characterized by acute and chronic inflammation of the bowel, which is thought to be caused by dysregulation of type I T-helper cell-mediated immune responses. Although treatments for Crohn's disease are available, including anti-inflammatory and immunosuppressive drugs, a certain proportion of patients do not respond to these approaches, or respond initially but then develop recurrent disease.

Preliminary data indicate that autologous HSCT might lead to clinical improvement in patients with Crohn's disease who are refractory to current treatment approaches. To investigate this further, Oyama et al. performed autologous HSCT in 12 patients who had failed to respond to conventional therapies (CROHN'S DISEASE ACTIVITY INDEX [CDAI] scores of ≥250). The procedure resulted in disease improvement in most patients and was well tolerated (apart from expected neutropenic or disease-related fevers). Neutrophil and platelet engraftment occurred at a median of 9.5 days (range 8–11) and 9 days (range 9–18), respectively. At a median follow-up time of 18.5 months only 1 patient had recurrent Crohn's disease and the remaining 11 patients remained in remission with CDAI scores of ≤150.

In this study, the authors also tested a new grading system for monitoring Crohn's disease severity, the Craig Crohn's Severity Index, which they designed to overcome some of the limitations of the CDAI system. Similar results were obtained using the two systems.

Oyama et al. conclude that: “Although longer follow-up is needed, further investigation of autologous HSCT for Crohn's disease appears warranted”.