Despite advances in immunosuppressive therapy, up to 10% of patients with severe Crohn's disease remain refractory to conventional treatment.
Limited evidence from pilot trials suggests that high-dose immunosuppression and autologous peripheral blood stem cell transplantation may induce remission in these patients, but there is substantial controversy regarding the safety and efficacy of this approach.
Professor Kreisel and colleagues from Germany performed a monocenter phase I/II trial of auto peripheral blood stem cell transplantation in patients with refractory Crohn's disease.
The research team reports on the outcome of 12 patients with refractory CD treated with auto peripheral blood stem cell transplantation.
|5 patients achieved remission within 6 months|
|Alimentary Pharmacology & Therapeutics|
The team harvested CD34+-selected peripheral blood stem cells after mobilization therapy with cyclophosphamide and granulocyte-colony stimulating factor.
Immunoablative conditioning therapy with high-dose cyclophosphamide followed by auto peripheral blood stem cell transplantation was applied, and clinical and endoscopic responses were analyzed after a mean follow-up of 3 years.
Peripheral blood stem cell transplantation harvest following mobilization chemotherapy was successful in 11 out of 12 patients, and resulted in a clinical and endoscopic improvement in 7 out of 12 patients.
The team performed subsequent conditioning and auto peripheral blood stem cell transplantation in 9 patients, and were relatively well tolerated.
Among those, 5 patients achieved a clinical and endoscopic remission within 6 months after auto peripheral blood stem cell transplantation.
The researchers observed that relapses occurred in 7 out of 9 patients during follow-up, but disease activity could be controlled by low-dose corticosteroids and conventional immunosuppressive therapy.
Professor Kreisel's team commented, "Immunoablation by cyclophosphamide and autologous peripheral blood stem cell transplantation is safe and effective to induce remission of refractory Crohn's disease, and should be further evaluated in randomised controlled trials."