9th Annual BMRP Investigator Meeting - Abstract
Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in Patients with Severe Anti-TNF Refractory Crohn’s Disease: Long-term Follow-up
Robert M. Craiga and Richard Burt
Northwestern University Feinberg School of Medicine (Chicago, Illinois, U.S.A.)
We evaluated the safety and clinical outcome of autologous non-myeloablative hematopoietic stem cell transplantation (HSCT) in patients with severe Crohn’s disease (CD) defined as a Crohn’s Disease Activity Index (CDAI) > 250, and/or Crohn’s Severity Index (CSI) > 16 despite anti-TNF therapy. Stem cells were mobilized from the peripheral blood using cyclophosphamide (2.0 g/m2) and G-CSF (10 ug/kg/day), enriched ex vivo by CD34+ selection, and re-infused after immune suppressive conditioning with cyclophosphamide (200 mg/kg) and either equine anti-thymocyte globulin (ATG) (90 mg/kg) or rabbit ATG (6 mg/kg). Eighteen of 24 patients are 5 or more years post transplant. All patients went into remission with CDAI < 150. The patient with the longest duration in remission is 9 years. There was no significant immediate complication from the HSCT except for leukopenic fever. Long-term, one had an abdominal abscess, one atypical mycobacterium pneumonia, and one H. zoster. There was no death from the procedure. The percentage of patients in remission (CDAI < 150), steroid free, or medication free at any post transplant evaluation interval over 5 years post transplant has remained at or above 70%, 80% and 60%, respectively. Percent clinical relapse-free survival defined as percent free of restarting CD medical therapy after transplant is 96% at 1 year, 73% at 2 years, 63% at 3 years, 50% at 4 years, and 36% at 5 years. The long-term results show that this therapy is a major advance for CD patients who have failed conventional therapy.
a Principal Investigator