Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease: Long-term follow-up

Richard K. Burt, Robert M. Craig, Francesca Milanetti, Kathleen Quigley, Paula Gozdziak, Jurate Bucha, Alessandro Testori, Amy Halverson, Larissa Verda, Willem J.S. De Villiers, Borko Jovanovic, Yu Oyama

Research output: Contribution to journalArticle

116 Scopus citations

Abstract

We evaluated the safety and clinical outcome of autologous nonmyeloablative hematopoietic stem cell transplantation (HSCT) in patients with severe Crohn disease (CD) defined as a Crohn Disease Activity Index (CDAI) greater than 250, and/or Crohn Severity Index greater than 16 despite anti-tumor necrosis factor therapy. Stem cells were mobilized from the peripheral blood using cyclophosphamide (2.0 g/m2) and G-CSF (10 μg/kg/day), enriched ex vivo by CD34+ selection, and reinfused after immune suppressive conditioning with cyclophosphamide (200 mg/kg) and either equine antithymocyte globulin (ATG, 90 mg/kg) or rabbit ATG (6 mg/kg). Eighteen of 24 patients are 5 or more years after transplantation. All patients went into remission with a CDAI less than 150. The percentage of clinical relapse-free survival defined as the percent free of restarting CD medical therapy after transplantation is 91% at 1 year, 63% at 2 years, 57% at 3 years, 39% at 4 years, and 19% at 5 years. The percentage of patients in remission (CDAI <150), steroid-free, or medication-free at any posttransplantation evaluation interval more than 5 years after transplantation has remained at or greater than 70%, 80%, and 60%, respectively. This trial was registered at www.clinicaltrials.gov as NCT0027853.

Original languageEnglish (US)
Pages (from-to)6123-6132
Number of pages10
JournalBlood
Volume116
Issue number26
DOIs
StatePublished - Dec 23 2010

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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    Burt, R. K., Craig, R. M., Milanetti, F., Quigley, K., Gozdziak, P., Bucha, J., Testori, A., Halverson, A., Verda, L., De Villiers, W. J. S., Jovanovic, B., & Oyama, Y. (2010). Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease: Long-term follow-up. Blood, 116(26), 6123-6132. https://doi.org/10.1182/blood-2010-06-292391