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 L Halverson, Larissa Verda, Willem J.S. De Villiers, Borko Jovanovic, Yu Oyama

Research output: Contribution to journalArticle

105 Citations (Scopus)

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

Fingerprint

Hematopoietic Stem Cell Transplantation
Stem cells
Crohn Disease
Refractory materials
Transplantation
Cyclophosphamide
Antilymphocyte Serum
Granulocyte Colony-Stimulating Factor
Horses
Blood
Stem Cells
Tumor Necrosis Factor-alpha
Steroids
Rabbits
Safety
Recurrence
Survival
Therapeutics

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Burt, Richard K ; Craig, Robert M. ; Milanetti, Francesca ; Quigley, Kathleen ; Gozdziak, Paula ; Bucha, Jurate ; Testori, Alessandro ; Halverson, Amy L ; Verda, Larissa ; De Villiers, Willem J.S. ; Jovanovic, Borko ; Oyama, Yu. / Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease : Long-term follow-up. In: Blood. 2010 ; Vol. 116, No. 26. pp. 6123-6132.
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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.",
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Burt, RK, Craig, RM, Milanetti, F, Quigley, K, Gozdziak, P, Bucha, J, Testori, A, Halverson, AL, Verda, L, De Villiers, WJS, 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, vol. 116, no. 26, pp. 6123-6132. https://doi.org/10.1182/blood-2010-06-292391

Autologous nonmyeloablative hematopoietic stem cell transplantation in patients with severe anti-TNF refractory Crohn disease : Long-term follow-up. / Burt, Richard K; Craig, Robert M.; Milanetti, Francesca; Quigley, Kathleen; Gozdziak, Paula; Bucha, Jurate; Testori, Alessandro; Halverson, Amy L; Verda, Larissa; De Villiers, Willem J.S.; Jovanovic, Borko; Oyama, Yu.

In: Blood, Vol. 116, No. 26, 23.12.2010, p. 6123-6132.

Research output: Contribution to journalArticle

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AU - Burt, Richard K

AU - Craig, Robert M.

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AU - Quigley, Kathleen

AU - Gozdziak, Paula

AU - Bucha, Jurate

AU - Testori, Alessandro

AU - Halverson, Amy L

AU - Verda, Larissa

AU - De Villiers, Willem J.S.

AU - Jovanovic, Borko

AU - Oyama, Yu

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N2 - 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.

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