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A pilot feasibility study of non-myeloablative allogeneic hematopoietic stem cell transplantation for refractory Crohn Disease

Bone Marrow Transplantation volume 55, pages 2343–2346 (2020)Cite this article

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Refractory Crohn disease (CD) has been treated with autologous hematopoietic stem cell transplantation (HSCT) using a cyclophosphamide and anti-thymocyte globulin conditioning regimen with or without CD34 selection of the autologous peripheral blood stem cells (PBSC) [1,2,3,4,5,6,7,8]. This regimen provided a treatment-free remission, but histologic evidence of disease activity persisted, and clinical relapses occurred in ~50% and 90–100% by 3 and 5 years, respectively [1,2,3,4,5,6,7,8,9,10]. In the only randomized clinical trial to date, the difference between autologous HSCT and the comparator arm in sustained remission defined as a Crohn disease activity index (CDAI) < 150 on no immune drugs and no endoscopic or radiologic evidence of disease was not significant [9, 10]. There have been no reports of sustained 5-year treatment-free remission without clinical, radiologic, endoscopic, or histologic evidence of disease after autologous HSCT.

We undertook a pilot study of non-myeloablative allogeneic HSCT using unselected matched sibling PBSC in three patients and umbilical cord blood (UCB) in six patients when a matched sibling donor was not available. UCB was 6/6 matched in four patients and 5/6 matched in two patients. Two UCB units were available for five patients. The trial was approved by the Institutional Review Board and the Food and Drug Administration and registered with www.clinicaltrials.gov NCT 01288053.

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Authors and Affiliations

  1. Division of Immunotherapy, Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA

    Richard K. Burt, Kathleen Quigley, Indira Arnautovic & Xiaoqiang Han

  2. Division of Gastroenterology and Hepatology, Department of Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA

    Robert Craig & Laura Yun

  3. Department of Surgery, Northwestern University Feinberg School of Medicine, Chicago, IL, USA

    Amy Halverson

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  1. Richard K. Burt
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  2. Robert Craig
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  7. Xiaoqiang Han
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Correspondence to Richard K. Burt.

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Burt, R.K., Craig, R., Yun, L. et al. A pilot feasibility study of non-myeloablative allogeneic hematopoietic stem cell transplantation for refractory Crohn Disease. Bone Marrow Transplant 55, 2343–2346 (2020). https://doi.org/10.1038/s41409-020-0953-y

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