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Tocilizumab for the treatment of severe steroid-refractory acute graft-versus-host disease of the lower gastrointestinal tract

Bone Marrow Transplantation volume 54, pages 212–217 (2019)Cite this article

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Abstract

Steroid-refractory (SR) acute gastrointestinal (GI) graft-versus-host disease (GVHD) is associated with significant mortality in allogeneic hematopoietic cell transplantation recipients. We retrospectively evaluated the efficacy of tocilizumab for the treatment of SR biopsy-proven acute lower GI GVHD in 16 consecutive adult transplant recipients between October 2015 and July 2016. Tocilizumab 8 mg/kg was administered every 2 weeks until achievement of complete response, defined as resolution of all manifestations of GI GVHD, or until patients had progression or initiation of other therapy. Ten of 16 patients (62.5%; 95% CI, 0.39–82) achieved a complete response after a median time of 11 days (range, 2–28 days) from tocilizumab initiation. The median time to response onset (improvement in stage by at least 1) was 1 day (range, 1–4 days). Tocilizumab was administered at a median of 9 days (range, 3–75 days) from GVHD diagnosis and 10 days (range, 3–75 days) from initiation of high-dose steroids. At a median follow-up of 7.6 months (range, 0.8–27.7 months) from initiation of tocilizumab, 6/16 (37.5%) patients are alive and free of their underlying hematologic malignancy. Tocilizumab appears to be a highly active agent for the treatment of severe SR lower GI acute GVHD.

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Authors and Affiliations

  1. Department of Pharmacy, Hospital of the University of Pennsylvania, Philadelphia, PA, USA

    Alex Ganetsky, Colleen Timlin & Craig W. Freyer

  2. Blood and Marrow Transplantation Program, Abramson Cancer Center and the Division of Hematology and Oncology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA

    Noelle V. Frey, Elizabeth O. Hexner, Alison W. Loren, Saar I. Gill, Selina M. Luger, James K. Mangan, Mary Ellen Martin, Daria V. Babushok, Jacqueline Smith, Edward A. Stadtmauer & David L. Porter

  3. Division of Hematology and Oncology, Department of Internal Medicine, The Medical College of Wisconsin, Milwaukee, WI, USA

    William R. Drobyski

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  1. Alex Ganetsky
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Correspondence to Alex Ganetsky.

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Ganetsky, A., Frey, N.V., Hexner, E.O. et al. Tocilizumab for the treatment of severe steroid-refractory acute graft-versus-host disease of the lower gastrointestinal tract. Bone Marrow Transplant 54, 212–217 (2019). https://doi.org/10.1038/s41409-018-0236-z

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