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

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.

Authors:   Alex Ganetsky; Noelle V. Frey; Elizabeth O. Hexner; Alison W. Loren; Saar I. Gill; Selina M. Luger; James K. Mangan; Mary Ellen Martin; Daria V. Babushok; William R. Drobyski; Jacqueline Smith; Colleen Timlin; Craig W. Freyer; Edward A. Stadtmauer; David L. Porter
Journal:   Bone Marrow Transplantation
Year:   2018
Pages:   1
DOI:   10.1038/s41409-018-0236-z
Publication date:   24-May-2018
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