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Safety analysis of patients who received ruxolitinib for steroid-refractory acute or chronic graft-versus-host disease in an expanded access program

Bone Marrow Transplantation volume 57, pages 975–981 (2022)Cite this article

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Abstract

Outside of clinical trials and before commercial availability for acute and chronic graft-versus-host disease (GVHD), the Janus kinase (JAK) 1/JAK2 inhibitor ruxolitinib was available to US patients with steroid-refractory GVHD through an open-label, multicenter expanded access program (EAP) sponsored by Incyte Corporation. To assess the safety of ruxolitinib, data on serious adverse events (SAEs) reported among patients in the EAP were collected. Patients ≥12 years old who received allogeneic hematopoietic cell transplantation for a hematologic malignancy and developed any-grade acute or chronic steroid-refractory GVHD received ruxolitinib at a starting dose of 5 mg twice daily (BID; acute GVHD) or 10 mg BID (chronic GVHD). At data extraction (May 8, 2020), 60 patients with acute GVHD and 549 with chronic GVHD were enrolled. In the acute and chronic GVHD cohorts, 41 (68.3%) and 186 (33.9%) patients, respectively, had ≥1 SAE. Sepsis (8.3%) and respiratory failure (6.7%) were the most common SAEs in the acute GVHD cohort, and pneumonia (4.9%), sepsis (3.8%), and lung infection (3.5%) in chronic GVHD. Infection SAEs were reported in 23.3% and 20.0% of patients with acute and chronic GVHD, respectively. Overall, these safety findings demonstrate the tolerability of ruxolitinib in steroid-refractory GVHD.

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Data availability

Incyte Corporation (Wilmington, DE, USA) is committed to data sharing that advances science and medicine while protecting patient privacy. Qualified external scientific researchers may request anonymized datasets owned by Incyte for the purpose of conducting legitimate scientific research. Researchers may request anonymized datasets from any interventional study (except Phase 1 studies) for which the product and indication have been approved on or after 1—January 2020 in at least one major market (e.g., US, EU, JPN). Data will be available for request after the primary publication or 2 years after the study has ended. Information on Incyte’s clinical trial data-sharing policy and instructions for submitting clinical trial data requests are available at: https://www.incyte.com/Portals/0/Assets/Compliance%20and%20Transparency/clinical-trial-data-sharing.pdf?ver=2020-05-21-132838-960.

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Acknowledgements

Support for this study was provided by Incyte Corporation (Wilmington, DE, USA). Writing assistance was provided by Jane Kovalevich, Ph.D., an employee of ICON (Blue Bell, PA, USA), and was funded by Incyte Corporation.

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Authors and Affiliations

  1. Division of Oncology, Washington University School of Medicine in St. Louis, St. Louis, MO, USA

    Mark A. Schroeder & John F. DiPersio

  2. Division of Hematology and Oncology, Medical College of Wisconsin, Milwaukee, WI, USA

    Parameswaran N. Hari

  3. US Medical Affairs, Incyte Corporation, Wilmington, DE, USA

    Amy Blithe, Dilan Paranagama & Valkal Bhatt

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  1. Mark A. Schroeder
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Contributions

MAS contributed to data acquisition, data interpretation, and paper writing. PNH contributed to data acquisition, data interpretation, and paper writing. AB contributed to study conceptualization, data management, and paper writing. DP contributed to study conceptualization, statistical analyses, data interpretation, and paper writing. VB contributed to study conceptualization, data interpretation, and paper writing. JFD contributed to data acquisition, data interpretation, and paper writing. All authors approved the final submitted version of the paper.

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Correspondence to Mark A. Schroeder.

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Competing interests

MAS has served on advisory boards and received honoraria or consultancy fees unrelated to the present work from CareDx, Incyte Corporation, and Sanofi Genzyme. PNH has received consulting fees from Amgen, BMS, GSK, Incyte Corporation, Janssen, Karyopharm, and Takeda. AB, DP, and VB are employees and shareholders of Incyte Corporation. JFD served on advisory boards for Arch, Bioline, Incyte Corporation, Macrogenics, and Rivervest and is a cofounder of Magenta Therapeutics and WUGEN.

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Schroeder, M.A., Hari, P.N., Blithe, A. et al. Safety analysis of patients who received ruxolitinib for steroid-refractory acute or chronic graft-versus-host disease in an expanded access program. Bone Marrow Transplant 57, 975–981 (2022). https://doi.org/10.1038/s41409-022-01673-y

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