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Ruxolitinib–ECP combination treatment for refractory severe chronic graft-versus-host disease


Glucocorticoid-refractory (SR) chronic (c) graft-versus-host disease (GVHD) is a multisystem immunological disease and the leading cause of non-relapse mortality (NRM) in patients surviving longer than 2 years after allogeneic hematopoietic cell transplantation. Both ruxolitinib (RUX) and extracorporeal photopheresis (ECP) have shown activity for SR-cGVHD which motivated us to treat refractory cGHVD patients with the RUX–ECP combination. In this retrospective survey, 23 patients received RUX–ECP as salvage therapy for SR-cGVHD. The best response (CR or PR) at any time point during treatment was 74% (17/23) including 9% (2/23) CR and 65% (15/23) PR. The 24-months-survival was 75% (CI 56.0–94.1). Newly diagnosed cytopenia occurred in 22% (5/23) and CMV reactivation was observed in 26% (6/23) of the patients. Serum levels of soluble interleukin-2 receptor (sIL-2R) correlated with response. Our retrospective analysis shows that the RUX–ECP combination is safe and has activity in a fraction of patients with SR-cGVHD, which needs validation in a prospective trial.

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Fig. 1: The combination of ruxolitinib and ECP improves symptoms in patients with SR-cGVHD.


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This study was supported by the Deutsche Forschungsgemeinschaft, Germany, SFB1160 (project P14 to RZ and Z02 to KMB), DFG under Germany’s Excellence Strategy—EXC-2189—Project ID: 390939984, GVHDCure (ERC consolidator grant to RZ), the Wilhelm Sander Stiftung (2008.046.5) and the Jose-Carreras Leukemia foundation grant number DJCLS 01R/2019 (RZ).

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Correspondence to Robert Zeiser.

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Conflict of interest

RZ received speakers fees from Incyte, Novartis, and Mallinckroth. The other authors have no conflict of interest with this manuscript to disclose.

Ethical approval

The analysis of soluble IL2R was approved by the ethics committee of the University Medical Center Freiburg, ethics approval number: 547/14. Ruxolitinib and ECP are used in clinical routine for cGVHD treatment based on multiple reports showing efficacy. Prior to treatment with ruxolitinib and ECP informed consent was obtained from each patient.

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Maas-Bauer, K., Kiote-Schmidt, C., Bertz, H. et al. Ruxolitinib–ECP combination treatment for refractory severe chronic graft-versus-host disease. Bone Marrow Transplant 56, 909–916 (2021).

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