ECP versus ruxolitinib in steroid-refractory acute GVHD - a retrospective study by the EBMT transplant complications working party: original article

Introduction: Extracorporal Photophoresis (ECP) is in clinical use for steroid-refractory and steroid-dependent acute GVHD (SR-aGVHD). Based on recent Phase-III study results, ruxolitinib has become the new standard of care for SR-aGVHD. Our aim was to collect comparative data between ruxolitinib an...

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Main Authors: Penack, Olaf (Author) , Peczynski, Christophe (Author) , Boreland, William (Author) , Lemaitre, Jessica (Author) , Afanasyeva, Ksenia (Author) , Kornblit, Brian (Author) , Jurado, Manuel (Author) , Martinez, Carmen (Author) , Natale, Annalisa (Author) , Pérez-Simón, Jose Antonio (Author) , Brunello, Lucia (Author) , Avenoso, Daniele (Author) , Klein, Stefan (Author) , Ozkurt, Zubeyde Nur (Author) , Herrera, Concha (Author) , Wichert, Stina (Author) , Chiusolo, Patrizia (Author) , Gavriilaki, Eleni (Author) , Basak, Grzegorz W. (Author) , Schoemans, Hélène (Author) , Koenecke, Christian (Author) , Moiseev, Ivan (Author) , Peric, Zinaida (Author)
Format: Article (Journal)
Language:English
Published: 11 December 2023
In: Frontiers in immunology
Year: 2023, Volume: 14, Pages: 1-9
ISSN:1664-3224
DOI:10.3389/fimmu.2023.1283034
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.3389/fimmu.2023.1283034
Verlag, kostenfrei, Volltext: https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2023.1283034/full
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Author Notes:Olaf Penack, Christophe Peczynski, William Boreland, Jessica Lemaitre, Ksenia Afanasyeva, Brian Kornblit, Manuel Jurado, Carmen Martinez, Annalisa Natale, Jose Antonio Pérez-Simón, Lucia Brunello, Daniele Avenoso, Stefan Klein, Zubeyde Nur Ozkurt, Concha Herrera, Stina Wichert, Patrizia Chiusolo, Eleni Gavriilaki, Grzegorz W. Basak, Hélène Schoemans, Christian Koenecke, Ivan Moiseev and Zinaida Peric

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520 |a Introduction: Extracorporal Photophoresis (ECP) is in clinical use for steroid-refractory and steroid-dependent acute GVHD (SR-aGVHD). Based on recent Phase-III study results, ruxolitinib has become the new standard of care for SR-aGVHD. Our aim was to collect comparative data between ruxolitinib and ECP in SR-aGVHD in order to improve the evidence base for clinical decision making. Methods: We asked EBMT centers if they were willing to participate in this study by completing a data form (Med-C) with detailed information on GVHD grading, -therapy, -dosing, -response and complications for each included patient. Results: 31 centers responded positively (14%) and we included all patients receiving alloSCT between 1/2017-7/2019 and treated with ECP or ruxolitinib for SR-aGVHD grades II-IV from these centers. We identified 53 and 40 patients with grades II-IV SR-aGVHD who were treated with ECP and ruxolitinib, respectively. We performed multivariate analyses adjusted on grading and type of SR-aGVHD (steroid dependent vs. refractory). At day+90 after initiation of treatment for SR-aGVHD we found no statistically significant differences in overall response. The odds ratio in the ruxolitinib group to achieve overall response vs. the ECP group was 1.13 (95% CI = [0.41; 3.22], p = 0.81). In line, we detected no statistically significant differences in overall survival, progression-free survival, non-relapse mortality and relapse incidence. Discussion: The clinical significance is limited by the retrospective study design and the current data can’t replace prospective studies on ECP in SR-aGVHD. However, the present results contribute to the accumulating evidence on ECP as an effective treatment option in SR-aGVHD. 
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