Abstract
Ruxolitinib has been approved for the treatment of adults and pediatric patients ≥12 years with steroid refractory graft-versus-host disease (GvHD). However, real-life studies are needed to confirm the results of clinical trials and further assess its efficacy in special populations. We performed a descriptive, retrospective, multi-center study of 352 adults and 42 pediatric patients treated with ruxolitinib for steroid-refractory acute or chronic GvHD. Among 119 and 233 adult patients with acute and chronic GvHD, overall response rate (ORR) was 58.8% (CR 33.6%) and 65.7% (CR 18.5%), respectively. Corticosteroids were withdrawn in 59.2% and 40.1%, and ruxolitinib in 47.2% and 34.8% in the acute and chronic groups of responders. Among 29 and 13 pediatric patients with acute and chronic GvHD, ORR was 82.7% (CR 51.7%) and 100% (CR 23%), respectively. Among responder patients, corticosteroids were withdrawn in 72.7% and 50%, and ruxolitinib in 75% and 30.7% in both groups respectively. Ruxolitinib in the real world setting, showed similar results as compared to clinical trials. Its efficacy is maintained in subsequent lines of treatment. In the pediatric population, the data are more favorable. In the long-term follow-up, corticosteroids, ruxolitinib and other inmunosuppressive drugs could be eliminated in a remarkably proportion of patients.
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Fig. 1: Number of patients receiving immunosuppressive drugs among responder patients in the adult and pediatric groups.
Fig. 2: Overall survival between responder and non-responder patients.
Fig. 3: Event-free survival.
Data availability
The datasets generated during and/or analysed during the current study are available from the corresponding author on reasonable request.
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Acknowledgements
This project has been carried out through the GETH-TC (Spanish Group for Transplantation and Cellular Therapy—Grupo Español de Trasplante Hematopoyético y Terapia Celular) and funded by Novartis.
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Authors and Affiliations
Servicio de hematologia, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBIS)/CSIC, Universidad de Sevilla, Sevilla, Spain
Virginia Escamilla-Gómez, Patricia Alcalde-Mellado, Francisco Manuel Martín-Domínguez, Jose A. Perez-Simon & Francisco Manuel Martín Domínguez
Hematology Service, Hospital Universitario Ramón y Cajal. IRYCIS, Universidad de Alcalá, Madrid, Spain
Valentín García Gutiérrez, Beatriz Astibia-Mahillo & Anabelle Chinea-Rodriguez
Department of Hematology, Hospital Clínico Universitario de Salamanca (CAUSA/IBSAL), Salamanca, Spain
Lucía López-Corral & Marina Acera-Gómez
Hematology Department, Hospital Universitario de Gran Canaria Doctor Negrín, Las Palmas de Gran Canaria, Spain
Melissa Karina Torres Ochando, Asunción Borrero Borrego & Leslie González Pinedo
Hematology Department, Hospital Universitario de Navarra, Pamplona, Spain
Teresa Zudaire Ripa
Hematopoietic Transplant Department, Hospital Universitario Niño Jesús, Madrid, Spain
Marta González Vicent
Hematology Department, Hospital Clínico Universitario de Valencia, Instituto de Investigación Sanitaria INCLIVA, Valencia, Valencia, Spain
Ana Benzaquén & Ana Benzaquén Vallejos
Hematology Department, Hospital Universitario Miguel Servet, Zaragoza, Spain
Isabel Izquierdo Garcia & Isabel Izquierdo Garcia
Hematology Department, Hospital Universitario y Politécnico La Fe, Valencia, Spain
Pedro Asensi Cantó, Juan Montoro & Pedro Asensi Cantó
Hematology Department, HospitalUniversitario Vall d´Hebron, Barcelona, Spain
Guillermo Orti & David Valcárcel
Hematology and Oncology Department, Division of Pediatric Hematology and Oncology, Hospital Universitario Vall d´Hebron, Barcelona, Spain
Maria Isabel Benitez-Carabante, Cristina Diaz-de-Heredia & Maria Isabel Benítez Carabante
Hematology Deparment, HospitalUniversitario Germans Trias i Pujol - Institut Català d’Oncologia, Barcelona, Spain
Eloi Cañamero, Christelle Ferrá & Eloi Cañamero Giro
Hematology Department, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain
Irene García-Cadenas & Sara Redondo
Hemato-Oncology Pediatric Service, HospitalUniversitario La Paz, Madrid, Spain
Luisa Sisinni, Antonio Perez-Martínez & Antonio Perez Martínez
Department of Hematology, Hospital Duran i Reynals, Instituto Catalán de Oncología, L’Hospitalet De Llobregat, Barcelona, Spain
Alberto Mussetti
Hospital Universitario Son Espases, Islas Baleares, Spain
Lucía Garcia-Mañó & Lucía García Maño
Hematology Department, Hospital Universitario Central de Asturias, Oviedo, Spain
María Del Pilar Palomo-Moraleda
Hospital Universitario Virgen de las Nieves, Instituto de Investigación Biosanitaria, Granada, Spain
Pedro Antonio González-Sierra, Manuel Jurado & Manuel Jurado Chacón
Authors
Virginia Escamilla-Gómez
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2. Valentín García Gutiérrez
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Consortia
on behalf of the GETH-TC
Virginia Escamilla-Gómez
, Valentín García Gutiérrez
, Patricia Alcalde-Mellado
, Beatriz Astibia-Mahillo
, Anabelle Chinea-Rodriguez
, Lucía López-Corral
, Marina Acera-Gómez
, Melissa Karina Torres Ochando
, Asunción Borrero Borrego
, Leslie González Pinedo
, Teresa Zudaire Ripa
, Marta González Vicent
, Ana Benzaquén Vallejos
, Isabel Izquierdo Garcia
, Pedro Asensi Cantó
, Juan Montoro
, Francisco Manuel Martín Domínguez
, Guillermo Orti
, David Valcárcel
, Maria Isabel Benítez Carabante
, Cristina Diaz-de-Heredia
, Eloi Cañamero Giro
, Christelle Ferrá
, Irene García-Cadenas
, Sara Redondo
, Luisa Sisinni
, Antonio Perez Martínez
, Alberto Mussetti
, Lucía García Maño
, María Del Pilar Palomo-Moraleda
, Pedro Antonio González-Sierra
, Manuel Jurado Chacón
& Jose A. Perez-Simon
Contributions
VEG, VGG and JAPS contributed equally to this work. They were responsable for designing the review protocol, writing the protocol and report, conducting the research, screening potentially eligible candidates, collect data in the eCRF, extracting and analysing data, interpreting results and writing and review the paper. PAM, BAM, ACR, LLC, MAG, MKTO, ABB, LGP, TZR, MGV, AB, IIG, PAC, JM, FMMD, GO, DV, MIBC, CD-H, EC, CF, IGC, SR, LS, APM, AM, LGM, MDPPM, PAGS, MJ were responsable for screening potentially eligible candidates, collect data in the eCRF and review the paper. GETH-TC provided the electronic data base and served as an intermediary among investigators. Moreover, all the researchers belong to the aforementioned consortium.
Corresponding authors
Correspondence to Valentín García Gutiérrez or Jose A. Perez-Simon.
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Competing interests
The study was funded by Novartis Company. VGG and GETH-TC received support from Novartis Company. The rest of the authors declare no competing financial interests.
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Escamilla-Gómez, V., García Gutiérrez, V., Alcalde-Mellado, P. et al. Ruxolitinib in acute and chronic graft-versus-host disease: real life long-term experience in a multi-center study for adult and pediatric patients, on behalf of the GETH-TC. Bone Marrow Transplant (2024). https://doi.org/10.1038/s41409-024-02483-0
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Received:16 June 2024
Revised:13 November 2024
Accepted:19 November 2024
Published:11 December 2024
DOI:https://doi.org/10.1038/s41409-024-02483-0
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