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Clinical outcomes of ruxolitinib treatment in 595 intermediate-1 risk patients with myelofibrosis: The RUX-MF Real-World Study.
Palandri, Francesca; Elli, Elena M; Morsia, Erika; Benevolo, Giulia; Tiribelli, Mario; Beggiato, Eloise; Bonifacio, Massimiliano; Farina, Mirko; Martino, Bruno; Caocci, Giovanni; Pugliese, Novella; Tieghi, Alessia; Crugnola, Monica; Binotto, Gianni; Cavazzini, Francesco; Abruzzese, Elisabetta; Iurlo, Alessandra; Isidori, Alessandro; Bosi, Costanza; Guglielmana, Veronica; Venturi, Marta; Dedola, Alessandra; Loffredo, Michele; Fontana, Gabriele; Duminuco, Andrea; Moioli, Alessia; Tosoni, Luca; Scalzulli, Emilia; Cattaneo, Daniele; Lemoli, Roberto M; Cilloni, Daniela; Bocchia, Monica; Pane, Fabrizio; Heidel, Florian H; Vianelli, Nicola; Cavo, Michele; Palumbo, Giuseppe A; Branzanti, Filippo; Breccia, Massimo.
Afiliación
  • Palandri F; IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia "Seràgnoli", Bologna, Italy.
  • Elli EM; Fondazione IRCCS San Gerardo dei Tintori, divisione di ematologia e unità trapianto di midollo, Monza, Italy.
  • Morsia E; Hematology Unit, Department of Clinical and Molecular Sciences, DISCLIMO, Università Politecnica delle Marche, Ancona, Italy.
  • Benevolo G; University Hematology Division, Città della Salute e della Scienza Hospital, Torino, Italy.
  • Tiribelli M; Division of Hematology and BMT, Department of Medicine, University of Udine, Udine, Italy.
  • Beggiato E; Unit of Hematology, Department of Oncology, University of Torino, Torino, Italy.
  • Bonifacio M; Department of Engineering for Innovation Medicine, Section of Innovation Biomedicine, Hematology Area, University of Verona, Verona, Italy.
  • Farina M; Unit of Blood Diseases and Stem Cells Transplantation, Department of Clinical and Experimental Sciences, University of Brescia, ASST Spedali Civili of Brescia, Brescia, Italy.
  • Martino B; Division of Hematology, Azienda Ospedaliera 'Bianchi Melacrino Morelli', Reggio Calabria, Italy.
  • Caocci G; Ematologia, Ospedale Businco, Università degli studi di Cagliari, Cagliari, Italy.
  • Pugliese N; Department of Clinical Medicine and Surgery, Federico II University Medical School, Naples, Italy.
  • Tieghi A; Department of Hematology, Azienda USL - IRCCS di Reggio Emilia, Reggio Emilia, Italy.
  • Crugnola M; Haematology and BMT Centre, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy.
  • Binotto G; Unit of Hematology and Clinical Immunology, University of Padova, Padova, Italy.
  • Cavazzini F; Division of Hematology, University of Ferrara, Ferrara, Italy.
  • Abruzzese E; Division of Hematology, Ospedale S. Eugenio, Roma, Italy.
  • Iurlo A; Hematology Division, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Isidori A; Hematology and Stem Cell Transplant Center, AORMN Hospital, Pesaro, Italy.
  • Bosi C; Division of Hematology, AUSL di Piacenza, Piacenza, Italy.
  • Guglielmana V; Fondazione IRCCS San Gerardo dei Tintori, divisione di ematologia e unità trapianto di midollo, Monza, Italy.
  • Venturi M; Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale, Università di Bologna, Bologna, Italy.
  • Dedola A; Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale, Università di Bologna, Bologna, Italy.
  • Loffredo M; Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale, Università di Bologna, Bologna, Italy.
  • Fontana G; Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale, Università di Bologna, Bologna, Italy.
  • Duminuco A; Postgraduate School of Hematology, University of Catania, Catania, Italy.
  • Moioli A; Department of Engineering for Innovation Medicine, Section of Innovation Biomedicine, Hematology Area, University of Verona, Verona, Italy.
  • Tosoni L; Division of Hematology and BMT, Department of Medicine, University of Udine, Udine, Italy.
  • Scalzulli E; Hematology, Department of Translational and Precision Medicine, Az. Policlinico Umberto I-Sapienza University, Rome, Italy.
  • Cattaneo D; Hematology Division, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Lemoli RM; IRCCS Ospedale Policlinico San Martino, Genoa, Italy.
  • Cilloni D; Dipartimento di Medicina Interna e Specialità Mediche, Università di Genova, Genova, Italy.
  • Bocchia M; Department of Clinical and Biological Sciences, University of Turin, Turin, Italy.
  • Pane F; Hematology Unit, Azienda Ospedaliera Universitaria Senese, University of Siena, Siena, Italy.
  • Heidel FH; Department of Clinical Medicine and Surgery, Federico II University Medical School, Naples, Italy.
  • Vianelli N; Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School (MHH), Hannover, Germany.
  • Cavo M; IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia "Seràgnoli", Bologna, Italy.
  • Palumbo GA; IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia "Seràgnoli", Bologna, Italy.
  • Branzanti F; Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale, Università di Bologna, Bologna, Italy.
  • Breccia M; Department of Scienze Mediche, Chirurgiche e Tecnologie Avanzate "G.F. Ingrassia", University of Catania, Catania, Italy.
Cancer ; 2024 Jul 30.
Article en En | MEDLINE | ID: mdl-39078647
ABSTRACT

BACKGROUND:

Ruxolitinib (RUX) is a JAK1/2 inhibitor approved for the therapy of myelofibrosis (MF) based on clinical trials including only intermediate2-high risk (INT2/HIGH) patients. However, RUX is commonly used in intermediate-1 (INT1) patients, with scarce information on responses and outcome.

METHODS:

The authors investigated the benefit of RUX in 1055 MF patients, included in the "RUX-MF" retrospective study.

RESULTS:

At baseline (BL), 595 (56.2%) patients were at INT1-risk according to DIPSS (PMF) or MYSEC-PM (SMF). The spleen was palpable at <5 cm, between 5 and 10 cm, and >10 cm below costal margin in 5.9%, 47.4%, and 39.7% of patients, respectively; 300 (54.1%) were highly symptomatic (total symptom score ≥20). High-molecular-risk (HMR) mutations (IDH1/2, ASXL-1, SRSF2, EZH2, U2AF1Q157) were detected in 77/167 patients. A total of 101 (19.2%) patients had ≥1 cytopenia (Hb < 10 g/dL n.36; PLT <100 x 109/L n = 43; white blood cells <4 x 109/L n = 40). After 6 months on RUX, IWG-MRT-defined spleen and symptoms response rates were 26.8% and 67.9%, respectively. In univariate analysis, predictors of SR at 6 months were no HMR mutations odds ratio [OR], 2.0, p = .05], no cytopenia (OR, 2.10; p = .01), and blasts <1% (OR, 1.91; p = .01). In multivariate analysis, absence of HMR maintained a significant association (OR, 2.1 [1.12-3.76]; p = .01).

CONCLUSIONS:

In INT1 patients, responses were more frequent and durable, whereas toxicity rates were lower compared to INT2/high-risk patients. Presence of HMR mutations, cytopenia, and peripheral blasts identified less-responsive INT1 patients, who may benefit for alternative therapeutic strategies.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Cancer Año: 2024 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Cancer Año: 2024 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Estados Unidos