Clinical outcomes of ruxolitinib treatment in 595 intermediate‐1 risk patients with myelofibrosis: The RUX‐MF Real‐World Study-Reference-Cited by-同舟云学术

Clinical outcomes of ruxolitinib treatment in 595 intermediate‐1 risk patients with myelofibrosis: The RUX‐MF Real‐World Study

Published:2024-07-30 Issue: Volume: Page:
ISSN:0008-543X
Container-title:Cancer
language:en
Short-container-title:Cancer

Author:

Palandri Francesca¹^ORCID,Elli Elena M.²^ORCID,Morsia Erika³,Benevolo Giulia⁴^ORCID,Tiribelli Mario⁵^ORCID,Beggiato Eloise⁶,Bonifacio Massimiliano⁷^ORCID,Farina Mirko⁸^ORCID,Martino Bruno⁹,Caocci Giovanni¹⁰,Pugliese Novella¹¹^ORCID,Tieghi Alessia¹²,Crugnola Monica¹³,Binotto Gianni¹⁴,Cavazzini Francesco¹⁵,Abruzzese Elisabetta¹⁶^ORCID,Iurlo Alessandra¹⁷^ORCID,Isidori Alessandro¹⁸,Bosi Costanza¹⁹,Guglielmana Veronica²,Venturi Marta²⁰,Dedola Alessandra²⁰,Loffredo Michele²⁰,Fontana Gabriele²⁰,Duminuco Andrea²¹^ORCID,Moioli Alessia⁷,Tosoni Luca⁵,Scalzulli Emilia²²,Cattaneo Daniele¹⁷,Lemoli Roberto M.²³²⁴,Cilloni Daniela²⁵,Bocchia Monica²⁶,Pane Fabrizio¹¹,Heidel Florian H.²⁷,Vianelli Nicola¹,Cavo Michele¹²⁰,Palumbo Giuseppe A.²⁸,Branzanti Filippo²⁰,Breccia Massimo²²^ORCID

Affiliation:

1. IRCCS Azienda Ospedaliero‐Universitaria di Bologna Istituto di Ematologia “Seràgnoli” Bologna Italy

2. Fondazione IRCCS San Gerardo dei Tintori divisione di ematologia e unità trapianto di midollo Monza Italy

3. Hematology Unit Department of Clinical and Molecular Sciences DISCLIMO Università Politecnica delle Marche Ancona Italy

4. University Hematology Division Città della Salute e della Scienza Hospital Torino Italy

5. Division of Hematology and BMT Department of Medicine University of Udine Udine Italy

6. Unit of Hematology Department of Oncology University of Torino Torino Italy

7. Department of Engineering for Innovation Medicine Section of Innovation Biomedicine Hematology Area University of Verona Verona Italy

8. Unit of Blood Diseases and Stem Cells Transplantation Department of Clinical and Experimental Sciences University of Brescia ASST Spedali Civili of Brescia Brescia Italy

9. Division of Hematology Azienda Ospedaliera 'Bianchi Melacrino Morelli' Reggio Calabria Italy

10. Ematologia Ospedale Businco Università degli studi di Cagliari Cagliari Italy

11. Department of Clinical Medicine and Surgery Federico II University Medical School Naples Italy

12. Department of Hematology Azienda USL ‐ IRCCS di Reggio Emilia Reggio Emilia Italy

13. Haematology and BMT Centre Azienda Ospedaliero‐Universitaria di Parma Parma Italy

14. Unit of Hematology and Clinical Immunology University of Padova Padova Italy

15. Division of Hematology University of Ferrara Ferrara Italy

16. Division of Hematology Ospedale S. Eugenio Roma Italy

17. Hematology Division Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy

18. Hematology and Stem Cell Transplant Center AORMN Hospital Pesaro Italy

19. Division of Hematology AUSL di Piacenza Piacenza Italy

20. Dipartimento di Medicina Specialistica Diagnostica e Sperimentale Università di Bologna Bologna Italy

21. Postgraduate School of Hematology University of Catania Catania Italy

22. Hematology Department of Translational and Precision Medicine Az. Policlinico Umberto I‐Sapienza University Rome Italy

23. IRCCS Ospedale Policlinico San Martino Genoa Italy

24. Dipartimento di Medicina Interna e Specialità Mediche Università di Genova Genova Italy

25. Department of Clinical and Biological Sciences University of Turin Turin Italy

26. Hematology Unit Azienda Ospedaliera Universitaria Senese University of Siena Siena Italy

27. Hematology Hemostasis Oncology and Stem Cell Transplantation Hannover Medical School (MHH) Hannover Germany

28. Department of Scienze Mediche Chirurgiche e Tecnologie Avanzate “G.F. Ingrassia” University of Catania Catania Italy

Abstract

AbstractBackgroundRuxolitinib (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.MethodsThe authors investigated the benefit of RUX in 1055 MF patients, included in the “RUX‐MF” retrospective study.ResultsAt 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).ConclusionsIn 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.

Funder

Ministero della Salute

Publisher

Wiley

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