Clinical Outcomes of Ruxolitinib Treatment in Patients With IPSS Intermediate‐1‐Risk Myelofibrosis: Interim Analysis From an Italian, Prospective Study (ROMEI)

Ruxolitinib (RUX), a JAK1/2 inhibitor, demonstrated treatment benefits for myelofibrosis (MF) in intermediate-1 (Int-1)-risk patients with a significant disease burden; however, the evidence is scarce. This interim analysis investigated the efficacy and safety of ruxolitinib in patients with Int-1-risk MF. ROMEI, a multicenter, observational, prospective study, enrolled 508 adult patients with MF receiving ruxolitinib according to approved indications. The present interim analysis was focused on 107 eligible patients in the Int-1-risk group. Primary endpoints included changes in symptoms response and health-related quality of life scores. Secondary endpoints included spleen response evaluation, overall survival, and safety including dosing pattern and dose interruptions. Among the 107 Int-1-risk patients with a median age of 63 years, 65.5% were highly symptomatic (total symptoms score: ≥ 20), while the spleen was palpable at ≥ 5 cm and ≥ 10 cm in 74% and 27% of patients, respectively, with baseline EuroQol visual analogue scale (EQ-VAS) score of 65.1 ± 19.4. After RUX treatment, 42.1% and 43.9% of patients demonstrated a symptom response at 24 and 48 weeks, while 38.9% and 46.8% showed a spleen response at 24 and 48 weeks, respectively. EQ-VAS increased to 71.8 ± 16.3 at 24 weeks and 69.3 ± 19.2 at 48 weeks. Furthermore, 11.2% and 25.2% of patients reported temporary and permanent discontinuation, respectively with no new adverse events reported. The interim analysis showed that ruxolitinib provided clinical benefits, a manageable safety profile, and improved quality of life for Int-1-risk subgroup patients with frequent and sustained responses with acceptable toxicity.

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Journal
Hematological Oncology
Published
2026-03-01
DOI
https://doi.org/10.1002/hon.70178
Primary Topic
Myeloproliferative Neoplasms: Diagnosis and Treatment
Type
article
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article

Clinical Outcomes of Ruxolitinib Treatment in Patients With IPSS Intermediate‐1‐Risk Myelofibrosis: Interim Analysis From an Italian, Prospective Study (ROMEI)

Barbara Anaclerico, Marina Landoni, Alessandro Allegra, Nicola Di Renzo et al.
Hematological Oncology
Myeloproliferative Neoplasms: Diagnosis and Treatment
article

Clinical Outcomes of Ruxolitinib Treatment in Patients With IPSS Intermediate‐1‐Risk Myelofibrosis: Interim Analysis From an Italian, Prospective Study (ROMEI)

Barbara Anaclerico, Marina Landoni, Alessandro Allegra, Nicola Di Renzo, Agostino Tafuri, Paola Ranalli, Angelo Gardellini, Marco Brociner, Antonietta Falcone, Diletta Valsecchi, Stefana Impera, Paolo Ditonno, Barbara Pocali, Simona Tomassetti, Daniele Vallisa, Fabrizio Pane, V. De Stefano, Nicola Orofino, Silvia Sibilla, Francesco Mendicino, D Pastore, Alessandro Lucchesi, Michela Magnoli, Giuseppe Carli, Erika Morsia, S. Siragusa, Maria Langella, Paolo Sportoletti, Alessandra Malato, Alessandra Ricco, Maurizio Musso, Elena Maria Elli, Maurizio Martelli, Marco De Gobbi, Paola Guglielmelli, Marta Brunoventre, Francesco Cavazzini, Serena Mazzotta, Massimiliano Bonifacio, Francesca Palandri, Giulia Benevolo, Chiara Castiglioni, Bruno Martino, Carmine Selleri, Tiziana Urbano, Filippo Gherlinzoni, Olga Mulas, Monica Crugnola, Francesca Lunghi, Elisabetta Abruzzese, Anna Marina Liberati, Gianni Binotto, Francesco Passamonti, Massimo Breccia, Mario Tiribelli, Giuseppe A. Palumbo
article en

Abstract

Ruxolitinib (RUX), a JAK1/2 inhibitor, demonstrated treatment benefits for myelofibrosis (MF) in intermediate-1 (Int-1)-risk patients with a significant disease burden; however, the evidence is scarce. This interim analysis investigated the efficacy and safety of ruxolitinib in patients with Int-1-risk MF. ROMEI, a multicenter, observational, prospective study, enrolled 508 adult patients with MF receiving ruxolitinib according to approved indications. The present interim analysis was focused on 107 eligible patients in the Int-1-risk group. Primary endpoints included changes in symptoms response and health-related quality of life scores. Secondary endpoints included spleen response evaluation, overall survival, and safety including dosing pattern and dose interruptions. Among the 107 Int-1-risk patients with a median age of 63 years, 65.5% were highly symptomatic (total symptoms score: ≥ 20), while the spleen was palpable at ≥ 5 cm and ≥ 10 cm in 74% and 27% of patients, respectively, with baseline EuroQol visual analogue scale (EQ-VAS) score of 65.1 ± 19.4. After RUX treatment, 42.1% and 43.9% of patients demonstrated a symptom response at 24 and 48 weeks, while 38.9% and 46.8% showed a spleen response at 24 and 48 weeks, respectively. EQ-VAS increased to 71.8 ± 16.3 at 24 weeks and 69.3 ± 19.2 at 48 weeks. Furthermore, 11.2% and 25.2% of patients reported temporary and permanent discontinuation, respectively with no new adverse events reported. The interim analysis showed that ruxolitinib provided clinical benefits, a manageable safety profile, and improved quality of life for Int-1-risk subgroup patients with frequent and sustained responses with acceptable toxicity.

Hematological OncologyVol. 44(2)
University of Verona (IT), University of Parma (IT), University of Udine (IT), University of Padua (IT), Vita-Salute San Raffaele University (IT), University of Perugia (IT), Azienda Ospedaliera San Giovanni Addolorata (IT), Casa Sollievo della Sofferenza (IT), Policlinico Umberto I (IT), Ospedale di Circolo e Fondazione Macchi (IT), Agostino Gemelli University Polyclinic (IT), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT), University of Catania (IT), University of Chieti-Pescara (IT), Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT), Azienda Universitaria Ospedaliera Consorziale - Policlinico Bari (IT), Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IT), Azienda ospedaliera "Bianchi-Melacrino-Morelli" (IT), Ospedale Vincenzo Cervello (IT), Ospedale A. Perrino (IT), Ospedale Garibaldi (IT), Ospedali Riuniti di Ancona (IT), Ospedale di Santo Spirito (IT), Azienda Ospedaliera San Gerardo (IT), Azienda Ospedaliero-Universitaria Careggi (IT), Istituto di Ematologia di Bologna (IT), Presidio Ospedaliero (IT), Azienda Ospedaliera S.Maria (IT), Azienda Ospedaliera Sant'Andrea (IT), Arcispedale Sant'Anna (IT), University of Campania "Luigi Vanvitelli" (IT), Ospedale Infermi di Rimini (IT), Azienda Ospedaliera Universitaria Università degli Studi della Campania Luigi Vanvitelli (IT), Istituto Tumori Bari (IT), Ospedale Annunziata di Cosenza (IT), Ospedali Riuniti San Giovanni di Dio e Ruggi d'Aragona (IT), Nini Hospital (LB), Ospedaliera di Piacenza (IT), Tecnologie Avanzate (Italy) (IT), Azienda Ospedaliera Universitaria Policlinico "G. Martino" (IT), Novartis (Italy) (IT), St. Eugenio Hospital (IT), Azienda di Rilievo Nazionale ed Alta Specializzazione (IT), Ca' Foncello Hospital (IT), Azienda Ospedaliera Universitaria Policlinico "Paolo Giaccone" di Palermo (IT), Institute of Studies for the Integration of Systems (IT), Ospedale San Giuseppe (IT), Azienda Ospedaliera Fatebenefratelli e Oftalmico (IT), Ospedale San Bortolo (IT), Ospedale Vito Fazzi (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT), Ospedale San Luigi Gonzaga (IT), ASST Fatebenefratelli Sacco (IT), Gruppo Italiano per il Trapianto di Midollo Osseo (IT), Ospedale Valduce (IT), Azienda Ospedaliera Ospedale Civile di Legnano (IT), La Maddalena (IT), Ospedali Riuniti Umberto I (IT), Azienda Ospedaliera G. Brotzu (IT), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele (IT), University of Bari Aldo Moro (IT)
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Myeloproliferative Neoplasms: Diagnosis and Treatment
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