Dosing and clinical outcomes of ruxolitinib in patients with myelofibrosis in a real‐world setting: Interim results of the Italian observational study (ROMEI)
Massimo Breccia, Francesca Palandri, Maurizio Martelli, Francesco Mendicino, Alessandra Malato, Giuseppe A. Palumbo, Silvia Sibilla, Nicola Di Renzo, Elisabetta Abruzzese, Sergio Siragusa, Monica Crugnola, Carmine Selleri, Fabrizio Pane, Paolo Sportoletti, Bruno Martino, Stefana Impera, Alessandra Ricco, Maria Langella, Paolo Ditonno, Giuseppe Carli, Federico Itri, Anna Marina Liberati, Tiziana Urbano, Agostino Tafuri, Vita Polizzi, Domenico Pastore, Erika Morsia, Giulia Benevolo, Giorgia Micucci, Gabriella Farina, Massimiliano Bonifacio, Elena Maria Elli, Angelo Gardellini, Valerio De Stefano, Giovanni Caocci, Antonietta Falcone, Daniele Vallisa, Marco Brociner, Mario Tiribelli, Gianni Binotto, Barbara Pocali, Francesco Cavazzini, Simona Tomassetti, Francesca Lunghi, Mauro Di Ianni, Alessandro Allegra, Barbara Anaclerio, Serena Mazzotta, Nicola Orofino, Filíppo Gherlinzoni, Chiara Castiglioni, Marina Landoni, Diletta Valsecchi, Michela Magnoli, Paola Guglielmelli, Francesco Passamonti
Abstract
BACKGROUND: Myelofibrosis (MF) significantly impacts patients' overall survival (OS) and quality of life (QOL). This prospective study analyzed ruxolitinib dosing patterns and associated clinical outcomes in patients with MF over 12 months. METHODS: ROMEI, a multicenter, observational, ongoing study, enrolled 508 adult patients with MF treated with ruxolitinib. For the current interim analysis, eligible patients with baseline platelet values were categorized into two groups based on ruxolitinib starting dosage: as expected (AsEx, n = 174) and lower than expected (LtEx, n = 132); ruxolitinib dose changes, interruptions and time to permanent discontinuation were analyzed, along with symptoms response, health-related QOL scores, spleen response, OS, and safety. RESULTS: Forty-three percent of patients started at a lower-than-expected dose. Both groups showed reduction in average daily ruxolitinib doses over 12 months. Symptoms response rate was similar in both groups at week 48 (40.8% AsEx vs 40.9% LtEx). The AsEx group demonstrated higher spleen response rates at both 24 weeks (50.0% vs 30.2%) and 48 weeks (57.7% vs 45.8%) with a shorter median time to first response (3.3 vs 11.1 months, p = .019) when compared to the LtEx group. Both groups showed upward trends in health-related QOL values. Estimated median OS was not reached for the AsEx group versus 4.7 years in the LtEx group (p = .014). Adverse events were reported in 87.4% and 84.9% of patients in the AsEx and LtEx groups, respectively. CONCLUSIONS: The ROMEI study demonstrated the importance of optimal ruxolitinib dosage in patients with MF for maximum effectiveness and improved OS, with manageable safety.