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

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.