ROMEI: Interim analysis from a real-world study of ruxolitinib in patients with MF

The ongoing, prospective, observational ROMEI study (CINC424AIT04) aimed to investigate outcomes with ruxolitinib in patients with MF in the real-world setting (N = 508). A 24-week interim analysis confirmed the symptom control, QoL benefits, and spleen size reduction of ruxolitinib. It also showed suboptimal treatment adherence in 25–40% of patients, potentially causing undertreatment and reduced disease control and survival outcomes.

A recent interim analysis of the ROMEI study investigated dosing patterns and correlation with clinical outcomes in patients who completed the first 12 months of follow-up or prematurely discontinued the study. Results were published by Breccia et al. in Cancer. The analysis included 306 patients with baseline platelets counts available, stratified into two groups: assigned ruxolitinib dose as expected (AsEx; n = 174) and lower than expected (LtEx; n = 132). The primary outcome was to assess changes in symptoms and HRQoL using MPN-10 TSS and EQ-5D-5L. 

Key learnings

Mean MPN-10 TSSs were 19.3 and 20.3 at 24 weeks and 19.7 and 21.1 at 48 weeks in the AsEx and LtEx groups, respectively. Mean EQ-VAS scores increased from 62.9 and 59.5 at baseline to 65.3 and 64.4 at 48 weeks, respectively. 

Response rates were higher in the AsEx vs LtEx groups at 4 weeks (38.5% vs 27.3%); however, the AsEx and LtEx groups showed similar response rates at 48 weeks (40.8% vs 40.9%).

The AsEx group showed 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 = 0.019) vs the LtEx group.

The AsEx group did not reach the estimated median OS compared with 4.7 years in the LtEx group (p = 0.014). 

AEs were reported in 87.4% and 84.9% of patients. Grade 3 anemia requiring dose adjustments was reported in 9.8% and 4.6% of patients in the AsEx and LtEx groups, respectively.

These findings highlight the importance of optimal ruxolitinib dosing, including initiating recommended doses to maximize OS in patients with MF. However, the observational and descriptive nature of the study limits the results.