Healthcare Utilization Databases for Real-World Outcome Analysis of Ruxolitinib Treatment in Myelofibrosis

Abstract

Introduction

Myelofibrosis (MF) is a BRC::ABL1-negative myeloproliferative neoplasm characterised by a heterogeneous clinical phenotype encompassing abnormalities in blood counts, bone marrow fibrosis, splenomegaly, constitutional symptoms and vascular complications. MF may progress to accelerated phase (AP) and blast phase (BP). MF includes primary MF, comprising prefibrotic and overt-PMF, and secondary MF, evolving from polycythemia vera or essential thrombocythemia. Survival estimates vary from 7 to 14 years according to MF subtype. Prognostic scores define patients’ suitability to stem cell transplant (SCT). Apart from SCT, disease burden control relies on JAK inhibitors (JAKis), among which Ruxolitinib (RUX) was the first approved in Italy. Because of its high cost, RUX dispensing data are routinely collected by regional health authorities through healthcare utilization databases (HCUDs), enabling the generation of real-world evidence.

Objectives

To assess overall survival (OS), time to RUX discontinuation and healthcare resource use in a population of intermediate2- and high-risk MF patients who initiated RUX outside a clinical trial, using regional HCUD.

Methods

We conducted a retrospective population-based cohort study using HCUD from three Italian regions (Lombardy, Lazio and Tuscany) accounting for 20 million inhabitants. The study included 652 intermediate2- and high-risk MF patients who initiated RUX between October 2014 and December 2017, followed through regional data availability (up to December 2023 in Lombardy and Lazio, July 2021 in Tuscany). We estimated OS and time to RUX discontinuation using the Kaplan-Maier estimator and assessed red blood cell (RBC) transfusions, SCT, hospitalization-related events, and average individual healthcare costs. We evaluated which factors among sex, age classes, year of RUX initiation, RUX starting dose, SCT (as time-dependent), and Multisource Comorbidity Score (MCS) classes were associated with mortality using a multivariable Cox proportional hazard model. Analyses were performed separately within each region, and region-specific estimates were combined using a two-stage meta-analysis.

Results

Over a median follow-up of 36.8 months, median OS was 48.0 months (95%CI: 43.2-51.6), and median time to RUX discontinuation was 31.2 months (95%CI: 26.4-36.0). In the first 6 months, 408 (69%) patients required no RBC transfusions, 172 (29%) received 1-5 units, and 14 (2%) received ≥6 units. 10.9% of patients underwent SCT. Incidence rates per 100 person-years were 10.30 for infections, 5.47 for solid tumors, 5.22 for AP/BP, 3.47 for bleeding events, and 1.56 for thrombosis. Mean annual cost per patient was 30,675€. Independent predictors of mortality were male sex (vs female: HR: 1.59, 95%CI: 1.12-2.23), age 70-79 years (vs <70: HR: 2.00, 95%CI: 1.60-2.49), age ≥80 years (vs <70: HR: 3.31, 95%CI: 2.11-5.19), poor MCS (≥15 vs 0-4: HR: 1.97, 95%CI: 1.45-2.68), and lower starting RUX dose (<20 vs ≥20 mg BID: HR: 1.42, 95%CI: 1.10-1.83).

Conclusions

HCUD provided information on RUX treatment in an unselected, real-world population of intermediate2- and high-risk MF patients. While disease-specific details (e.g., blood counts, mutation profile, karyotype) are unavailable in HCUD, these data are pivotal for understanding real-world drug use and supporting pharmacoeconomic evaluations.

Declaration: This abstract is based on the following previously published article:

Mora B, Franchi M, Margotto L, et al. Health Care Utilization Databases obtained from health system inform outcome for ruxolitinib treatment in patients with myelofibrosis. Hemasphere. 2026 Feb 24;10(2):e70316. doi: 10.1002/hem3.70316. PMID: 41743264; PMCID: PMC12931254.