Real-World Progression-Free Survival and Healthcare Resource Utilization Associated with Daratumumab Use in Transplant-Ineligible Multiple Myeloma Patients in Italy
Purpose: This study evaluated real-world clinical and economic outcomes associated with daratumumab-based regimens in patients with multiple myeloma (MM) ineligible for autologous stem cell transplantation (ASCT) in Italy. Differences in healthcare resource use according to daratumumab administration route (intravenous vs subcutaneous) were also explored.
Methods: A retrospective observational analysis was conducted using administrative healthcare databases from Italian Local Health Units covering ~12 million individuals (2018– 2023). Patients with MM receiving daratumumab in first-line (1L) or second-line (2L) therapy were included. Real-world progression-free survival (rwPFS), defined as time to treatment switch or death, was used as a proxy endpoint for disease progression, and healthcare resource utilization were compared between treatment regimens. Propensity score weighting was applied to to improve comparability between treatment groups. Direct healthcare costs captured in administrative databases were analyzed from the perspective of the Italian National Health System.
Results: Among 790 non-ASCT MM patients, 344 received 1L DaraRd and 413 received 2L DaraRd. In the 1L setting, median rwPFS was not reached for DaraRd and was 25.7 months for Rd (p=0.011). In 2L, median rwPFS was 42.9 months for DaraRd versus 19.1 months for Rd (p < 0.001). In 1L, DaraRd was associated with lower hospitalization costs compared with Rd for both all-cause (€ 4324 vs € 7971) and MM-related (€ 2862 vs € 6693) admissions. In 2L, differences in hospitalization costs were smaller and not statistically significant.
Conclusion: In this real-world analysis, daratumumab-based regimens were associated with longer rwPFS compared with Rd among transplant-ineligible MM patients, in both 1L and 2 L settings. In 1L treatment, these outcomes were accompanied by lower hospitalization costs. Given the retrospective design and the use of administrative healthcare data, these findings should be interpreted with caution, but they contribute to the growing body of real-world evidence on treatment outcomes and resource utilization in routine clinical practice.
Characteristics, therapeutic pathway and the economicburden of patients with drug-resistantepilepsy: A real-world analysis following the introduction of cenobamate inItaly
Abstract Objectives: Drug-resistant epilepsy (DRE) remains a major clinical challenge, affecting approximately one-thirdof patients with epilepsy. Cenobamate, a novel antiseizure medication (ASM) approved in Italy in 2022, has shown promisein clinical trials. However, real-world data on its use, especially economic evaluations, remain limited. This analysis assessed the impact of cenobamate in Italian clinical practice, focusing… Read More »Characteristics, therapeutic pathway and the economicburden of patients with drug-resistantepilepsy: A real-world analysis following the introduction of cenobamate inItaly
Analisi del percorso terapeutico e del consumo di risorse sanitarie in pazienti diabetici in trattamento insulinico: uno studio di real-world evidence su un campione di ASL della regione Sicilia
Italian reports indicate that more than one-third of patients with type 2 diabetes (T2D) require insulin therapy within 8 years of diagnosis, and commonly basal insulin represents the frontline option. An observational analysis was conducted with the aim of investigating patients treated with basal insulin alone in a sample of Local health Units of Sicily… Read More »Analisi del percorso terapeutico e del consumo di risorse sanitarie in pazienti diabetici in trattamento insulinico: uno studio di real-world evidence su un campione di ASL della regione Sicilia
Characteristics and drug utilization of patients with hereditary angioedema in Italy, a real world-analysis
This real-world analysis investigated the characteristics and treatment patterns of patientswith hereditary angioedema (HAE) in Italy using the administrative data of health units across Italy.Patients were identified via exemption code or HAE-specific treatments (thus, all known forms, typeI, II and, III, were included). The index date was that of first prescription of HAE treatments withinthe… Read More »Characteristics and drug utilization of patients with hereditary angioedema in Italy, a real world-analysis