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.
Differenze di genere in trattamento, aderenza e costi sanitari nei pazienti in terapia ipolipemizzante: un’analisi real-world
Introduzione
Nonostante i benefici cardiovascolari delle terapie ipolipemizzanti siano comparabili tra i sessi, persistono disparità nella gestione della dislipidemia. Questa analisi valuta differenze negli schemi terapeutici, nell’aderenza e nei costi sanitari tra uomini e donne nella pratica clinica italiana.
Metodi
È stata condotta un’analisi retrospettiva osservazionale su database amministrativi (2017– 2022) coprenti circa 12 milioni di assistibili. Sono stati inclusi adulti con almeno una prescrizione di ipolipemizzanti (statine, ezetimibe). Sono stati analizzati: complessità del trattamento, aderenza (Proportion of Days Covered, PDC ≥80%) e costi, con analisi stratificate per rischio vascolare secondo le linee guida ESC/EAS 2019.
Risultati
Tra i 1.899.894 pazienti inclusi, le donne (51,8%) erano mediamente più anziane (68,9 vs 66,1 anni, p <0,001), ma con un indice di comorbidità di Charlson inferiore (0,5 vs 0,6; p <0,001). Alle donne venivano prescritte meno frequentemente statine ad alta intensità (es. atorvastatina 40/80 mg) o terapie combinate (es. statina + ezetimibe) (p <0,001). I costi annuali erano inferiori nelle donne (2.469 € vs 3.020 €, p <0,001), principalmente per minori ospedalizzazioni e consulti cardiologici.
Conclusioni
Le evidenze di questa analisi osservazionale suggeriscono che le donne con dislipidemia ricevono trattamenti ipolipemizzanti meno intensi e mostrano minore aderenza, anche dopo aggiustamento per età e rischio cardiovascolare. Queste disparità evidenziano la necessità di strategie di trattamento sensibili al genere per ottimizzare la gestione e gli esiti clinici.
Treatment Pathway and Clinical Outcomesof the Population with Muscle‑invasiveBladder Cancer in Italy: A Real‑world Analysiswith Administrative Databases
ABSTRACTIntroduction: In Italy, real-world data on muscle-invasive bladder cancer (MIBC) are scanty. This analysis exploited administrative databases to describe the clinical characteristics and treatment outcomes of patients with MIBC. Methods: Adults hospitalized for non-metastatic bladder cancer who underwent cystectomy in 2018 were proxied for MIBC. The following variables were examined: demographic and clinical characteristics, number… Read More »Treatment Pathway and Clinical Outcomesof the Population with Muscle‑invasiveBladder Cancer in Italy: A Real‑world Analysiswith Administrative Databases
Ranolazine in patients with chronic coronarysyndromes: real-world data provide newevidence on the antiarrhythmic properties of the drug
Aims: Ranolazine (Ran) is an anti-anginal drug inhibiting late sodium current, an action possibly hindering arrhythmias onset. Indeed, some evidence supports the anti-arrhythmic effects of Ran. The aim of this study, which evaluated Italian patients withchronic coronary syndrome (CCS), was to investigate whether Ran, as an add-on therapy, was associated with a lower incidenceof atrial… Read More »Ranolazine in patients with chronic coronarysyndromes: real-world data provide newevidence on the antiarrhythmic properties of the drug
A Real-World Analysis of Patientswith Triple Class Exposed Multiple Myeloma in Italy: Epidemiology Estimates, Treatment Pattern and Economic Burden
ABSTRACTOBJECTIVES: This research aimed to provide updated epidemiological estimates of multiple myeloma (MM) in Italy andto characterize the clinical journey, treatment patterns, and economic burden focusing specifically on the subset of patientswho have been exposed to all three major therapeutic classes: proteasome inhibitors, immunomodulatory drugs, and anti-CD38 monoclonal antibodies (triple-class exposed, TCE).METHODS: A retrospective analysis… Read More »A Real-World Analysis of Patientswith Triple Class Exposed Multiple Myeloma in Italy: Epidemiology Estimates, Treatment Pattern and Economic Burden
Treatment Pathways, Drug Utilization and Healthcare Resource Consumption in Patients with Metastatic Anaplastic Lymphoma Kinase-Positive Non-small Cell Lung Cancer: A Real-World Analysis with Administrative Databases in Italy
Background: Metastatic anaplastic lymphoma kinase-positive (ALK+) non-small cell lung cancer (NSCLC) is a rare but clinically distinct subtype of lung cancer with therapeutic sensitivity to ALK inhibitors (ALKi). Over the past decade, several ALKi have been introduced in Italy, yet real-world data on their utilization, patient characteristics, outcomes, and healthcare burden remain limited. Objectives: This… Read More »Treatment Pathways, Drug Utilization and Healthcare Resource Consumption in Patients with Metastatic Anaplastic Lymphoma Kinase-Positive Non-small Cell Lung Cancer: A Real-World Analysis with Administrative Databases in Italy
Real-World Evidence on Disease Burden and Economic Impact of Paroxysmal Nocturnal Haemoglobinuria in Italy
Background/Objectives: This analysis was conducted in Italy to estimate the epidemiology of paroxysmal nocturnal hemoglobinuria (PNH) and to describe the features and economic burden of PHN in the adult population considering the role of anti-complement therapy with C5/3-inhibitors (C5/3i). Methods: Administrative databases of healthcare entities covering approximately 12 million citizens were used to estimate the prevalence and incidence of PNH. Demographics, clinical characteristics and healthcare costs were analyzed among adults with PHN stratified by the presence/absence of C5/3i therapy. Results: The prevalence in Dec-2021 of PNH in adults was 17.6/1,000,000 people, and the incidence rate in the period 2011–2022 was 1.5/1,000,000/year. In 142 patients with at least 12 months of data available before and after inclusion (mean age: 50.7 years; 45.8% males), 27% received C5/3i therapy. The main baseline comorbidities were aplastic anemia and other bone marrow failure syndromes, found in 10.6% of patients and more common in C5/3i-treated than untreated patients (18.4% vs. 7.7%). Cost analysis showed that the average cost per patient per year (PPPY) was EUR 41,084, mainly driven by drug expenses (87% of total costs), especially anti-complement therapy (80%). RBC transfusions were the most impactive item among the hospitalization costs (EUR 1982 of EUR 4284 PPPY). The C5/3i-treated cohort was associated with higher total costs (EUR 133,472 vs. EUR 8089, p < 0.001), mainly due to drug expenses (EUR 127,180 vs. EUR 3217, p < 0.001). Conclusions: This real-world analysis confirmed a rising PNH prevalence in Italy, aligning with global data. Despite available therapies, many patients face a high disease burden, suggesting potential benefits from novel treatments targeting upstream complement components.