Management of hyperkalaemia in the Italian clinical practice: impact of diverse potassium binders on outcomes and costs in patients with chronic kidney disease and heart failure
Introduction Hyperkalemia is a common and clinically relevant condition in patients with chronic kidney disease (CKD)
and heart failure (HF), often limiting optimal use of renin–angiotensin–aldosterone system inhibitors (RAASi).
Methods This real-world study evaluated adherence, clinical outcomes, and healthcare costs among Italian patients initiating
patiromer, sodium zirconium cyclosilicate (SZC), or sodium polystyrene sulfonate (SPS) between January 2022 and
June 2024.
Results Administrative data covering about 10% of the Italian population identified 1953 patients: 249 on patiromer, 387 on
SZC, and 1317 on SPS. After full matching, 67.5% of patiromer patients were adherent at 6 months, compared with 20.4%
for SZC and 0.5% for SPS (p < 0.001). Over the total follow-up, adherence remained higher with patiromer (42.5% vs 13.4%
and 0.5%). During the first 6 months, HF-related hospitalization rates were lower with patiromer versus SZC (5.4 vs 12.8
events per 100 person-years; p = 0.079), becoming significant over overall follow-up (5.3 vs 15.7; p = 0.003). Composite
HF outcomes at 6 months were also lower with patiromer compared with SZC (5.3 vs 17.4; p = 0.012) and SPS (3.4 vs 24.8;
p = 0.005). At 12 months, total healthcare costs were significantly lower with patiromer than with SZC and SPS (€5645 vs
€8500 and €4736 vs €6234; p < 0.001). Overall, patiromer use was associated with better adherence, fewer HF-related events,
and reduced healthcare expenditure.
Conclusions These findings suggest a patiromer potential role for sustained potassium binder therapy in supporting RAASi
optimization and improving clinical management in routine practice, but confirmation through direct comparative prospective
randomized trials is needed
First-Line Treatment of Chronic Lymphocytic Leukemia in Italy: Real-World Evidence on Utilization, Outcomes, and Healthcare Costs
Background: Chronic lymphocytic leukemia (CLL) is the most common adult leukemia in Western countries, mainly affecting older
people. Targeted agents have reshaped first-line (1L) strategies, making real-world evidence important to complement clinical trials.
Objective: To estimate the incidence of Italian patients initiating first-line CLL therapy (2019–2022) and describe demographics/
clinical profile, treatment patterns, adherence, outcomes (overall survival [OS], time to next treatment [TTNT]), and healthcare costs
from the perspective of the Italian National Health System (NHS).
Methods: A retrospective observational study using administrative healthcare databases (~9 million residents) was conducted on CLL
patients starting 1L therapy (index-date) for CLL. Baseline characteristics were assessed in the 12 months pre-index; follow-up was
≥12 months. Drug use, adherence (medication possession ratio), dose adjustments, OS, TTNT, and direct costs were analyzed with
descriptive and multivariable methods.
Results: A total of 1479 patients initiated 1L therapy: 63.9% chemotherapy (CHT), 23.2% ibrutinib, 3.2% acalabrutinib, and 9.7%
other regimens. CHT remained common, especially among older and more comorbid patients. Ibrutinib showed lower mortality versus
CHT (HR 0.663; p=0.002) and longer TTNT (median not reached). Dose adjustments were frequent; extended refill intervals did not
appear to reduce drug survival. Mean annual cost per patient was €38,573, mainly driven by drug acquisition; ibrutinib users had lower
hospitalization and outpatient costs than other 1L groups.
Conclusion: In Italian practice, ibrutinib was the main targeted 1L option and was associated with improved survival and delayed
progression versus CHT. Despite higher drug costs, reduced hospital-based resource use suggests favourable overall clinical and
economic impact.
Keywords: chronic lymphocytic leukemia, first-line treatment, healthcare costs, ibrutinib, real-world evidence, targeted therapy
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.
Impatto clinico ed economico dell’emicrania in Italia: un’analisi di real-world evidence
Introduction: This retrospective observational analysis evaluated the clinical, demographic, and economic burdenof migraine patients in real-world Italian clinical practice.Methods: A retrospective observational analysis was conducted using Italian administrative databases, involvingapproximately 12 million beneficiaries. All adult patients with at least one hospital discharge diagnosis ofmigraine (ICD-9-CM = 346) or at least one prescription for migraine-specific medications… Read More »Impatto clinico ed economico dell’emicrania in Italia: un’analisi di real-world evidence
Clinical and economic burden of migraine in Italy: a real-world evidence analysis
Introduction: This retrospective observational analysis evaluated the clinical, demographic, and economic burdenof migraine patients in real-world Italian clinical practice.Methods: A retrospective observational analysis was conducted using Italian administrative databases, involvingapproximately 12 million beneficiaries. All adult patients with at least one hospital discharge diagnosis ofmigraine (ICD-9-CM = 346) or at least one prescription for migraine-specific medications (ATC =… Read More »Clinical and economic burden of migraine in Italy: a real-world evidence analysis
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
Epidemiologic Characteristics Determining the Choice ofDirect-Acting Antiviral Therapy in HCV Patients: An Italian Real-World Evidence Study
AbstractPangenotypic direct-acting antivirals (pDAAs) have transformed hepatitis C virus (HCV) treatment. In Italy, sofosbuvir/velpatasvir (SOF/VEL) and glecaprevir/pibrentasvir (GLE/PIB) are available. While both show similar efficacy, differences in patient profilesand potential drug–drug interactions (DDIs) may influence treatment choice. This study examined factors affecting pDAA selection and potential prescribing gaps. Using administrative databases (2018–2023) covering 3.7 million… Read More »Epidemiologic Characteristics Determining the Choice ofDirect-Acting Antiviral Therapy in HCV Patients: An Italian Real-World Evidence Study
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
The economic impact of multimorbidity in Italy:evaluation of direct costs and scenario analysis ofpatients with type 2 diabetes, heart failure, andchronic kidney disease using real-world data
ABSTRACTObjectives: This study aimed to evaluate the healthcare costs associated with managing type 2 diabetes (T2D),chronic kidney disease (CKD), and heart failure (HF) in Italy. Specifically, the research investigated the economicimpact on the Italian National Health System due to the increased clinical complexity and multimorbidity amongpatients with these conditions.Methods: A predictive model was developed to… Read More »The economic impact of multimorbidity in Italy:evaluation of direct costs and scenario analysis ofpatients with type 2 diabetes, heart failure, andchronic kidney disease using real-world data