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
Treatment Switching and Drug Survival of Biologic Therapies in Psoriasis: A Real-World Italian Study Across Biologic Classes
Background/Objectives: Psoriasis is a chronic inflammatory skin disease leading to substantial
psycho-physical and social burden and reduced quality of life. Biologic agents
have transformed its therapeutic landscape. This real-world Italian study described the
pattern of treatment with biologic drugs in patients with psoriasis. Methods: A retrospective
observational study was conducted using administrative databases from Italian
Local Health Units, covering nearly 12 million individuals. The study included adults
with psoriasis identified from January 2015 to March 2025 by hospitalization, co-payment
exemption code, or topical anti-psoriatic prescriptions. Patients initiating a biologic drug
(anti-TNFα, anti-IL12/23, anti-IL17, and anti-IL23) were selected and further analyzed
in terms of treatment switching, drug survival, and healthcare resource utilization and
related costs within the first year after biologic initiation, and compared. Results: A total
of 10,270 biologic-naïve adult patients was included in the analysis (anti-TNFα N = 5078;
anti-IL12/23 N = 767; anti-IL17 N = 2574; anti-IL23 N = 1851). Most patients (95.0%) starting
an anti-IL23 agent did not switch. Compared with anti-TNFα, initiating an anti-IL23
inhibitor was associated with a significant reduced risk of switching (HR = 0.186; 95%CI:
0.144–0.240; p < 0.001). According to the cost analysis stratified by switching status, remaining
on the index biologic was associated with a lower economic burden. Although
differences between switchers vs. non-switchers among anti-IL23 users did not reach
statistical significance (€12,052 vs. €11,406, respectively, p = 0.132), data support the economic
advantage associated with greater treatment stability. Conclusions: Anti-IL23 agents
showed effective, durable first-line use with potential long-term clinical and economic
benefits in moderate-to-severe psoriasis.
Unlocking the benefits of allopurinol: the role of timely initiation and adherence in cardiovascular and renal outcomes in hyperuricemia
Background: Increasing evidence links elevated serum uric acid levels to adverse cardiorenal outcomes. To date,
limited data exist on whether the benefits of urate-lowering therapies (ULTs) depend on timely treatment
initiation and sustained adherence in patients at high cardiovascular risk.
Methods: An economic evaluation was conducted, based on a simulation model populated by Italian real-world
patient-level data from the Uric Acid Right for Heart Health (URRAH) cohort to evaluate long-term cardiovascular,
renal, and economic outcomes associated with timely allopurinol use. 10,000 patients with hyperuricemia
(serum uric acid >5.6 mg/dL) and elevated cardiovascular risk were simulated over a 20-year time horizon.
Timely initiation of allopurinol (before the first gout flare) was compared with treatment initiated only after
symptomatic disease onset. Scenarios reflecting current and improved adherence to ULTs were assessed. Outcomes
included major adverse cardiovascular events, end-stage renal disease onset, and economic outcomes from
the payer’s perspective.
Results: According to our model, timely initiation of allopurinol was associated with modest but consistent health
gain (0.04 quality adjusted life years) including reductions in gout flares, cardiovascular- and renal endpoints.
Timely allopurinol initiation was cost-saving and, from a health economic perspective, dominant over current
practice. Improved ULT adherence amplified these benefits.
Conclusion: Our hypothesis-generating research suggests that, in hyperuricemic patients at high cardiovascular
risk, the potential benefits of allopurinol also depend on timely initiation and sustained adherence. While these
findings do not constitute evidence of causal clinical benefit, they support a broader cardiorenal clinical
approach to hyperuricemia management that extends beyond gout prevention
Atogepant reduces triptan use: a pharmacoeconomic analysis in migraine prevention
Background Migraine is a leading cause of disability worldwide, impairing quality of life and productivity. Preventive
therapies aim to reduce attack frequency, severity and the need for acute medications. Oral atogepant (60 mg), a
calcitonin gene-related peptide (CGRP) receptor antagonist, has recently expanded migraine prevention options. This
study evaluated changes in triptan use after atogepant initiation using real-world data.
Methods This retrospective observational study used administrative healthcare databases from the Local Health Unit
of Modena (Italy). Adults with chronic or high-frequency episodic migraine initiating atogepant between November
2023 and December 2024 were identified. Triptan use and related costs were assessed in the 6 months before and
after treatment initiation (index date), measured as dispensed dosage units.
Results Among 95 patients (86% female; mean age 53.5 years), 55 triptan users were included in the analysis. Triptan
use decreased in 45 patients (82%), with ≥ 60% reduction in 27 (49%) and complete discontinuation in 10. Median
consumption decreased from 76 to 24 dosage units, and mean consumption from 88.1 to 45.2. This reduction was
statistically significant (Wilcoxon signed-rank test, p < 0.001). This reduction corresponded to a decrease of €2,591 in
triptan-related pharmaceutical expenditure within the study cohort.
Conclusion Atogepant preventive treatment may substantially reduce acute migraine medication use in real-world
practice, with potential clinical and economic benefits.
Keywords CGRP, Gepants, Headache, Medication-overuse headache, Pain
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