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.
Burden of Illness in Follicular Lymphoma with Multiple Lines of Treatment, Italian RWE Analysis
This real-world analysis investigated patients with follicular lymphoma in Italy receivingthree or more treatment lines (3L), focusing on therapeutic pathways with their rebounds onhealthcare resource consumptions and costs. Data were retrieved from administrative databases fromhealthcare entities covering about 13.3 million residents. Adults diagnosed with follicular lymphomawere identified between January 2015 and June 2020, and among… Read More »Burden of Illness in Follicular Lymphoma with Multiple Lines of Treatment, Italian RWE Analysis
Real-world analysis of the economic and therapeutic burden in advanced breast cancer patients in Italy
Background: This real-world analysis evaluated drug utilization focusing on wastage and healthcare costsfor treatment of patients with advanced breast cancer (aBC) hormone receptor-positive (HR+)/humanepidermal growth factor receptor-2 negative (HER2-) in Italy.Methods: A retrospective analysis was conducted on administrative data covering about 13.3 millionhealth-assisted individuals. Across January/2017–June/2021, all patients with HR+/HER2-aBC wereidentified by ≥ 1 prescription… Read More »Real-world analysis of the economic and therapeutic burden in advanced breast cancer patients in Italy
Real-world Evaluation of the Management, Treatment Pathways and Outcome of Melanoma Patients with Target Therapies in Italy
In recent years, an increasing trend in the incidence of melanoma has been observed in Europe. Although early diagnosis and prompt intervention with local resection often results in positive outcomes, conversely, metastatic disease is still clinically challenging with a poor prognosis and a 5-year survival of around 30%. The growing awareness of melanoma biology and… Read More »Real-world Evaluation of the Management, Treatment Pathways and Outcome of Melanoma Patients with Target Therapies in Italy
Real-World Evidence on Disease Burden and Economic Impact of Sickle Cell Disease in Italy
A real-world analysis was conducted in Italy among sickle cell disease (SCD) patients to evaluate the epidemiology of SCD, describe patients’ characteristics and the therapeutic and economic burden. A retrospective analysis of administrative databases of various Italian entities was carried out. All patients with ≥1 hospitalization with SCD diagnosis were included from 01/2010-12/2017 (up to… Read More »Real-World Evidence on Disease Burden and Economic Impact of Sickle Cell Disease in Italy
The Economic Burden of Chronic Myeloid Leukemia in Patients with Later Lines: Findings from a Real-World Analysis in Italy
Introduction: Chronic myeloid leukemia (CML) is a hematopoietic myeloproliferative disorder that accounts for 20% of all leukemias of adults. The introduction of tyrosine kinase inhibitors (TKIs) (imatinib, bosutinib, dasatinib, nilotinib, ponatinib) has yielded significant benefits for patients with CML in terms of survival and quality of life. This real-world analysis evaluated the economic burden for managing… Read More »The Economic Burden of Chronic Myeloid Leukemia in Patients with Later Lines: Findings from a Real-World Analysis in Italy