Real world data are becoming a crucial tool to understand how cancer is treated in routine daily practice. This real-world analysis aims to describe the characteristics of patients with CML in 2nd or ≥3rd tyrosine kinase inhibitors (TKI) lines of therapy, to evaluate their treatment sequence and utilization in settings of Italian clinical practice in Italy. A retrospective analysis was performed using an administrative databases covering around 15.3 million cases. All adult patients prescribed with TKI as 2nd or ≥3rd lines (L) of therapy for CML during January 2015–December 2018 were included. A total of 491 patients in 2nd and 144 in ≥3rd L was included. In both cohorts, hypertension was the most reported comorbidity, followed by metabolic and blood count alterations. In each calendar inclusion year, an increment of 97.6% was observed in the number of patients treated in ≥3rd L. In the 2nd L cohort, 18.7% had a switch to 3rd L, while 26.4% of ≥3rd L patients switched to a subsequent line. Around 40% in both lines discontinued their treatment after a median time of 5.5 (2nd L) and 4.3 (≥3rd L) years. The results provided insights into CML management clinical practice, indicating a heavy disease burden for patients in later lines that showed an increasing complex management, and suggest that a need for novel treatment strategies might exists.

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 12/2018 for epidemiologic analysis). The index date corresponded to the first SCD diagnosis. In 2018, SCD incidence rate was 0.93/100,000, the prevalence was estimated at 13.1/100,000. Overall, 1816 patients were included. During the 1st year of follow-up, 50.7% of patients had one all-cause hospitalization, 27.8% had 2, 10.4% had 3, and 11.1% had ≥4. Over follow-up, 6.1–7.2% of patients were treated with SCD-specific, 58.4–69.4% with SCD-related, 60.7–71.3% with SCD-complications-related drugs. Mean annual number per patient of overall treatments was 14.9 ± 13.9, hospitalizations 1.1 ± 1.1, and out-patient services 5.3 ± 7.6. The total mean direct cost per patient was EUR 7918/year (EUR 2201 drugs, EUR 3320 hospitalizations, and EUR 2397 out-patient services). The results from this real-world analysis showed a high disease burden for SCD patients with multiple hospitalizations during the follow-up. High healthcare resource utilization and costs were associated with patient’ management and were most likely underestimated since indirect costs and Emergency Room admissions were not included.

This real-world analysis investigated patients with follicular lymphoma in Italy receiving three or more treatment lines (≥3L), focusing on therapeutic pathways with their rebounds on healthcare resource consumptions and costs. Data were retrieved from administrative databases from healthcare entities covering about 13.3 million residents. Adults diagnosed with follicular lymphoma were identified between January 2015 and June 2020, and among them 2434 patients with ≥3L of treatment during the data availability interval (January 2009 to June 2021) were included. Of them, 1318 were in 3L, 494 in 4L and 622 in ≥5L. A relevant proportion of patients (12–32%) switched to a later line within the same calendar year. At 3-year follow-up (median), 34% patients died. Total mean annual expenses were euro 14,508 in the year preceding inclusion and rose to euro 21,081 at 1-year follow-up (on average euro 22,230/patient/year for the whole follow-up), with hospitalization and drug expenses as weightiest cost items. In conclusion, the clinical and economic burden of follicular lymphoma increases along with later treatment lines. The high mortality rates indicate that further efforts are needed to optimize disease management.

Purpose: This phase IIIb study prospectively evaluated the prognostic and predictive value of baseline and dynamic circulating tumor DNA (ctDNA) in postmenopausal patients with hormone receptor–positive (HR+), human epidermal growth factor receptor 2–negative (HER2−) advanced breast cancer (ABC) treated with first-line ribociclib/letrozole. Experimental Design: A total of 287 patients were enrolled, with ctDNA analyzed at baseline (n = 263), day 15 of cycle 1 (C1D15; n = 238), C2D1 (n = 241), and first imaging (n = 206). The primary objective was to identify ctDNA alterations, characterize their evolution across treatment time points, and assess their association with progression-free survival (PFS). Results: Median PFS was 23.4 months (95% confidence interval, 20.8 to not estimable). At baseline, the most frequently altered genes were PIK3CA (22.1%) and TP53 (15.5%). Alterations in TP53, MYC, and HER− and cyclin-dependent kinase 4/6− pathway genes were linked to early progression. Absence of a detectable mutation at baseline (n = 150, 57%) was associated with a better prognosis [hazard ratio (HR) = 0.41]. Among patients with a detectable mutation at baseline (n = 104), early clearance (mutation undetectability) was observed in 47.1% at C1D15 and 52.4% at C2D1 and was associated with improved PFS (C1D15, HR = 0.51; C2D1, HR = 0.44). In patients without a detectable mutation at baseline, 22.7% (n = 34) developed new mutations at C1D15, C2D1, or first imaging. Patients without new mutations had a lower risk of progression (HR = 0.45). Conclusions: Pretreatment and early dynamics of ctDNA represent promising prognostic and predictive biomarkers in patients with HR+/HER2− ABC treated with ribociclib/letrozole. Early ctDNA dynamics seem to be a promising surrogate biomarker for treatment. Further studies are warranted to validate their clinical utility.

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.

Background: Published evidence on patient experiences, perceptions, and challenges related to early breast cancer (eBC) in Italy is limited. Understanding these aspects is critical for improving diagnosis, treatment outcomes, and quality of life (QoL). Objective: This study used social media listening (SML) to explore the patient journey, treatment perceptions, QoL, and unmet needs of patients with eBC, caregivers, and health care professionals (HCPs) in Italy. Methods: This retrospective noninterventional SML study analyzed publicly available posts from December 2021 to November 2023 using breast cancer–related keywords in English and Italian through Sprinklr, a web-based aggregator tool. Posts sourced from social media platforms, such as X (formerly known as Twitter), blogs, forums, Facebook, Instagram, and YouTube, were filtered by geographic location to include only users in Italy. Posts were filtered using natural language processing (NLP) for relevance and duplicates, followed by manual review and stakeholder identification (patients, caregivers, and HCPs). Key themes of discussion were identified through thematic analysis of posts across the stages of the patient journey (symptoms, diagnosis, treatment, etc). Ethical guidelines were followed by using anonymized, publicly available data. Descriptive statistics were used to analyze the data, and posts with missing data were excluded. Consequently, denominators varied across analyses and were adjusted based on data availability for specific variables. Results: Of the 20,008 posts initially extracted, 1580 posts were retained following NLP filtering, and 530 posts were included after manual screening. The majority (493/518, 95%) of the posts were sharing information about diagnosis and treatment journeys, emotional challenges, QoL concerns, and symptoms (eg, lumps, breast pain), while 27% (141/518) of the posts sought information on diagnostic dilemmas, treatment options, and second opinions. Patients contributed 60% (318/530) of the posts, and caregivers contributed 21% (111/530) of the posts, with over half (57/107, 53%) discussing their mothers’ diagnosis and treatment struggles. HCPs contributed 16% (85/530) of the posts, primarily sharing clinical trial updates, drug approvals, and disease awareness efforts. A total of 88 posts included discussions on QoL, and eBC significantly impacted patients’ emotional, physical, functional, and social well-being. Discussions revealed key unmet needs, including limited awareness of adjuvant therapy options, lack of peer support groups, suboptimal patient-HCP communication, and insufficient access to specialty care facilities. Conclusions: This study highlights gaps in eBC management related to patient education, HCP communication, and access to specialty care and describes an associated worsening of QoL for patients as reflected in social media posts. Within the limitations of an observational SML design, increasing patient and caregiver awareness of available adjuvant therapies to improve adherence and reduce recurrence risk, alongside expanding access to regional breast cancer centers, may help optimize patient experiences and outcomes. Further research using complementary data sources is needed to confirm and extend these findings.