Arcispedale Sant'Anna

Patients with transfusion-dependent thalassemia (TDT) require lifelong blood transfusions; data on the psychosocial and economic impact on patients and their caregivers remain limited. The study aims to address this gap by describing the burden of TDT on pediatric, young, and adult patients, as well as their caregivers, within real-world settings in Italy. This is a cross-sectional, descriptive study conducted between May and December 2021. TDT patients and their caregivers from Italian sites were invited to complete an online survey with the Ratip and Modell questionnaire (1996) and another bespoke questionnaire. All subjects signed an online informed consent before responding to the questionnaires. Seventy-nine subjects completed the survey: 59 patients (43 adults and 16 children) and 20 caregivers (the majority of adult patients were aged 41-50 years). The age at diagnosis differed between adults and pediatric patients, being earlier in the latter (0-2 months in 43.8%). Nearly half of patients received more than one transfusion/month. More than half of the patients experienced blood shortages, particularly during the summer. School absenteeism was reported as a major issue. TDT continues to impose a significant burden, negatively impacting daily life, work, education, and social interactions. The availability of blood resources remains a critical issue, particularly in high-prevalence regions. The study highlights the need for ongoing research, innovative approaches, and enhanced support to ensure improved well-being for TDT patients and their caregivers.

To evaluate the clinical-demographic drivers of treatment choice among targeted synthetic (ts-) and biologic (b-) disease-modifying anti-rheumatic drugs (DMARDs) and therapeutic outcomes in a cohort of patients with rheumatoid arthritis (RA) before and after the European Medicines Agency (EMA) pronouncement on Janus Kinase Inhibitors (JAKis) in January 2023. This single-center, ambidirectional study enrolled patients with RA who were initiating a new course of b/tsDMARDs. Retrospective (2019-2022) and prospective cohorts (2023-2024) were compared, and disease activity and treatment response (achievement of remission or low disease activity) were evaluated at 3 and 6-month follow-up. The study analyzed 539 treatment courses (300 retrospective, 239 prospective) in 380 patients. Following the EMA guidelines, tumor necrosis factor-α inhibitors (TNFis) became the predominant bDMARD (from 32% to 41.4%), whilst JAKis prescriptions declined, particularly in the first-line settings (from 18% to 4.1%). The risk factors proposed by the EMA did not significantly prevent treatment with JAKis. However, we observed that a benefit associated with the first-line treatment in terms of target achievement (adjusted OR for non-response retrospective cohort 0.30, 95%CI 0.14-0.62) was mitigated in the prospective phase (OR 0.82, 95%CI 0.33-2.07). The 2023 EMA pronouncement on JAKis led to a shift in prescription patterns, promoting TNFis versus JAKis. The decline in JAKis prescription did not appear to be driven by EMA-identified baseline risk factors, suggesting a generalized, categorical change. This channeling may have compromised the appropriateness of treatment choices, diminishing effectiveness in the prospective cohort, particularly for first-line therapies.

Heart failure (HF) due to left ventricular systolic dysfunction (LVSD) remains a major clinical challenge, particularly among patients with chronic total occlusions (CTO). CTO are present in up to 30% of patients with LVSD undergoing coronary angiography and are independently associated with worse outcomes. Although advances in interventional techniques have increased success rates of CTO percutaneous coronary intervention (CTO-PCI), high-quality evidence supporting this procedure in patients with LVSD remains limited. Observational studies report potential benefits, including improved survival, alleviation of HF symptoms, and recovery of left ventricular ejection fraction (LVEF). However, randomized controlled trials (RCTs) have largely excluded patients with LVEF <35% and those with advanced, complex coronary artery disease (CAD), including CTO, thereby restricting generalizability. Assessment of myocardial viability remains central to patient select for CTO-PCI, its prognostic value for hard clinical endpoints has not been definitively established. The use of mechanical circulatory support (MCS) during high-risk CTO-PCI is increasing, particularly in patients with reduced LVEF and complex coronary anatomy; available data provides inconsistent evidence regarding its impact on clinical outcomes and appears to be largely influenced by individual patient characteristics. Finally, in the setting of acute coronary syndromes (ACS), the effect of CTO revascularization on clinical endpoints and arrhythmic risk is unclear, with conflicting observational data. Future research should prioritize this underrepresented high-risk cohort and be conducted in experienced centers within an integrated multidisciplinary care framework.

Psoriasis is a chronic inflammatory skin disease associated with significant physical and psychological burden. Tildrakizumab, an interleukin-23 p19 inhibitor, has demonstrated efficacy in treating moderate-to-severe plaque psoriasis both in clinical trials and real-world setting. However, limited data are available on the impact of the effective treatment of psoriasis on the psychological health of patients. The aim of this study was to assess changes in psychological well-being, as well as clinical and quality-of-life outcomes, in patients with moderate-to-severe plaque psoriasis treated with tildrakizumab in routine clinical practice in Italy. This was an interim analysis (IA) of a 52-week multicenter, prospective, observational study. Adults with moderate-to-severe plaque psoriasis initiating tildrakizumab were enrolled. Endpoints focused on well-being and psychological health and included changes, from baseline to week28, in Depression, Anxiety, and Stress Scale-21 (DASS-21) scores, Dermatology Life Quality Index (DLQI), European Social Survey (ESS) items, and World Health Organization-Five Well-Being Index (WHO-5). Effectiveness was also monitored via Psoriasis Area and Severity Index (PASI), and safety via treatment-emergent adverse event reporting. A total of 115 patients were included (mean age 52.5years, 60.8% male), 102 receiving ≥ 1 dose of tildrakizumab and completing DASS-21 evaluations at baseline and week28. At week28, improvements were observed in DASS-21 subscales [depression (-2.6, 95%CI -2.0 to -1.0), anxiety (-2.3, 95%CI -2.0 to -1.0), and stress (-3.4, 95%CI -4.0 to -2.0)], accompanied by marked PASI reduction (-13.7, 95%CI -12.8 to -10.1). DLQI, ESS, and WHO-5 scores also improved. Adverse events were generally mild or moderate, with no unexpected safety signals. In this real-world IA, tildrakizumab was observed to improve the psychological well-being of patients, reflected by a reduction in all items of the DASS-21 scale and, in parallel, confirmed its effectiveness in managing physical symptoms of psoriasis, establishing its role in the holistic management of psoriasis.

In Autoimmunology, interpretative comments can significantly improve the quality of information in laboratory reports. Laboratory autoimmunologists play an increasingly important role in supporting clinicians with the request and interpretation of tests, considering the growing complexity of autoimmune diagnostic and the increase in requests for autoimmune tests by non-specialist physicians. In 2024, the Study Group on Autoimmunology (GdS-AI) of the Italian Society of Clinical Pathology and Laboratory Medicine (SIPMeL) in collaboration with the Study Group of Extra-Analytical Quality (GdS-QuEA) developed a project with the aim to define interpretative comments for systemic autoimmune diseases and organ-specific autoimmune diseases. Based on the analysis of the comments used by GdS-AI laboratories in routine practice, the working group drafted a summary document including no. 47 interpretative comments (22 for systemic autoimmune diseases and 25 for organ-specific autoimmune diseases), representative of possible findings in autoimmune laboratory reports. The document is intended as a proposal of interpretative comments to support laboratory reporting. Furthermore, it defines the important role of the laboratory autoimmunologist in improving the informational quality of reports, while promoting harmonization and appropriateness of test ordering.

Introduction: The aim of the study was to evaluate the safety and feasibility of a novel surgical technique for subconjunctival burial of 10-0 Prolene sutures without conjunctival peritomy. Methods: A retrospective case series of 12 patients who underwent trans-scleral suture fixation or silicone oil retention suture placement using 10-0 Prolene sutures was conducted between January 2024 and February 2025 at Sant’Anna University Hospital, University of Ferrara, Italy. Inclusion criteria included adults requiring scleral fixation with 10-0 Prolene. Exclusion criteria included prior conjunctival surgery at the site, active ocular surface disease, or inability to complete follow-up. Instead of conjunctival peritomy, a linear high-temperature cautery was applied along the intended suture path to create a fibrotic track. Sutures were passed trans-scleral and left exposed on the cauterized zone, allowing spontaneous re-epithelialization. Follow-up included slit-lamp examination, anterior segment photography, and optical coherence tomography at 1 day, 1 week, and 1, 3, and 6 months. Results: A total of 12 patients (12 eyes) were included. Successful suture burial was achieved in all 12 eyes (100%) without exposure, extrusion, or conjunctival dehiscence during the 6-month follow-up. Re-epithelialization was complete by day 7 in all cases (12/12, 100%). No patient reported foreign body sensation after day 7. There were no infections, granulomas, or surgery revisions. Conclusions: The “Burn and Bury” technique is a safe and minimally invasive alternative to conjunctival peritomy for burying trans-scleral Prolene sutures. It preserves conjunctival integrity, reduces surgical trauma, promotes rapid epithelial healing, and enhances patient comfort. This technique may be broadly applicable in anterior segment procedures requiring scleral suture fixation and warrants prospective validation.

BACKGROUND:The pullback pressure gradient (PPG) is a novel physiological metric that quantifies coronary artery disease patterns as focal or diffuse on a scale from 0 to 1. This study assessed the relationship between PPG and residual angina at 1 year.METHODS:PPG Global is a prospective, investigator-initiated, single-arm, multicenter study that enrolled patients with at least 1 lesion with a fractional flow reserve ≤0.80 intended to be treated with percutaneous coronary intervention. After the PPG calculation, physicians could revise treatment assignment to medical therapy or coronary artery bypass graft surgery instead of percutaneous coronary intervention. Focal and diffuse disease were defined based on the median PPG value of 0.62. Patient-reported outcomes were assessed using the Seattle Angina Questionnaire at baseline and 1-year follow-up.RESULTS:The study included 947 patients with PPG and the Seattle Angina Questionnaire at 1 year. The mean age was 67.6±10.2 years, 24% were female, and 29% had diabetes. At 1 year, patients with focal coronary artery disease reported less angina than those with diffuse disease (Seattle Angina Questionnaire angina frequency score, 95.3±9.9 versus 92.5±15.0; P=0.006). PPG was independently associated with improvement in angina (P=0.017).CONCLUSIONS:In patients with flow-limiting coronary artery disease, a focal disease pattern defined by high PPG was associated with greater symptomatic relief at 1 year compared with diffuse disease (low PPG). By capturing the underlying pathophysiologic distribution of epicardial disease and its relation to post-treatment symptom relief, PPG may support a more tailored revascularization decision-making and percutaneous coronary intervention strategy.

ABSTRACTPurposeAtrial fibrillation (AF) is highly prevalent in patients with transfusion‐dependent beta‐thalassemia (TDT). Speckle tracking echocardiography (STE) provides detailed information about left ventricular (LV) and atrial function, however its role in TDT patients with AF has not been completely investigated. This study aimed to assess differences in cardiac mechanical parameters between thalassemia patients with and without history of AF.Methods and results223 TDT patients in sinus rhythm were enrolled and, among them, 26 (11%) had history of AF. A complete STE analysis with the evaluation of global longitudinal strain (GLS), peak atrial longitudinal strain (PALS) and myocardial work (MW) indices were performed. The primary endpoint was the difference in STE parameters. The secondary endpoint was the prevalence of cardiac mechanical dysfunction. Patients with history of AF showed significantly lower values of GLS (19% vs 21%, p = 0.01) and PALS (24% vs 35%, p < 0.001) compared to those without AF. AF patients showed higher prevalence of both ventricular and atrial mechanical dysfunction (respectively 27% vs 12%, p = 0.03 and 42% vs 11%, p < 0.001). PALS showed high discriminative ability (AUC 0.76, 95% CI 0.66–0.85) with an optimal cut‐off value of 25.9% to detect those with history of AF.ConclusionsAmong TDT patients, those with history of AF showed lower values of GLS and PALS. Both LV and atrial mechanical dysfunction were significantly prevalent in patients with history of AF and PALS showed high diagnostic accuracy for the detection of AF.Clinical trial registrationClinicalTrials.gov id NCT05508932