Epidemiology, management and patient needs in myasthenia gravis: an Italian multistakeholder consensus based on Delphi methodology.

Objectives: To provide comprehensive information on the burden of myasthenia gravis (MG) in Italy, including the unmet needs of patients and several other aspects related to the disease, based on skilled viewpoints of MG experts.

Design: Iterative analysis conducted in accordance with the best practices of the Delphi method, including anonymity, controlled feedback, and statistical stability of consensus.

Setting And Participants: 24 clinicians, 18 public health experts and 4 patient associations experts completed all the Delphi iterations between 18 April and 3 July 2023, for a total of 46 participants from several Italian Regions.

Faricimab versus the standard of care for neovascular age-related macular degeneration in Italy: an indirect treatment comparison.

Objectives: To assess through an indirect treatment comparison (ITC) the potential benefit of faricimab over the anti-vascular endothelial growth factor (VEGF) real-life scenario, hereby defined standard of care (SoC), in Italy, that is, aflibercept, bevacizumab, and ranibizumab, in patients with neovascular age-related macular degeneration (nAMD) naïve to any anti-VEGF treatment.

Methods: Individual patient-level data from the phase III clinical trials TENAYA and LUCERNE (faricimab cohort) and the real-world study RADIANCE (RADIANCE cohort) were used. Efficacy was evaluated with changes in best corrected visual acuity (BCVA) and central subfield thickness (CST) from baseline to 1 year (week 52 in the RADIANCE and week 48 in the faricimab cohorts, respectively).

Beyond the Fragility Index.

The results of randomized clinical trials (RCTs) are frequently assessed with the fragility index (FI). Although the information provided by FI may supplement the p value, this indicator presents intrinsic weaknesses and shortcomings. In this article, we establish an analysis of fragility within a broader framework so that it can reliably complement the information provided by the p value.

Identifying important and feasible primary care structures and processes in the US healthcare system: a modified Delphi study.

Objective: To identify primary care structures and processes that have the highest and lowest impact on chronic disease management and screening and prevention outcomes as well as to assess the feasibility of implementing these structures and processes into practice.

Design: A two-round Delphi study was conducted to establish consensus on the impact and feasibility of 258 primary care structures and processes.

Participants: 29 primary care providers, health system leaders and health services researchers in the USA.

The management of patients with inflammatory bowel disease-associated spondyloarthritis: Italian Group for the Study of Inflammatory Bowel Disease (IG-IBD) and Italian Society of Rheumatology (SIR) recommendations based on a pseudo-Delphi consensus.

Spondyloarthritis (SpA) is the most frequent extraintestinal manifestation in patients with inflammatory bowel diseases (IBD). When IBD and spondyloarthritis coexist, musculoskeletal and intestinal disease features should be considered when planning a therapeutic strategy. Treatment options for IBD and SpA have expanded enormously over the last few years, but randomized controlled trials with specific endpoints focused on SpA are not available in the IBD setting.

From Indication-Based Pricing to Blended Approach: Evidence on the Price and Reimbursement Negotiation in Italy.

Background: New indications for existing medicines are increasing over time. In most countries, drug pricing and reimbursement conditions are renegotiated every time a new indication is approved. There is a growing interest in the price negotiation model for new indications, specifically comparing an indication-based versus blended approach.

Study protocol of a clinical randomized controlled trial on the efficacy of an innovative Digital thErapy to proMote wEighT loss in patients with obesity by incReasing their Adherence to treatment: the DEMETRA study.

Despite the increasing importance of innovative medications and bariatric surgery for the treatment of obesity, lifestyle interventions (diet and physical activity) remain the first-line therapy for this disease. The use of digital devices in healthcare aims to respond to the patient's needs, in order to make obesity treatment more accessible, so our study aims to assess the safety and efficacy of a Digital Therapy for Obesity App (DTxO) for achieving weight loss and its maintenance in patients affected with obesity undergoing an experimental non-pharmacological treatment. Here we present the study protocol of a prospective, multicenter, pragmatic, randomized, double-arm, placebo-controlled, parallel, single-blind study on obese patients who will be treated with a new digital therapy to obtain an improvement in their disease condition through the application of different simultaneous strategies (a dietary regimen and personalized advice program, a tailored physical exercise program, a cognitive-behavioural assessment and program, alerts and reminders, dedicated section on prescribed drugs intake, and chat and online visits with clinical professionals).

Atopic Dermatitis in Italian Pre-School Children: Literature Review of Epidemiological Data with a Focus on Disease Prevalence and Severity.

Atopic dermatitis (AD) is one of the most common diseases worldwide. Severe AD has a major impact on the quality of life of patients. We performed a systematic literature review on the epidemiology of AD in Italian pre-school children (age 0-5 years) and we assessed the available data on the severity of AD.

Potential risk factors, clinicopathological features and determinants of survival for multiple primary melanoma patients compared to single primary melanoma: a large single-center Italian study.

Melanoma patients have a high risk of developing subsequent primary melanomas, a condition known as multiple primary melanoma (MPM). We aimed to compare risk factors of patients with MPM and single primary melanoma (SPM). Primary MPM and SPM consecutively treated at the National Cancer Institute in Milan, Italy, from 1978 to 2021 were retrospectively investigated.

The prognostic role of mitotic rate in cutaneous malignant melanoma: Evidence from a multicenter study on behalf of the Italian Melanoma Intergroup.

Background: This study aimed to improve the understanding of the prognostic value of tumor mitotic rate (TMR) in cutaneous melanoma and assessed its significance as a predictor for overall, melanoma-specific, and recurrence-free survival.

Patients And Methods: This is a retrospective multicenter Italian cohort study of 13,016 patients diagnosed with and treated for invasive primary melanoma between 2005 and 2020 with median follow-up of 5.5 years.

The Evaluation of Drug Innovativeness in Italy: Key Determinants and Internal Consistency.

Background: Innovative medicines are provided with dedicated funds and immediate market access in Italy. Innovativeness evaluation considers unmet need, added therapeutic value, and quality of the evidence.

Objective: We aimed to evaluate the internal consistency and drivers of the innovativeness appraisal process.

Adherence to European Association of Urology Guidelines and State of the Art of Glycosaminoglycan Therapy for the Management of Urinary Tract Infections: A Narrative Review and Expert Meeting Report.

Context: Urinary tract infections (UTIs) have a significant impact on patient's quality of life and society. Antibiotic therapy is the primary approach for the management of UTIs; however, it has major limits in the prevention of recurrent UTIs (rUTIs), also increasing the risk of development of multidrug-resistant micro-organisms.

Objective: The aim of this paper is to discuss the European Association of Urology guidelines for the management of UTIs/rUTIs, the level of adherence to these recommendations, and the available evidence on the use of glycosaminoglycans (GAGs) as a possible alternative treatment to prevent rUTIs.

Allium vegetables intake and the risk of gastric cancer in the Stomach cancer Pooling (StoP) Project.

Background: The role of allium vegetables on gastric cancer (GC) risk remains unclear.

Methods: We evaluated whether higher intakes of allium vegetables reduce GC risk using individual participant data from 17 studies participating in the "Stomach cancer Pooling (StoP) Project", including 6097 GC cases and 13,017 controls. Study-specific odds ratios (ORs) were pooled using a two-stage modelling approach.

Bone and body composition analyses by DXA in adults with GH deficiency: effects of long-term replacement therapy.

Purpose: The effects of growth hormone (GH) replacement on bone mass and body composition in adult with GH deficiency (AGHD) are still debated with regard to their persistence in the long term. Moreover, the impact of the gender on the response to GH is controversial. Aim of this study was to evaluate the long-term effects of rhGH replacement on bone mass and body composition in a monocentric cohort of patients with AGHD.

Key drivers of innovativeness appraisal for medicines: the Italian experience after the adoption of the new ranking system.

Objective: In 2017, the Italian Medicines Agency (Agenzia Italiana del Farmaco, AIFA) introduced a standardised process to appraise innovativeness of medicines. Innovative medicines are provided speeder market access and dedicated funds. Innovativeness criteria are: unmet therapeutic need, added therapeutic value and quality of the evidence (Grading of Recommendations Assessment, Development and Evaluation method).

Shortening the Edinburgh postnatal depression scale using optimal test assembly methods: Development of the EPDS-Dep-5.

Aims: This study used a large database to develop a reliable and valid shortened form of the Edinburgh Postnatal Depression Scale (EPDS), a self-report questionnaire used for depression screening in pregnancy and postpartum, based on objective criteria.

Methods: Item responses from the 10-item EPDS were obtained from 5157 participants (765 major depression cases) from 22 primary screening accuracy studies that compared the EPDS to the Structured Clinical Interview for DSM (SCID). Unidimensionality of the EPDS latent construct was verified using confirmatory factor analysis, and an item response theory model was fit.

Protocol Implementation for Normothermia in Surgery Settings in Italy: Budget-Impact Analysis.

Introduction: Hypothermia is a well-known risk of the perioperative period and considered a preventable effect of anesthesia care. Nevertheless, it is not fully controlled, causing a number of adverse outcomes following surgical operations and thus increasing length of stay in hospital and treatment costs. The aim of this study was to assess the budget impact (BI) of the implementation of proactive strategies to prevent inadvertent perioperative hypothermia (IPH) in surgical patients in Italy, as recommended by international guidelines and by a good clinical practice (GCP) guideline of the Italian Society of Anesthesia, Analgesia, Reanimation, and Intensive Care.

Assessing differential item functioning for the Social Appearance Anxiety Scale: a Scleroderma atient-centred Intervention Network (SPIN) Cohort Study.

Objectives: The Social Appearance Anxiety Scale (SAAS) is a 16-item questionnaire developed to evaluate fear of appearance-based evaluation by others. The primary objective of this research was to investigate the existence of differential item functioning (DIF) for the 16 SAAS items, comparing patients who completed the SAAS in English and French, either to confirm that scores are comparable or provide guidance on calculating comparable scores. A secondary research objective was to investigate the existence of DIF based on sex and disease status.

Statistical considerations for crowdsourced perceptual ratings of human speech productions.

Crowdsourcing has become a major tool for scholarly research since its introduction to the academic sphere in 2008. However, unlike in traditional laboratory settings, it is nearly impossible to control the conditions under which workers on crowdsourcing platforms complete tasks. In the study of communication disorders, crowdsourcing has provided a novel solution to the collection of perceptual ratings of human speech production.