Ruxolitinib crema nella vitiligine: efficacia reale oltre i dati clinici. #ICD2025 [Dermatologia]

A un anno dall’introduzione di ruxolitinib crema, primo trattamento topico specifico per la vitiligine non segmentale, si apre un nuovo capitolo nella gestione clinica della malattia. In occasione dell’incontro scientifico Sharing Real Practice in Vitiligo: Beyond Clinical Data, svoltosi durante il XIV International Congress of Dermatology, sono state condivise esperienze, casi clinici e dati real-world, confrontando l’efficacia terapeutica osservata nella pratica quotidiana con i risultati degli studi di fase 3. 

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Gestione della sepsi e dello shock settico, confronto internazionale a Bologna [Cardio]

Si è tenuto a Bologna il DEC-Day & BEYOND: FACING NEW CHALLENGES, un evento scientifico di rilievo internazionale promosso da AOP Health, dedicato alla gestione avanzata del paziente critico con sepsi e shock settico. Due giornate intense, ricche di contributi multidisciplinari, che hanno visto la partecipazione di clinici e ricercatori impegnati ad affrontare le sfide emergenti in area critica, alla luce delle più recenti evidenze e strategie terapeutiche.

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Neurofibromatosi di tipo 1, Progetto SENSE. Video Reportage di PharmaStar [Malattie Rare]

Il progetto SENSE (esperienza Sensoriale nella gestione e trattamento del Neurofibroma pleSsiformE), realizzato grazie al contributo non condizionante di Alexion, nasce dalla volontà di proporre un approccio più profondo, umano e multidisciplinare a una delle sfide più delicate nell’ambito delle malattie rare pediatriche: accompagnare i bambini con neurofibromatosi di tipo 1 (NF1) nella complessa gestione del neurofibroma plessiforme (PN).

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Vasculiti ANCA-associate, conferme per avacopan da studi di real life #EULAR2025 [Ortopedia e Reumatologia]

Nel corso del congresso EULAR, sono stati presentati nuovi dati che suffragano l’impiego di avacopan nel trattamento di due vasculiti ANCA-associate (la granulomatosi con poliangioite e la poliangioite microscopica – GPA ed MPA). Il primo (AVAC-EUR), uno studio osservazionale multicentrico retrospettivo europeo, ha mostrato come la continuazione del trattamento con il farmaco si accompagni ad una riduzione delle ospedalizzazioni e dei tassi di infezione rispetto al quanto osservato con l’ìnterruzione del trattamento entro un anno. Il secondo, uno studio prospettico multicentrico italiano di real life, ha dimostrato l’efficacia precoce e duratura, la sicurezza e i miglioramenti nei punteggi della qualità di vita associati al trattamento con avacopan nella gestione delle vasculiti sopra indicate. in una coorte di pazienti italiani.

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Eczema cronico delle mani, perché è necessario parlare di epidemiologia? [Dermatologia]

L’eczema cronico delle mani rappresenta una sfida significativa sia clinica che sociale, e la sua gestione non può prescindere da una solida conoscenza epidemiologica. Affrontare l’argomento dal punto di vista epidemiologico è fondamentale per molteplici ragioni, che vanno ben oltre la semplice descrizione clinica. E’ stato questo il tema della relazione della Dott.ssa Emanuela Martina, Specialista in Dermatologia e Venereologia, SOD Clinica di Dermatologia, AOU delle Marche, Ancona, in occasione della Special Edition del congresso SIDeMaST-ADOI che si è svolto in parallelo alla prima giornata del XIV International Congress of Dermatology.

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Gestione dell'infezione da HIV: importanza dell'ottimizzazione della terapia [Infettivologia]

La gestione dell’infezione da HIV ha fatto enormi progressi, portando oggi la maggior parte delle persone con HIV in trattamento a raggiungere e mantenere una viremia non rilevabile. Tuttavia, l’efficacia della terapia antiretrovirale (ART) non è più il solo parametro da considerare: l’ottimizzazione della terapia è diventata fondamentale per garantire che il trattamento sia ben tollerato, sostenibile e adattato alle esigenze individuali, migliorando così l’aderenza e la qualità di vita del soggetto, in una prospettiva di successo a lungo termine.

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Presentato in Senato il Position Paper sui PDTA per amiloidosi da transtiretina  [Cardio]

Un paziente con amiloidosi cardiaca da transtiretina (ATTR) oggi in Italia può ricevere diagnosi e cure molto diverse a seconda della Regione in cui vive. Eppure, questa malattia rara, spesso sottovalutata e diagnosticata troppo tardi, necessita di un percorso di cura rapido, personalizzato e multidisciplinare.
Per rispondere a questa sfida, Bistoncini Partners ha realizzato, con il contributo non condizionante di Pfizer, il Position Paper: “L’importanza dei PDTA personalizzati per la gestione del paziente con Amiloidosi da Transtiretina”, che viene oggi presentato nel corso di una conferenza stampa promossa dalla Senatrice Elena Murelli

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The Impact of Fontan Circulatory Failure on Heart Transplant Survival: A 20-Center Retrospective Cohort Study

Circulation, Ahead of Print. BACKGROUND:Fontan circulatory failure (FCF) is a chronic state in palliated single ventricle heart disease with high morbidity and mortality rates, including heart failure, multisystem end-organ disease, and need for heart transplant. Specific FCF morbidities have not been rigorously defined, limiting study of how FCF morbidities affect pre–heart transplant and post–heart transplant outcomes. We hypothesized that FCF-related morbidities affect survival from heart transplant waitlisting through 1 year after heart transplant.METHODS:This 20-center, retrospective cohort study collected demographic, medical/surgical history, waitlist, and peri- and post–heart transplant data, and a priori defined FCF-specific morbidities, in Fontan patients who were listed for heart transplant from 2008 through 2022. Univariate 2-group statistics compared surviving individuals with those who died anytime from waitlisting to 1 year after heart transplant, died on the waitlist, or underwent transplant and died within 1 year after transplant. Using covariates from both univariate analyses, multivariable logistic regression determined the primary study outcome of independent FCF risk factors for death between waitlist and 1 year after heart transplant.RESULTS:Of 409 waitlisted patients, 24 (5.9%) died on the waitlist. Of the 341 (83.4%) who underwent transplant, 27 (8.5%) did not survive to 1 year. Univariate risk factors for waitlist death included higher aortopulmonary collateral burden, >1 hospitalization in the previous year, younger age, sleep apnea, higher New York Heart Association class, nonenrollment in school or work, and single-parent home. Risk factors for 1-year post–heart transplant mortality included hypoplastic left heart syndrome diagnosis, patent fenestration, anatomic Fontan obstruction, clinical cyanosis (pulse oximetry 1 hospitalization in the year before waitlisting (adjusted odds ratio, 2.0 [95% CI, 1.0–4.1];P=0.05) and clinical cyanosis (adjusted odds ratio, 5.0 [95% CI, 1.8–13.4];P=0.002).CONCLUSIONS:Patients with Fontan palliation selected for heart transplant have substantial mortality rates from waitlisting through transplant. Among FCF-specific morbidities, cyanosis is associated with worsened survival and necessitates further study. Clinical morbidity of any type requiring repeated hospital admission also should prompt consideration of heart transplant.

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Tumore della prostata: presentata la campagna 'Allo Specchio della Salute' [Oncologia-Ematologia]

In Italia, il carcinoma della prostata rappresenta la neoplasia più diffusa tra gli uomini over 50, incidendo per circa il 30% su tutti i tumori maschili, con 40.192 nuovi casi stimati solo nel 2024. È stata presentata oggi, in Senato la campagna di sensibilizzazione ‘Allo Specchio della Salute – uno spazio di confronto sul carcinoma prostatico’, pensata per coinvolgere l’intera comunità nel dibattito intorno alla gestione e cura di questa patologia. Un’intera giornata dedicata al carcinoma della prostata suddivisa in due appuntamenti sinergici e xomplementari: la conferenza stampa seguita dalla campagna di awareness.

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Evaluating an intervention to promote access to mental healthcare for low language proficient migrants and refugees across Europe (MentalHealth4All): study protocol for a pretest-post-test cross-national survey study

Background
Migrants and refugees with low language proficiency (LLP) in the dominant language of their host country have a higher risk of suffering from certain mental health disorders compared with non-migrant populations. They are also more likely to experience a lack of access to mental healthcare due to language-related and culture-related barriers. As part of the MentalHealth4All project, a digital multilingual communication and information platform was developed to promote access to mental healthcare for LLP migrants and refugees across Europe. This paper describes the study protocol for evaluating the platform in practice, among both health and/or social care providers (HSCPs) and LLP migrants and refugees.

Methods and analysis
We will conduct a pretest–post-test cross-national survey study to evaluate the platform’s effect evaluation (primary objective) and process evaluation (secondary objective). The primary outcomes (measured at T0, T2 and T3) are four dimensions of access to mental healthcare services: availability, approachability, acceptability and appropriateness of mental healthcare. Secondary outcomes (measured at T2) are: actual usage of the platform (ie, tracking data), perceived ease of use, usefulness of content, comprehensibility of information, attractiveness of content and emotional support. Participants will be recruited from nine European countries: Belgium, Germany, Italy, Lithuania, the Netherlands, Poland, Slovakia, Spain and the UK. Using convenience sampling through professional networks/organisations and key figures, we aim to include at least 52 HSCPs (ie, 6–10 per country) and 260 LLP migrants (ie, 30–35 per country). After completing a pretest questionnaire (T0), participants will be requested to use the platform, and HSCPs will participate in an additional personalised training (T1). Next, participants will fill out a post-test questionnaire (T2) and will be requested to participate in a second post-test questionnaire (T3, about 6–8 weeks after T2) to answer additional questions on their experiences through a brief phone interview (T3 is optional for migrants/refugees).

Ethics and dissemination
For all nine countries, the ethical review board of the participating university (hospital) has assessed and approved the protocol. If successful, the MentalHealth4All platform will be made publicly available to help improve access to mental healthcare services, as well as HSCPs’ cultural competencies in delivering such services, for any LLP migrants and refugees across Europe (and beyond). Findings will also be disseminated through peer-reviewed journals and conferences.

Registration details
The ‘MHealth4All project’ was prospectively registered on Open Science Framework, DOI: 10.17605/OSF.IO/U4XSM.

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Implementation of an intervention to scale up coverage of pneumonia management in children younger than 5 years in a north Indian district: protocol for a quasi-experimental, mixed-methods, pre-post implementation study

Introduction
The National Family Health Survey-5 has reported an under-five mortality rate of 41.9 per 1000 live births in India. Pneumonia, one of the leading causes of under-five mortality, contributes substantially to this figure. The Indian government has made efforts through multiple national programmes, but pneumonia-specific mortality remains high. The Government of India revised their Childhood Pneumonia Management Guidelines in 2019 to improve under-five pneumonia prevention and management. This implementation study aims to achieve a high population-based coverage of pneumonia treatment for under 5 yearold children in the Palwal district of India.

Method and analysis
This implementation study uses a quasi-experimental pre-post design and a mixed-methods approach, conducted in three phases: (i) formative research, (ii) model optimisation through iterative testing in a learning block and (iii) scale-up and concurrent evaluation. The study is set in Palwal district, Haryana, and the primary catchment/study area will be the Health and Wellness Centres, the most accessible public health facilities for the community. Approximately 4167 households will be surveyed to capture ~2400 under-five children, among whom about 120 pneumonia cases (based on an estimated 5% prevalence) will be included in the analysis of treatment coverage and outcomes. Quantitative data will be analysed using descriptive statistics and generalised linear models, while qualitative data from focus group discussions and in-depth interviews will be thematically analysed using NVivo software.

Ethics and dissemination
Ethical approval was granted by the ethical committees of the Society for Applied Studies (ERC/IR Pneumonia/2021), the Regional Ethics Committee of Western Norway (2022/531608) and the WHO(ERC.0003652). Additionally, this study has obtained the Government of Haryana state (Memo no. HSHRC/2022/505) and Health Ministry steering committee (approval date: 19 Dec 2022, proposal id 2022–17596) approvals. Informed consent will be obtained from all participants, including caregivers and healthcare workers, prior to data collection. Dissemination meetings in the study country will share results with stakeholders, including Ministry of Health officials, health managers, families of under-five children, community leaders and academia, to discuss national health programme implications. Results will also be shared regionally and globally, with publications and presentations encouraged in national and international forums.

Study registration
Clinical Trials Registry – India, CTRI/2021/03/031622.

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Purastat post-EMR: unresolved questions from the PURPLE trial

We read with interest the recently published PURPLE trial evaluating the prophylactic use of Purastat to prevent delayed bleeding following EMR of large duodenal and colorectal lesions.1 While we acknowledge the importance of randomised trials in shaping clinical practice, several methodological concerns in this study limit the interpretability and clinical applicability of its findings. The trial was prematurely halted following an interim analysis involving only 200 patients. We believe this pragmatic interim analysis might have been conducted too early on too small a sample size, potentially underpowering the study to detect a clinically relevant effect. The initial hypothesis of an 80% reduction in delayed bleeding in the Purastat arm, based on results from a clipping study by Albéniz et al,2 appears overtly optimistic. The power calculation was based on an anticipated 2% delayed bleeding rate in the Purastat arm which is unrealistic given the inclusion…

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Stressed hepatocyte sustains alcohol-associated hepatitis progression by producing leukocyte cell-derived chemotaxin 2

Background
Neutrophil infiltration and hepatocyte damage are indispensable hallmarks in alcohol-associated hepatitis (AH), yet the underlying crosstalk between neutrophils and hepatocytes and its role in AH pathogenesis remain unclear.

Objective
We investigate the regulatory role of leucocyte cell-derived chemotaxin 2 (LECT2) in hepatocyte–neutrophil interaction and its impact on AH progression.

Design
We used bulk and single-cell RNA sequencing to identify hepatocyte-secreted factors targeting neutrophils. We analysed serum and liver samples from AH patients and employed genetically modified mice alongside in vitro studies.

Results
RNA-sequencing analysis identified several neutrophil chemokines that are elevated in hepatocytes from AH patients, including LECT2 whose role in AH remains largely unknown. AH patients exhibited increased levels of LECT2 in hepatocytes, positively correlating with the severity of AH. Ethanol-fed mice also exhibited elevated liver LECT2, which was abolished by inhibiting endoplasmic reticulum stress. Functional studies revealed that ethanol-induced liver injury was ameliorated in Lect2-deficient mice but was exacerbated in mice with hepatic overexpression of Lect2. Furthermore, LECT2 exacerbated ethanol-induced liver injury by promoting reactive oxygen species (ROS) through its interaction with prohibitin 2 (PHB2), a neutrophil membrane protein. By directly binding to PHB2, LECT2 disrupts the stable structure of PHB1/PHB2 heterodimerisation, consequently leading to PHB2 degradation, ROS accumulation, neutrophil activation and neutrophil extracellular trap formation. Moreover, therapeutic intervention of LECT2 via Lect2 shRNA ameliorated ethanol-induced liver injury.

Conclusion
Our studies identified a novel vicious cycle between neutrophils and hepatocytes through the LECT2–PHB2 interaction, presenting a promising therapeutic intervention by targeting LECT2 to mitigate AH in patients.

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