Cellule staminali nella cura di Crohn e colite ulcerosa: tra speranza e realtà clinica [Gastroenterologia ed epatologia]

Le malattie infiammatorie croniche intestinali (MICI), come la malattia di Crohn e la colite ulcerosa, rappresentano una sfida terapeutica complessa. Attualmente, il trattamento si basa sul controllo dell’infiammazione attraverso la modulazione del sistema immunitario. Tuttavia, una nuova frontiera terapeutica, quella delle cellule staminali, si sta facendo largo nella ricerca clinica, con l’obiettivo di rigenerare i tessuti danneggiati e superare i limiti delle terapie immunosoppressive. Sebbene i risultati siano ancora contrastanti e non sufficienti per un’applicazione su larga scala, alcuni studi mostrano segnali promettenti, soprattutto per i casi refrattari di Crohn.

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PASSIoN Trial (Perinatal Arterial Stroke Treated With Intranasal Stromal Cells): 2-Year Safety and Neurodevelopment

Stroke, Ahead of Print. BACKGROUND:The PASSIoN study (Perinatal Arterial Stroke Treated With Stromal Cells Intranasally) demonstrated the feasibility and short-term safety of single-dose allogeneic mesenchymal stromal cells (MSCs) administered intranasally to neonates with perinatal arterial ischemic stroke between February 2020 and April 2021. In this study, we assessed long-term safety and neurodevelopmental outcomes and explored outcome differences with a non–MSC-treated cohort.METHODS:In this post hoc analysis, we evaluated the safety of MSC administration by assessing brain tissue loss, adverse events, and neurodevelopmental outcomes of PASSIoN participants (n=10). The tissue loss ratio was calculated using semi-automatic segmentation based on neonatal and 3-month magnetic resonance imaging. At the age of 2 years, we assessed the occurrence of cerebral palsy, motor and cognitive delays (Zscore

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Epilessie e sport: attività fisica sì, ma con consapevolezza. Parola agli esperti LICE [Neurologia e Psichiatria]

Epilessie e sport, un binomio possibile. L’attività fisica, infatti, rappresenta un importante alleato per il benessere psico-fisico delle persone con epilessia, purché si scelgano discipline adatte e si adottino le giuste precauzioni. Per maggiori informazioni è possibile consultare Raccomandazioni riguardo a “Epilessia e Attività Sportive” redatte congiuntamente con la Federazione Medico Sportiva Italiana (FMSI)

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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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Finerenone ottiene il via libera Fda per lo scompenso cardiaco con frazione di eiezione ventricolare sinistra ≥40% [Fda]

L’Agenzia statunitense Fda ha recentemente approvato una nuova indicazione terapeutica per finerenone, antagonista non steroideo del recettore dei mineralcorticoidi (MRA), destinato ora anche ai pazienti con scompenso cardiaco con frazione di eiezione ventricolare sinistra (LVEF) pari o superiore al 40%.  Il via libera dell’Fda si basa sui risultati dello studio FINEARTS-HF.

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Uso prolungato del cerotto alla capsaicina migliora dolore e qualità della vita nella neuropatia periferica [Dolore]

Secondo i risultati di uno studio pubblicato su Pain and Therapy, applicazioni ripetute di un cerotto ad alta concentrazione di capsaicina (HCCP, 179 mg) possono determinare miglioramenti duraturi nel dolore, nella qualità del sonno, nello stato emotivo e nella qualità di vita dei pazienti con neuropatia periferica da lesione nervosa chirurgica o traumatica (PNI).

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Osteogenesi imperfetta, bisognerà attendere per la chiusura della fase III di setrusumab. Delusione in Borsa. [Ortopedia e Reumatologia]

Le azioni di Ultragenyx Pharmaceutical Inc. e Mereo BioPharma Group plc sono crollate giovedì, rispettivamente di circa il 27% e oltre il 40%, dopo che le due aziende hanno annunciato che lo studio clinico di Fase 3 ORBIT, che valuta setrusumab (UX143) in pazienti pediatrici e giovani adulti affetti da osteogenesi imperfetta (OI), non verrà interrotto anticipatamente per efficacia. Una decisione che ha deluso le aspettative del mercato, che sperava in una conclusione precoce del trial sulla base di risultati intermedi positivi.

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Obesità con e senza diabete di tipo 2, triplo agonista sperimentale retatrutide migliora i profili metabolici [Diabete]

Nelle persone obese, con o senza diabete di tipo 2, il farmaco sperimentale a somministrazione settimanale retatrutide, che agisce come agonista recettoriale del GIP, del GLP-1 e del glucagone, ha migliorato i profili metabolici. Sono i risultati di un’analisi esplorativa dei biomarcatori di uno studio di fase II presentati al congresso 2025 dell’American Diabetes Association (ADA).

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Principal investigators experience of COVID-19 therapeutic clinical trials in Japan: a qualitative study

Objective
During the COVID-19 pandemic, many clinical trials were conducted to identify effective COVID-19 therapeutics. However, while a large amount of resources was invested and significant numbers of patients participated, this did not necessarily have an impact on clinical practice. To face these issues, initiatives such as the 100 Days Mission have been set out globally. Yet, limited data exist on the context surrounding the implementation of clinical trials at a national level during a health emergency. The study explored experiences and perceptions of principal investigators in conducting clinical trials for COVID-19 therapeutics in Japan.

Design
A qualitative study was conducted using semistructured interviews. The obtained data were inductively analysed using thematic analysis.

Setting and participants
We interviewed 15 principal investigators between September and November 2022 who conducted investigator-initiated clinical trials on the development of COVID-19 therapeutics in Japan.

Results
Three themes were generated: structural barriers, fragmented efforts and limited evidence generation. Structural barriers and fragmented efforts comprised four subthemes: individual, institutional, interinstitutional and policy/regulatory levels. Structural barriers at all levels included (1) limitations of individual capabilities, (2) the double burden of clinical practice and research, (3) inefficient interinstitutional collaboration and (4) regulatory frameworks and available resources that interrupt stakeholders’ actions, leading to limited evidence generation despite the fragmented efforts of principal investigators and other stakeholders.

Conclusions
This study illustrated that the efforts of Japanese principal investigators did not necessarily pay off in identifying therapeutics. A strategic and systematic approach for an improved national clinical trial ecosystem must be sought during the interpandemic period to overcome structural barriers in harmonisation with the global stakeholders.

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PREACT-digital: study protocol for a longitudinal, observational multicentre study on digital phenotypes of non-response to cognitive behavioural therapy for internalising disorders

Introduction
Cognitive behavioural therapy (CBT) serves as a first-line treatment for internalising disorders (ID), encompassing depressive, anxiety or obsessive-compulsive disorders. Nonetheless, a substantial proportion of patients do not experience sufficient symptom relief. Recent advances in wearable technology and smartphone integration enable new, ecologically valid approaches to capture dynamic processes in real time. By combining ecological momentary assessment (EMA) with passive sensing of behavioural and physiological information, this project seeks to track daily fluctuations in symptom-associated constructs like affect, emotion regulation (ER) and physical activity. Our central goal is to determine whether dynamic, multimodal markers derived from EMA and passive sensing can predict treatment non-response and illuminate key factors that drive or hinder therapeutic change.

Methods and analysis
PREACT-digital is a subproject of the Research Unit FOR 5187 (PREACT), a large multicentre observational study in four outpatient clinics. PREACT channels state-of-the-art machine learning techniques identify predictors of non-response to CBT in ID. The study is currently running and will end in June 2026. Patients seeking CBT at one of four participating outpatient clinics are invited to join PREACT-digital. They can take part in (1) a short version with a 14-day EMA and passive sensing phase prior to therapy, or (2) a long version in which the short version’s assessments are extended throughout the therapy. It is estimated that 468 patients take part in PREACT-digital, of which 350 opt for the long version of the study. Participants are provided with a smartwatch and a customised study app. We collect passive data on heart rate, physical activity, sleep and location patterns. EMA assessments cover affect, ER strategies, context and therapeutic agency. Primary outcomes on (non)-response are assessed after 20 therapy sessions and therapy end. We employ predictive and exploratory analyses. Predictive analyses focus on classification of non-response using basic algorithms (ie, logistic regression and gradient boosting) for straightforward interpretability and advanced methods (LSTM, DSEM) to capture complex temporal and hierarchical patterns. Exploratory analyses investigate mechanistic links, examine the interplay of variables over time and analyse change trajectories. Study findings will inform more personalised and ecologically valid approaches to CBT for ID.

Ethics and dissemination
The study has received ethical approval from the Institutional Ethics Committee of the Department of Psychology at Humboldt Universität zu Berlin (Approval No. 2021–01) and the Ethics Committee of Charité-Universitätsmedizin Berlin (Approval No. EA1/186/22). Written informed consent will be obtained from all participants prior to enrolment. Results will be disseminated through peer-reviewed journals and presentations at national and international conferences.

Trial registration number
DRKS00030915; OSF PREACT: http://osf.io/bcgax; OSF PREACT-digital: https://osf.io/253nb.

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Bimekizumab nell'idrosadenite suppurativa: miglioramento clinicamente rilevante mantenuto fino a due anni.  [Dermatologia]

L’idrosadenite suppurativa è una malattia cutanea cronica e infiammatoria che interessa i follicoli piliferi. Si presenta con noduli infiammatori profondi e dolorosi, ascessi, tunnel drenanti (draining tunnels, DTs) e cicatrici, localizzati tipicamente in aree intertriginose del corpo1. Bimekizumab (BKZ), anticorpo monoclonale umanizzato diretto contro le interleuchine IL-17A e IL-17F, ha mostrato risultati promettenti nel trattamento dell’HS. I dati dello studio di estensione in aperto BE HEARD EXT, presentati durante la XIV edizione dell’International Congress of Dermatology confermano un miglioramento clinicamente rilevante delle lesioni e dei tunnel drenanti mantenuto fino a due anni.2

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