Mieloma multiplo ricaduto/refrattario, triplette con belantamab mafodotin confermano i loro benefici, anche nei pazienti ad alto rischio. #EHA25 [Oncologia-Ematologia]

Nei pazienti con mieloma multiplo ricaduto/refrattario che presentano almeno un’alterazione citogenetica associata a un alto rischio di progressione della malattia, il trattamento con l’anticorpo farmaco-coniugato (ADC) anti-BCMA belantamab mafodotin combinato con bortezomib e desametasone (tripletta BVd) offre un beneficio rispetto alla tripletta standard daratumumab-bortezomib-desametasone (DVd) sia in termini di sopravvivenza libera da progressione (PFS) e sopravvivenza globale (OS) sia in termini di tasso di risposte profonde.

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SIMG – diabete, scompenso cardiaco, malattia renale cronica: apprezzamento per la decisione di Aifa di abrogare i Piani Terapeutici [Italia]

La Società Italiana di Medicina Generale e delle Cure Primarie esprime pieno apprezzamento per la decisione comunicata dall’Agenzia Italiana del Farmaco (Aifa) il 30 giugno con cui vengono riclassificate le gliflozine (SGLT2-i: inibitori del trasportatore sodio/glucosio di tipo 2) in fascia A e contestualmente abrogati, per l’intera classe, i Piani Terapeutici per il loro utilizzo nel trattamento del diabete di tipo 2, dello scompenso cardiaco e della malattia renale cronica.

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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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Nuova sfida della Fondazione Lilly: formare i leader della Sanità del futuro. VideoReportage di PharmaStar [Diabete]

Un investimento strategico per il futuro della Sanità pubblica italiana, pensato per colmare il divario in ricerca e innovazione e costruire competenze che sappiano guidare il cambiamento. È questo il cuore della nuova missione della Fondazione Lilly per l’Italia, che con il piano “30×30” nei prossimi 10 anni investirà oltre 1,5 milioni di euro per finanziare 30 dottorati di ricerca in 30 università italiane, in tutte le regioni del Paese.

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[Review] Measurable residual disease in hematologic malignancies: a biomarker in search of a standard

Over the past decade, measurable residual disease (MRD) has emerged as a critical tool for detecting and monitoring a variety of cancers, but particularly hematologic malignancies. The rapid adaptation of this novel approach to monitoring disease status is intuitively appealing: it offers significantly greater sensitivity than traditional methods for detecting the small population of malignant cells that persist after treatment, and which are undetectable by standard approaches such as monitoring abnormalities on radiographic scans or assessing morphologic or karyotypic changes from bone marrow sampling.

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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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Theory-based chatbot for promoting colorectal cancer screening in a community setting in Hong Kong: study protocol of a randomised controlled trial

Background
Colorectal cancer (CRC) is the third most common cancer and the second leading cause of cancer mortality worldwide. Despite the organised CRC screening programme, the uptake rate of the population-based CRC screening was still low. Thus, we will conduct a randomised controlled trial in a community setting to evaluate the effectiveness of a theory-based chatbot in promoting CRC screening uptake.

Methods and analysis
A total of 500 eligible participants will be randomly assigned to a WhatsApp Messenger-initiated chatbot outreach group or a standard text reminder group at a ratio of 1:1. The intervention group will deliver Chinese culturally tailored education texts and videos developed based on the Health Belief Model and the Trans-Theoretical Model. The control group will deliver a standard text reminder of information about the Hong Kong organised CRC screening programme. In addition to the baseline assessment and postintervention assessment, all subjects will be followed up for 3 months and 6 months, respectively. The primary outcome will be the CRC screening uptake rate at the 3 month and 6 month follow-up. The secondary outcomes will be the intention to undergo CRC screening uptake, time interval to participate in and complete screening after recruitment, and reasons for not participating in screening at the 3 month and 6 month follow-up. Quantitative data will be analysed using Student’s t-test, Pearson’s 2 test or Fisher’s exact test. Qualitative data will be analysed by thematic analysis.

Ethics and dissemination
Ethical approval of this trial was granted by the Joint Chinese University of Hong Kong-New Territories East Cluster Clinical Research Ethics Committee (2022.614). Written informed consent will be obtained from study participants before enrolment. The findings will be disseminated through peer-reviewed journals.

Trial registration number
The study was registered on clinicaltrials.gov (NCT06192862).

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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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Cancro della prostata non metastatico ormono-sensibile, Aifa approva  la rimborsabilità di enzalutamide. Cambia il paradigma di cura [Italia]

L’Agenzia Italiana del Farmaco (Aifa) ha riconosciuto la rimborsabilità di un’ulteriore indicazione terapeutica per enzalutamide, sulla base dei risultati dello studio di Fase 3 EMBARK. Con questa autorizzazione enzalutamide diventa il primo ed unico inibitore del segnale del recettore degli androgeni che ha ottenuto la rimborsabilità per il trattamento di uomini adulti con cancro della prostata non metastatico ormono-sensibile (nmHSPC) con recidiva biochimica (BCR) ad alto rischio non idonei alla radioterapia di salvataggio

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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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Understanding Predictors of Lifelong Initiation and Follow-up Treatment for adolescents and youth living with HIV (UPLIFT): an integrated prospective cohort in Eastern Cape, South Africa

Purpose
Adolescents living with HIV (ALHIV) are a priority population for achieving global HIV prevention and treatment targets but experience poorer outcomes than adults. Long-term follow-up is essential to understand their transition into adulthood. By linking self-reported survey data with routine laboratory records, we established a social science clinical cohort of ALHIV South Africa’s Eastern Cape to explore factors shaping their long-term health and well-being.

Participants
Eligible participants were adolescents who were part of a three-wave quantitative cohort of ALHIV and not living with HIV (2014–2018) and had consented (adolescent and caregiver) to having their self-reported interviews linked with routine health records (n=1563). Adolescents were recruited into the existing three-wave cohort through clinic and community-based methods (97% enrolment, >90% retention over three waves). Between 2019 and 2022, we abstracted laboratory test records from the National Health Laboratory Services database for all eligible participants, with matching based on demographic variables. Individuals with at least one HIV-related record form our ‘lifelong social science cohort’, a total of 956 ALHIV (852 of 1107 ALHIV and 104 of 456 HIV-uninfected).

Findings to date
A total of 32 886 laboratory test records from 2004 to 2023 were matched through three rounds of data extraction, using iteratively refined record-linking searches. Most records were viral load (8864) and CD4 count (6801) results, with a median of 10 (IQR: 7–14) and 8 (IQR: 5–11) tests per matched adolescent, respectively. Overall, 956 of 1563 adolescents (61%) were successfully linked to laboratory data, including 852 of 1107 (77%) ALHIV. Analysis of the matched cohort survey-laboratory data provided several insights. Self-reported antiretroviral therapy adherence was strongly associated with viral suppression, even after adjusting for covariates. The strongest predictors of suppression were not reporting missed doses in the past 3 days, past week and not missing clinic appointments in the past year. Among adolescent girls and young women living with HIV, access to safe and affordable facilities, and kind and respectful staff were associated with a higher likelihood of multiple improved HIV-related outcomes, including viral suppression. Exposure to sexual and intimate partner violence predicted worse viral load outcomes among adolescents.

Future plans
This integrated prospective cohort provides an opportunity to characterise long-term HIV treatment outcomes among ALHIV in Africa. We will investigate how individual, familial, community and healthcare experiences in childhood, and adolescence shape these outcomes. Since the COVID-19 pandemic happened during the period of matched data, we will also investigate the potential effect of the COVID-19 pandemic on adolescent HIV treatment outcomes, with potential subgroup analyses for individuals with available COVID-19-related results.

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Association between loneliness and depression, anxiety and anger during the COVID-19 pandemic: a nationwide population-based survey

Objectives
This study aims to determine the association between loneliness and depression, anxiety and anger with a representative sample of the general population in Korea, which are the most prevalent mental health problems during the pandemic.

Design
Cross-sectional study.

Setting
National survey across all 17 provinces in South Korea between December 2021 and January 2022.

Participants
We conducted a national survey on 2699 participants aged 19–84 years using proportional stratified sampling. Using the UCLA Loneliness Scale and standardised questionnaires for depression (Patient Health Questionnaire-9), anxiety (Generalised Anxiety Disorder-7) and anger (Patient-Reported Outcomes Measurement Information System-Anger), we explored the prevalence and association of loneliness with these mental health outcomes.

Primary and secondary outcome measures
Primary outcomes included the prevalence and co-occurrence of depression, anxiety and anger across different levels of loneliness.

Results
Of total, 20.7% and 2.1% experienced moderately high and high levels of loneliness, respectively. Among participants with high levels of loneliness, 11.8%, 5.9% and 11.8% had depression, anxiety and anger, respectively, and 28.7% of them had depression, anxiety and anger together. The adjusted prevalence of depression was 0.2 (95% CI 0.0 to 0.5), 8.2 (95% CI 6.7 to 9.7), 31.3 (95% CI 27.4 to 35.3) and 63.5 (95% CI 50.1 to 76.8) for low, moderate, moderately high and high levels of loneliness, respectively. Similarly, increased adjusted prevalence of anxiety and anger was observed ㅈwith higher levels of loneliness.

Conclusions
Lonely people have a higher risk of depression, anxiety and anger. Identifying individuals who may be vulnerable to loneliness is important for early intervention.

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