Presse Medicale最新文献_第4页

Diagnosis and management of infantile hemangiomas: Tremendous advances over the last 15 years 婴儿血管瘤的诊断和治疗：在过去的15年中取得了巨大的进步。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-09-01 Epub Date: 2025-05-28 DOI: 10.1016/j.lpm.2025.104291

Dorine Canu , Sorilla Mary-Prey , Christine Leauté-Labreze

引用次数: 0

Genetic aspects of vascular malformations 血管畸形的遗传方面。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-09-01 Epub Date: 2025-05-29 DOI: 10.1016/j.lpm.2025.104295

Nicole Revencu , Julien Coulie , Laurence M. Boon , Miikka Vikkula

引用次数: 0

Syndromic vascular anomalies: A challenge 综合征性血管异常：一个挑战。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-09-01 Epub Date: 2025-05-30 DOI: 10.1016/j.lpm.2025.104294

Stephanie Bowe , Veronika Dvorakova

引用次数: 0

Peripheral arteriovenous malformations: Diagnosis and future prospects 外周动静脉畸形：诊断及未来展望。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-09-01 Epub Date: 2025-06-09 DOI: 10.1016/j.lpm.2025.104296

Julien Coulie , Miikka Vikkula , Laurence M. Boon

{"title":"Peripheral arteriovenous malformations: Diagnosis and future prospects","authors":"Julien Coulie , Miikka Vikkula , Laurence M. Boon","doi":"10.1016/j.lpm.2025.104296","DOIUrl":"10.1016/j.lpm.2025.104296","url":null,"abstract":"<div><div>Peripheral arteriovenous malformations (AVMs) are rare, high-flow vascular anomalies caused by errors in vasculogenesis or angiogenesis, frequently driven by mutations in the RAS/RAF/MEK/ERK signaling pathway. These lesions exhibit progressive behavior, leading to diverse clinical presentations ranging from localized pain, swelling and important bleeding to severe systemic complications such as high-output cardiac failure. AVMs pose significant diagnostic and therapeutic challenges due to their heterogeneous nature and high recurrence rates. Accurate diagnosis relies on advanced imaging modalities, including Doppler ultrasonography, magnetic resonance imaging and angiography, and digital subtraction angiography, which provide critical insights into lesion extent and flow dynamics. Current management strategies involve a combination of preoperative embolization, surgical excision, and endovascular therapies. However, incomplete treatment frequently results in recurrence, emphasizing the need for complete nidus removal. Emerging antiangiogenic therapies, such as thalidomide and MEK inhibitors, show promise as adjuncts to invasive treatments, potentially reducing recurrence rates and enhancing long-term outcomes. This review highlights the need for standardized treatment protocols, integrating clinical, anatomical, and genetic insights. Syndromic AVMs require broader diagnostic and therapeutic considerations, while advances in molecular genetics pave the way for targeted pharmacologic therapies. Future research should focus on refining combination therapies and optimizing individualized care through multidisciplinary approaches. While challenges remain, these developments represent significant steps toward improving outcomes for patients with this complex and debilitating vascular anomaly.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 3","pages":"Article 104296"},"PeriodicalIF":3.2,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144275801","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Editorial QMR vascular malformations 血管畸形。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-09-01 Epub Date: 2025-05-28 DOI: 10.1016/j.lpm.2025.104290

Anne Dompmartin

引用次数: 0

Capillary malformations updates on aetiopathogenesis, diagnosis, and management 毛细血管畸形的病因、诊断和治疗的最新进展。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-09-01 Epub Date: 2025-05-29 DOI: 10.1016/j.lpm.2025.104292

Eulalia Baselga, Luisa F. Montenegro Morillo

引用次数: 0

French evaluation of innovative health technologies: Early access and fundings 法国对创新卫生技术的评价：尽早获得和供资

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-06-01 Epub Date: 2025-04-08 DOI: 10.1016/j.lpm.2025.104284

Capucine Serain , Sylvie Chevret , Yann Chambon , Vanessa Hernando , Françoise Lucet , Samuel Seksik , Hassan Serrier , Elodie Velzenberger Maquart , Pierre Cochat

{"title":"French evaluation of innovative health technologies: Early access and fundings","authors":"Capucine Serain , Sylvie Chevret , Yann Chambon , Vanessa Hernando , Françoise Lucet , Samuel Seksik , Hassan Serrier , Elodie Velzenberger Maquart , Pierre Cochat","doi":"10.1016/j.lpm.2025.104284","DOIUrl":"10.1016/j.lpm.2025.104284","url":null,"abstract":"<div><h3>Background</h3><div>To accelerate the availability of innovative healthcare technologies for patients with the potential for significant clinical benefits in a context of high unmet needs, derogatory programs have been elaborated. The aim of this paper is to describe the different pathways developed in France to accelerate access to innovation, efficiently handle uncertainties while controlling the risks for patients.</div></div><div><h3>Methods</h3><div>We first describe the different early and temporary accesses to innovation in France involving the HAS. Feedback on these pathways based on the decisions provided by the HAS up until June 2024, is summarised and discussed. Subsequent emphasis is placed on the challenges of the evaluation process.</div></div><div><h3>Findings</h3><div>French derogatory pathways for innovation distinguish between medicinal products, medical devices (MDs) and procedures, as well as the funding mechanism. Early funding is dedicated to MDs, in vitro diagnostic MDs and procedures. Later fast-track access is dedicated to medicinal products but also to (digital) MDs. Based on the submitted files from 2015 to 2021, the derogatory access was approved about 70% for medicinal products and 30% for MDs/procedures.</div></div><div><h3>Conclusions and Relevance</h3><div>While fast-track processes appear widely used and understood for medicinal products, the different pathways available for MDs and procedures remain under-used and sometimes misunderstood. Whichever the product, the main limitation factor of approval was data quality and maturity, in concordance with reports on accelerated approvals from the FDA. The main challenge is to find the right balance between rapid access to innovation and patient safety, while addressing ethical challenges posed by new therapeutic approaches.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 2","pages":"Article 104284"},"PeriodicalIF":3.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924138","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Taking ethics into account: the HAS perspective 考虑伦理：HAS视角

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-06-01 Epub Date: 2025-04-07 DOI: 10.1016/j.lpm.2025.104283

Maxence Lyonnet , Robert Gelli , Hubert Galmiche

{"title":"Taking ethics into account: the HAS perspective","authors":"Maxence Lyonnet , Robert Gelli , Hubert Galmiche","doi":"10.1016/j.lpm.2025.104283","DOIUrl":"10.1016/j.lpm.2025.104283","url":null,"abstract":"<div><div>The HAS is unique in France due to its status as an independent public authority. Its closest equivalent is the National Institute for Health and Care Excellence (NICE) in England and Wales. It stands out from other French health agencies by its independence and wide scope, covering health and the social and medico-social sectors. Its independence, guaranteed by law, is combined with regular dialogue with public authorities and external partners. Independence does not come without accountability; it must report its actions to the state and its control bodies (Parliament ant Justice in particular) and more broadly to society and health actors. Scientific rigor, independence, and transparency are at the heart of its values. The 2011 health law, adopted following the French Mediator scandal, established a common legal framework for preventing conflicts of interest in health. The has also developed its own complementary prevention tools. It ensures that the composition of its expert committees balances different schools of thought. In terms of transparency, the HAS publishes all its opinions and decisions, as well as methodological and practical guides. In a demanding context where scientific expertise is particularly scrutinized and the risk of a health crisis, such as Covid-19, is imminent, the HAS must maintain the highest standards of conflict of interest prevention and transparency. This is essential for the quality of expertise and public trust.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 2","pages":"Article 104283"},"PeriodicalIF":3.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143924137","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Health technology assessment in Europe: A comparison of organizations and introduction to the European regulation 欧洲卫生技术评估：组织比较和欧洲法规介绍

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-06-01 Epub Date: 2025-04-07 DOI: 10.1016/j.lpm.2025.104282

Judith Fernandez, Paul de Boissieu, Margaret Galbraith

{"title":"Health technology assessment in Europe: A comparison of organizations and introduction to the European regulation","authors":"Judith Fernandez, Paul de Boissieu, Margaret Galbraith","doi":"10.1016/j.lpm.2025.104282","DOIUrl":"10.1016/j.lpm.2025.104282","url":null,"abstract":"<div><div>Health Technology Assessment (HTA) is a multidisciplinary process that evaluates the medical, social, economic, legal, and ethical aspects of health technologies to determine their value throughout their lifecycle. With rising healthcare costs in Europe, robust HTA processes are crucial for making informed decisions that promote an equitable and efficient health system. HTA practices date back to 1967 in France and have expanded across Europe, with most countries adopting HTA models to guide pricing and reimbursement decisions. An analysis of European Health Technology Assessment bodies (HTAb) was conducted through an online survey to showcase the diversity of HTA systems while highlighting their shared goals. The survey, sent to 33 HTAb from July 8 to August 25, 2024, included 11 multiple-choice questions about their organization and processes, allowing for optional free text responses. Data collected were self-reported and analysed using descriptive statistics, with minimal verification of responses. Despite some differences in remit and scope, European HTAb remain steadfast in their resolve to collaborate. The European Commission and EU member states have fostered collaboration among HTA bodies through initiatives like EUnetHTA, culminating in the adoption of the HTA Regulation (EU) 2021/2282, which will be implemented starting January 12, 2025 with the production on joint clinical assessment and joint scientific consultation for some medicinal products and high-risk medical devices. The HTAR offers numerous opportunities for collaboration. Joint productions will foster a culture of mutual learning, allowing countries to benefit from shared expertise and data while ensuring the rigorous and transparent assessment of new health technologies. Moreover, a more unified approach to HTA could accelerate the adoption of new and effective technologies at the continental level, ultimately improving patient outcomes across Europe.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 2","pages":"Article 104282"},"PeriodicalIF":3.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143942793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The French way of HTA: Between scientific rigor, independence and transparency 法国的HTA方式：在科学严谨、独立和透明之间。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-06-01 Epub Date: 2025-04-05 DOI: 10.1016/j.lpm.2025.104281

Joachim Baba , Nathan Guerrier , Irena Guzina , Vanessa Hernando , Vassilia Ressencourt , Ivana Spasojevic , Dominic Thorrington , Charlotte Masia

{"title":"The French way of HTA: Between scientific rigor, independence and transparency","authors":"Joachim Baba , Nathan Guerrier , Irena Guzina , Vanessa Hernando , Vassilia Ressencourt , Ivana Spasojevic , Dominic Thorrington , Charlotte Masia","doi":"10.1016/j.lpm.2025.104281","DOIUrl":"10.1016/j.lpm.2025.104281","url":null,"abstract":"<div><h3>Context</h3><div>The Haute Autorité de Santé (HAS) is an independent body responsible for advising the government on the appropriateness of national funding for healthcare technologies and interventions. To fulfil this role, HAS evaluates their efficacy and safety, especially in comparison to existing alternatives.</div></div><div><h3>Methods</h3><div>We first described how the HAS is organised to carry out this mission, then outlined the different stages of the evaluation process, highlighting specific features according to the type of health technology being assessed We also reviewed the HAS's activities for 2023.</div></div><div><h3>Results</h3><div>The HAS relies on international methodological standards to assess health technologies and interventions, on the expertise of healthcare professionals and patient experiences, and input from various stakeholders where appropriate and necessary.</div></div><div><h3>Conclusion</h3><div>Through this rigorous, independent, and transparent scientific evaluation process, the HAS aims to ensure that patients have access to the best available treatments and care, and, when relevant, to the most efficient options for the French healthcare system, while guaranteeing patient safety.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 2","pages":"Article 104281"},"PeriodicalIF":3.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143804102","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0