Presse Medicale最新文献_第5页

Health technology assessment in France 法国的卫生技术评估

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-06-01 Epub Date: 2025-04-08 DOI: 10.1016/j.lpm.2025.104278

Lionel Collet , Pierre Cochat

引用次数: 0

French HTA and appropriateness of care: Beyond reimbursement 法国的 HTA 和护理的适当性：超越报销。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-06-01 Epub Date: 2025-04-05 DOI: 10.1016/j.lpm.2025.104280

Sophie Blanchard , Valérie Lindecker-Cournil , Bertrand Mussetta , Fréderic Nahmias , Cédric Carbonneil

{"title":"French HTA and appropriateness of care: Beyond reimbursement","authors":"Sophie Blanchard , Valérie Lindecker-Cournil , Bertrand Mussetta , Fréderic Nahmias , Cédric Carbonneil","doi":"10.1016/j.lpm.2025.104280","DOIUrl":"10.1016/j.lpm.2025.104280","url":null,"abstract":"<div><div>Heath Technology Assessment (HTA) is mainly used for reimbursement/disinvestment and access to innovation purposes. In addition, HTA is a useful tool to promote appropriateness of care, as it describes the updated level of validation of assessed health technologies. Thus, several tools (proper use sheets, drug-indexed decision support systems, supervision of practices or use) based on HTA are developed to promote the appropriateness of use of drugs, medical devices or diagnostic and therapeutic procedures among healthcare professionals.</div><div>HTA can also support the development and the update of clinical practice guidelines (CPG) which are the main tool of clinical practice improvement. In addition, HTA can increase CPG implementation through reimbursement of recommended health technologies. Therefore, HTA and CPGs can be perfectly articulated to enable synergistic synchronic or asynchronic action to improve the relevance of care by enabling more dynamic and efficient actions, particularly in terms of robustness and updating.</div><div>However, HTA shares with CPGs the same difficulties of implementation in the field. To face them, whatever the implementation strategy defined, it requires a multifaceted approach involving iterative communication and the involvement of healthcare professionals, patients and stakeholders, both upstream and downstream of the HTA stages.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 2","pages":"Article 104280"},"PeriodicalIF":3.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143804101","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

French reimbursement of health technologies: assessment is at the heart of the procedure 法国对保健技术的报销：评估是这一程序的核心

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-06-01 Epub Date: 2025-04-09 DOI: 10.1016/j.lpm.2025.104279

Cédric Carbonneil , Corinne Collignon , Hubert Galmiche , Andrea Lasserre , Charlotte Masia , Floriane Pelon

{"title":"French reimbursement of health technologies: assessment is at the heart of the procedure","authors":"Cédric Carbonneil , Corinne Collignon , Hubert Galmiche , Andrea Lasserre , Charlotte Masia , Floriane Pelon","doi":"10.1016/j.lpm.2025.104279","DOIUrl":"10.1016/j.lpm.2025.104279","url":null,"abstract":"<div><h3>Background</h3><div>In France, health technologies can be reimbursed by the national health insurance system. However, not all are eligible, and several steps are required. This article aims at deciphering the various procedures involved.</div></div><div><h3>Methods</h3><div>We first described the most common steps for reimbursement procedures, from authorisation to health technology assessment, including clinical evaluation and reimbursement decision. We then discussed certain specificities related to the nature of the health technologies (for example, vaccines among drugs, or digital medical devices (DMDs) among medical devices), and to the status of the procedure (derogatory vs. standard).</div></div><div><h3>Findings</h3><div>France benefits from several procedures aimed at reimbursing health products, and their number is increasing over time. They all share one common step, assessment, which is systematically performed from a clinical perspective and sometimes includes an organisational or an economic viewpoint, by a single actor: the French National Authority for Health (Haute Autorité de santé, HAS).</div></div><div><h3>Conclusion and relevance</h3><div>HAS evaluations are essential tools for informing healthcare decision-making, and ensuring that patients have access to safe, effective, and affordable treatments. The system will have to face new challenges in the coming years: the European regulation will pool certain procedural steps, there will be more self-directed DMDs on the market and new organizations set up to support innovation as well as scarce human and financial resources. Ultimately, it is all about constantly adapting to meet patients' needs, introducing new health technologies that offer added value and limited risks, while making the best possible use of available resources.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 2","pages":"Article 104279"},"PeriodicalIF":3.2,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143916741","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

Primary systemic amyloidosis: A brief overview 原发性系统性淀粉样变性：简要概述。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-03-01 Epub Date: 2024-12-11 DOI: 10.1016/j.lpm.2024.104267

Michael Sang Hughes, Suzanne Lentzsch

引用次数: 0

Imaging in multiple myeloma 多发性骨髓瘤的影像学表现。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-03-01 Epub Date: 2024-12-09 DOI: 10.1016/j.lpm.2024.104263

Jas Virk , Jens Hillengass

{"title":"Imaging in multiple myeloma","authors":"Jas Virk , Jens Hillengass","doi":"10.1016/j.lpm.2024.104263","DOIUrl":"10.1016/j.lpm.2024.104263","url":null,"abstract":"<div><div>Multiple myeloma (MM) is the second most common adult hematologic malignancy, characterized by clonal proliferation of malignant plasma cells mostly in the bone marrow. The presence of destructive changes of the mineralized bone is a hallmark feature of the condition and a sign of end-organ damage. Due to this, imaging plays an integral role in the diagnosis, prognostication, and treatment monitoring of patients undergoing therapy for MM as well as surveillance of patients with early-stage disease. While conventional radiography has traditionally been the mainstay of initial evaluation of patients suspected of having MM, the advent of more sensitive imaging techniques such as computed tomography (CT), magnetic resonance imaging (MRI), and positron emission tomography (PET) have taken its place in assessing patients. While either CT alone or as part of a PET/CT examination is the initial radiographic method of choice, MRI remains the gold-standard modality in assessing bone marrow involvement, especially in early disease stages. PET/CT also provides valuable information regarding assessment of response to therapy and extramedullary manifestations of the disease. There is however increasing evidence that functional MRI techniques, albeit limitedly available, might be superior to PET/CT for treatment monitoring. This review summarizes current knowledge on the use of different imaging techniques in monoclonal plasma cell disorders and discusses future developments in this area of research.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 1","pages":"Article 104263"},"PeriodicalIF":3.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142814015","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

Kidney disease in multiple myeloma 多发性骨髓瘤肾病。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-03-01 Epub Date: 2024-12-09 DOI: 10.1016/j.lpm.2024.104264

Frank Bridoux , Nelson Leung , Samih H. Nasr , Arnaud Jaccard , Virginie Royal , International Kidney and Monoclonal Gammopathy Research Group

{"title":"Kidney disease in multiple myeloma","authors":"Frank Bridoux , Nelson Leung , Samih H. Nasr , Arnaud Jaccard , Virginie Royal , International Kidney and Monoclonal Gammopathy Research Group","doi":"10.1016/j.lpm.2024.104264","DOIUrl":"10.1016/j.lpm.2024.104264","url":null,"abstract":"<div><div>Renal disease is a frequent complication of symptomatic multiple myeloma, that increases morbidity and reduces quality of life and overall survival. It may result from various lesions, the most frequent being light chain cast nephropathy (LCCN), related to precipitation of monoclonal free light chains (FLC) with uromodulin in distal tubules. Rapid identification of the type of kidney disease with appropriate management is key. LCCN typically reveals the underlying myeloma and manifests with severe acute kidney injury, high serum FLC level (>500 mg/l) and predominant light chain proteinuria (urine albumin/creatinine ratio <10 %). Urgent therapy is required, based on vigorous fluid expansion, correction of precipitating factors and introduction of efficient anti-myeloma therapy which choice should consider renal elimination of each agent and patient frailty. Early and deep reduction in serum FLC level conditions renal recovery, warranting assessment of efficacy by serial serum FLC level monitoring. In newly diagnosed patients, the combination of bortezomib, high-dose dexamethasone and an anti-CD38 monoclonal antibody is commonly used. The benefit to risk balance of quadruplets incorporating cyclophosphamide or an immunodulatory agent requires to be evaluated in prospective studies. In patients with severe acute kidney injury, reinforcing chemotherapy with FLC removal through plasma exchange or high-cutoff hemodialysis may increase the probability of renal response, despite controversial data from randomized trials. Histological assessment of the extent of cast formation and interstitial fibrosis/tubular atrophy may help evaluating renal prognosis and refining therapy. Thanks to improved overall survival, renal transplantation may be considered in selected candidates with end-stage kidney disease.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 1","pages":"Article 104264"},"PeriodicalIF":3.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142813998","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

Patient-reported outcome measures: A key to patient-tailored and outcome-driven care in pulmonary embolism survivors 患者报告的结果测量：肺栓塞幸存者患者定制和结果驱动护理的关键。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-03-01 Epub Date: 2025-01-22 DOI: 10.1016/j.lpm.2025.104271

Sophie N.M. ter Haar , Cindy M.M. de Jong , Francis Couturaud , Thijs E. van Mens , Frederikus A. Klok

引用次数: 0

Minimal residual disease in multiple myeloma 多发性骨髓瘤的微小残留病。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-03-01 Epub Date: 2024-12-09 DOI: 10.1016/j.lpm.2024.104261

Nour Moukalled , Florent Malard , Ali Bazarbachi , Mohamad Mohty

{"title":"Minimal residual disease in multiple myeloma","authors":"Nour Moukalled , Florent Malard , Ali Bazarbachi , Mohamad Mohty","doi":"10.1016/j.lpm.2024.104261","DOIUrl":"10.1016/j.lpm.2024.104261","url":null,"abstract":"<div><div>Minimal Residual Disease (MRD) in multiple myeloma has emerged as a significant prognostic factor, guiding treatment strategies and enhancing patient outcomes. Despite advancements in therapies such as proteasome inhibitors, immunomodulatory drugs, monoclonal antibodies, CAR-T cell therapy, and bispecific antibodies, complete eradication of malignant plasma cells remains challenging. MRD refers to a small number of residual cancer cells that persist after treatment and require sensitive methods like next-generation flow cytometry (NGF) and next-generation sequencing (NGS) for detection. MRD negativity has been associated with improved progression-free survival (PFS) and overall survival (OS), making it a key marker in clinical trials.</div><div>The clinical utility of MRD lies in its ability to predict outcomes, with sustained MRD negativity linked to prolonged survival. Furthermore, it will likely help in tailoring treatment approaches, such as therapy escalation for high-risk patients or de-escalation for those achieving MRD negativity. Despite its prognostic value, challenges remain in standardizing MRD testing, ensuring its widespread availability, and addressing variability in results based on different detection methods. Future research aims to refine MRD-guided treatment and explore novel detection techniques, such as liquid biopsies, to improve patient monitoring in multiple myeloma.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 1","pages":"Article 104261"},"PeriodicalIF":3.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142814001","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

Updates on mechanisms of disease progression in precursor myeloma: Monoclonal gammopathy of undermined significance and smoldering myeloma 前体骨髓瘤疾病进展机制的最新进展：单克隆γ病和阴燃骨髓瘤。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-03-01 Epub Date: 2025-01-18 DOI: 10.1016/j.lpm.2025.104268

Cynthia Saade , Irene M. Ghobrial

{"title":"Updates on mechanisms of disease progression in precursor myeloma: Monoclonal gammopathy of undermined significance and smoldering myeloma","authors":"Cynthia Saade , Irene M. Ghobrial","doi":"10.1016/j.lpm.2025.104268","DOIUrl":"10.1016/j.lpm.2025.104268","url":null,"abstract":"<div><div>Monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM) are premalignant stages in the development of multiple myeloma (MM). Advances in detection, risk stratification, and therapeutic intervention have transformed our understanding of disease progression. Sensitive techniques like mass spectrometry have identified smaller monoclonal gammopathies, such as monoclonal gammopathy of indeterminate potential (MGIP), which may precede MGUS. Risk stratification models for MGUS and SMM, including the Mayo Clinic, PETHEMA, 2/20/20, IMWG, and PANGEA models, leverage tumor burden markers and cytogenetic abnormalities to predict prognosis. Genomic studies have revealed mutations, structural changes, and mutational signatures that predict progression. Immune microenvironmental alterations underscore the multifactorial nature of disease evolution, while epigenetics is emerging as a source of tumoral and microenvironmental changes. Therapies for high-risk SMM, including lenalidomide, daratumumab, and next-generation immunotherapies, demonstrate efficacy in delaying progression to MM but raise concerns regarding safety in asymptomatic patients. Future research must refine prognostic models, integrate genomic and immunophenotypic data, and establish consensus on optimal strategies for early intervention. This comprehensive review highlights the biological, clinical, and therapeutic advancements in MM and its precursors, emphasizing the importance of early risk assessment and targeted treatment to improve outcomes.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 1","pages":"Article 104268"},"PeriodicalIF":3.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143010474","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

Evolving strategies in the management of transplant-eligible patients with newly diagnosed multiple myeloma 新诊断的符合移植条件的多发性骨髓瘤患者的管理策略的演变。

IF 3.2 3区医学

Presse Medicale Pub Date : 2025-03-01 Epub Date: 2024-12-09 DOI: 10.1016/j.lpm.2024.104262

Aurore Perrot

{"title":"Evolving strategies in the management of transplant-eligible patients with newly diagnosed multiple myeloma","authors":"Aurore Perrot","doi":"10.1016/j.lpm.2024.104262","DOIUrl":"10.1016/j.lpm.2024.104262","url":null,"abstract":"<div><div>Multiple myeloma treatment has evolved significantly with the introduction of triplet and quadruplet regimens, notably incorporating anti-CD38 antibodies. While autologous stem cell transplantation remains a cornerstone of therapy, its role in the context of increasingly effective upfront treatments is debated. Current guidelines still recommend transplant for all eligible patients, especially those with high-risk features at diagnosis, despite concerns regarding the lack of overall survival benefits and the potential long-term toxicities associated with high-dose melphalan. Delaying transplantation until first relapse has been proposed, but this approach carries the risk of patients becoming ineligible for transplantation due to worsening health or disease progression. Consolidation therapy after transplant is not strongly endorsed in recent guidelines, and studies show mixed results regarding its efficacy. Some data suggests a progression-free survival advantage with post-ASCT consolidation; others found no significant differences in outcomes among various strategies. Nonetheless, tandem transplant may be beneficial for high-risk patients. Maintenance therapy, particularly with lenalidomide, has proven effective, offering substantial progression-free and overall survival benefits. While lenalidomide remains the standard, emerging data indicate that combinations with proteasome inhibitors or anti-CD38 antibodies could enhance outcomes, particularly in high-risk populations. As our understanding of myeloma biology deepens, tailoring treatment approaches based on risk profiles and response depth will be crucial for optimizing patient outcomes.</div></div>","PeriodicalId":20530,"journal":{"name":"Presse Medicale","volume":"54 1","pages":"Article 104262"},"PeriodicalIF":3.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142814012","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0