The 7ᵗʰ Joint Symposium between the European Association for the Study of Diabetes (EASD) and the Asian Association for the Study of Diabetes (AASD) took place on the 2ⁿᵈ–6ᵗʰ October in Hamburg, Germany. In an engaging session examining the impact of COVID-19 on new healthcare models, experts provided insights into whole-system approaches for diabetes, and opportunities for improvement in patient outcomes via telemedicine.
{"title":"Can COVID-19 Help Us Deliver Whole-System Holistic Healthcare?","authors":"Ada Enesco","doi":"10.33590//emj/10304838","DOIUrl":"https://doi.org/10.33590//emj/10304838","url":null,"abstract":"The 7ᵗʰ Joint Symposium between the European Association for the Study of Diabetes (EASD) and the Asian Association for the Study of Diabetes (AASD) took place on the 2ⁿᵈ–6ᵗʰ October in Hamburg, Germany. In an engaging session examining the impact of COVID-19 on new healthcare models, experts provided insights into whole-system approaches for diabetes, and opportunities for improvement in patient outcomes via telemedicine.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139179496","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Methotrexate is a common first-line treatment for rheumatoid arthritis, yet its widespread and habitual usage often leads physicians to overlook the choice of administration route when planning management strategies. A recent survey involving 30 consultant rheumatologists from France, Germany, Italy, Poland, Spain, and the UK, identified variation in the utilisation and perceptions regarding oral versus subcutaneous delivery for methotrexate. In November 2023, EMJ interviewed Roberto Caporali, Professor of Rheumatology at the University of Milan, and Head of the Department of Rheumatology and Medical Sciences at Gaetano Pini Hospital, Milan, Italy. Caporali’s expertise is in clinical practice, teaching, and research in rheumatology, mainly rheumatoid arthritis and other immune-mediated inflammatory diseases, with a focus on prognostic factors, biomarkers, and treatment for patients with moderate-to-severe active rheumatoid arthritis. During this interview, Caporali discussed the decision-making process for treating rheumatoid arthritis, with a particular focus on the use of methotrexate. The purpose was to gain insights from a rheumatology expert regarding the prevalence and management goals of the disease, and available treatment options. The interview considered key decision-making drivers and barriers to healthcare professionals when selecting the route of administration. Caporali suggested that the efficacy and safety profile of methotrexate when delivered subcutaneously may be the optimal choice for patients, often resulting in higher adherence compared to oral dosing. Caporali recommended education and re-evaluation of local guidelines to improve patient outcomes by better understanding the optimal use and efficacy of methotrexate.
{"title":"Exploring Methotrexate Route of Administration Decisions for Adults with Rheumatoid Arthritis","authors":"Hannah Moir","doi":"10.33590/emj/10308161","DOIUrl":"https://doi.org/10.33590/emj/10308161","url":null,"abstract":"Methotrexate is a common first-line treatment for rheumatoid arthritis, yet its widespread and habitual usage often leads physicians to overlook the choice of administration route when planning management strategies. A recent survey involving 30 consultant rheumatologists from France, Germany, Italy, Poland, Spain, and the UK, identified variation in the utilisation and perceptions regarding oral versus subcutaneous delivery for methotrexate. In November 2023, EMJ interviewed Roberto Caporali, Professor of Rheumatology at the University of Milan, and Head of the Department of Rheumatology and Medical Sciences at Gaetano Pini Hospital, Milan, Italy. Caporali’s expertise is in clinical practice, teaching, and research in rheumatology, mainly rheumatoid arthritis and other immune-mediated inflammatory diseases, with a focus on prognostic factors, biomarkers, and treatment for patients with moderate-to-severe active rheumatoid arthritis. During this interview, Caporali discussed the decision-making process for treating rheumatoid arthritis, with a particular focus on the use of methotrexate. The purpose was to gain insights from a rheumatology expert regarding the prevalence and management goals of the disease, and available treatment options. The interview considered key decision-making drivers and barriers to healthcare professionals when selecting the route of administration. Caporali suggested that the efficacy and safety profile of methotrexate when delivered subcutaneously may be the optimal choice for patients, often resulting in higher adherence compared to oral dosing. Caporali recommended education and re-evaluation of local guidelines to improve patient outcomes by better understanding the optimal use and efficacy of methotrexate.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139180196","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Objectives: This study assessed the effect of liraglutide as a monotherapy and add-on to metformin on weight loss and BMI, among patients with Type 2 diabetes (T2D) who are overweight or obese. Methods: The following databases were assessed to identify relevant papers published until July 2023: Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE (PubMed), clinicaltrial.gov, and Web of Science. All clinical trials evaluating the effect of liraglutide on weight loss and BMI in patients with T2D who are obese or overweight, treated for at least 2 months, were included in the review. All analysis and risk of bias assessment was done using Cochrane Review Manager software, version 5.4.1 (Cochrane, London, UK). A random-effects model with inverse variance was used to synthesise the results. Results: In total, 10 randomised controlled trials involving 945 participants were included in the meta-analysis. Treatment with liraglutide with or without metformin for more than 2 months led to a significant weight loss (mean difference: -4.75 kg; 95% confidence interval: -7.02–-2.48; p<0.01). Liraglutide supplementation also led to a significant decrease in BMI (mean difference: -2.07; 95% confidence interval: -2.75–-1.39; p<0.01). However, the decrease in weight and BMI was not statistically significant as compared to treatment with other oral hypoglycaemic drugs or placebo. Conclusion: Liraglutide used alone or as adjunctive therapy to metformin produces reduction in weight and BMI when administered in adult patients with T2D who are obese or overweight.
{"title":"Effect of Liraglutide on Weight Loss and BMI Among Patients Who Are Overweight and Obese with Type 2 Diabetes: A Systematic Review and Meta-analysis","authors":"C. Zorampari, Rachna Gupta, Lalit K. Gupta","doi":"10.33590/emj/10306493","DOIUrl":"https://doi.org/10.33590/emj/10306493","url":null,"abstract":"Objectives: This study assessed the effect of liraglutide as a monotherapy and add-on to metformin on weight loss and BMI, among patients with Type 2 diabetes (T2D) who are overweight or obese. Methods: The following databases were assessed to identify relevant papers published until July 2023: Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE (PubMed), clinicaltrial.gov, and Web of Science. All clinical trials evaluating the effect of liraglutide on weight loss and BMI in patients with T2D who are obese or overweight, treated for at least 2 months, were included in the review. All analysis and risk of bias assessment was done using Cochrane Review Manager software, version 5.4.1 (Cochrane, London, UK). A random-effects model with inverse variance was used to synthesise the results. Results: In total, 10 randomised controlled trials involving 945 participants were included in the meta-analysis. Treatment with liraglutide with or without metformin for more than 2 months led to a significant weight loss (mean difference: -4.75 kg; 95% confidence interval: -7.02–-2.48; p<0.01). Liraglutide supplementation also led to a significant decrease in BMI (mean difference: -2.07; 95% confidence interval: -2.75–-1.39; p<0.01). However, the decrease in weight and BMI was not statistically significant as compared to treatment with other oral hypoglycaemic drugs or placebo. Conclusion: Liraglutide used alone or as adjunctive therapy to metformin produces reduction in weight and BMI when administered in adult patients with T2D who are obese or overweight.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139179465","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
A. A. Raghavan, Ian W Gibson, Robert Wightman, Piotr Czaykowski, Jeffrey Graham
Renal cell carcinoma (RCC) is the most common primary tumour of the kidney. RCC is a clinically and pathologically heterogenous entity, which has traditionally been classified under two broad categories: clear-cell and non-clear cell. With improved molecular diagnostic methodologies and genetic testing, the classification of RCC has shifted from a morphological basis to a molecular/genetic focus, and has been systematically updated to reflect these advancements. The new 2022 World Health Organization (WHO) classification of RCC is the most recent of these updates, and contains significant changes, as compared to the previous 2016 classification. The most substantial of these changes is the establishment of a new category of molecularly-defined RCC, including TFE3-rearranged RCC, TFEB-altered RCC, ELOC-mutated RCC, fumarate hydratase-deficient RCC, succinate dehydrogenase-deficient RCC, ALK-rearranged RCC, and SMARCB1-deficient renal medullary carcinoma. In this narrative review, the authors briefly summarise the histopathological characteristics, clinical course, current treatment standards, and future treatment directions of each of these molecularly-defined RCC subtypes.
{"title":"Redefining Renal Cell Carcinoma: A Molecular Perspective on Classification and Clinical Implications","authors":"A. A. Raghavan, Ian W Gibson, Robert Wightman, Piotr Czaykowski, Jeffrey Graham","doi":"10.33590/emj/10301071","DOIUrl":"https://doi.org/10.33590/emj/10301071","url":null,"abstract":"Renal cell carcinoma (RCC) is the most common primary tumour of the kidney. RCC is a clinically and pathologically heterogenous entity, which has traditionally been classified under two broad categories: clear-cell and non-clear cell. With improved molecular diagnostic methodologies and genetic testing, the classification of RCC has shifted from a morphological basis to a molecular/genetic focus, and has been systematically updated to reflect these advancements. The new 2022 World Health Organization (WHO) classification of RCC is the most recent of these updates, and contains significant changes, as compared to the previous 2016 classification. The most substantial of these changes is the establishment of a new category of molecularly-defined RCC, including TFE3-rearranged RCC, TFEB-altered RCC, ELOC-mutated RCC, fumarate hydratase-deficient RCC, succinate dehydrogenase-deficient RCC, ALK-rearranged RCC, and SMARCB1-deficient renal medullary carcinoma. In this narrative review, the authors briefly summarise the histopathological characteristics, clinical course, current treatment standards, and future treatment directions of each of these molecularly-defined RCC subtypes.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139180422","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
This symposium was held as part of the 28th International Annual Congress of the World Muscle Society (WMS), held in Charleston, South Carolina, USA. Speakers reviewed the natural history of Becker muscular dystrophy (Becker), outlined the top line, 12-month data from the ARCH open label study of investigational agent EDG-5506, and aimed to put those results into clinical context. Becker is a serious rare disease with significant physical, emotional, financial, and social impact on the affected individuals and their caregivers. Once function begins to decline, males affected by the progressive X-linked genetic disorder continue to irreversibly lose muscle, ultimately leading to the loss of ambulatory and cardiopulmonary function. After discussing the aetiology of the condition, Erik Niks, Paediatric and Adult Neurologist, Leiden University Medical Center (LUMC), the Netherlands, presented the findings of natural history studies. They showed that while the age at which decline begins varies, once it does start, patients tend to experience a consistent decline in function equivalent to around 1.2–1.3 North Star Ambulatory Assessment (NSAA) points each year. This finding, combined with data on using MRI as a biomarker of disease progression, provides an evidence-based framework for clinical trial design, he argued. Sam Collins, Vice President of clinical development, Edgewise Therapeutics, Boulder, Colorado, USA, then presented topline 12-month data from the ARCH study. It found that EDG-5506 was well tolerated, and, importantly, recorded the stabilisation of functional assessments, including the NSAA, with a trend towards improvement, as well as rapid, sustained, and significant decreases in biomarkers of progression, including those related to muscle damage. Putting the ARCH study data into context, Barry Byrne, Director of the Health Center for Advanced Therapeutics and Powell Gene Therapy Center, University of Florida (UF), Gainesville, USA, explained exactly how declining NSAA status translated into life-altering function loss. Stabilising function, or even reducing the speed of decline, was an important goal for patients, he said, adding that meeting it could help to address significant unmet medical need.
{"title":"Becker Muscular Dystrophy: Could Altering the Natural History of Decline Help Tackle Unmet Medical Need?","authors":"Amanda Barrell","doi":"10.33590/emj/10300460","DOIUrl":"https://doi.org/10.33590/emj/10300460","url":null,"abstract":"This symposium was held as part of the 28th International Annual Congress of the World Muscle Society (WMS), held in Charleston, South Carolina, USA. Speakers reviewed the natural history of Becker muscular dystrophy (Becker), outlined the top line, 12-month data from the ARCH open label study of investigational agent EDG-5506, and aimed to put those results into clinical context. Becker is a serious rare disease with significant physical, emotional, financial, and social impact on the affected individuals and their caregivers. Once function begins to decline, males affected by the progressive X-linked genetic disorder continue to irreversibly lose muscle, ultimately leading to the loss of ambulatory and cardiopulmonary function. After discussing the aetiology of the condition, Erik Niks, Paediatric and Adult Neurologist, Leiden University Medical Center (LUMC), the Netherlands, presented the findings of natural history studies. They showed that while the age at which decline begins varies, once it does start, patients tend to experience a consistent decline in function equivalent to around 1.2–1.3 North Star Ambulatory Assessment (NSAA) points each year. This finding, combined with data on using MRI as a biomarker of disease progression, provides an evidence-based framework for clinical trial design, he argued. Sam Collins, Vice President of clinical development, Edgewise Therapeutics, Boulder, Colorado, USA, then presented topline 12-month data from the ARCH study. It found that EDG-5506 was well tolerated, and, importantly, recorded the stabilisation of functional assessments, including the NSAA, with a trend towards improvement, as well as rapid, sustained, and significant decreases in biomarkers of progression, including those related to muscle damage. Putting the ARCH study data into context, Barry Byrne, Director of the Health Center for Advanced Therapeutics and Powell Gene Therapy Center, University of Florida (UF), Gainesville, USA, explained exactly how declining NSAA status translated into life-altering function loss. Stabilising function, or even reducing the speed of decline, was an important goal for patients, he said, adding that meeting it could help to address significant unmet medical need.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139180480","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
While awareness of the issues facing women leading up to, during, and following the menopause has increased in recent years, it remains a condition of significant unmet need. Reasons include a stigma around asking for help, and a lack of awareness of the symptoms and associated risks among women and healthcare professionals alike. Standard medical training includes limited education on perimenopausal and post-reproductive healthcare, meaning clinicians are often ill-prepared to intervene. However, there is much that healthcare professionals, regardless of their specialty, can do to help women entering their post-reproductive years, say Rossella Nappi, Chief of the Research Centre for Reproductive Medicine and Director of the Gynecological Endocrinology and Menopause Unit at IRCCS San Matteo Foundation, University of Pavia, in Italy; and Katrin Schaudig, co-founder of the Centre for Gynaecological Endocrinology, Hormone Hamburg, Germany, and President of the German Menopause Society. Moreover, perimenopause represents an important opportunity to engage, inform, and empower women to take charge of their health for years to come. In this key opinion leader interview, Nappi and Schaudig explain the physiological changes associated with menopause, their clinical manifestations, and their associated risk factors. They talk about the need to tackle the stigma that is often attached to this phase of life, and why healthcare professionals have a duty to work with women to spot the signs of menopausal transition from an early stage. They argue that holistic care, which focuses as much on the prevention of chronic disease as it does on the treatment of menopausal symptoms, is key to ensuring women stay physically and mentally fit and healthy as they get older.
近年来，人们对妇女在更年期前、更年期中和更年期后所面临的问题的认识有所提高，但更年期仍然是一个需求严重得不到满足的问题。究其原因，包括求助是一种耻辱，以及妇女和医疗保健专业人员对症状和相关风险缺乏认识。标准医学培训中有关围绝经期和生育后保健的教育非常有限，这意味着临床医生往往没有做好干预的准备。意大利帕维亚大学圣马特奥基金会（IRCCS San Matteo Foundation）生殖医学研究中心主任兼妇科内分泌和更年期科主任罗塞拉-纳皮（Rossella Nappi）和德国汉堡荷尔蒙妇科内分泌中心创始人之一、德国更年期协会主席卡特琳-肖迪格（Katrin Schaudig）表示，无论哪个专业的医护人员，都可以做很多事情来帮助进入更年期的妇女。此外，围绝经期也是一个重要的机会，可以让妇女参与其中、获得信息并增强能力，从而在未来的岁月里掌握自己的健康。 在这次关键意见领袖访谈中，Nappi 和 Schaudig 解释了与更年期有关的生理变化、临床表现及其相关风险因素。他们谈到有必要消除人们对更年期的成见，以及为什么医疗保健专业人员有责任与妇女合作，从早期阶段就发现更年期转变的迹象。她们认为，整体护理既要关注慢性疾病的预防，也要关注更年期症状的治疗，这是确保妇女在步入老年后保持身心健康的关键。
{"title":"Perimenopause and Menopause: An Opportunity to Engage, Inform, and Empower Women to Live Well","authors":"Amanda Barrell","doi":"10.33590/emj/10306944","DOIUrl":"https://doi.org/10.33590/emj/10306944","url":null,"abstract":"While awareness of the issues facing women leading up to, during, and following the menopause has increased in recent years, it remains a condition of significant unmet need. Reasons include a stigma around asking for help, and a lack of awareness of the symptoms and associated risks among women and healthcare professionals alike. Standard medical training includes limited education on perimenopausal and post-reproductive healthcare, meaning clinicians are often ill-prepared to intervene. However, there is much that healthcare professionals, regardless of their specialty, can do to help women entering their post-reproductive years, say Rossella Nappi, Chief of the Research Centre for Reproductive Medicine and Director of the Gynecological Endocrinology and Menopause Unit at IRCCS San Matteo Foundation, University of Pavia, in Italy; and Katrin Schaudig, co-founder of the Centre for Gynaecological Endocrinology, Hormone Hamburg, Germany, and President of the German Menopause Society. Moreover, perimenopause represents an important opportunity to engage, inform, and empower women to take charge of their health for years to come. In this key opinion leader interview, Nappi and Schaudig explain the physiological changes associated with menopause, their clinical manifestations, and their associated risk factors. They talk about the need to tackle the stigma that is often attached to this phase of life, and why healthcare professionals have a duty to work with women to spot the signs of menopausal transition from an early stage. They argue that holistic care, which focuses as much on the prevention of chronic disease as it does on the treatment of menopausal symptoms, is key to ensuring women stay physically and mentally fit and healthy as they get older.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139180179","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
IgA nephropathy (IgAN) is a common form of glomerular disease, with wide heterogeneity of symptom occurrence and progression. Diagnosis is based on kidney biopsy findings. IgAN initiates in the mucosa with development of galactose-deficient IgA1 (Gd-IgA1) and anti-Gd-IgA1 autoantibodies, leading to deposition of these complexes in glomerular mesangium with resulting fibrosis, inflammation, tubulointerstitial scarring, and glomerular injury. This can lead to chronic kidney disease (CKD), kidney failure, and death. IgAN treatment involves optimised supportive care, including individualised strategies to address symptoms, such as high blood pressure control and cardiovascular risks. Drug treatment includes renin-angiotensin-aldosterone system (RAAS) inhibitors and immunosuppressant therapies. While the latter can successfully lower proteinuria, and have a positive effect on estimated glomerular filtration rate (eGFR), adverse effects can limit treatment duration, and increasing proteinuria and decreasing eGFR can return following treatment discontinuation. New formulations of immunosuppressant therapies include delayed-release budesonide with targeted release in the lower part of the small intestine where Gd-IgA1 production occurs. Although treatment with this drug can reduce proteinuria and sustain eGFR levels, similar to other immunosuppressant therapies, effects seem to be predominantly limited to the active treatment period. Targeting a different mechanism, sparsentan is a dual endothelin A receptor (ETA) and angiotensin II receptor type 1 (AT1) blocker that targets endothelin-1 (ET-1) and angiotensin II, both involved in IgAN progression. Initial Phase III trial results show significant differences, favouring sparsentan, compared with the AT1 blocker irbesartan, on proteinuria, with similar adverse event profiles. These agents, and several other drugs in development, will widen the armamentarium of therapies for people with IgAN, which, when used in combination, can target different aspects of IgAN pathogenesis for a more individualised treatment approach.
IgA 肾病（IgAN）是一种常见的肾小球疾病，其症状发生和发展具有广泛的异质性。诊断依据是肾活检结果。IgAN 起病于粘膜，会产生半乳糖缺乏性 IgA1（Gd-IgA1）和抗 Gd-IgA1 自身抗体，导致这些复合物沉积在肾小球系膜中，造成纤维化、炎症、肾小管间质瘢痕和肾小球损伤。这可能导致慢性肾病（CKD）、肾衰竭和死亡。IgAN 的治疗包括优化支持性护理，包括针对症状的个体化策略，如控制高血压和心血管风险。药物治疗包括肾素-血管紧张素-醛固酮系统（RAAS）抑制剂和免疫抑制剂疗法。虽然后者可以成功降低蛋白尿，并对估计肾小球滤过率（eGFR）产生积极影响，但不良反应会限制治疗持续时间，而且在停止治疗后，蛋白尿增加和 eGFR 下降的情况可能会再次出现。免疫抑制剂疗法的新配方包括在产生 Gd-IgA1 的小肠下部定向释放的缓释布地奈德。虽然与其他免疫抑制剂疗法类似，使用这种药物治疗可以减少蛋白尿并维持 eGFR 水平，但效果似乎主要局限于积极治疗期间。针对不同的机制，sparsentan 是一种双重内皮素 A 受体（ETA）和血管紧张素 II 受体 1 型（AT1）阻断剂，其作用靶点是内皮素-1（ET-1）和血管紧张素 II，两者都参与了 IgAN 的进展。初步的 III 期试验结果显示，与 AT1 受体阻滞剂厄贝沙坦相比，斯帕生坦在蛋白尿方面有显著差异，但不良反应情况相似。这些药物和其他几种在研药物将拓宽IgAN患者的治疗手段，联合使用时可针对IgAN发病机制的不同方面，采取更加个体化的治疗方法。
{"title":"New Horizons in IgA Nephropathy: A Focus on Current Treatment and Emerging Solutions","authors":"Eleanor Roberts","doi":"10.33590/emj/10303661","DOIUrl":"https://doi.org/10.33590/emj/10303661","url":null,"abstract":"IgA nephropathy (IgAN) is a common form of glomerular disease, with wide heterogeneity of symptom occurrence and progression. Diagnosis is based on kidney biopsy findings. IgAN initiates in the mucosa with development of galactose-deficient IgA1 (Gd-IgA1) and anti-Gd-IgA1 autoantibodies, leading to deposition of these complexes in glomerular mesangium with resulting fibrosis, inflammation, tubulointerstitial scarring, and glomerular injury. This can lead to chronic kidney disease (CKD), kidney failure, and death. IgAN treatment involves optimised supportive care, including individualised strategies to address symptoms, such as high blood pressure control and cardiovascular risks. Drug treatment includes renin-angiotensin-aldosterone system (RAAS) inhibitors and immunosuppressant therapies. While the latter can successfully lower proteinuria, and have a positive effect on estimated glomerular filtration rate (eGFR), adverse effects can limit treatment duration, and increasing proteinuria and decreasing eGFR can return following treatment discontinuation. New formulations of immunosuppressant therapies include delayed-release budesonide with targeted release in the lower part of the small intestine where Gd-IgA1 production occurs. Although treatment with this drug can reduce proteinuria and sustain eGFR levels, similar to other immunosuppressant therapies, effects seem to be predominantly limited to the active treatment period. Targeting a different mechanism, sparsentan is a dual endothelin A receptor (ETA) and angiotensin II receptor type 1 (AT1) blocker that targets endothelin-1 (ET-1) and angiotensin II, both involved in IgAN progression. Initial Phase III trial results show significant differences, favouring sparsentan, compared with the AT1 blocker irbesartan, on proteinuria, with similar adverse event profiles. These agents, and several other drugs in development, will widen the armamentarium of therapies for people with IgAN, which, when used in combination, can target different aspects of IgAN pathogenesis for a more individualised treatment approach.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139179235","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
More than half of survivors of stroke experience some degree of motor impairment, and spasticity can develop within days of the initial event. Patients with post-stroke spasticity (PSS) report a lower quality of life than those without spasticity, and they require regular long-term follow-up and monitoring within the healthcare system. This symposium supported a non-promotional discussion regarding the prevalence, burden, consequences, and need for identification of PSS. The benefits of PSS identification within 3 months of stroke were discussed by a panel of key opinion leaders, including Ted Wein, Neurologist and Assistant Professor of Neurology and Neurosurgery at McGill University, Montréal, Quebec, Canada; Ganesh Bavikatte, Consultant and Clinical Lead in rehabilitation medicine at the Walton Centre, Liverpool, and Honorary Senior Clinical Lecturer at the University of Liverpool, UK; and Sean Savitz, Professor of Neurology and Physical Medicine and Rehabilitation, Frank M. Yatsu MD Chair in Neurology, and Director of the Institute for Stroke and Cerebrovascular Diseases, University of Texas Health Science Center at Houston (UTHealth), Texas, US. These key opinion leaders explained that early prediction of PSS could be improved by increased awareness of the associated risk factors and tools, such as the Post-Stroke Checklist (PSC), the Spasticity Screening Tool, and the PSS Referral Tool. Finally, potential barriers to the early identification of PSS were presented, alongside strategies to overcome these barriers.
半数以上的中风幸存者都会出现一定程度的运动障碍，而且痉挛可能在中风发生后数天内出现。与没有痉挛的患者相比，卒中后痉挛（PSS）患者的生活质量较低，他们需要医疗系统的定期长期随访和监测。 本次研讨会支持就 PSS 的发病率、负担、后果和识别需求进行非宣传性讨论。由主要意见领袖组成的小组讨论了在卒中后 3 个月内识别 PSS 的益处，这些意见领袖包括加拿大魁北克省蒙特利尔市麦吉尔大学神经病学家兼神经病学和神经外科助理教授 Ted Wein、利物浦沃尔顿中心康复医学顾问和临床负责人兼英国利物浦大学名誉高级临床讲师 Ganesh Bavikatte、神经病学和物理医学与康复学教授兼 Frank M. Yatsu MD 神经病学讲座教授 Sean Savitz。Yatsu MD 神经病学讲座教授、美国得克萨斯州休斯敦大学健康科学中心（UTHealth）中风与脑血管疾病研究所所长肖恩-萨维茨。这些主要意见领袖解释说，可以通过提高对相关风险因素和工具（如卒中后核对表 (PSC)、痉挛筛查工具和 PSS 转诊工具）的认识来改善对 PSS 的早期预测。最后，介绍了早期识别 PSS 的潜在障碍以及克服这些障碍的策略。
{"title":"Spasticity Matters: A Call to Action Following an Acute Stroke","authors":"Nicola Humphry","doi":"10.33590/emj/10304502","DOIUrl":"https://doi.org/10.33590/emj/10304502","url":null,"abstract":"More than half of survivors of stroke experience some degree of motor impairment, and spasticity can develop within days of the initial event. Patients with post-stroke spasticity (PSS) report a lower quality of life than those without spasticity, and they require regular long-term follow-up and monitoring within the healthcare system. This symposium supported a non-promotional discussion regarding the prevalence, burden, consequences, and need for identification of PSS. The benefits of PSS identification within 3 months of stroke were discussed by a panel of key opinion leaders, including Ted Wein, Neurologist and Assistant Professor of Neurology and Neurosurgery at McGill University, Montréal, Quebec, Canada; Ganesh Bavikatte, Consultant and Clinical Lead in rehabilitation medicine at the Walton Centre, Liverpool, and Honorary Senior Clinical Lecturer at the University of Liverpool, UK; and Sean Savitz, Professor of Neurology and Physical Medicine and Rehabilitation, Frank M. Yatsu MD Chair in Neurology, and Director of the Institute for Stroke and Cerebrovascular Diseases, University of Texas Health Science Center at Houston (UTHealth), Texas, US. These key opinion leaders explained that early prediction of PSS could be improved by increased awareness of the associated risk factors and tools, such as the Post-Stroke Checklist (PSC), the Spasticity Screening Tool, and the PSS Referral Tool. Finally, potential barriers to the early identification of PSS were presented, alongside strategies to overcome these barriers.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139179367","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
A spontaneous subscapular hepatic haematoma is a rare condition that has not widely been reported in literature. Subscapular hepatic haematoma has a high mortality rate, especially if the haematoma ruptures, so early diagnosis is imperative. In this case report, the authors present an unusual case of subscapular hepatic haematoma of a female in her 70s, who, in her first few days of admission for the management of acute calculus cholecystitis, developed acute onset right upper quadrant and epigastric pain radiating to her back. Her haemoglobin dropped from 112 g/L to 54 g/L, and her liver function tests and coagulation studies became deranged. Abdominal and pelvic CT and angiography showed a subscapular liver haematoma without active bleeding. The patient received a blood transfusion and was managed conservatively, with no obvious cause being identified.
{"title":"Spontaneous Subcapsular Hepatic Haematoma: A Rare Case Report","authors":"Roisin Burrows-O’Donoghue, Rowena Donnison, Emmanuel D’Almeida","doi":"10.33590/emj/10300184","DOIUrl":"https://doi.org/10.33590/emj/10300184","url":null,"abstract":"A spontaneous subscapular hepatic haematoma is a rare condition that has not widely been reported in literature. Subscapular hepatic haematoma has a high mortality rate, especially if the haematoma ruptures, so early diagnosis is imperative. In this case report, the authors present an unusual case of subscapular hepatic haematoma of a female in her 70s, who, in her first few days of admission for the management of acute calculus cholecystitis, developed acute onset right upper quadrant and epigastric pain radiating to her back. Her haemoglobin dropped from 112 g/L to 54 g/L, and her liver function tests and coagulation studies became deranged. Abdominal and pelvic CT and angiography showed a subscapular liver haematoma without active bleeding. The patient received a blood transfusion and was managed conservatively, with no obvious cause being identified.","PeriodicalId":505023,"journal":{"name":"European Medical Journal","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139239603","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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