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Real-World Safety and Effectiveness of Dimethyl Fumarate in Patients with MS: Results from the ESTEEM Phase 4 and PROCLAIM Phase 3 Studies with a Focus on Older Patients. 富马酸二甲酯对多发性硬化症患者的实际安全性和有效性:以老年患者为重点的 ESTEEM 第 4 期和 PROCLAIM 第 3 期研究结果。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-21 DOI: 10.1007/s12325-024-03047-w
Yang Mao-Draayer, Amit Bar-Or, Konstantin Balashov, John Foley, Kyle Smoot, Erin E Longbrake, Derrick Robertson, Jason P Mendoza, James B Lewin, Nicholas Everage, Ivan Božin, Jennifer Lyons, Oksana Mokliatchouk, Eris Bame, Fabrizio Giuliani

Introduction: Real-world studies in the USA report that 41-56% of patients with multiple sclerosis (MS) are ≥ 50 years old, yet data on their response to disease-modifying therapies (DMTs) is limited. Dimethyl fumarate (DMF) is an oral DMT approved for treating relapsing MS. This analysis evaluated the safety, efficacy, and immunophenotype changes of DMF in patients ≥ 50 years compared with patients < 50 years.

Methods: ESTEEM, a 5-year, real-world, observational phase 4 study, assessed the safety and effectiveness of DMF, including treatment-emergent serious adverse events (SAEs) and adverse events (AEs) leading to treatment discontinuation. Absolute lymphocyte counts (ALCs) were recorded from a subset of patients. The PROCLAIM study, a phase 3b interventional study, reported safety outcomes and lymphocyte subset changes in patients with relapsing-remitting MS (RRMS) treated with DMF. The study evaluated safety outcomes by analyzing the incidence of SAEs and detailed changes in CD4+ and CD8+ T cell compartments over 96 weeks of DMF treatment.

Results: ESTEEM included 4020 patients aged < 50 years and 1069 aged ≥ 50 years. AEs leading to discontinuation were reported by 19.6% patients < 50 years and 29.6% of patients ≥ 50 years, with gastrointestinal disorders being the most common. SAEs were reported by 5.2% of patients < 50 years and 8.9% those ≥ 50 years. In PROCLAIM, SAEs were reported in 13% of patients < 50 years and 10% of those ≥ 50 years. Median ALC decreased by 35% in patients < 50 years and 50% in those ≥ 50 years in ESTEEM, with similar patterns observed in PROCLAIM.

Conclusions: ESTEEM found no unexpected safety signals in older patients and annualized relapse rates (ARRs) were significantly reduced in both age groups. Both studies indicated that DMF is efficacious and has a favorable safety profile in patients with RRMS aged ≥ 50 years.

Clinical trial registration: ESTEEM (NCT02047097), PROCLAIM (NCT02525874).

导言:美国的实际研究报告显示,41%-56%的多发性硬化症(MS)患者年龄≥50岁,但有关他们对改变病情疗法(DMT)的反应的数据却很有限。富马酸二甲酯(DMF)是一种获准用于治疗复发性多发性硬化症的口服 DMT。这项分析评估了 DMF 对年龄≥ 50 岁患者的安全性、疗效和免疫表型变化,并与患者进行了比较:ESTEEM是一项为期5年的真实世界观察性4期研究,评估了DMF的安全性和有效性,包括治疗引发的严重不良事件(SAE)和导致治疗中止的不良事件(AE)。对部分患者的绝对淋巴细胞计数(ALC)进行了记录。PROCLAIM研究是一项3b期干预研究,报告了接受DMF治疗的复发性缓解型多发性硬化症(RRMS)患者的安全性结果和淋巴细胞亚群变化。该研究通过分析SAE的发生率以及DMF治疗96周期间CD4+和CD8+T细胞群的详细变化来评估安全性结果:结果:ESTEEM共纳入了4020名患者:ESTEEM在老年患者中未发现意外安全信号,两个年龄组的年复发率(ARR)均显著降低。这两项研究均表明,DMF对年龄≥50岁的RRMS患者具有良好的疗效和安全性:临床试验注册: esteem (NCT02047097)、Proclaim (NCT02525874)。
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引用次数: 0
A Response to: Letter to the Editor Regarding "Comparing Cardiovascular Outcomes and Costs of Perindopril-, Enalapril- or Losartan-Based Antihypertensive Regimens in South Africa: Real-World Medical Claims Database Analysis". 回应:致编辑的信,内容涉及 "比较南非基于培哚普利、依那普利或洛沙坦的降压方案的心血管疗效和成本:真实世界医疗索赔数据库分析"。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-19 DOI: 10.1007/s12325-024-03045-y
Jacques R Snyman, Freedom Gumedze, Erika S W Jones, Olufunke A Alaba, Nqoba Tsabedze, Alykhan Vira, Ntobeko A B Ntusi
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引用次数: 0
Oral Corticosteroid-Related Healthcare Resource Utilization and Associated Costs in Patients with COPD. 慢性阻塞性肺病患者与口服皮质类固醇相关的医疗资源利用率和相关成本。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-19 DOI: 10.1007/s12325-024-03024-3
Gary Tse, Cono Ariti, Mona Bafadhel, Alberto Papi, Victoria Carter, Jiandong Zhou, Derek Skinner, Xiao Xu, Hana Müllerová, Benjamin Emmanuel, David Price

Introduction: Oral corticosteroids (OCS) are used to manage chronic obstructive pulmonary disease (COPD) exacerbations but are associated with adverse outcomes that may increase healthcare resource utilization and costs. We compared attendance/costs associated with OCS-related adverse outcomes in patients who ever used OCS versus those who never used OCS and examined associations between cumulative OCS exposure and attendance/costs.

Methods: This direct matched observational cohort study used the UK Clinical Practice Research Datalink GOLD database (data range 1987-2019). Patients with a COPD diagnosis on/after April 1, 2003, and Hospital Episode Statistics linkage were included. Emergency room, specialist or primary care outpatient, and inpatient attendance were analyzed. Costs, estimated using Health and Social Care 2019 and National Health Service Reference Costs 2019-2020 reports, were adjusted for sex, age, exacerbation number, and inhaler type used in the 12 months before index date.

Results: The OCS cohort had higher annualized disease-specific (excluding respiratory) total attendance/costs versus the non-OCS cohort (adjusted incidence rate ratio [aIRR] with 95% confidence intervals [CIs]) ranging from 37% (1.37 [1.31, 1.43]) for emergency room attendances to 149% (2.49 [2.36, 2.63]) for specialist consultations. Disease-specific (excluding respiratory) attendance/costs increased in a positive dose-response relationship for most attendance categories versus the < 0.5 g reference dose. For the 0.5 to < 1.0 g cumulative dose category, the greatest increases in disease-specific (excluding respiratory) attendance/costs occurred for primary care consultations (aIRR [95% CI] 1.38 [1.32, 1.44]). For the ≥ 10 g cumulative dose category, the greatest increases were observed for primary care consultations (aIRR [95% CI] 2.83 [2.66, 3.00]), non-elective long stays (≥ 2 days; 2.54 [2.15, 2.99]), and non-elective short stays (≤ 1 day; 2.51 [2.12, 2.98]). Similar findings were observed for all-cause attendance/costs.

Conclusion: Among patients with COPD, OCS-related adverse outcomes were associated with higher attendance and costs, with a positive dose-response relationship. A graphical abstract is available with this article.

导言:口服皮质类固醇(OCS)用于控制慢性阻塞性肺疾病(COPD)的恶化,但其不良后果可能会增加医疗资源的利用率和成本。我们比较了曾经使用过口服皮质类固醇与从未使用过口服皮质类固醇的患者中与口服皮质类固醇相关不良后果有关的就诊/费用,并研究了累积口服皮质类固醇暴露与就诊/费用之间的关联:这项直接匹配的观察性队列研究使用了英国临床实践研究数据链 GOLD 数据库(数据范围为 1987-2019)。研究纳入了在 2003 年 4 月 1 日/之后诊断出慢性阻塞性肺病的患者,并与医院病历统计进行了连接。对急诊室、专科或初级保健门诊以及住院病人的就诊情况进行了分析。使用《2019年健康与社会保健》和《2019-2020年国家卫生服务参考成本》报告估算成本,并根据指数日期前12个月中使用的吸入器类型对性别、年龄、加重次数进行调整:与非OCS队列相比,OCS队列的年化特定疾病(不包括呼吸系统)总就诊人次/费用较高(调整后发病率比[aIRR],95%置信区间[CIs]),从急诊就诊人次的37% (1.37 [1.31, 1.43])到专家会诊的149% (2.49 [2.36, 2.63])不等。与结论相比,大多数就诊类别的疾病特异性(不包括呼吸系统)就诊人次/费用呈正剂量反应关系:在慢性阻塞性肺病患者中,OCS相关不良后果与就诊率和费用的增加有关,且呈正剂量反应关系。本文附有图表摘要。
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引用次数: 0
Letter to the Editor Regarding "Comparing Cardiovascular Outcomes and Costs of Perindopril-, Enalapril- or Losartan-Based Antihypertensive Regimens in South Africa: Real-World Medical Claims Database Analysis". 致编辑的信,内容涉及 "比较南非基于培哚普利、依那普利或洛沙坦的降压方案的心血管预后和成本:真实世界医疗索赔数据库分析"。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-19 DOI: 10.1007/s12325-024-03025-2
Trudy D Leong, Sumayyah Ebrahim, Tamara Kredo
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引用次数: 0
Understanding Influenza Vaccine Clinical Performance: A Podcast. 了解流感疫苗的临床表现:播客
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-18 DOI: 10.1007/s12325-024-03021-6
Nihar R Desai, Pier L Lopalco

Despite well-established vaccination programs, seasonal influenza is still causing substantial clinical, economic and societal burdens. As part of strategies to continually improve influenza vaccine clinical performance, several new approaches are being examined, including high-dose vaccines, adjuvanted vaccines, egg-free vaccines, nasal spray vaccines and mRNA vaccines. Given this range of influenza vaccines, coupled with various vaccine hesitancy concerns, healthcare professionals' understanding and confidence in the clinical performance of influenza vaccines remain key. In this podcast, we discuss the challenges for healthcare professionals in understanding the clinical performance of influenza vaccines and the importance of education in this area, particularly to address perceptions of influenza vaccine failure. We also explore several elements that should be considered in the assessment of influenza vaccine clinical performance: (1) assessing relevant clinical outcomes, such as hospitalization data, (2) utilizing robust methodology in influenza vaccine trials to ensure high quality evidence and (3) approaches used when considering the full body of evidence.

尽管疫苗接种计划已经完善,但季节性流感仍然造成了巨大的临床、经济和社会负担。作为不断改进流感疫苗临床表现策略的一部分,目前正在研究几种新方法,包括高剂量疫苗、佐剂疫苗、无蛋疫苗、鼻喷疫苗和 mRNA 疫苗。鉴于流感疫苗种类繁多,再加上各种疫苗犹豫不决的问题,医疗保健专业人员对流感疫苗临床表现的理解和信心仍是关键。在本期播客中,我们将讨论医护专业人员在了解流感疫苗临床表现方面所面临的挑战,以及在这一领域开展教育的重要性,尤其是在解决人们对流感疫苗失效的看法方面。我们还探讨了在评估流感疫苗临床表现时应考虑的几个因素:(1) 评估相关的临床结果,如住院数据;(2) 在流感疫苗试验中采用可靠的方法以确保高质量的证据;(3) 在考虑全部证据时所采用的方法。
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引用次数: 0
Predicting Asthma Exacerbations Using Machine Learning Models. 利用机器学习模型预测哮喘恶化。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-18 DOI: 10.1007/s12325-024-03053-y
Gianluca Turcatel, Yi Xiao, Scott Caveney, Gilles Gnacadja, Julie Kim, Nestor A Molfino

Introduction: Although clinical, functional, and biomarker data predict asthma exacerbations, newer approaches providing high accuracy of prognosis are needed for real-world decision-making in asthma. Machine learning (ML) leverages mathematical and statistical methods to detect patterns for future disease events across large datasets from electronic health records (EHR). This study conducted training and fine-tuning of ML algorithms for the real-world prediction of asthma exacerbations in patients with physician-diagnosed asthma.

Methods: Adults with ≥ 2 ICD9/10 asthma codes within 1 year and at least 30 days apart were identified from the Optum Panther EHR database between 2016 and 2023. An emergency department (ED), urgent care, or inpatient visit for asthma, while on systemic administration of corticosteroids, was considered an exacerbation. To predict factors associated with exacerbations in a 6-month study period, clinical information from patients was retrieved in the preceding 6-month baseline period. Clinical information included demographics, lab results, diagnoses, medications, immunizations, and allergies. Three models built using Extreme Gradient Boosting (XGBoost), Long Short-Term Memory (LSTM), and Transformers algorithms were trained and tested on independent datasets. Predictions were explained using the SHAP (SHapley Additive exPlanations) library.

Results: Of 1,331,934 patients with asthma, 16,279 (1.2%) experienced ≥ 1 exacerbation. XGBoost was the best predictive algorithm (area under the curve [AUC] = 0.964). Factors associated with exacerbations included a prior history of exacerbation, prednisone usage, high-dose albuterol usage, and elevated troponin I. Reduced probability of exacerbations was associated with receiving inhaled albuterol, vitamins, aspirin, statins, furosemide, and influenza vaccination.

Conclusion: This ML-based study on asthma in the real world confirmed previously known features associated with increased exacerbation risk for asthma, while uncovering not entirely understood features associated with reduced risk of asthma exacerbations. These findings are hypothesis-generating and should contribute to ongoing discussion of the strengths and limitations of ML and other supervised learning models in patient risk stratification.

简介:虽然临床、功能和生物标记物数据可以预测哮喘恶化,但在哮喘的实际决策中需要更新的方法来提供高准确度的预后。机器学习(ML)利用数学和统计方法从电子健康记录(EHR)的大型数据集中检测未来疾病事件的模式。本研究对 ML 算法进行了训练和微调,以便在现实世界中预测医生诊断的哮喘患者的哮喘恶化情况:从 Optum Panther EHR 数据库中识别出 2016 年至 2023 年间在 1 年内≥ 2 个 ICD9/10 哮喘代码且间隔至少 30 天的成人。因哮喘到急诊科(ED)、紧急护理中心或住院部就诊,同时全身使用皮质类固醇的患者被视为病情加重。为了预测与 6 个月研究期间病情加重相关的因素,我们检索了患者在前 6 个月基线期间的临床信息。临床信息包括人口统计学、化验结果、诊断、药物、免疫接种和过敏症。使用极端梯度提升(XGBoost)、长短期记忆(LSTM)和变形算法建立的三个模型在独立的数据集上进行了训练和测试。预测使用 SHAP(SHapley Additive exPlanations)库进行解释:在 1,331,934 名哮喘患者中,有 16,279 人(1.2%)经历了≥ 1 次病情加重。XGBoost 是最佳预测算法(曲线下面积 [AUC] = 0.964)。与病情恶化相关的因素包括既往有病情恶化史、使用泼尼松、使用大剂量阿布特罗以及肌钙蛋白 I 升高:这项基于 ML 的真实世界哮喘研究证实了以前已知的与哮喘恶化风险增加有关的特征,同时发现了与哮喘恶化风险降低有关的尚未完全了解的特征。这些研究结果提出了一些假设,有助于继续讨论 ML 和其他监督学习模型在患者风险分层中的优势和局限性。
{"title":"Predicting Asthma Exacerbations Using Machine Learning Models.","authors":"Gianluca Turcatel, Yi Xiao, Scott Caveney, Gilles Gnacadja, Julie Kim, Nestor A Molfino","doi":"10.1007/s12325-024-03053-y","DOIUrl":"https://doi.org/10.1007/s12325-024-03053-y","url":null,"abstract":"<p><strong>Introduction: </strong>Although clinical, functional, and biomarker data predict asthma exacerbations, newer approaches providing high accuracy of prognosis are needed for real-world decision-making in asthma. Machine learning (ML) leverages mathematical and statistical methods to detect patterns for future disease events across large datasets from electronic health records (EHR). This study conducted training and fine-tuning of ML algorithms for the real-world prediction of asthma exacerbations in patients with physician-diagnosed asthma.</p><p><strong>Methods: </strong>Adults with ≥ 2 ICD9/10 asthma codes within 1 year and at least 30 days apart were identified from the Optum Panther EHR database between 2016 and 2023. An emergency department (ED), urgent care, or inpatient visit for asthma, while on systemic administration of corticosteroids, was considered an exacerbation. To predict factors associated with exacerbations in a 6-month study period, clinical information from patients was retrieved in the preceding 6-month baseline period. Clinical information included demographics, lab results, diagnoses, medications, immunizations, and allergies. Three models built using Extreme Gradient Boosting (XGBoost), Long Short-Term Memory (LSTM), and Transformers algorithms were trained and tested on independent datasets. Predictions were explained using the SHAP (SHapley Additive exPlanations) library.</p><p><strong>Results: </strong>Of 1,331,934 patients with asthma, 16,279 (1.2%) experienced ≥ 1 exacerbation. XGBoost was the best predictive algorithm (area under the curve [AUC] = 0.964). Factors associated with exacerbations included a prior history of exacerbation, prednisone usage, high-dose albuterol usage, and elevated troponin I. Reduced probability of exacerbations was associated with receiving inhaled albuterol, vitamins, aspirin, statins, furosemide, and influenza vaccination.</p><p><strong>Conclusion: </strong>This ML-based study on asthma in the real world confirmed previously known features associated with increased exacerbation risk for asthma, while uncovering not entirely understood features associated with reduced risk of asthma exacerbations. These findings are hypothesis-generating and should contribute to ongoing discussion of the strengths and limitations of ML and other supervised learning models in patient risk stratification.</p>","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142646660","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
Drug Survival, Retention, and Persistence of Dupilumab in Adults and Adolescents with Atopic Dermatitis: A Narrative Literature Review. 成人和青少年特应性皮炎患者使用杜匹单抗的药物存活率、保留率和持续率:文献综述
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-15 DOI: 10.1007/s12325-024-03052-z
Mariateresa Rossi, Silvia M Ferrucci, Piergiacomo Calzavara-Pinton, Angelo V Marzano, Ketty Peris, Elena Nicoli, Devis Moretti, Andrea Chiricozzi

Atopic dermatitis (AD) is a chronic, relapsing inflammatory skin condition that can have a negative impact on a patient's quality of life. Long-term effectiveness is required to manage the symptoms of AD (skin inflammation, eczematous lesions, and itching). Because some of the systemic immunosuppressants used to treat AD have been associated with serious adverse events (AEs), other safer, more effective options, including dupilumab, have been proven effective long-term for treatment of adult and adolescent patients with moderate-to-severe AD. The long-term safety and effectiveness of a drug are usually confirmed in real-world studies by evaluating its performance over time. Measures such as drug survival, drug retention, drug persistence, or retention rates reflect whether treatment may be considered as satisfactory by both patients and physicians, meeting key clinical needs. This review aimed to describe the survival, retention, or persistence of dupilumab therapy in adults and adolescents with moderate-to-severe AD by conducting a PubMed search in March 2023 and screening for relevant publications. Globally, real-world studies with dupilumab have regularly reported high drug survival rates after 1, 2, and 3 years of observation, being consistently at 80-90%, with low rates of treatment discontinuation due to lack of efficacy or AEs. These findings are notably higher than 1- and 2-year drug survival rates of systemic immunosuppressants (including cyclosporine [37% and 20%, respectively] and methotrexate [41% and 33%, respectively]). Overall, real-world data on drug survival have confirmed that dupilumab provides long-term sustained efficacy and acceptable safety in patients with moderate-to-severe AD.

特应性皮炎(AD)是一种慢性、复发性炎症性皮肤病,会对患者的生活质量产生负面影响。治疗特应性皮炎的症状(皮肤炎症、湿疹和瘙痒)需要长期的疗效。由于一些用于治疗 AD 的全身性免疫抑制剂与严重的不良事件(AEs)有关,因此包括杜比单抗在内的其他更安全、更有效的选择已被证明对治疗中度至重度 AD 的成人和青少年患者长期有效。药物的长期安全性和有效性通常在实际研究中通过评估其长期表现来确认。药物存活率、药物保留率、药物持续率或保留率等指标反映了患者和医生是否认为治疗令人满意,是否满足了关键的临床需求。本综述旨在通过在2023年3月进行PubMed检索并筛选相关出版物,描述中重度AD成人和青少年患者接受杜比单抗治疗后的存活率、留药率或持续率。在全球范围内,使用杜必鲁单抗进行的真实世界研究定期报告称,经过1年、2年和3年的观察后,药物存活率较高,始终保持在80%-90%,因缺乏疗效或AEs而中断治疗的比例较低。这些结果明显高于全身性免疫抑制剂(包括环孢素[分别为 37% 和 20%]和甲氨蝶呤[分别为 41% 和 33%])的 1 年和 2 年药物存活率。总体而言,药物存活期的实际数据证实,dupilumab 对中重度 AD 患者具有长期持续的疗效和可接受的安全性。
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引用次数: 0
Correction to: Inside ANEMIA of CKD: Projecting the Future Burden of Anemia of Chronic Kidney Disease and Benefits of Proactive Management: A Microsimulation Model of the Chinese Population. 更正:慢性肾脏病贫血内幕:预测慢性肾脏病贫血的未来负担和积极治疗的益处:中国人口的微观模拟模型。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-14 DOI: 10.1007/s12325-024-03012-7
Lise Retat, Dunming Xiao, Laura Webber, Alexander Martin, Joshua Card-Gowers, Jiaqi Yao, Yuzheng Zhang, Chalet Zhang, Juan Jose Garcia Sanchez, Claudia Cabrera, Susan Grandy, Naveen Rao, Yiqing Wu, Zuo Li, Jianwei Xuan
{"title":"Correction to: Inside ANEMIA of CKD: Projecting the Future Burden of Anemia of Chronic Kidney Disease and Benefits of Proactive Management: A Microsimulation Model of the Chinese Population.","authors":"Lise Retat, Dunming Xiao, Laura Webber, Alexander Martin, Joshua Card-Gowers, Jiaqi Yao, Yuzheng Zhang, Chalet Zhang, Juan Jose Garcia Sanchez, Claudia Cabrera, Susan Grandy, Naveen Rao, Yiqing Wu, Zuo Li, Jianwei Xuan","doi":"10.1007/s12325-024-03012-7","DOIUrl":"https://doi.org/10.1007/s12325-024-03012-7","url":null,"abstract":"","PeriodicalId":7482,"journal":{"name":"Advances in Therapy","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142612015","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
Using Chronic Kidney Disease as a Model Framework to Estimate Healthcare-Related Environmental Impact. 将慢性肾脏病作为估算医疗相关环境影响的模型框架。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-14 DOI: 10.1007/s12325-024-03039-w
Juan Jose Garcia Sanchez, Katherine A Barraclough, Aleix Cases, Roberto Pecoits-Filho, Celine Germond-Duret, Carmine Zoccali, Nina Embleton, Antony Wright, Luke Hubbert, Lindsay Nicholson, Salvatore Barone, Nigel Budgen, Claudia Cabrera, Viknesh Selvarajah, Matthew J Eckelman

Introduction: While the economic and clinical burden of chronic diseases are well documented, their environmental impact remains poorly understood. We developed a framework to estimate the environmental impact of a disease care pathway using chronic kidney disease (CKD) as an example.

Methods: A life cycle assessment framework was developed for the CKD care pathway and validated by experts. Life cycle stages were characterised for resource utilisation based on a literature review and ecoinvent database inputs, in ten countries. The ReCiPe impact assessment method was used to calculate impacts across multiple environmental dimensions.

Results: At CKD stage 5, kidney replacement therapies (KRT) have highest impact; emissions ranged between 3.5 and 43.9 kg carbon dioxide equivalents (CO2e) per session depending on dialysis modality, and 336-2022 kg CO2e for kidney transplant surgery, depending on donor type. Hospitalisations have a substantial environmental impact: a 1-day intensive care stay had highest impact (66.4-143.6 kg CO2e), followed by a 1-day hospital stay (28.8-63.9 kg CO2e) and an 8-h emergency room visit (14.4-27.5  kg CO2e). Patient transport to and from healthcare sites was a key driver of environmental impact for all life cycle stages, representing up to 99.5% of total CO2e emissions.

Conclusion: Full care pathways should be analysed alongside specific healthcare processes. Application of this framework enables quantification of the environmental benefits of preventative medicine and effective management of chronic diseases. For CKD, early diagnosis, and proactive management to reduce the need for KRT and hospitalisations could improve patient outcomes and reduce environmental burden.

导言:虽然慢性病造成的经济和临床负担有据可查,但其对环境的影响却鲜为人知。我们以慢性肾脏病(CKD)为例,开发了一个估算疾病护理路径对环境影响的框架:方法:我们为慢性肾脏病护理路径开发了一个生命周期评估框架,并由专家进行了验证。在文献综述和 ecoinvent 数据库输入的基础上,对 10 个国家的生命周期阶段进行了资源利用特征描述。采用 ReCiPe 影响评估方法计算多个环境维度的影响:结果:在 CKD 第 5 阶段,肾脏替代疗法(KRT)的影响最大;根据透析方式的不同,每次透析的二氧化碳排放量在 3.5 至 43.9 千克二氧化碳当量(CO2e)之间;根据捐献者类型的不同,肾脏移植手术的二氧化碳排放量在 336 至 2022 千克二氧化碳当量之间。住院对环境的影响很大:1 天重症监护住院对环境的影响最大(66.4-143.6 千克二氧化碳当量),其次是 1 天住院(28.8-63.9 千克二氧化碳当量)和 8 小时急诊(14.4-27.5 千克二氧化碳当量)。在所有生命周期阶段,患者往返医疗机构的交通都是造成环境影响的主要因素,占二氧化碳排放总量的 99.5%:结论:在分析具体医疗流程的同时,还应分析完整的医疗路径。应用该框架可量化预防医学和慢性病有效管理对环境的益处。对于慢性肾脏病而言,早期诊断和积极管理以减少对 KRT 和住院的需求,可改善患者预后并减轻环境负担。
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引用次数: 0
Integrating Genetic Insights, Technological Advancements, Screening, and Personalized Pharmacological Interventions in Childhood Obesity. 整合儿童肥胖症的遗传学见解、技术进步、筛查和个性化药物干预。
IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-11-13 DOI: 10.1007/s12325-024-03057-8
Robert Šket, Barbara Slapnik, Primož Kotnik, Klementina Črepinšek, Barbara Čugalj Kern, Tine Tesovnik, Barbara Jenko Bizjan, Blaž Vrhovšek, Žiga I Remec, Maruša Debeljak, Tadej Battelino, Jernej Kovač

Childhood obesity is a significant global health challenge with rising prevalence over the past 50 years, affecting both immediate and long-term health outcomes. The increase in prevalence from 0.7% to 5.6% in girls and 0.9% to 7.8% in boys highlights the urgency of addressing this epidemic. By 2025, it is estimated that 206 million children and adolescents aged 5-19 years will be living with obesity. This review explores the complex interplay of genomics and genetics in pediatric obesity, transitioning from monogenic and polygenic obesity to epigenetics, and incorporating advancements in omics technologies. The evolutionary purpose of adiposity, systemic evaluation of hyperphagia, and the role of various genetic factors are discussed. Technological advancements in genotyping offer new insights and interventions. The integration of genetic screening into clinical practice for early identification and personalized treatment strategies is emphasized.

儿童肥胖症是一项重大的全球性健康挑战,在过去 50 年中发病率不断上升,影响了近期和长期的健康结果。女孩的患病率从 0.7% 上升到 5.6%,男孩的患病率从 0.9% 上升到 7.8%,这凸显了应对这一流行病的紧迫性。据估计,到 2025 年,将有 2.06 亿 5-19 岁的儿童和青少年患有肥胖症。本综述探讨了基因组学和遗传学在小儿肥胖症中的复杂相互作用,从单基因和多基因肥胖症过渡到表观遗传学,并结合了 omics 技术的进步。文章讨论了肥胖的进化目的、多食症的系统评估以及各种遗传因素的作用。基因分型技术的进步提供了新的见解和干预措施。强调将基因筛查纳入临床实践,以实现早期识别和个性化治疗策略。
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引用次数: 0
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Advances in Therapy
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