Real-world evidence

真实世界的证据
  • 文章类型: Journal Article
    aHCC的治疗前景近年来有所发展,需要对治疗模式进行全面分析,临床结果,HCCU,以及将新出现的治疗方法情境化的成本。本研究旨在使用安大略省的实际数据调查这些结果,加拿大。这项回顾性队列研究是使用2010年4月至2020年3月的关联管理数据库进行的。包括诊断为aHCC的患者,并分析了他们的临床和人口统计学特征,以及治疗模式,生存,HCCU,和经济负担。在7322名患者中,802例aHCC患者符合纳入研究的资格标准。治疗亚组包括1L全身治疗(53.2%),其他全身治疗(4.5%),轻轨(9.0%),无治疗(33.3%)。中位年龄为66岁,大多数是男性(82%)。从诊断开始整个队列的mOS为6.5个月。然而,接受1L全身治疗的患者的mOS为9.0个月,显着高于其他三个亚组。每位接受HCC治疗的患者的平均费用为49,640加元,口服药物和住院是最大的成本驱动因素。结果强调了在不断发展的治疗选择时代,需要对HCC管理策略进行持续评估和优化。
    The therapeutic landscape for aHCC has evolved in recent years, necessitating a comprehensive analysis of treatment patterns, clinical outcomes, HCRU, and costs to contextualize emerging treatments. This study aimed to investigate these outcomes using real-world data from Ontario, Canada. This retrospective cohort study was conducted using linked administrative databases from April 2010 to March 2020. Patients diagnosed with aHCC were included, and their clinical and demographic characteristics were analyzed, as well as treatment patterns, survival, HCRU, and economic burden. Among 7322 identified patients, 802 aHCC patients met the eligibility criteria for inclusion in the study. Treatment subgroups included 1L systemic therapy (53.2%), other systemic treatments (4.5%), LRT (9.0%), and no treatment (33.3%). The median age was 66 years, and the majority were male (82%). The mOS for the entire cohort from diagnosis was 6.5 months. However, patients who received 1L systemic therapy had an mOS of 9.0 months, which was significantly higher than the other three subgroups. The mean cost per aHCC-treated patient was $49,640 CAD, with oral medications and inpatient hospitalizations as the largest cost drivers. The results underscore the need for the continuous evaluation and optimization of HCC management strategies in the era of evolving therapeutic options.
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  • 文章类型: Journal Article
    据估计,抗生素耐药性(AMR)每年导致全球近500万人死亡,到2050年将达到1000万人。耐碳青霉烯类鲍曼不动杆菌(CRAB)感染是全球因抗菌素耐药性引起的第四大死亡原因。但是仍然缺乏标准化的治疗方法。在考虑的抗生素中,舒巴坦/durlobactam似乎是替代当前骨干药物的最佳候选药物。头孢地洛可在联合治疗方案中起关键作用。由于毒性和药代动力学/药效学(PK/PD)的限制,粘菌素(或多粘菌素B)应用作替代药物(当没有其他选择时)。替加环素(或米诺环素)和磷霉素可以代表两种NBL的合适伴侣。需要进行随机临床试验(RCT),以更好地评估NBLs在CRAB感染治疗中的作用,并比较替加环素和磷霉素作为伴侣抗生素的疗效。应测试NBLs与“旧”药物(利福平和甲氧苄啶/磺胺甲恶唑)之间的协同作用。应该做出巨大的努力,以加快对具有改善的肺活动的更安全的多粘菌素候选物的临床前和临床研究,以及IV利福布汀配方。在这篇叙述性评论中,鉴于新开发的β-内酰胺类药物(NBLs),我们重点关注CRAB感染的抗生素治疗.
    It is estimated that antimicrobial resistance (AMR) is responsible for nearly 5 million human deaths worldwide each year and will reach 10 million by 2050. Carbapenem-resistant Acinetobacter baumannii (CRAB) infections represent the fourth-leading cause of death attributable to antimicrobial resistance globally, but a standardized therapy is still lacking. Among the antibiotics under consideration, Sulbactam/durlobactam seems to be the best candidate to replace current back-bone agents. Cefiderocol could play a pivotal role within combination therapy regimens. Due to toxicity and the pharmacokinetics/pharmacodynamics (PK/PD) limitations, colistin (or polymyxin B) should be used as an alternative agent (when no other options are available). Tigecycline (or minocycline) and fosfomycin could represent suitable partners for both NBLs. Randomized clinical trials (RCTs) are needed to better evaluate the role of NBLs in CRAB infection treatment and to compare the efficacy of tigecycline and fosfomycin as partner antibiotics. Synergism should be tested between NBLs and \"old\" drugs (rifampicin and trimethoprim/sulfamethoxazole). Huge efforts should be made to accelerate pre-clinical and clinical studies on safer polymyxin candidates with improved lung activity, as well as on the iv rifabutin formulation. In this narrative review, we focused the antibiotic treatment of CRAB infections in view of newly developed β-lactam agents (NBLs).
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  • 文章类型: Journal Article
    越来越多的人使用数字减肥服务(DWLS)来治疗超重和肥胖。尽管人们普遍认为数字模式总体上改善了获得护理的机会,肥胖利益相关者仍然担心许多DWLSs不够全面或可持续,无法提供有意义的健康结果.这项研究采用了混合方法的方法来评估患者为什么以及在多长时间后倾向于停止澳大利亚最大的DWLS,在多学科护理团队的指导下,结合行为和药物治疗的计划。我们发现,在2022年1月至6月开始桉树DWLS的一组患者中(n=5604),平均计划依从性为171.2(±158.2)天.患者所需的葡萄糖样肽-1受体激动剂药物供应不足是停药的最常见原因(43.7%),其次是项目成本(26.2%),结果不满意(9.9%),和服务不满意(7.2%)。统计测试表明,种族和年龄都对患者的依从性有显着影响。这些发现表明,DWLS有可能改善获得全面、持续的肥胖护理,但是护理模型需要改进在澳大利亚桉树DWLS中观察到的模型,以减轻常见的现实世界计划减员因素。
    Increasingly large numbers of people are using digital weight loss services (DWLSs) to treat being overweight and obesity. Although it is widely agreed that digital modalities improve access to care in general, obesity stakeholders remain concerned that many DWLSs are not comprehensive or sustainable enough to deliver meaningful health outcomes. This study adopted a mixed methods approach to assess why and after how long patients tend to discontinue Australia\'s largest DWLS, a program that combines behavioural and pharmacological therapy under the guidance of a multidisciplinary care team. We found that in a cohort of patients who commenced the Eucalyptus DWLS between January and June 2022 (n = 5604), the mean program adherence was 171.2 (±158.2) days. Inadequate supplying of a patient\'s desired glucose-like peptide-1 receptor agonist medication was the most common reason for discontinuation (43.7%), followed by program cost (26.2%), result dissatisfaction (9.9%), and service dissatisfaction (7.2%). Statistical tests revealed that ethnicity and age both had a significant effect on patient adherence. These findings suggest that DWLSs have the potential to improve access to comprehensive, continuous obesity care, but care models need to improve upon the one observed in the Eucalyptus Australia DWLS to mitigate common real-world program attrition factors.
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  • 文章类型: Journal Article
    Niraparib最近在加拿大获得资助,用于铂类化疗后卵巢癌的维持治疗。然而,该药物在现实世界中的安全性仍不确定。我们进行了一项队列研究,以描述在加拿大四个省(安大略省,艾伯塔省,不列颠哥伦比亚省[BC],和魁北克)。我们使用了安大略省卫生部的行政数据和电子病历,艾伯塔省卫生服务,和BC癌症,和来自ExactisInnovation的注册数据。我们使用描述性统计数据总结基线特征,并使用累积发生率报告安全性结果。我们确定了514名接受尼拉帕尼的患者。平均年龄为67岁,大多数以每天100或200mg/天的剂量开始。3/4级贫血,中性粒细胞减少症,和血小板减少发生率为11-16%.在安大略省,3/4级血小板减少症的三个月累积发生率为11.6%(95%CI,8.3-15.4%),中性粒细胞减少率为7.1%(95%CI,4.6-10.4%),贫血为11.3%(95%CI,8.0-15.2%)。其余省份的累积发病率相似。在现实世界中,初始日剂量和血液学不良事件的比例很低,可能与临床医生的谨慎处方和密切监测有关。
    Niraparib was recently funded in Canada for the maintenance treatment of ovarian cancer following platinum-based chemotherapy. However, the drug\'s safety profile in the real world remains uncertain. We conducted a cohort study to describe the patient population using niraparib and the proportion that experienced adverse events between June 2019 and December 2022 in four Canadian provinces (Ontario, Alberta, British Columbia [BC], and Quebec). We used administrative data and electronic medical records from Ontario Health, Alberta Health Services, and BC Cancer, and registry data from Exactis Innovation. We summarized baseline characteristics using descriptive statistics and reported safety outcomes using cumulative incidence. We identified 514 patients receiving niraparib. Mean age was 67 years and most were initiated on a daily dose of 100 or 200 mg/day. Grade 3/4 anemia, neutropenia, and thrombocytopenia occurred in 11-16% of the cohort. In Ontario, the three-month cumulative incidence of grade 3/4 thrombocytopenia was 11.6% (95% CI, 8.3-15.4%), neutropenia was 7.1% (95% CI, 4.6-10.4%), and anemia was 11.3% (95% CI, 8.0-15.2%). Cumulative incidences in the remaining provinces were similar. Initial daily dose and proportions of hematological adverse events were low in the real world and may be related to cautious prescribing and close monitoring by clinicians.
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  • 文章类型: Journal Article
    寻常型牛皮癣与重大的医疗保健负担有关,随着时间的推移,随着疾病的进展而增加。这次回顾的目的,以人群为基础的注册研究旨在描述芬兰银屑病患者使用生物制剂和口服免疫抑制剂(常规药物)的医疗资源利用情况.
    该研究队列包括2012-2018年间在二级医疗机构中诊断为寻常型银屑病的所有患者,这些患者在2013-2017年间开始了生物学(n=1,297)或常规(n=4,753)治疗。关于一级和二级HCCU的数据是从全国医疗保健注册中心收集的。
    结果表明,开始生物制剂治疗的患者在开始治疗后与皮肤科医生的接触显着减少(开始治疗前平均每年每人接触5.4人,开始治疗后平均每年接触2.3人),但不是常规(3.3和3.2)治疗。对于传统的初学者,在治疗切换的时间与皮肤科医生有很高的联系,这对于生物起始物没有观察到。
    总的来说,在开始或切换治疗后,初级和其他二级护理接触没有减少.结果突出了彻底考虑最佳治疗方案的重要性,考虑到患者和医疗保健系统的总体疾病负担。
    UNASSIGNED: Psoriasis vulgaris is associated with a significant healthcare burden, which increases over time as the disease progresses. The aim of this retrospective, population-based registry study was to characterize healthcare resource utilization (HCRU) in patients with psoriasis using biologics and oral immunosuppressants (conventionals) in Finland.
    UNASSIGNED: The study cohort included all patients with a diagnosis of psoriasis vulgaris in the secondary healthcare setting between 2012-2018, who initiated a biologic (n=1,297) or conventional (n=4,753) treatment between 2013-2017. Data on primary and secondary HCRU were collected from nationwide healthcare registries.
    UNASSIGNED: The results indicated a remarkable decrease in contacts with a dermatologist after the treatment initiation among patients starting biologic (mean annual number of contacts 5.4 per person before and 2.3 after the initiation), but not conventional (3.3 and 3.2) treatment. For conventional starters there was a high level of contacts with a dermatologist surrounding times of treatment switching, which was not observed for biologic starters.
    UNASSIGNED: Overall, primary and other secondary care contacts did not decrease after the initiation or switch of treatment. The results highlight the importance of thorough consideration of the most optimal treatment alternatives, considering the overall disease burden to patients and healthcare systems.
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  • 文章类型: Editorial
    暂无摘要。
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  • 文章类型: Journal Article
    背景/目的:塞马鲁肽是目前唯一的每日一次口服胰高血糖素样受体激动剂。这项研究的目的是描述2型糖尿病(T2D)患者的临床特征,开始口服司马鲁肽,评估其对血糖控制的影响,体重(BW)及其在常规临床实践中的耐受性。方法:评估了两家意大利糖尿病诊所的电子病历。在口服司马鲁肽处方之前和之后6个月,评估了T2D成人的平均糖化血红蛋白(HbA1c)和BW。报告了治疗中止和安全性数据。结果:总共192名开始口服司马鲁肽的患者(44%为女性)的平均年龄为66岁,糖尿病持续10年,HbA1c为7.9%,BW为82.6kg。几乎50%的患者肥胖。从基线到随访的平均HbA1c和BW变化分别为-0.7%和-2.6kg,分别。基线HbA1c≥8%且糖尿病病程<5年的患者HbA1c下降幅度更大。22.5%的参与者实现了HbA1c≤7%和体重减轻≥5%的复合终点。共有40例患者(20.8%)停止治疗:26例因胃肠道不良事件,和10由于降低HbA1c和/或BW的有效性有限。结论:在真实的临床环境中,开始口服司马鲁肽的患者表现出次优的代谢控制,糖尿病病程短和肥胖;HbA1c和BW的显着改善主要在最近诊断为糖尿病的患者中实现,支持在疾病早期使用口服司马鲁肽。
    Background/Objectives: Semaglutide is the unique once-daily oral glucagon-like receptor agonist presently available. Aims of this study were to describe clinical characteristics of patients with type 2 diabetes (T2D) initiating oral semaglutide, to assess its effects on glycemic control, body weight (BW) and its tolerability in routine clinical practice. Methods: Electronic medical records from two Italian diabetes clinics were evaluated. Mean glycated hemoglobin (HbA1c) and BW were assessed in adults with T2D before and 6 months after oral semaglutide prescription. Treatment discontinuation and safety data were reported. Results: A total of 192 patients initiating oral semaglutide (44% female) presented a mean age of 66 years, a diabetes duration of 10 years, HbA1c of 7.9% and a BW of 82.6 kg. Almost 50% of patients were obese. Mean HbA1c and BW changes from baseline to follow up were -0.7% and -2.6 kg, respectively. Greater HbA1c reduction was observed in patients with baseline HbA1c ≥ 8% and with diabetes duration <5 years. The composite endpoint of HbA1c ≤7% and a weight loss ≥5% was achieved in 22.5% of the participants. A total of 40 patients (20.8%) discontinued treatment: 26 because of gastrointestinal adverse events, and 10 due to limited effectiveness in lowering HbA1c and/or BW. Conclusions: In a real clinical setting, patients initiating oral semaglutide showed suboptimal metabolic control, short diabetes duration and obesity; a significant improvement in HbA1c and BW was achieved mainly in patients with a more recent diabetes diagnosis, supporting the use of oral semaglutide in the early phase of the disease.
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  • 文章类型: Journal Article
    背景:腕管综合征(CTS),正中神经压迫引起的压迫性神经病,是一种可导致生活质量下降的进行性疾病。研究表明CTS与关节炎之间存在关联;然而,以前检查骨关节炎(OA)和CTS的研究数量有限,范围和研究设计。这项研究估计了OA患者中CTS的发生率和风险。整体和特定的关节,在美国一个以人口为基础的大型队列中。
    方法:来自Optum索赔数据库的患者年龄≥45岁,在2018年1月1日至2022年12月31日之间诊断为OA,符合OA队列的条件。非OA队列包括在索引日期未诊断为OA且索引前12个月无OA病史的患者。使用倾向评分匹配来平衡基线特征。使用Cox回归估计的发病率和调整后的风险比评估OA和非OA队列中的CTS风险。
    结果:应用纳入/排除标准后,诊断为OA的6,023,384名成年人中的3,610,240人仍留在OA队列中。在倾向得分匹配后,每个队列包括1,033,439例个体.每1000人年CTS的发病率在OA队列中为7.35(95%置信区间[CI]7.21-7.49),在非OA队列中为1.44(95%CI1.38-1.50)。OA患者发生CTS的风险约为无OA患者的4倍(风险比=3.80;95%CI3.54-4.07)。在所有OA关节类型中都发现了这种增加的风险,手/手腕的OA具有最高的CTS风险。此外,与单个受影响关节相比,多个OA关节的风险更高.
    结论:OA会增加CTS的风险,但这不仅限于手/腕部OA患者,提示OA对CTS的系统性影响。虽然手/腕部OA患者的风险似乎最高,膝关节或髋关节等受影响关节较远的患者发生CTS的风险也增加.
    BACKGROUND: Carpal tunnel syndrome (CTS), an entrapment neuropathy caused by pressure of the median nerve, is a progressive condition that can lead to a decreased quality of life. Studies suggest an association between CTS and arthritis; however, previous studies examining osteoarthritis (OA) and CTS are limited in number, scope and study design. This study estimated the incidence and risk of CTS among patients with OA, both overall and by specific joints, in a large population-based cohort in the United States.
    METHODS: Patients from the Optum claims database aged ≥ 45 years and diagnosed with OA between January 1, 2018, and December 31, 2022, were eligible for the OA cohort. The non-OA cohort included those without a diagnosis of OA at the index date and no history of OA for 12 months pre-index. Baseline characteristics were balanced using propensity score matching. The risk of CTS in the OA and non-OA cohort were evaluated using incidence rates and adjusted hazard ratios that were estimated using Cox regression.
    RESULTS: After applying the inclusion/exclusion criteria, 3,610,240 of the 6,023,384 adults with a diagnosis of OA remained in the OA cohort. After propensity-score matching, each cohort included 1,033,439 individuals. The incidence rates for CTS per 1000 person-years were 7.35 (95% confidence interval [CI] 7.21-7.49) in the OA cohort and 1.44 (95% CI 1.38-1.50) in the non-OA cohort. The risk of developing CTS in patients with OA was ~ 4 times that of patients without (hazard ratio = 3.80; 95% CI 3.54-4.07). This increased risk was found across all OA joint types, with OA of the hand/wrist having the highest risk for CTS. Additionally, multiple OA joints presented a higher risk compared with a single affected joint.
    CONCLUSIONS: OA increases the risk of CTS, but this is not limited to patients with hand/wrist OA, suggesting a systemic impact of OA on CTS. While the risk appears highest for patients with hand/wrist OA, patients with more distant affected joints like knee or hip also have an increased risk of CTS.
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  • 文章类型: Journal Article
    这份ISPOR良好实践报告提供了一个框架,用于评估电子健康记录数据是否适合用于卫生技术评估(HTA)。尽管电子健康记录(EHR)数据可以填补证据空白并改善决策,几个重要的局限性会影响其有效性和相关性。ISPOR框架包括2个组件:数据描绘和数据适合性。数据描绘通过描述(1)数据特征;(2)数据来源;和(3)数据治理来提供对数据的完整理解和对其可信度的评估。适用性包括(1)数据可靠性项目,ie,如何准确和完整的估计是回答手头的问题和(2)数据相关性项目,从决策的角度评估数据适合回答特定问题的程度。该报告包括特定于EHR数据报告的检查表:ISPOR适用性检查表。它还为HTA机构和政策制定者提供了建议,以随着时间的推移改善EHR衍生数据的使用。报告最后讨论了该领域的局限性和未来方向,包括大型语言模型和生成人工智能的扩散和能力的实质性和快速发展带来的潜在影响。该报告的直接受众是HTA证据开发人员和用户。我们预计它也将对其他利益相关者有用,特别是监管机构和制造商,在未来。
    This ISPOR Good Practices report provides a framework for assessing the suitability of electronic health records data for use in health technology assessments (HTAs). Although electronic health record (EHR) data can fill evidence gaps and improve decisions, several important limitations can affect its validity and relevance. The ISPOR framework includes 2 components: data delineation and data fitness for purpose. Data delineation provides a complete understanding of the data and an assessment of its trustworthiness by describing (1) data characteristics; (2) data provenance; and (3) data governance. Fitness for purpose comprises (1) data reliability items, ie, how accurate and complete the estimates are for answering the question at hand and (2) data relevance items, which assess how well the data are suited to answer the particular question from a decision-making perspective. The report includes a checklist specific to EHR data reporting: the ISPOR SUITABILITY Checklist. It also provides recommendations for HTA agencies and policy makers to improve the use of EHR-derived data over time. The report concludes with a discussion of limitations and future directions in the field, including the potential impact from the substantial and rapid advances in the diffusion and capabilities of large language models and generative artificial intelligence. The report\'s immediate audiences are HTA evidence developers and users. We anticipate that it will also be useful to other stakeholders, particularly regulators and manufacturers, in the future.
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  • 文章类型: Journal Article
    评估西班牙队列中使用不同的单片丙烯酸单焦点人工晶体进行白内障手术后长达五年的掺钕钇铝石榴石激光(Nd:YAG)囊切开术的发生率。
    数据是从电子病历中提取的。符合条件的参与者年龄≥65岁,使用五种不同的丙烯酸单焦点IOL(AlconAcrySof,AJLLLASY60,MediconturBi-flex,IOLTechStabibag和ZeissAsphina),和超过六个月的基线数据。参与者从手术后随访5年,从Nd:YAG随访6个月。在IOL之间比较Nd:YAG的发生率,并进行多变量分析以确定白内障手术后五年Nd:YAG发生率的预测因素。
    最初的队列包括9545名患者,14,519只眼(53%为女性,平均年龄75岁)。其中,白内障手术五年后,有3955只眼可用于分析。在手术后的五年里,AlconAcrysofIOL的Nd:YAG发病率始终低于其他IOL。五年时,AlconAcrysof的Nd:YAG发病率为8.8%。相比之下,AJLLLASY60的发生率为47.4%(OR=9.54,95%CI[6.57,13.84]),蔡司Asphina为44.3%(OR=8.35,95%CI[5.85,11.94]),IOLTechStabibag为44.0%(OR=8.02,95%CI[4.60,13.84])。
    AlconAcrySofIOL在白内障手术后的长期随访期内,Nd:YAG发病率始终较低,强调IOL选择对患者长期预后的重要性。
    UNASSIGNED: To estimate the incidence of neodymium-doped yttrium aluminum garnet laser (Nd:YAG) capsulotomy up to five years after cataract surgery with different single-piece acrylic monofocal IOLs in a Spanish cohort.
    UNASSIGNED: Data were extracted from electronic medical records. Eligible participants were aged ≥65, had cataract surgery with one of five different acrylic monofocal IOLs (Alcon AcrySof, AJL LLASY60, Medicontur Bi-flex, IOL Tech Stabibag and Zeiss Asphina), and more than six months baseline data. Participants were followed up to five years from surgery and up to six months from Nd:YAG. The incidence of Nd:YAG was compared between the IOLs and multivariate analyses were conducted to identify predictors of Nd:YAG incidence at five-years after cataract surgery.
    UNASSIGNED: The initial cohort included 9545 patients with 14,519 eyes (53% female, average age 75 years). Of those, 3955 eyes were available for analysis five years after cataract surgery. Throughout the five years post-surgery, Nd:YAG incidence was consistently lower with Alcon Acrysof IOLs than the other IOLs. At five years the Nd:YAG incidence rate for Alcon Acrysof was 8.8%. In comparison, the incidence was 47.4% for AJL LLASY60 (OR = 9.54, 95% CI [6.57, 13.84]), 44.3% for Zeiss Asphina (OR = 8.35, 95% CI [5.85, 11.94]) and 44.0% for IOL Tech Stabibag (OR = 8.02, 95% CI [4.60, 13.84]).
    UNASSIGNED: Alcon AcrySof IOLs have a consistently lower risk of Nd:YAG incidence over a long follow-up period after cataract surgery, highlighting the importance of IOL choice for patients\' long-term outcomes.
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