Clinical Burden

临床负担
  • 文章类型: Systematic Review
    背景:这项系统研究旨在评估全球流行病学,经济,与所有类型的遗传性血管性水肿相关的人文疾病负担。
    方法:系统搜索报告流行病学的文章,经济,从开始至2022年5月23日,使用英文和中文文献数据库对HAE患者的人文负担进行了研究。对选定的研究进行质量和偏倚风险评估。该研究是根据系统评价和荟萃分析的首选报告项目进行的,并在国际系统评价前瞻性注册中心(PROSPERO;CRD42022352377)注册。
    结果:总计,符合检索纳入标准的65篇文章报道了10310例HAE患者,其中5861人为女性患者。总之,4312例患者(81%)和479例患者(9%)患有1型和2型HAE,分别,而422例患者(8%)有HAE-正常C1-INH。所有类型的HAE的总体患病率为每100,000例0.13至1.6例。从HAE症状的首次发作到确诊的平均或中值延迟范围为3.9至26年。HAE患者窒息死亡的估计风险为8.6%。住院治疗,药物,不必要的手术,看医生,专业服务,护理费用是导致经济负担不断增加的直接费用。间接成本主要是由于缺少工作(3402美元/年)和生产力损失(5750美元/年)。此外,观察到患者报告结局报告的QoL受损.QoL措施确定抑郁症,焦虑,压力是成人患者和儿童最常见的症状。
    结论:本研究强调了早期诊断的重要性以及提高卫生保健专业人员意识以减轻HAE对患者和社会的负担的必要性。
    BACKGROUND: This systematic study aims to assess the global epidemiologic, economic, and humanistic burden of illness associated with all types of hereditary angioedema.
    METHODS: A systematic search for articles reporting the epidemiologic, economic, and humanistic burden associated with patients with HAE was conducted using English and Chinese literature databases from the inception to May 23, 2022. The selected studies were assessed for their quality and risk of bias. The study was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses and registered with the International Prospective Register of Systematic Reviews (PROSPERO; CRD42022352377).
    RESULTS: In total, 65 articles that met the search inclusion criteria reported 10,310 patients with HAE, of whom 5861 were female patients. Altogether, 4312 patients (81%) and 479 patients (9%) had type 1 and type 2 HAE, respectively, whereas 422 patients (8%) had HAE-normal C1-INH. The overall prevalence of all types of HAE was between 0.13 and 1.6 cases per 100,000. The mean or median delay from the first onset of a symptom of HAE to confirmed diagnosis ranged from 3.9 to 26 years. The estimated risk of death from asphyxiation was 8.6% for patients with HAE. Hospitalization, medication, unnecessary surgeries, doctor visits, specialist services, and nursing costs are direct expenses that contribute to the growing economic burden. The indirect cost accounted mostly due to missing work ($3402/year) and loss of productivity ($5750/year). Furthermore, impairment of QoL as reported by patient-reported outcomes was observed. QoL measures identified depression, anxiety, and stress to be the most common symptoms for adult patients and children.
    CONCLUSIONS: This study highlights the importance of early diagnosis and the need for improving awareness among health care professionals to reduce the burden of HAE on patients and society.
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  • 文章类型: Journal Article
    焦虑和抑郁在慢性阻塞性肺疾病(COPD)患者中很常见,尤其是老年患者。这会使疾病进展复杂化并导致增加的临床和经济负担。我们试图调查老年COPD患者中与焦虑和/或抑郁症状相关的临床和经济负担。
    我们根据2017年全球倡议慢性阻塞性肺疾病(GOLD)指南,筛选了579名年龄超过60岁并通过肺功能测试诊断为COPD的患者。入院时使用医院焦虑和抑郁量表(HADS)通过面对面访谈来测量焦虑和抑郁。在出院后6、12、18、24和36个月通过电话进行随访,以评估临床和经济负担。使用倾向评分将COPD-焦虑和/或抑郁患者与没有焦虑和抑郁(仅COPD)的患者进行匹配。使用多因素回归模型比较COPD-焦虑和/或抑郁组和仅COPD组的临床和经济负担。
    与仅COPD患者相比,COPD患者并发焦虑和/或抑郁增加了临床负担,包括COPD相关门诊量较高,COPD相关住院,与COPD相关的住院时间(p<0.001)。此外,他们也增加了经济负担,包括较高的年度医疗总费用,医疗费用,和药品费用(p<0.001)。
    伴有焦虑或抑郁的老年COPD患者的临床和经济负担明显高于没有这些合并症的患者。这些发现值得进一步探索,可能对制定相关医疗政策有用。
    UNASSIGNED: Anxiety and depression are common in patients with chronic obstructive pulmonary disease (COPD), especially older adult patients. This can complicate the disease progression and lead to increased clinical and economic burden. We sought to investigate the clinical and economic burdens associated with the presence of anxious and/or depressive symptoms among older adult COPD patients.
    UNASSIGNED: We screened 579 patients aged over 60 years and diagnosed with COPD via a lung function test following the 2017 Global Initiative Chronic Obstructive Lung Disease (GOLD) guidelines. Anxiety and depression were measured using the Hospital Anxiety and Depression Scale (HADS) through face-to-face interviews at admission. Follow-up was conducted by telephone calls at 6, 12, 18, 24, and 36 months after discharge to assess clinical and economic burden. COPD-anxiety and/or depression patients were matched to patients without anxiety and depression (COPD-only) using propensity scores. Multivariate regression models were used to compare clinical and economic burden between COPD-anxiety and/or depression and COPD-only groups.
    UNASSIGNED: Compared with COPD-only patients, COPD patients complicated with anxiety and/or depression had increased clinical burden, including higher COPD-related outpatient visits, COPD-related hospitalizations, and length of COPD-related hospitalizations (p < 0.001). Moreover, they also had an increased economic burden, including higher annual total healthcare costs, medical costs, and pharmacy costs (p < 0.001).
    UNASSIGNED: Older adult COPD patients with anxiety or depression had significantly higher clinical and economic burdens than patients without these comorbidities. These findings deserve further exploration and may be useful for the formulation of relevant healthcare policies.
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  • 文章类型: Review
    目的:脑膜疾病(LMD)与低生存率和健康相关生活质量(HRQoL)相关。迫切需要在该领域进行临床研究以改善结果。本研究的目的是总结LMD的活跃临床研究领域,确定知识差距,并提出了未来的研究方向。
    方法:对LMD的临床试验进行叙述性回顾,是基于临床领域的检索。政府在“状况或疾病”下使用搜索词“软脑膜”。标记为“尚未招募”的实体恶性肿瘤引起的LMD患者的临床试验,“招募”,\"通过邀请注册\"或\"活动,不招募“被包括在内。选择对LMD未来研究方向有重大影响的研究进行讨论。
    共纳入38项临床试验。在这38项试验中,在这篇综述中讨论了19个,重点关注他们的研究问题和对未来研究方向的影响。大多数未被选择用于讨论的研究都集中在生物标志物驱动的干预措施上。确定了四个关键研究领域,即(I)诊断,LMD的反应评估或分子谱分析(n=2);(II)放射治疗的进展(n=3);(III)鞘内治疗(n=13);(IV)用于全身治疗的新型药物载体(n=1)。19个讨论的临床试验中的研究问题包括LMD的肿瘤微环境,新型分子技术在LMD中的作用,放疗与药物的结合,和基于细胞的免疫疗法。在这19项研究中,11个是第一阶段试验,3个是2期或1/2期试验,2个为3期或2/3期试验,其余3个研究未报告研究阶段。讨论了现有的知识差距,包括缺乏主要的特定部位的预后工具,成本效益研究,用于LMD和治疗排序的专用HRQoL评估工具。
    结论:目前LMD的临床试验为改善LMD患者的诊断和治疗结果提供了希望。需要更多的研究来克服当前治疗中的潜在障碍,并弥合本综述中确定的知识差距。提高患者生存的数量和质量。
    OBJECTIVE: Leptomeningeal disease (LMD) is associated with poor survival and health-related quality of life (HRQoL). There is an urgent need for clinical research in this area to improve the outcomes. The purpose of this study is to summarize the areas of active clinical research in LMD, identify the knowledge gap, and suggest future research directions.
    METHODS: A narrative review of clinical trials in LMD was conducted based on a search in clinicatrials.gov using the search term \"leptomeningeal\" under \"condition or disease\". Clinical trials in patients with LMD arising from solid malignancy that were labelled as \"not yet recruiting\", \"recruiting\", \"enrolling by invitation\" or \"active, not recruiting\" were included. Studies which were deemed to have significant impact on future research direction in LMD were selected for discussion.
    UNASSIGNED: A total of 38 clinical trials were included. Of these 38 trials, 19 are discussed in this review, with focus on their research questions and impact on future research directions. Most of the studies that were not selected for discussion focused on biomarker-driven interventions. Four key areas of research were identified, namely the (I) diagnosis, response assessment or molecular profiling of LMD (n=2); (II) advances in radiotherapy (n=3); (III) intrathecal treatment (n=13); (IV) novel drug carrier for systemic treatment (n=1). The research questions in the 19 discussed clinical trials included the tumour microenvironment of LMD, the role of novel molecular techniques in LMD, combination of radiotherapy with drugs, and cell-based immunotherapy. Among these 19 studies, 11 were phase 1 trials, 3 were phase 2 or phase 1/2 trials, 2 were phase 3 or phase 2/3 trials and the study phase was not reported in the remaining 3 studies. The existing knowledge gaps are discussed, including the lack of primary site-specific prognostic tools, cost-effectiveness studies, dedicated HRQoL assessment tools for LMD and sequencing of treatment.
    CONCLUSIONS: The current clinical trials in LMD offer the promise to improve the diagnosis and treatment outcomes of patients with LMD. More research is needed to overcome the potential hurdles in the current treatment and bridge the knowledge gaps as identified in this review, to improve patients\' quantity and quality of survival.
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  • 文章类型: Case Reports
    目的:毛霉菌病是一种机会性真菌感染,发病率低,死亡率高。很少有研究显示中国毛霉菌病的治疗和疾病负担。这项研究旨在收集所有报告的病例,以描述特征和治疗模式,并评估中国毛霉菌病的经济负担。
    方法:我们对中国患者的毛霉菌病病例报告进行了文献综述,以总结该疾病在中国的特点和治疗方式。建立了一个经济模型来评估每人毛霉菌病的总成本,包括直接医疗费用,直接非医疗成本和间接成本。
    结论:共有676例病例报告显示,最常见的毛霉菌病类型是肺毛霉菌病(299/676,44.2%),鼻脑毛霉菌病的病死率最高(122/185,68.5%)。在那些使用经验性疗法的人中,48.8%(231/473)不包括抗粘膜药物;79.8%(336/421)的治疗包括两性霉素B(AMB)或AMB-lipo,在检测到粘液菌后;98.6%(69/70)的报告不良事件与AMB和AMB-lipo相关。治疗时间为90至180天;住院时间为22至95天。每位患者的平均总费用为人民币16.6万元,其中93.1%为直接医疗费用(15.5万元人民币)。
    结论:在中国,毛霉菌病的抗真菌治疗方案数量有限。这项研究强调了引入创新和更广谱的抗真菌药物以提高安全性的迫切需要。临床疗效较好,更容易给药,减轻了中国毛霉菌病患者的经济负担。
    OBJECTIVE: Mucormycosis is an opportunistic fungal infection associated with low incidence but high mortality. Few studies have shown the treatment and disease burden of mucormycosis in China. This study aims at collecting all the reported cases to describe the characteristics and treatment patterns and to assess the economic burden of mucormycosis in China.
    METHODS: We conducted a literature review of mucormycosis case reports in Chinese patients to summarize the characteristics and treatment patterns of the disease in China. An economic model was built to evaluate the total cost of mucormycosis per person, including direct medical cost, direct non-medical cost and indirect cost.
    CONCLUSIONS: A total of 676 case reports showed that the most common type of mucormycosis was pulmonary mucormycosis (299/676, 44.2%), and rhinocerebral mucormycosis had the highest case fatality rate (122/185, 68.5%). Among those who used empiric therapies, 48.8% (231/473) did not include anti-mucor drugs; 79.8% (336/421) of the therapies include amphotericin B (AMB) or AMB-lipo after detection of mucormycetes; 98.6% (69/70) of the reported adverse events were associated with AMB and AMB-lipo. The duration of treatment ranged from 90 to 180 days; the length of stay ranged from 22 to 95 days. The average total cost per patient was 166 thousand Chinese Yuan (CNY), of which 93.1% was the direct medical cost (155 thousand CNY).
    CONCLUSIONS: There are a limited number of antifungal treatment options for mucormycosis in China. This study highlights the critical need to introduce innovative and broader spectrum antifungal drugs with improved safety, better clinical efficacy, easier administration and reduced economic burden to Chinese mucormycosis patients.
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  • 文章类型: Journal Article
    Background: Carbapenem resistant Klebsiella pneumoniae (CRKP), Pseudomonas aeruginosa (CRPA), and Acinetobacter baumannii (CRAB) pose significant threats to public health. However, the clinical and economic impacts of CRKP, CRPA, and CRAB remain largely uninvestigated in China. This study aimed to examine the clinical and economic burden of CRKP, CRPA, and CRAB compared with carbapenem susceptible cases in China. Method: We conducted a retrospective and multicenter study among inpatients hospitalized at four tertiary hospitals between 2013 and 2015 who had K. pneumoniae, P. aeruginosa, and A. baumannii positive clinical samples. Propensity score matching (PSM) was used to balance the impact of potential confounding variables, including age, sex, insurance, number of diagnosis, comorbidities (disease diagnosis, and Charlson comorbidity index), admission to intensive care unit, and surgeries. The main indicators included economic costs, length of stay (LOS), and mortality rate. Results: We included 12,022 inpatients infected or colonized with K. pneumoniae, P. aeruginosa, and A. baumannii between 2013 and 2015, including 831 with CRKP and 4328 with carbapenem susceptible K. pneumoniae (CSKP), 1244 with CRPA and 2674 with carbapenem susceptible P. aeruginosa (CSPA), 1665 with CRAB and 1280 with carbapenem susceptible A. baumannii (CSAB). After PSM, 822 pairs, 1155 pairs, and 682 pairs, respectively were generated. Compared with carbapenem-susceptible cases, those with CRKP, CRPA, and CRAB were associated with statistically significantly increased total hospital cost ($14,252, p < 0.0001; $4605, p < 0.0001; $7277, p < 0.0001) and excess LOS (13.2 days, p < 0.0001; 5.4 days, p = 0.0003; 15.8 days, p = 0.0004). In addition, there were statistically significantly differences in hospital mortality rate between CRKP and CSKP, and CRAB and CSAB group (2.94%, p = 0.024; 4.03%, p = 0.03); however, the difference between CRPA and CSPA group was marginal significant (2.03%, p = 0.052). Conclusion: It highlights the clinical and economic impact of CRKP, CRPA, and CRAB to justify more resources for implementing antibiotic stewardship practices to improve clinical outcomes and to reduce economic costs.
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  • 文章类型: Journal Article
    BACKGROUND: The epidemiology of acute kidney injury (AKI) in nonexacerbated chronic obstructive pulmonary disease (NECOPD) patients is unknown. This study investigated the factors associated with AKI and the association between AKI and in-hospital mortality in the hospitalized NECOPD population.
    METHODS: The electronic medical records of 2897 patients hospitalized with NECOPD were analyzed retrospectively. Demographic information, medicine used before AKI, diagnosis records and laboratory data were collected. AKI was classified as community-acquired (CA-) or hospital-acquired (HA-) AKI according to the serum creatinine criteria. Risk factors for HA-AKI and in-hospital mortality were analyzed by logistic regression analyses. To avoid an interaction between cor pulmonale and AKI, the association between AKI and in-hospital morality was further analyzed with cor pulmonale stratification.
    RESULTS: The incidence rates of CA- and HA-AKI were 7.1 and 12.0%, respectively. Increased age, female sex, cor pulmonale comorbidity, chronic kidney disease stage, diuretic and glycopeptide use before AKI and iodine-containing contrast medium exposure were independently associated with HA-AKI. A total of 5.7% of the patients died. After adjustment for age, sex, cor pulmonale, chronic kidney disease, Charlson comorbidity index score (without renal disease) and hemoglobin level, HA-AKI was an independent risk factor for in-hospital mortality [OR 13.909 (95% CI 8.699-22.238) in non-cor pulmonale subgroup; OR 26.604 (95% CI 12.166-58.176) in cor pulmonale subgroup], whereas CA-AKI was not.
    CONCLUSIONS: AKI is common in the NECOPD population. Diuretics and contrast media are associated with HA-AKI in this population. The patients with HA-AKI have a higher mortality risk than the patients without AKI.
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