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Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom 迟发性庞贝病的生活质量:英国的定性访谈和一般公共效用评估
Q2 ECONOMICS Pub Date : 2023-03-03 DOI: 10.36469/jheor.2023.68157
Lena Hubig, Anna-Katrine Sussex, Alasdair MacCulloch, Derralynn Hughes, Ryan Graham, Liz Morris, Syed Raza, Andrew Lloyd, Amanda Sowinski, Katy Gallop
Background: Late-onset Pompe disease (LOPD) is a rare, progressive neuromuscular condition typically characterized by weakness of skeletal muscles, including those involved in respiration and diaphragmatic dysfunction. Individuals with LOPD typically eventually require mobility and/or ventilatory support. Objectives: This study aimed to develop health state vignettes and estimate health state utility values for LOPD in the United Kingdom. Methods: Vignettes were developed for 7 health states of LOPD with states defined in terms of mobility and/or ventilatory support. Vignettes were drafted based on patient-reported outcome data from the Phase 3 PROPEL trial (NCT03729362) and supplemented by a literature review. Qualitative interviews with individuals living with LOPD and clinical experts were conducted to explore the health-related quality-of-life (HRQoL) impact of LOPD and to review the draft vignettes. Vignettes were finalized following a second round of interviews with individuals living with LOPD and used in health state valuation exercises with people of the UK population. Participants rated the health states using the EQ-5D-5L, visual analogue scale, and time trade-off interviews. Results: Twelve individuals living with LOPD and 2 clinical experts were interviewed. Following the interviews, 4 new statements were added regarding dependence on others, bladder control problems, balance issues/fear of falling, and frustration. One hundred interviews with a representative UK population sample were completed. Mean time trade-off utilities ranged from 0.754 (SD = 0.31) (no support) to 0.132 (SD = 0.50) (invasive ventilatory and mobility support–dependent). Similarly, EQ-5D-5L utilities ranged from 0.608 (SD = 0.12) to -0.078 (SD = 0.22). Discussion: The utilities obtained in the study are consistent with utilities reported in the literature (0.670-0.853 for nonsupport state). The vignette content was based on robust quantitative and qualitative evidence and captured the main HRQoL impacts of LOPD. The general public rated the health states consistently lower with increasing disease progression. There was greater uncertainty around utility estimates for the severe states, suggesting that participants found it harder to rate them. Conclusion: This study provides utility estimates for LOPD that can be used in economic modeling of treatments for LOPD. Our findings highlight the high disease burden of LOPD and reinforce the societal value of slowing disease progression.
背景:迟发性庞贝病(LOPD)是一种罕见的进行性神经肌肉疾病,典型特征为骨骼肌无力,包括呼吸和膈功能障碍。LOPD患者通常最终需要活动和/或呼吸支持。目的:本研究旨在开发健康状态小片段,并估计英国LOPD的健康状态效用值。方法:针对LOPD的7种健康状态制定了小视频,这些状态根据活动能力和/或通气支持来定义。研究人员根据3期PROPEL试验(NCT03729362)患者报告的结果数据起草了小样本,并辅以文献综述。对LOPD患者和临床专家进行了定性访谈,以探讨LOPD对健康相关生活质量(HRQoL)的影响,并审查草稿。在与LOPD患者进行第二轮访谈后,最终确定了小插曲,并将其用于英国人口的健康状况评估练习。参与者使用EQ-5D-5L、视觉模拟量表和时间权衡访谈对健康状态进行评级。结果:对12名LOPD患者和2名临床专家进行了访谈。在访谈之后,增加了4项新的陈述,涉及对他人的依赖、膀胱控制问题、平衡问题/害怕跌倒和沮丧。对具有代表性的英国人口样本进行了100次访谈。平均时间权衡效用范围从0.754 (SD = 0.31)(无支持)到0.132 (SD = 0.50)(有创通气和活动支持依赖)。同样,EQ-5D-5L的效用范围从0.608 (SD = 0.12)到-0.078 (SD = 0.22)。讨论:本研究获得的效用与文献报道的效用一致(不支持状态为0.670-0.853)。小插曲的内容基于可靠的定量和定性证据,并捕获了LOPD对HRQoL的主要影响。随着疾病进展的加剧,公众对健康状况的评价一直较低。对于那些严重的州,效用估计存在更大的不确定性,这表明参与者发现更难对它们进行评级。结论:本研究提供了LOPD的效用估计,可用于LOPD治疗的经济建模。我们的研究结果强调了LOPD的高疾病负担,并加强了减缓疾病进展的社会价值。
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引用次数: 0
Outcomes, Healthcare Resource Utilization, and Costs of Overall, Community-Acquired, and Hospital-Acquired Acute Kidney Injury in COVID-19 Patients COVID-19患者整体、社区获得性和医院获得性急性肾损伤的结局、医疗资源利用和成本
Q2 ECONOMICS Pub Date : 2023-02-23 DOI: 10.36469/jheor.2023.57651
Jay Koyner, Rachel Mackey, Ning Rosenthal, Leslie Carabuena, J. Patrick Kampf, Paul McPherson, Toni Rodriguez, Aarti Sanghani, Julien Textoris
Background: In hospitalized patients with COVID-19, acute kidney injury (AKI) is associated with higher mortality, but data are lacking on healthcare resource utilization (HRU) and costs related to AKI, community-acquired AKI (CA-AKI), and hospital-acquired AKI (HA-AKI). Objectives: To quantify the burden of AKI, CA-AKI, and HA-AKI among inpatients with COVID-19. Methods: This retrospective cohort study included inpatients with COVID-19 discharged from US hospitals in the Premier PINC AI™ Healthcare Database April 1–October 31, 2020, categorized as AKI, CA-AKI, HA-AKI, or no AKI by ICD-10-CM diagnosis codes. Outcomes were assessed during index (initial) hospitalization and 30 days postdischarge. Results: Among 208 583 COVID-19 inpatients, 30%, 25%, and 5% had AKI, CA-AKI, and HA-AKI, of whom 10%, 7%, and 23% received dialysis, respectively. Excess mortality, HRU, and costs were greater for HA-AKI than CA-AKI. In adjusted models, for patients with AKI vs no AKI and HA-AKI vs CA-AKI, odds ratios (ORs) (95% CI) were 3.70 (3.61-3.79) and 4.11 (3.92-4.31) for intensive care unit use and 3.52 (3.41-3.63) and 2.64 (2.52-2.78) for in-hospital mortality; mean length of stay (LOS) differences and LOS ratios (95% CI) were 1.8 days and 1.24 (1.23-1.25) and 5.1 days and 1.57 (1.54-1.59); and mean cost differences and cost ratios were $7163 and 1.35 (1.34-1.36) and $19 127 and 1.78 (1.75-1.81) (all P < .001). During the 30 days postdischarge, readmission LOS was ≥6% longer for AKI vs no AKI and HA-AKI vs CA-AKI; outpatient costs were ≥41% higher for HA-AKI vs CA-AKI or no AKI. Only 30-day new dialysis (among patients without index hospitalization dialysis) had similar odds for HA-AKI vs CA-AKI (2.37-2.8 times higher for AKI, HA-AKI, or CA-AKI vs no AKI). Discussion: Among inpatients with COVID-19, HA-AKI had higher excess mortality, HRU, and costs than CA-AKI. Other studies suggest that interventions to prevent HA-AKI could decrease excess morbidity, HRU, and costs among inpatients with COVID-19. Conclusions: In adjusted models among COVID-19 inpatients, AKI, especially HA-AKI, was associated with significantly higher mortality, HRU, and costs during index admission, and higher dialysis and longer readmission LOS during the 30 days postdischarge. These findings support implementation of interventions to prevent HA-AKI in COVID-19 patients.
背景:在住院的COVID-19患者中,急性肾损伤(AKI)与较高的死亡率相关,但缺乏与AKI、社区获得性AKI (CA-AKI)和医院获得性AKI (HA-AKI)相关的医疗资源利用率(HRU)和成本的数据。目的:量化COVID-19住院患者AKI、CA-AKI和HA-AKI的负担。方法:本回顾性队列研究纳入了2020年4月1日至10月31日在Premier PINC AI™医疗保健数据库中从美国医院出院的COVID-19住院患者,根据ICD-10-CM诊断代码分类为AKI、CA-AKI、HA-AKI或无AKI。在指数(初始)住院期间和出院后30天评估结果。结果:208583例COVID-19住院患者中,有AKI、CA-AKI和HA-AKI的分别占30%、25%和5%,其中接受透析治疗的分别占10%、7%和23%。HA-AKI的额外死亡率、HRU和费用高于CA-AKI。在调整后的模型中,对于AKI患者与无AKI患者、HA-AKI患者与CA-AKI患者,重症监护病房使用的优势比(or) (95% CI)分别为3.70(3.61-3.79)和4.11(3.92-4.31),院内死亡率的优势比(or) (95% CI)分别为3.52(3.41-3.63)和2.64 (2.52-2.78);平均住院时间(LOS)差异和LOS比值(95% CI)分别为1.8天和1.24天(1.23-1.25)和5.1天和1.57天(1.54-1.59);平均成本差异和成本比分别为7163美元和1.35美元(1.34-1.36)和19 127美元和1.78美元(1.75-1.81)(P <措施)。出院后30天,AKI组与无AKI组、HA-AKI组与CA-AKI组再入院LOS延长≥6%;HA-AKI患者的门诊费用比CA-AKI患者或无AKI患者高41%以上。只有30天新透析(在没有住院透析指标的患者中)发生HA-AKI与CA-AKI的几率相似(AKI、HA-AKI或CA-AKI与无AKI的几率高2.37-2.8倍)。讨论:在COVID-19住院患者中,HA-AKI的超额死亡率、HRU和成本高于CA-AKI。其他研究表明,预防HA-AKI的干预措施可以降低COVID-19住院患者的超额发病率、HRU和成本。结论:在调整后的COVID-19住院患者模型中,AKI,特别是HA-AKI,与指数入院期间的死亡率、HRU和成本显著升高,以及出院后30天内透析率升高和再入院LOS延长相关。这些发现支持在COVID-19患者中实施预防HA-AKI的干预措施。
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引用次数: 0
Outcomes, Healthcare Resource Utilization, and Costs of Overall, Community-Acquired, and Hospital-Acquired Acute Kidney Injury in COVID-19 Patients. 新冠肺炎患者的总体、社区获得性和住院获得性急性肾损伤的结果、医疗资源利用和成本。
Q2 ECONOMICS Pub Date : 2023-02-23 eCollection Date: 2023-01-01 DOI: 10.36469/001c.57651
Jay L Koyner, Rachel H Mackey, Ning A Rosenthal, Leslie A Carabuena, J Patrick Kampf, Paul McPherson, Toni Rodriguez, Aarti Sanghani, Julien Textoris

Background: In hospitalized patients with COVID-19, acute kidney injury (AKI) is associated with higher mortality, but data are lacking on healthcare resource utilization (HRU) and costs related to AKI, community-acquired AKI (CA-AKI), and hospital-acquired AKI (HA-AKI). Objectives: To quantify the burden of AKI, CA-AKI, and HA-AKI among inpatients with COVID-19. Methods: This retrospective cohort study included inpatients with COVID-19 discharged from US hospitals in the Premier PINC AI™ Healthcare Database April 1-October 31, 2020, categorized as AKI, CA-AKI, HA-AKI, or no AKI by ICD-10-CM diagnosis codes. Outcomes were assessed during index (initial) hospitalization and 30 days postdischarge. Results: Among 208 583 COVID-19 inpatients, 30%, 25%, and 5% had AKI, CA-AKI, and HA-AKI, of whom 10%, 7%, and 23% received dialysis, respectively. Excess mortality, HRU, and costs were greater for HA-AKI than CA-AKI. In adjusted models, for patients with AKI vs no AKI and HA-AKI vs CA-AKI, odds ratios (ORs) (95% CI) were 3.70 (3.61-3.79) and 4.11 (3.92-4.31) for intensive care unit use and 3.52 (3.41-3.63) and 2.64 (2.52-2.78) for in-hospital mortality; mean length of stay (LOS) differences and LOS ratios (95% CI) were 1.8 days and 1.24 (1.23-1.25) and 5.1 days and 1.57 (1.54-1.59); and mean cost differences and cost ratios were $7163 and 1.35 (1.34-1.36) and $19 127 and 1.78 (1.75-1.81) (all P < .001). During the 30 days postdischarge, readmission LOS was ≥6% longer for AKI vs no AKI and HA-AKI vs CA-AKI; outpatient costs were ≥41% higher for HA-AKI vs CA-AKI or no AKI. Only 30-day new dialysis (among patients without index hospitalization dialysis) had similar odds for HA-AKI vs CA-AKI (2.37-2.8 times higher for AKI, HA-AKI, or CA-AKI vs no AKI). Discussion: Among inpatients with COVID-19, HA-AKI had higher excess mortality, HRU, and costs than CA-AKI. Other studies suggest that interventions to prevent HA-AKI could decrease excess morbidity, HRU, and costs among inpatients with COVID-19. Conclusions: In adjusted models among COVID-19 inpatients, AKI, especially HA-AKI, was associated with significantly higher mortality, HRU, and costs during index admission, and higher dialysis and longer readmission LOS during the 30 days postdischarge. These findings support implementation of interventions to prevent HA-AKI in COVID-19 patients.

背景:在新冠肺炎住院患者中,急性肾损伤(AKI)与较高的死亡率相关,但缺乏医疗资源利用(HRU)和与AKI、社区获得性AKI(CA-AKI)和医院需要性AKI相关的成本的数据。目的:量化新冠肺炎住院患者AKI、CA-AKI和HA-AKI的负担。方法:这项回顾性队列研究包括了从美国派米雷医院出院的新冠肺炎住院患者™ 医疗保健数据库2020年4月1日至10月31日,根据ICD-10-CM诊断代码分类为AKI、CA-AKI、HA-AKI或无AKI。在指数(初始)住院期间和出院后30天评估结果。结果:208 583名新冠肺炎住院患者,30%、25%和5%患有AKI、CA-AKI和HA-AKI,其中分别有10%、7%和23%接受透析。HA-AKI的超额死亡率、HRU和成本高于CA-AKI。在调整后的模型中,对于有AKI与无AKI和HA-AKI与CA-AKI的患者,重症监护室使用的比值比(OR)(95%CI)分别为3.70(3.61-3.79)和4.11(3.92-4.31),住院死亡率分别为3.52(3.41-3.63)和2.64(2.52-2.78);平均住院时间(LOS)差异和LOS比率(95%CI)分别为1.8天和1.24(1.23-1.25)、5.1天和1.57(1.54-1.59);平均成本差异和成本比率分别为7163美元和1.35美元(1.34-1.36)和19美元 127和1.78(1.75-1.81)(均P 讨论:在新冠肺炎住院患者中,HA-AKI的超额死亡率、HRU和费用高于CA-AKI。其他研究表明,预防HA-AKI的干预措施可以降低新冠肺炎住院患者的超额发病率、HRU和费用。结论:在新冠肺炎住院患者的调整模型中,AKI,尤其是HA-AKI,与指数入院期间显著更高的死亡率、HRU和费用,以及出院后30天内更高的透析和更长的再入院LOS相关。这些发现支持在新冠肺炎患者中实施预防HA-AKI的干预措施。
{"title":"Outcomes, Healthcare Resource Utilization, and Costs of Overall, Community-Acquired, and Hospital-Acquired Acute Kidney Injury in COVID-19 Patients.","authors":"Jay L Koyner, Rachel H Mackey, Ning A Rosenthal, Leslie A Carabuena, J Patrick Kampf, Paul McPherson, Toni Rodriguez, Aarti Sanghani, Julien Textoris","doi":"10.36469/001c.57651","DOIUrl":"10.36469/001c.57651","url":null,"abstract":"<p><p><b>Background:</b> In hospitalized patients with COVID-19, acute kidney injury (AKI) is associated with higher mortality, but data are lacking on healthcare resource utilization (HRU) and costs related to AKI, community-acquired AKI (CA-AKI), and hospital-acquired AKI (HA-AKI). <b>Objectives:</b> To quantify the burden of AKI, CA-AKI, and HA-AKI among inpatients with COVID-19. <b>Methods:</b> This retrospective cohort study included inpatients with COVID-19 discharged from US hospitals in the Premier PINC AI™ Healthcare Database April 1-October 31, 2020, categorized as AKI, CA-AKI, HA-AKI, or no AKI by ICD-10-CM diagnosis codes. Outcomes were assessed during index (initial) hospitalization and 30 days postdischarge. <b>Results:</b> Among 208 583 COVID-19 inpatients, 30%, 25%, and 5% had AKI, CA-AKI, and HA-AKI, of whom 10%, 7%, and 23% received dialysis, respectively. Excess mortality, HRU, and costs were greater for HA-AKI than CA-AKI. In adjusted models, for patients with AKI vs no AKI and HA-AKI vs CA-AKI, odds ratios (ORs) (95% CI) were 3.70 (3.61-3.79) and 4.11 (3.92-4.31) for intensive care unit use and 3.52 (3.41-3.63) and 2.64 (2.52-2.78) for in-hospital mortality; mean length of stay (LOS) differences and LOS ratios (95% CI) were 1.8 days and 1.24 (1.23-1.25) and 5.1 days and 1.57 (1.54-1.59); and mean cost differences and cost ratios were $7163 and 1.35 (1.34-1.36) and $19 127 and 1.78 (1.75-1.81) (all <i>P</i> < .001). During the 30 days postdischarge, readmission LOS was ≥6% longer for AKI vs no AKI and HA-AKI vs CA-AKI; outpatient costs were ≥41% higher for HA-AKI vs CA-AKI or no AKI. Only 30-day new dialysis (among patients without index hospitalization dialysis) had similar odds for HA-AKI vs CA-AKI (2.37-2.8 times higher for AKI, HA-AKI, or CA-AKI vs no AKI). <b>Discussion:</b> Among inpatients with COVID-19, HA-AKI had higher excess mortality, HRU, and costs than CA-AKI. Other studies suggest that interventions to prevent HA-AKI could decrease excess morbidity, HRU, and costs among inpatients with COVID-19. <b>Conclusions:</b> In adjusted models among COVID-19 inpatients, AKI, especially HA-AKI, was associated with significantly higher mortality, HRU, and costs during index admission, and higher dialysis and longer readmission LOS during the 30 days postdischarge. These findings support implementation of interventions to prevent HA-AKI in COVID-19 patients.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 1","pages":"31-40"},"PeriodicalIF":0.0,"publicationDate":"2023-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9961448/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10806767","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Outcomes, Healthcare Resource Utilization, and Costs of Overall, Community-Acquired, and Hospital-Acquired Acute Kidney Injury in COVID-19 Patients COVID-19患者整体、社区获得性和医院获得性急性肾损伤的结局、医疗资源利用和成本
Q2 ECONOMICS Pub Date : 2023-02-23 DOI: 10.36469/jheor.2023.
Jay Koyner, Rachel Mackey, Ning Rosenthal, Leslie Carabuena, J. Patrick Kampf, Paul McPherson, Toni Rodriguez, Aarti Sanghani, Julien Textures
Background: In hospitalized patients with COVID-19, acute kidney injury (AKI) is associated with higher mortality, but data are lacking on healthcare resource utilization (HRU) and costs related to AKI, community-acquired AKI (CA-AKI), and hospital-acquired AKI (HA-AKI). Objectives: To quantify the burden of AKI, CA-AKI, and HA-AKI among inpatients with COVID-19. Methods: This retrospective cohort study included inpatients with COVID-19 discharged from US hospitals in the Premier PINC AI™ Healthcare Database April 1–October 31, 2020, categorized as AKI, CA-AKI, HA-AKI, or no AKI by ICD-10-CM diagnosis codes. Outcomes were assessed during index (initial) hospitalization and 30 days postdischarge. Results: Among 208 583 COVID-19 inpatients, 30%, 25%, and 5% had AKI, CA-AKI, and HA-AKI, of whom 10%, 7%, and 23% received dialysis, respectively. Excess mortality, HRU, and costs were greater for HA-AKI than CA-AKI. In adjusted models, for patients with AKI vs no AKI and HA-AKI vs CA-AKI, odds ratios (ORs) (95% CI) were 3.70 (3.61-3.79) and 4.11 (3.92-4.31) for intensive care unit use and 3.52 (3.41-3.63) and 2.64 (2.52-2.78) for in-hospital mortality; mean length of stay (LOS) differences and LOS ratios (95% CI) were 1.8 days and 1.24 (1.23-1.25) and 5.1 days and 1.57 (1.54-1.59); and mean cost differences and cost ratios were $7163 and 1.35 (1.34-1.36) and $19 127 and 1.78 (1.75-1.81) (all P < .001). During the 30 days postdischarge, readmission LOS was ≥6% longer for AKI vs no AKI and HA-AKI vs CA-AKI; outpatient costs were ≥41% higher for HA-AKI vs CA-AKI or no AKI. Only 30-day new dialysis (among patients without index hospitalization dialysis) had similar odds for HA-AKI vs CA-AKI (2.37-2.8 times higher for AKI, HA-AKI, or CA-AKI vs no AKI). Discussion: Among inpatients with COVID-19, HA-AKI had higher excess mortality, HRU, and costs than CA-AKI. Other studies suggest that interventions to prevent HA-AKI could decrease excess morbidity, HRU, and costs among inpatients with COVID-19. Conclusions: In adjusted models among COVID-19 inpatients, AKI, especially HA-AKI, was associated with significantly higher mortality, HRU, and costs during index admission, and higher dialysis and longer readmission LOS during the 30 days postdischarge. These findings support implementation of interventions to prevent HA-AKI in COVID-19 patients.
背景:在住院的COVID-19患者中,急性肾损伤(AKI)与较高的死亡率相关,但缺乏与AKI、社区获得性AKI (CA-AKI)和医院获得性AKI (HA-AKI)相关的医疗资源利用率(HRU)和成本的数据。目的:量化COVID-19住院患者AKI、CA-AKI和HA-AKI的负担。方法:本回顾性队列研究纳入了2020年4月1日至10月31日在Premier PINC AI™医疗保健数据库中从美国医院出院的COVID-19住院患者,根据ICD-10-CM诊断代码分类为AKI、CA-AKI、HA-AKI或无AKI。在指数(初始)住院期间和出院后30天评估结果。结果:208583例COVID-19住院患者中,有AKI、CA-AKI和HA-AKI的分别占30%、25%和5%,其中接受透析治疗的分别占10%、7%和23%。HA-AKI的额外死亡率、HRU和费用高于CA-AKI。在调整后的模型中,对于AKI患者与无AKI患者、HA-AKI患者与CA-AKI患者,重症监护病房使用的优势比(or) (95% CI)分别为3.70(3.61-3.79)和4.11(3.92-4.31),院内死亡率的优势比(or) (95% CI)分别为3.52(3.41-3.63)和2.64 (2.52-2.78);平均住院时间(LOS)差异和LOS比值(95% CI)分别为1.8天和1.24天(1.23-1.25)和5.1天和1.57天(1.54-1.59);平均成本差异和成本比分别为7163美元和1.35美元(1.34-1.36)和19 127美元和1.78美元(1.75-1.81)(P <措施)。出院后30天,AKI组与无AKI组、HA-AKI组与CA-AKI组再入院LOS延长≥6%;HA-AKI患者的门诊费用比CA-AKI患者或无AKI患者高41%以上。只有30天新透析(在没有住院透析指标的患者中)发生HA-AKI与CA-AKI的几率相似(AKI、HA-AKI或CA-AKI与无AKI的几率高2.37-2.8倍)。讨论:在COVID-19住院患者中,HA-AKI的超额死亡率、HRU和成本高于CA-AKI。其他研究表明,预防HA-AKI的干预措施可以降低COVID-19住院患者的超额发病率、HRU和成本。结论:在调整后的COVID-19住院患者模型中,AKI,特别是HA-AKI,与指数入院期间的死亡率、HRU和成本显著升高,以及出院后30天内透析率升高和再入院LOS延长相关。这些发现支持在COVID-19患者中实施预防HA-AKI的干预措施。
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引用次数: 0
PROMETHEUS: Long-Term Exacerbation and Mortality Benefits of Implementing Single-Inhaler Triple Therapy in the US COPD Population. PROMETHEUS:在美国慢性阻塞性肺病患者中实施单吸入器三联疗法的长期恶化和死亡率效益。
Q2 ECONOMICS Pub Date : 2023-01-24 eCollection Date: 2023-01-01 DOI: 10.36469/001c.55635
Gerard Criner, Fernando Martinez, Hitesh Gandhi, Bruce Pyenson, Norbert Feigler, Matthew Emery, Umang Gupta, Muthiah Vaduganathan

Background: The US population includes 24 million to 29 million people with diagnosed and undiagnosed chronic obstructive pulmonary disease (COPD). Studies have demonstrated the safety and efficacy of single-inhaler triple therapy (SITT) in reducing COPD exacerbations. Long-term population implications of SITT use have not been quantified. Objectives: This simulation-based projection aimed to estimate the potential impact of widespread SITT use on the US COPD population. Methods: Exacerbation and all-cause mortality reductions reported in the Efficacy and Safety of Triple Therapy in Obstructive Lung Disease trial (ETHOS; NCT02465567) were used to project clinical outcomes in US patients meeting ETHOS trial eligibility criteria (ETHOS-Eligible) and patients meeting a practical definition of SITT eligibility (Expanded ETHOS-Eligible). The US COPD population was modeled with 1000 simulations of patient progression over 10 years. Agent characteristics were based on literature and claims analysis of the 2016-2018 Medicare 100% fee-for-service and IBM MarketScan® databases. Agent annual characteristics reflected incident cases, changes in COPD severity, treatment, mortality, and exacerbations under status quo treatment patterns and scenarios for the adoption of SITT. The scenarios assumed the reduced exacerbation and mortality rates associated with SITT according to ETHOS trial outcomes mean values. Results: Higher than current SITT adoption over 10 years would be expected to substantially reduce COPD exacerbation-associated hospitalizations by 2 million. Applying mean improvements reported in ETHOS for SITT would extend average patient life expectancy 2.2 years for ETHOS-Eligible patients and 1.7 years for Expanded ETHOS-Eligible patients. The number needed to treat to extend the average patient life by 1 year was 8 for the ETHOS-Eligible population and 10 for the Expanded ETHOS-Eligible population. Discussion: Widespread SITT adoption may be impeded by competitive pressures from generic treatments and nonadherence, and efficacy observed in clinical trials may not occur in real-world populations. Conclusions: Assuming ETHOS treatment effects and adherence translate to clinical practice, higher than current use of SITT can substantially reduce COPD exacerbations and hospitalizations and extend survival. These results should be viewed cautiously, because the improved outcomes for SITT in the ETHOS final retrieved vital statistics data were not statistically significant for all comparator therapy groups.

背景:美国有 2400 万至 2900 万确诊和未确诊的慢性阻塞性肺病(COPD)患者。研究表明,单吸入器三联疗法(SITT)在减少慢性阻塞性肺病恶化方面具有安全性和有效性。单次吸入器三联疗法对人群的长期影响尚未量化。目标:该模拟预测旨在估算广泛使用 SITT 对美国 COPD 患者的潜在影响。方法:利用阻塞性肺病三联疗法的有效性和安全性试验(ETHOS;NCT02465567)中报告的病情恶化和全因死亡率降低情况,预测符合 ETHOS 试验资格标准(ETHOS-Eligible)的美国患者和符合 SITT 资格实用定义(扩展 ETHOS-Eligible)的患者的临床结果。对美国慢性阻塞性肺病人群进行了建模,模拟了 1000 次患者在 10 年内的病情进展情况。代理特征基于对 2016-2018 年医疗保险 100% 付费服务和 IBM MarketScan® 数据库的文献和索赔分析。代理人的年度特征反映了事件病例、慢性阻塞性肺病严重程度的变化、治疗、死亡率以及在现状治疗模式和采用 SITT 情景下的病情加重情况。根据 ETHOS 试验结果的平均值,假设采用 SITT 会降低恶化率和死亡率。结果:如果在 10 年内采用高于目前水平的 SITT,预计将大幅减少 200 万例慢性阻塞性肺疾病恶化相关的住院治疗。应用 ETHOS 报告的 SITT 平均改善效果,将使符合 ETHOS 条件的患者平均预期寿命延长 2.2 年,使符合扩展 ETHOS 条件的患者平均预期寿命延长 1.7 年。将患者平均寿命延长 1 年所需的治疗人数在符合 ETHOS 条件的人群中为 8 人,在符合 ETHOS 扩展条件的人群中为 10 人。讨论:SITT的广泛应用可能会受到来自非专利治疗的竞争压力和不依从性的阻碍,临床试验中观察到的疗效可能不会在实际人群中出现。结论:假定 ETHOS 的治疗效果和依从性转化为临床实践,高于当前水平的 SITT 使用率可大幅减少慢性阻塞性肺疾病的恶化和住院,并延长生存期。但应谨慎看待这些结果,因为在 ETHOS 最终检索到的生命统计数据中,SITT 对所有比较治疗组的治疗效果改善并不具有统计学意义。
{"title":"PROMETHEUS: Long-Term Exacerbation and Mortality Benefits of Implementing Single-Inhaler Triple Therapy in the US COPD Population.","authors":"Gerard Criner, Fernando Martinez, Hitesh Gandhi, Bruce Pyenson, Norbert Feigler, Matthew Emery, Umang Gupta, Muthiah Vaduganathan","doi":"10.36469/001c.55635","DOIUrl":"10.36469/001c.55635","url":null,"abstract":"<p><p><b>Background:</b> The US population includes 24 million to 29 million people with diagnosed and undiagnosed chronic obstructive pulmonary disease (COPD). Studies have demonstrated the safety and efficacy of single-inhaler triple therapy (SITT) in reducing COPD exacerbations. Long-term population implications of SITT use have not been quantified. <b>Objectives:</b> This simulation-based projection aimed to estimate the potential impact of widespread SITT use on the US COPD population. <b>Methods:</b> Exacerbation and all-cause mortality reductions reported in the Efficacy and Safety of Triple Therapy in Obstructive Lung Disease trial (ETHOS; NCT02465567) were used to project clinical outcomes in US patients meeting ETHOS trial eligibility criteria (ETHOS-Eligible) and patients meeting a practical definition of SITT eligibility (Expanded ETHOS-Eligible). The US COPD population was modeled with 1000 simulations of patient progression over 10 years. Agent characteristics were based on literature and claims analysis of the 2016-2018 Medicare 100% fee-for-service and IBM MarketScan<sup>®</sup> databases. Agent annual characteristics reflected incident cases, changes in COPD severity, treatment, mortality, and exacerbations under status quo treatment patterns and scenarios for the adoption of SITT. The scenarios assumed the reduced exacerbation and mortality rates associated with SITT according to ETHOS trial outcomes mean values. <b>Results:</b> Higher than current SITT adoption over 10 years would be expected to substantially reduce COPD exacerbation-associated hospitalizations by 2 million. Applying mean improvements reported in ETHOS for SITT would extend average patient life expectancy 2.2 years for ETHOS-Eligible patients and 1.7 years for Expanded ETHOS-Eligible patients. The number needed to treat to extend the average patient life by 1 year was 8 for the ETHOS-Eligible population and 10 for the Expanded ETHOS-Eligible population. <b>Discussion:</b> Widespread SITT adoption may be impeded by competitive pressures from generic treatments and nonadherence, and efficacy observed in clinical trials may not occur in real-world populations. <b>Conclusions:</b> Assuming ETHOS treatment effects and adherence translate to clinical practice, higher than current use of SITT can substantially reduce COPD exacerbations and hospitalizations and extend survival. These results should be viewed cautiously, because the improved outcomes for SITT in the ETHOS final retrieved vital statistics data were not statistically significant for all comparator therapy groups.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 1","pages":"20-27"},"PeriodicalIF":0.0,"publicationDate":"2023-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9879267/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10666848","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
PROMETHEUS: Long-Term Exacerbation and Mortality Benefits of Implementing Single-Inhaler Triple Therapy in the US COPD Population PROMETHEUS:在美国COPD人群中实施单吸入器三联治疗的长期恶化和死亡率益处
Q2 ECONOMICS Pub Date : 2023-01-24 DOI: 10.36469/jheor.2023.55635
Criner Gerard, Fernando Martinez, Hitesh Gandhi, Bruce Pyenson, Norbert Feigler, Matthew Emery, Umang Gupta, Muthiah Vaduganathan
Background: The US population includes 24 million to 29 million people with diagnosed and undiagnosed chronic obstructive pulmonary disease (COPD). Studies have demonstrated the safety and efficacy of single-inhaler triple therapy (SITT) in reducing COPD exacerbations. Long-term population implications of SITT use have not been quantified. Objectives: This simulation-based projection aimed to estimate the potential impact of widespread SITT use on the US COPD population. Methods: Exacerbation and all-cause mortality reductions reported in the Efficacy and Safety of Triple Therapy in Obstructive Lung Disease trial (ETHOS; NCT02465567) were used to project clinical outcomes in US patients meeting ETHOS trial eligibility criteria (ETHOS-Eligible) and patients meeting a practical definition of SITT eligibility (Expanded ETHOS-Eligible). The US COPD population was modeled with 1000 simulations of patient progression over 10 years. Agent characteristics were based on literature and claims analysis of the 2016-2018 Medicare 100% fee-for-service and IBM MarketScan® databases. Agent annual characteristics reflected incident cases, changes in COPD severity, treatment, mortality, and exacerbations under status quo treatment patterns and scenarios for the adoption of SITT. The scenarios assumed the reduced exacerbation and mortality rates associated with SITT according to ETHOS trial outcomes mean values. Results: Higher than current SITT adoption over 10 years would be expected to substantially reduce COPD exacerbation-associated hospitalizations by 2 million. Applying mean improvements reported in ETHOS for SITT would extend average patient life expectancy 2.2 years for ETHOS-Eligible patients and 1.7 years for Expanded ETHOS-Eligible patients. The number needed to treat to extend the average patient life by 1 year was 8 for the ETHOS-Eligible population and 10 for the Expanded ETHOS-Eligible population. Discussion: Widespread SITT adoption may be impeded by competitive pressures from generic treatments and nonadherence, and efficacy observed in clinical trials may not occur in real-world populations. Conclusions: Assuming ETHOS treatment effects and adherence translate to clinical practice, higher than current use of SITT can substantially reduce COPD exacerbations and hospitalizations and extend survival. These results should be viewed cautiously, because the improved outcomes for SITT in the ETHOS final retrieved vital statistics data were not statistically significant for all comparator therapy groups.
背景:美国人口包括2400万至2900万确诊和未确诊的慢性阻塞性肺疾病(COPD)患者。研究已经证明了单吸入器三联疗法(SITT)在减少COPD恶化方面的安全性和有效性。使用SITT对人口的长期影响尚未量化。目的:这项基于模拟的预测旨在评估SITT广泛使用对美国COPD人群的潜在影响。方法:阻塞性肺疾病三联疗法疗效和安全性试验(ETHOS;NCT02465567)用于预测符合ETHOS试验资格标准(ETHOS- eligible)和符合SITT资格实际定义(扩展ETHOS- eligible)的美国患者的临床结果。美国慢性阻塞性肺病人群在10年内进行了1000次患者进展模拟。代理人特征基于2016-2018年医疗保险100%按服务收费和IBM MarketScan®数据库的文献和索赔分析。代理人的年度特征反映了在现状治疗模式和采用SITT的情况下的发病率、COPD严重程度、治疗、死亡率和恶化的变化。根据ETHOS试验结果的平均值,假设与SITT相关的恶化和死亡率降低。结果:高于目前SITT的采用率超过10年,预计将大大减少与COPD恶化相关的住院人数200万。应用ETHOS报告的SITT平均改善将使符合ETHOS条件的患者的平均预期寿命延长2.2年,扩展符合ETHOS条件的患者的平均预期寿命延长1.7年。在符合ethos条件的人群中,需要治疗的患者平均寿命延长1年的人数为8人,而在扩大的符合ethos条件的人群中,需要治疗的人数为10人。讨论:SITT的广泛采用可能受到仿制治疗和不依从性的竞争压力的阻碍,并且在临床试验中观察到的疗效可能不会出现在现实人群中。结论:假设ETHOS治疗效果和依从性转化为临床实践,高于目前SITT的使用可以显著减少COPD恶化和住院,延长生存期。这些结果应该谨慎看待,因为在ETHOS最终检索的生命统计数据中,SITT的改善结果在所有比较治疗组中都没有统计学意义。
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引用次数: 1
Economic Burden of HIV in a Commercially Insured Population in the United States. 美国商业保险人群中艾滋病毒的经济负担。
IF 2.3 Q2 ECONOMICS Pub Date : 2023-01-19 eCollection Date: 2023-01-01 DOI: 10.36469/001c.56928
Cindy Y Chen, Prina Donga, Alicia K Campbell, Babafemi Taiwo

Background: With advances in antiretroviral therapy (ART), people with HIV infection are living longer. Pre-exposure prophylaxis (PrEP) to reduce HIV infection risk continues to be underutilized in high-risk individuals. Recent data on economic burden for patients with newly diagnosed HIV-1 or initiated with PrEP are limited. Objectives: To assess characteristics, healthcare resource utilization (HRU), and costs among adults and adolescents either with newly diagnosed HIV-1 or initiated with PrEP. Methods: This retrospective observational study utilized data from the IBM MarketScan® Commercial Claims and Encounters database. Adults with newly diagnosed HIV-1 or those initiated with PrEP were included (index date was the first HIV diagnosis or PrEP prescription, respectively, between January 1, 2016, and April 30, 2021). Corresponding cohorts of adolescents were considered exploratory. Descriptive analyses were conducted to assess baseline demographics and clinical characteristics, and all-cause and HIV-related HRU and costs per patient per month (PPPM) during follow-up. Results: Data from 18 154 adults and 220 adolescents with newly diagnosed HIV and 34 123 adults and 175 adolescents initiated with PrEP were included. Approximately 70% of adolescents and 9% of adults receiving PrEP were female. Baseline depression/anxiety was present in 16.1% and 24.6% of adults and 14.5% and 45.1% of adolescents in the HIV and PrEP cohorts, respectively. Substance abuse in the HIV and PrEP cohorts, respectively, was reported in 10.1% and 7.0% of adults, and 2.7% and 17.7% of adolescents. During follow-up, among adults with newly diagnosed HIV, mean (SD) total all-cause and HIV-related PPPM costs were $2657 ($5954) and $1497 ($4463), respectively; pharmacy costs represented 47% of all-cause costs and 67% of HIV-related costs, but only 37% of patients had an HIV-related prescription. All-cause costs PPPM for adults with PrEP were $1761 ($1938), with pharmacy costs accounting for 71%. Conclusions: Despite advances in ART, patients with newly diagnosed HIV and at-risk patients receiving PrEP continue to incur HRU costs. The chronic nature of HIV warrants further exploration of factors contributing to disease burden and opportunities to improve prevention strategies.

背景:随着抗逆转录病毒疗法(ART)的进步,HIV 感染者的寿命越来越长。在高危人群中,旨在降低 HIV 感染风险的暴露前预防疗法(PrEP)仍未得到充分利用。有关新诊断出的 HIV-1 患者或开始使用 PrEP 的患者的经济负担的最新数据很有限。研究目的评估新确诊 HIV-1 患者或开始使用 PrEP 的成人和青少年的特征、医疗资源利用率 (HRU) 和成本。方法:这项回顾性观察研究利用了 IBM MarketScan® 商业索赔和遭遇数据库中的数据。研究对象包括新诊断出 HIV-1 的成人或开始使用 PrEP 的成人(索引日期分别为 2016 年 1 月 1 日至 2021 年 4 月 30 日期间首次诊断出 HIV 或开具 PrEP 处方的日期)。相应的青少年队列被认为是探索性的。我们进行了描述性分析,以评估基线人口统计学和临床特征,以及随访期间全因和 HIV 相关的 HRU 和每位患者每月的费用 (PPPM)。结果:研究纳入了 18 154 名成人和 220 名青少年新诊断出的 HIV 感染者的数据,以及 34 123 名成人和 175 名青少年开始使用 PrEP 的数据。在接受 PrEP 的青少年和成人中,约 70% 为女性,9% 为女性。在 HIV 感染者和 PrEP 群体中,分别有 16.1% 和 24.6% 的成年人和 14.5% 和 45.1% 的青少年患有抑郁症/焦虑症。在 HIV 和 PrEP 群体中,分别有 10.1% 和 7.0% 的成年人和 2.7% 和 17.7% 的青少年报告滥用药物。在随访期间,在新诊断出艾滋病毒的成年人中,平均(标度)全因成本和艾滋病毒相关 PPPM 总成本分别为 2657 美元(5954 美元)和 1497 美元(4463 美元);药房成本占全因成本的 47%,占艾滋病毒相关成本的 67%,但只有 37% 的患者拥有艾滋病毒相关处方。使用 PrEP 的成人的全因成本 PPPM 为 1761 美元(1938 美元),其中药房成本占 71%。结论:尽管抗逆转录病毒疗法取得了进步,但新诊断出的 HIV 患者和接受 PrEP 的高危患者仍需承担 HRU 费用。艾滋病毒的慢性特性要求我们进一步探讨造成疾病负担的因素以及改进预防策略的机会。
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引用次数: 0
Economic Burden of HIV in a Commercially Insured Population in the United States 美国商业保险人群中HIV的经济负担
Q2 ECONOMICS Pub Date : 2023-01-19 DOI: 10.36469/jheor.2023.56928
Cindy Chen, Prina Donga, Alicia Campbell, Babafemi Taiwo
Background: With advances in antiretroviral therapy (ART), people with HIV infection are living longer. Pre-exposure prophylaxis (PrEP) to reduce HIV infection risk continues to be underutilized in high-risk individuals. Recent data on economic burden for patients with newly diagnosed HIV-1 or initiated with PrEP are limited. Objectives: To assess characteristics, healthcare resource utilization (HRU), and costs among adults and adolescents either with newly diagnosed HIV-1 or initiated with PrEP. Methods: This retrospective observational study utilized data from the IBM MarketScan® Commercial Claims and Encounters database. Adults with newly diagnosed HIV-1 or those initiated with PrEP were included (index date was the first HIV diagnosis or PrEP prescription, respectively, between January 1, 2016, and April 30, 2021). Corresponding cohorts of adolescents were considered exploratory. Descriptive analyses were conducted to assess baseline demographics and clinical characteristics, and all-cause and HIV-related HRU and costs per patient per month (PPPM) during follow-up. Results: Data from 18 154 adults and 220 adolescents with newly diagnosed HIV and 34 123 adults and 175 adolescents initiated with PrEP were included. Approximately 70% of adolescents and 9% of adults receiving PrEP were female. Baseline depression/anxiety was present in 16.1% and 24.6% of adults and 14.5% and 45.1% of adolescents in the HIV and PrEP cohorts, respectively. Substance abuse in the HIV and PrEP cohorts, respectively, was reported in 10.1% and 7.0% of adults, and 2.7% and 17.7% of adolescents. During follow-up, among adults with newly diagnosed HIV, mean (SD) total all-cause and HIV-related PPPM costs were $2657 ($5954) and $1497 ($4463), respectively; pharmacy costs represented 47% of all-cause costs and 67% of HIV-related costs, but only 37% of patients had an HIV-related prescription. All-cause costs PPPM for adults with PrEP were $1761 ($1938), with pharmacy costs accounting for 71%. Conclusions: Despite advances in ART, patients with newly diagnosed HIV and at-risk patients receiving PrEP continue to incur HRU costs. The chronic nature of HIV warrants further exploration of factors contributing to disease burden and opportunities to improve prevention strategies.
背景:随着抗逆转录病毒治疗(ART)的进展,艾滋病毒感染者的寿命延长了。在高危人群中,减少艾滋病毒感染风险的暴露前预防(PrEP)仍然没有得到充分利用。最近关于新诊断的HIV-1患者或开始使用PrEP的患者经济负担的数据有限。目的:评估新诊断HIV-1或开始PrEP的成人和青少年的特征、医疗资源利用率(HRU)和成本。方法:本回顾性观察性研究利用了IBM MarketScan®商业索赔和遭遇数据库的数据。新诊断为HIV-1的成年人或开始使用PrEP的成年人被纳入(索引日期分别为2016年1月1日至2021年4月30日之间的首次HIV诊断或PrEP处方)。相应的青少年队列被认为是探索性的。进行描述性分析以评估基线人口统计学和临床特征,以及随访期间全因和hiv相关的HRU和每个患者每月的费用(PPPM)。结果:纳入18 154名成人和220名青少年新诊断艾滋病毒和34 123名成人和175名青少年开始PrEP的数据。大约70%接受PrEP的青少年和9%的成年人是女性。在HIV和PrEP队列中,分别有16.1%和24.6%的成年人以及14.5%和45.1%的青少年存在基线抑郁/焦虑。在HIV和PrEP队列中,分别有10.1%和7.0%的成年人以及2.7%和17.7%的青少年报告了药物滥用。在随访期间,在新诊断为HIV的成年人中,全因和HIV相关PPPM的平均(SD)总成本分别为2657美元(5954美元)和1497美元(4463美元);药费占全因费用的47%,占艾滋病毒相关费用的67%,但只有37%的患者有与艾滋病毒相关的处方。使用PrEP的成人的全因成本PPPM为1761美元(1938美元),其中药房成本占71%。结论:尽管抗逆转录病毒治疗取得了进展,但新诊断的艾滋病毒患者和接受PrEP的高危患者继续承担HRU费用。艾滋病毒的慢性性质需要进一步探索造成疾病负担的因素和改进预防战略的机会。
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引用次数: 0
Use of Minimal Residual Disease Status to Reduce Uncertainty in Estimating Long-term Survival Outcomes for Newly Diagnosed Multiple Myeloma Patients. 使用最小残留疾病状态来减少估计新诊断多发性骨髓瘤患者长期生存结果的不确定性。
Q2 ECONOMICS Pub Date : 2023-01-06 eCollection Date: 2023-01-01 DOI: 10.36469/001c.56072
Naomi van Hest, Peter Morten, Keith Stubbs, Nicola Trevor

Background: Demonstrating the cost-effectiveness of new treatments for multiple myeloma (MM) often relies on the extrapolation of overall survival (OS) trial data. This method can introduce uncertainty in long-term survival estimates if OS data are immature, as is often the case in newly diagnosed MM (NDMM). We explore the use of the relationship between minimal residual disease (MRD) status and OS to reduce uncertainty of long-term survival outcomes. Objectives: To evaluate if uncertainty in long-term modeled outcomes in NDMM is reduced using a response-based partitioned survival model (PSM), whereby patients were categorized as MRD-positive or -negative, relative to a standard PSM, when OS data are immature. Methods: Standard and response-based PSMs, estimating patient life-years (LYs) over a lifetime horizon, were developed for NDMM patients treated with bortezomib, thalidomide, and dexamethasone (BTd) with or without daratumumab as induction and consolidation therapy. In the standard PSM, LYs were determined by extrapolations from individual patient data from CASSIOPEIA. In the response-based PSM, survival was dependent on MRD status at the time of the response assessment via a landmark analysis. Cox-proportional hazard ratios from external sources and CASSIOPEIA informed the relationship for OS between MRD-positive and MRD-negative, and between patients receiving BTd and daratumumab plus BTd, respectively. Uncertainty was assessed by comparing LYs and OS extrapolations from deterministic and probabilistic analyses. Results: This response-based PSM demonstrated reduced uncertainty in long-term survival outcomes compared with the standard PSM (range across extrapolations of 3.4 and 7.7 LYs for daratumumab plus BTd and BTd, respectively, vs 14.8 and 11.8 LYs for the standard PSM). It also estimated a narrower interquartile range of LYs in the probabilistic analyses for the majority of parametric extrapolations. Discussion: Alternative methods to estimate long-term survival outcomes, such as a response-based PSM, can reduce uncertainty in modeling predictions around cost-effectiveness estimates for health technology assessment bodies and payers, thereby supporting faster market access for novel therapies with immature survival data. Conclusions: Use of MRD status in a response-based PSM reduces uncertainty in modeling long-term survival in patients with NDMM and provides a greater number of clinically plausible extrapolations compared with a standard PSM.

背景:证明多发性骨髓瘤(MM)新疗法的成本效益通常依赖于总生存期(OS)试验数据的外推。如果OS数据不成熟,这种方法可能会给长期生存估计带来不确定性,就像新诊断的MM (NDMM)一样。我们探索最小残留病(MRD)状态与OS之间的关系,以减少长期生存结果的不确定性。目的:评估使用基于反应的分区生存模型(PSM)是否减少了NDMM长期建模结果的不确定性,当OS数据不成熟时,相对于标准PSM,患者被分类为mrd阳性或阴性。方法:针对采用或不采用达拉单抗作为诱导和巩固治疗的波特佐米、沙利度胺和地塞米松(BTd)治疗的NDMM患者,开发了标准和基于反应的psm,估计患者生命年(LYs)。在标准PSM中,LYs是通过从CASSIOPEIA的个体患者数据推断确定的。在基于反应的PSM中,生存率取决于通过里程碑式分析进行反应评估时的MRD状态。来自外部来源和CASSIOPEIA的Cox-proportional风险比分别告知了mrd阳性和mrd阴性患者以及接受BTd和daratumumab加BTd患者之间的OS关系。通过比较确定性和概率分析的LYs和OS推断来评估不确定性。结果:与标准PSM相比,这种基于反应的PSM显示出长期生存结果的不确定性降低(daratumumab加BTd和BTd的外推范围分别为3.4和7.7 LYs,而标准PSM为14.8和11.8 LYs)。在大多数参数外推的概率分析中,它还估计了较窄的LYs的四分位数范围。讨论:评估长期生存结果的替代方法,如基于反应的PSM,可以减少卫生技术评估机构和付款人围绕成本效益估算建模预测的不确定性,从而支持具有不成熟生存数据的新疗法更快进入市场。结论:与标准PSM相比,在基于反应的PSM中使用MRD状态减少了NDMM患者长期生存模型的不确定性,并提供了更多临床可信的推断。
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引用次数: 0
Healthcare Resource Utilization and Costs Among Commercially Insured Patients With Advanced or Recurrent Endometrial Cancer Initiating First-Line Therapy in the United States 美国商业保险晚期或复发子宫内膜癌患者开始一线治疗的医疗资源利用和成本
Q2 ECONOMICS Pub Date : 2023-01-01 DOI: 10.36469/jheor.2023.88419
Monica Kobayashi, Jamie Garside, Joehl Nguyen
Background: Endometrial cancer (EC) represents a substantial economic burden for patients in the United States. Patients with advanced or recurrent EC have a much poorer prognosis than patients with early-stage EC. Data on healthcare resource utilization (HCRU) and costs for patients with advanced or recurrent EC specifically are lacking. Objectives: To describe HCRU and costs associated with first-line (1L) therapy for commercially insured patients with advanced or recurrent EC in the United States. Methods: This was a retrospective cohort study of adult patients with advanced or recurrent EC using the MarketScan® database. Treatment characteristics, HCRU, and costs were assessed from the first claim in the patient record for 1L therapy for advanced or recurrent EC (index) until initiation of a new anti-cancer therapy, disenrollment from the database, or the end of data availability. Baseline demographics were determined during the 12 months before the patient’s index date. Results: A total of 7932 patients were eligible for inclusion. Overall, mean age at index was 61 years, most patients (77.3%) had received prior surgery for EC, and the most common 1L regimen was carboplatin/paclitaxel (59.1%). During the observation period, most patients had at least one healthcare visit (all-cause, 99.9%; EC-related, 82.8%), most commonly outpatient visits (all-cause, 91.4%; EC-related, 68.7%). The highest mean (SD) costs (US dollars) were for inpatient hospitalization for both all-cause and EC-related events ($8396 [$15,130] and $9436 [$16,784], respectively). Total costs were higher for patients with a diagnosis of metastasis at baseline than for those without a diagnosis of metastasis. Discussion: For patients with advanced or recurrent EC in the United States, 1L therapy is associated with considerable HCRU and economic burden. They are particularly high for patients with metastatic disease. Conclusions: This study highlights the need for new cost-effective treatments for patients with newly diagnosed advanced or recurrent EC.
背景:子宫内膜癌(EC)对美国患者来说是一个巨大的经济负担。晚期或复发性EC患者的预后比早期EC患者差得多。缺乏关于晚期或复发性EC患者的医疗资源利用(HCRU)和费用的数据。目的:描述美国商业保险晚期或复发性EC患者的HCRU和与一线(1L)治疗相关的费用。方法:这是一项使用MarketScan®数据库的晚期或复发性EC成年患者的回顾性队列研究。治疗特征、HCRU和费用从患者记录中首次申请晚期或复发性EC的1L治疗(指数)开始评估,直到开始新的抗癌治疗、从数据库注销或数据可用性结束。基线人口统计数据在患者索引日期前的12个月内确定。结果:共有7932例患者符合纳入条件。总体而言,平均年龄为61岁,大多数患者(77.3%)之前接受过EC手术,最常见的1L方案是卡铂/紫杉醇(59.1%)。在观察期间,大多数患者至少进行过一次医疗保健访问(全因,99.9%;与ec相关,82.8%),最常见的门诊就诊(全因,91.4%;EC-related, 68.7%)。最高的平均(SD)费用(美元)是全因和ec相关事件的住院治疗(分别为8396美元[15,130美元]和9436美元[16,784美元])。基线诊断为转移的患者的总费用高于未诊断为转移的患者。讨论:对于美国晚期或复发性EC患者,1L治疗与相当大的HCRU和经济负担相关。对于患有转移性疾病的患者来说,它们尤其高。结论:这项研究强调了新诊断的晚期或复发性EC患者需要新的经济有效的治疗方法。
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引用次数: 0
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Journal of Health Economics and Outcomes Research
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