Cost-effectiveness of in-home automated external defibrillators for children with cardiac conditions associated with risk for sudden cardiac death.
- 作者列表："Haag MB","Hersh AR","Toffey DE","Sargent JA","Stecker EC","Heitner SB","Caughey AB","Balaji S
BACKGROUND:While children at high risk of sudden cardiac death (SCD) (>6% over 5 years) receive implanted cardioverter defibrillators (ICDs), there are no guidelines for those at lower risk. For children at intermediate risk of SCD (4-6% over 5 years), the utility and cost effectiveness of in-home automated external defibrillators (AED) are unclear. OBJECTIVE:To assess the cost effectiveness of in-home AED for children at intermediate risk of SCD. METHODS:Utilizing hypertrophic cardiomyopathy (HCM) as the proxy disease, a theoretical cohort of 1,550 ten-year old children with HCM was followed for 69 years. Baseline annual risk of SCD was 0.8%. Outcomes were sudden cardiac death (SCD), severe neurological morbidity (SNM), cost, and quality-adjusted life years (QALYs). Model inputs were derived from the literature, with a willingness-to-pay threshold of $100,000 per QALY. RESULTS:Among children at intermediate risk of SCD, in-home AED resulted in 31 fewer cases of SCD, but 3 more cases of SNM. There were 319 QALYs gained. Although costs were higher by $28 million, the incremental cost-effectiveness ratio was $86,458, below the willingness-to-pay threshold. CONCLUSIONS:For children at intermediate risk of SCD and HCM, in-home AED is cost effective, resulting in fewer deaths and increased QALYS for a cost below the willingness-to-pay threshold. These findings highlight the economic benefits of in-home AED utilization in this population.
背景: 虽然心脏性猝死 (SCD) 高危儿童 (5 岁以上> 6%) 接受了植入心律转复除颤器 (icd)，但没有针对低风险儿童的指南。对于 SCD 中等风险的儿童 (5 岁以上 4-6%)，家庭自动体外除颤器 (AED) 的效用和成本效益尚不清楚。 目的: 评估家庭 AED 治疗 SCD 中危儿童的成本效益。 方法: 以肥厚型心肌病 (HCM) 为代表疾病，对 1,550 例 10 岁 HCM 患儿进行 69 年的理论队列随访。SCD 的基线年风险为 0.8%。结局为心源性猝死 (SCD) 、严重神经系统疾病 (SNM) 、成本和质量调整生命年 (QALYs)。模型投入来源于文献，支付意愿阈值为每 QALY 100,000 美元。 结果: 在 SCD 中等风险的儿童中，家庭 AED 导致 SCD 病例减少 31 例，但 SNM 病例增加 3 例。增加了 319 个 QALYs。尽管成本较高 2800万美元，但增量成本效益比为 86,458 美元，低于支付意愿门槛值。 结论: 对于 SCD 和 HCM 中等风险的儿童，家庭 AED 具有成本效益，导致死亡人数减少，费用低于支付意愿阈值的 QALYS 增加。这些发现强调了在该人群中使用家庭 AED 的经济效益。
METHODS:BACKGROUND:Preterm birth is a risk factor for elevated blood pressure in childhood and the development of hypertension and cardiometabolic disease in adulthood; however, mechanisms for the development of both are poorly understood. Rapid weight gain early in childhood may serve as a driver directly and indirectly through cortisol levels found to be elevated in early childhood in individuals born preterm. OBJECTIVES:The objective of this pilot study was to examine the effect sizes of the relationships between weight gain and blood pressure in toddlers born very preterm. A secondary aim was to note any mediating effect of cortisol on the relationships between weight gain and blood pressure. METHODS:A cross-sectional design with a convenience sample of 36 toddlers who were born very preterm was used to examine the relationships between postnatal weight gain, cortisol, and blood pressure at follow-up. RESULTS:Many of the participants experienced rapid weight gain in the first 12 months of life. Mean systolic and diastolic readings were 94 and 56.6, respectively. Diastolic blood pressure readings were obtained from 23 participants and the majority were elevated. Weight gain was associated with diastolic blood pressure with a medium effect size. A mediating role with cortisol was not supported.Although findings need to be validated in a larger sample, the blood pressure elevations in this sample were alarming. If readings continue to amplify as these children age, the fact that elevations are already present during the toddler period could indicate more significant cardiovascular disease in adulthood for this population. Rapid weight gain in early life may be a driver for elevated blood pressure even during early childhood in individuals born preterm.
METHODS:This article presents a case series of n = 21 models of fetal cardiovascular anatomies obtained from post mortem microfocus computed tomography (micro-CT) data. The case series includes a broad range of diagnoses (e.g., tetralogy of Fallot, hypoplastic left heart syndrome, dextrocardia, double outlet right ventricle, atrio-ventricular septal defect) and cases also had a range of associated extra-cardiac malformations (e.g., VACTERL syndrome, central nervous system anomalies, renal anomalies). All cases were successfully reconstructed from the microfocus computed tomography data, demonstrating the feasibility of the technique and of the protocols, including in-house printing with a desktop 3D printer (Form2, Formlabs). All models were printed in 1:1 scale as well as with the 5-fold magnification, to provide insight into the intra-cardiac structures. Possible uses of the models include education and training.
METHODS:Abstract Background Congenital heart disease (CHD) affects ~ 1% of live births and is the most common birth defect. Although the genetic contribution to the CHD has been long suspected, it has only been well established recently. De novo variants are estimated to contribute to approximately 8% of sporadic CHD. Methods CHD is genetically heterogeneous, making pathway enrichment analysis an effective approach to explore and statistically validate CHD-associated genes. In this study, we performed novel gene and pathway enrichment analyses of high-impact de novo variants in the recently published whole-exome sequencing (WES) data generated from a cohort of CHD 2645 parent-offspring trios to identify new CHD-causing candidate genes and mutations. We performed rigorous variant- and gene-level filtrations to identify potentially damaging variants, followed by enrichment analyses and gene prioritization. Results Our analyses revealed 23 novel genes that are likely to cause CHD, including HSP90AA1, ROCK2, IQGAP1, and CHD4, and sharing biological functions, pathways, molecular interactions, and properties with known CHD-causing genes. Conclusions Ultimately, these findings suggest novel genes that are likely to be contributing to CHD pathogenesis.