BACKGROUND:Continuous outpatient inotrope infusion therapy (COIIT) can be used as palliative or interim treatment in patients with advanced heart failure (AHF). Despite widespread use, there is a relative lack of data informing best practices. This study aimed to examine whether patterns of COIIT use differed by region and to explore whether observed differences influenced clinical outcomes. METHODS:Retrospective study of AHF patients receiving COIIT from May 2009 through June 2016. The primary outcome was regional difference, the secondary outcome was persistence (duration) on therapy. Cox proportional hazards model was used to calculate hazard ratios for treatment regimens. RESULTS:There were 3,286 patients, mean (SD) age 61.9 (14.4) years and 74.0% (2,433) male. Inotrope selection and beta blocker use varied by region by chi square (χ2 (21) = 166.9, p < 0.001). Persistence was greater on milrinone compared to dobutamine (HR (for discontinuation) 0.54, CI 0.41-0.70, p < 0.001). Concurrent beta-blocker was associated with greater persistence for patients receiving milrinone (HR 0.13, CI 0.08-0.20, p < 0.001) and dobutamine (HR 0.36, CI 0.18-0.71, p < 0.001). CONCLUSIONS:Patterns of COIIT use varied by region, and variations in use were associated with differences in clinical outcomes.

Background Online longitudinal surveys may be subject to potential biases due to sample attrition. This study was designed to identify potential predictors of attrition using a longitudinal panel survey collected during the COVID-19 pandemic. Methods Three waves of data were collected using Amazon Mechanical Turk (MTurk), an online crowd-sourced platform. For each wave, the study sample was collected by referencing a US national representative sample distribution of age, gender, and race, based on US census data. Variables included respondents’ demographics, medical history, socioeconomic status, COVID-19 experience, changes of health behavior, productivity, and health-related quality of life (HRQoL). Results were compared to pre-pandemic US norms. Measures that predicted attrition at different times of the pandemic were identified via logistic regression with stepwise selection. Results 1467 of 2734 wave 1 respondents participated in wave 2 and, 964 of 2454 wave 2 respondents participated in wave 3. Younger age group, Hispanic origin (p ≤ 0.001) and higher self-rated survey difficulty (p ≤ 0.002) consistently predicted attrition in the following wave. COVID-19 experience, employment, productivity, and limited physical activities were commonly observed variables correlated with attrition with specific measures varying by time periods. From wave 1, mental health conditions, average daily hours worked (p = 0.004), and COVID-19 impact on work productivity (p < 0.001) were associated with a higher attrition rate at wave 2, additional to the aforementioned factors. From wave 2, support of social distancing (p = 0.032), being Republican (p < 0.001), and having just enough money to make ends meet (p = 0.003) were associated with predicted attrition at wave 3. Conclusions Attrition in this longitudinal panel survey was not random. Besides commonly identified demographic factors that contribute to panel attrition, COVID-19 presented novel opportunities to address sample biases by correlating attrition with additional behavioral and HRQoL factors in a constantly evolving environment. While age, ethnicity, and survey difficulty consistently predicted attrition, other factors, such as COVID-19 experience, changes of employment, productivity, physical health, mental health, and financial situation impacted panel attrition during the pandemic at various degrees.

Objective The use of therapeutic hypothermia (TH) for mild hypoxic-ischaemic encephalopathy (HIE) remains controversial and inconsistent. We analysed trends in TH and maternal and infant characteristics associated with short-term outcomes of infants with mild HIE. Design Retrospective cohort analysis of the California Perinatal Quality Care Collaborative database 2010-2018. E-value analysis was conducted to determine the potential impact of unmeasured confounding. Setting California neonatal intensive care units. Patients 1364 neonates with mild HIE. Interventions Supportive care versus TH. Main outcome measures Factors associated with TH and mortality. Results The proportion of infants receiving TH increased from 46% in 2010 to 79% in 2018. TH was more likely in the setting of singleton birth (OR 2.69, 95% CI 1.21 to 5.39), no major birth defects (OR 2.18, 95% CI 1.42 to 3.30), operative vaginal delivery (OR 3.04, 95% CI 1.80 to 5.10) and 5-minute Apgar score <= 5 (OR 3.17, 95% CI 2.43 to 4.13). Mortality was associated with small for gestational age (OR 5.79, 95% CI 1.90 to 18.48), <38 weeks' gestation (OR 7.31 95% CI 2.39 to 24.93), major birth defects (OR 11.62, 95% CI 3.97 to 38.00), inhaled nitric oxide (OR 12.73, 95% CI 4.00 to 44.53) and nosocomial infection (OR 7.98, 95% CI 1.15 to 47.03). E-value analyses suggest that unmeasured confounding may have contributed to some of the observed effects. Conclusions Variation in management of mild HIE persists, but therapeutic drift has become more prevalent over time. Further studies are needed to assess long-term outcomes alongside resource utilisation to inform evidence-based practice. This manuscript reports trends in the use of therapeutic hypothermia for babies with mild HIE in the NICUs in the CPQCC catchment, and assesses odds ratios for maternal and infant factors associated with the use of TH. There was an increase in the use of TH for mild HIE increased over time as a form of therapeutic creep.

Background The duration of extracorporeal membrane oxygenation (ECMO) has been historically confined in many centers to two weeks. We evaluated the cost-effectiveness of additional weeks on ECMO beyond two weeks for newborns with congenital diaphragmatic hernia (CDH) who may require longer stays to maximize survival potential. Methods We modeled lifetime outcomes using a decision tree from the US societal perspective. Survival at discharge, probability of long-term sequelae, direct medical costs, indirect costs, and quality-adjusted life years (QALY) for long-term disability were considered. Considering the nature of severity of CDH, we used $200,000 per QALY as the willingness-to-pay threshold in the base case. Results The lifetime costs per CDH infant generated from staying on ECMO for ≤2 weeks, 2–3 weeks, and >3 weeks are $473,334, $654,771, $1,007,476, respectively (2018 USD), and the total QALYs gained from each treatment arm are 1.83, 3.6, and 5.05. In the base case, the net monetary benefits are −$108,034 for ECMO ≤2 weeks, $64,258 for 2–3 weeks, and $2955 for >3 weeks. In probabilistic simulations, a duration of ≤2 weeks is dominated by a duration of 2–3 weeks in 65.3% of cases and dominated by > 3 weeks in 60.2% of cases. A duration of 2–3 weeks is more cost-effective than >3 weeks in 68.6% of simulations. Conclusion Our findings suggest that 2–3 weeks of ECMO may be the most cost-effective for CDH infants that are unable to wean off at 2 weeks from the US societal perspective. Regardless of ECMO duration, ECMO use generates positive incremental NMB at WTP of $200,000 if the survival probability is greater than 0.3. Future research must be conducted to evaluate the long-term outcomes and sequelae of CDH patients post-discharge to better inform the clinical decision-making in neonatal intensive care unit.

BackgroundOnline longitudinal surveys may be subject to potential biases due to sample attrition. This study was designed to identify potential predictors of attrition using a longitudinal panel survey collected during the COVID-19 pandemic.MethodsThree waves of data were collected online during the pandemic using Amazon MTurk. Variables included respondents’ demographics, medical history, socioeconomic status, COVID-19 experience, behavioral change, employment change, financial spending change, and health-related quality of life (HRQoL). Results were compared to US pre-pandemic norms. Measures that predicted attrition in the following wave were identified via logistic regression with stepwise selection.Results1,467 out of 2,734 wave 1 respondents participated in wave 2 and, 964 out of 2,454 wave 2 respondents participated in wave 3. Younger age group, Hispanic origin (wave 1: p<0.001; wave 2: p=0.001), and higher self-rated survey difficulty (wave 1: p=0.002; wave 2: p<0.001) at waves 1 and 2 consistently predicted attrition in the following wave, respectively. COVID-19 experience, employment status, and limited physical activities were commonly observed factors contributed to attrition while specific measures change to reflect the most concerning matter at the time. In addition, mental health, average hours worked per day (p=0.004), and COVID-19 impact on work productivity (p<0.001) at wave 1 were found to be correlated with a higher attrition rate at wave 2. Support of social distancing (p=0.032), being Republican (p<0.001), and having just enough money to make ends meet (p=0.003) were some of the remaining key characteristics that predicted attrition at wave 3.ConclusionsAttritions found in this longitudinal COVID-19 panel survey was not at random. Besides commonly identified demographic factors that contribute to panel attrition, COVID-19 presented new challenges to address sample biases by correlating attrition with additional factors in a constantly evolving environment. BackgroundOnline longitudinal surveys may be subject to potential biases due to sample attrition. This study was designed to identify potential predictors of attrition using a longitudinal panel survey collected during the COVID-19 pandemic. Online longitudinal surveys may be subject to potential biases due to sample attrition. This study was designed to identify potential predictors of attrition using a longitudinal panel survey collected during the COVID-19 pandemic. MethodsThree waves of data were collected online during the pandemic using Amazon MTurk. Variables included respondents’ demographics, medical history, socioeconomic status, COVID-19 experience, behavioral change, employment change, financial spending change, and health-related quality of life (HRQoL). Results were compared to US pre-pandemic norms. Measures that predicted attrition in the following wave were identified via logistic regression with stepwise selection. Three waves of data were collected online during the pandemic using Amazon MTurk. Variables included respondents’ demographics, medical history, socioeconomic status, COVID-19 experience, behavioral change, employment change, financial spending change, and health-related quality of life (HRQoL). Results were compared to US pre-pandemic norms. Measures that predicted attrition in the following wave were identified via logistic regression with stepwise selection. Results1,467 out of 2,734 wave 1 respondents participated in wave 2 and, 964 out of 2,454 wave 2 respondents participated in wave 3. Younger age group, Hispanic origin (wave 1: p<0.001; wave 2: p=0.001), and higher self-rated survey difficulty (wave 1: p=0.002; wave 2: p<0.001) at waves 1 and 2 consistently predicted attrition in the following wave, respectively. COVID-19 experience, employment status, and limited physical activities were commonly observed factors contributed to attrition while specific measures change to reflect the most concerning matter at the time. In addition, mental health, average hours worked per day (p=0.004), and COVID-19 impact on work productivity (p<0.001) at wave 1 were found to be correlated with a higher attrition rate at wave 2. Support of social distancing (p=0.032), being Republican (p<0.001), and having just enough money to make ends meet (p=0.003) were some of the remaining key characteristics that predicted attrition at wave 3. 1,467 out of 2,734 wave 1 respondents participated in wave 2 and, 964 out of 2,454 wave 2 respondents participated in wave 3. Younger age group, Hispanic origin (wave 1: p<0.001; wave 2: p=0.001), and higher self-rated survey difficulty (wave 1: p=0.002; wave 2: p<0.001) at waves 1 and 2 consistently predicted attrition in the following wave, respectively. COVID-19 experience, employment status, and limited physical activities were commonly observed factors contributed to attrition while specific measures change to reflect the most concerning matter at the time. In addition, mental health, average hours worked per day (p=0.004), and COVID-19 impact on work productivity (p<0.001) at wave 1 were found to be correlated with a higher attrition rate at wave 2. Support of social distancing (p=0.032), being Republican (p<0.001), and having just enough money to make ends meet (p=0.003) were some of the remaining key characteristics that predicted attrition at wave 3. ConclusionsAttritions found in this longitudinal COVID-19 panel survey was not at random. Besides commonly identified demographic factors that contribute to panel attrition, COVID-19 presented new challenges to address sample biases by correlating attrition with additional factors in a constantly evolving environment. Attritions found in this longitudinal COVID-19 panel survey was not at random. Besides commonly identified demographic factors that contribute to panel attrition, COVID-19 presented new challenges to address sample biases by correlating attrition with additional factors in a constantly evolving environment.

Background To assess the price range in which fexapotide triflutate (FT), a novel injectable, is cost-effective relative to current oral pharmacotherapy (5 α -reductase inhibitor, α -blocker, 5 α-reductase inhibitor and α-blocker combination therapy) as initial therapy followed by surgery for moderate-to-severe benign prostate hyperplasia patients with lower urinary tract symptoms (BPH-LUTS). Methods We developed a microsimulation decision-analytic model to track the progression of BPH-LUTS and associated costs and quality-adjusted life years in the target population. The cost-effectiveness analysis was performed from Medicare’s perspective with a time horizon of 4 years using 2019 US dollars for all costs. The microsimulation model considered treatment patterns associated with nonadherence to oral medication and progression to surgery. Model parameters were estimated from large randomized controlled trials, literature and expert opinion. For each initial treatment option, simulations were performed with 1000 iterations, with 1000 patients per iteration. Results Three upfront oral pharmacotherapy options are close in cost-effectiveness, with combination therapy being the most cost-effective option. Relative to upfront oral pharmacotherapy options, FT slightly increases quality-adjusted life years (QALY) per patient (1.870 (95% CI, 1.868 to 1.872) vs. 1.957 (95% CI, 1.955 to 1.959) QALYs). Under the willingness-to-pay (WTP) threshold of $150,000 per QALY, at price per injection of $14,000, FT is about as cost-effective as upfront oral pharmacotherapy options with net monetary benefit (NMB) $279,168.54. Under the WTP threshold of $50,000 per QALY, at price per injection of $5,000, FT is about as cost-effective as upfront oral pharmacotherapy options with NMB $92,135.18. In an alternative 10-year time horizon scenario, FT price per injection at $11,000 and $4,500 makes FT as cost-effective as oral pharmacotherapies. One-way sensitivity analysis showed this result is most sensitive to upfront therapy prices, FT efficacy and initial IPSS. At price per injections of $5,000, $10,000 and $15,000, the probability that FT is either cost-effective or dominant compared to upfront oral pharmacotherapy options using a WTP threshold of $150,000 per QALY is 100%, 93% and 40%, respectively. Conclusions Compared to upfront oral pharmacotherapy options, FT would be cost-effective at a price per injection below $14,000, assuming a WTP threshold of $150,000 per QALY.

To assess and compare the impact of the COVID-19 pandemic on health-related quality-of-life (HRQoL) in the United States, Sweden and Norway. Two waves of web-based survey were conducted in April 2020 and January 2021 to collect demographic data, COVID-19 status, behavior and employment changes related to COVID-19 in each country (EuroQol Grant: 246-2020RA). EQ-5D-5L was used to assess health status of respondents. Results were compared between the two waves to measure changes in HRQoL. One-way ANOVA was used to detect significant differences between countries, and t-tests for differences between waves. We collected 2,734, 1,003 and 1,020 responses in Wave 1, and 2,252, 1,013 and 1,011 responses in Wave 2 for the US, Sweden, and Norway respectively. Corresponding mean (SD) EQ-VAS scores were 74.6 (±19.2), 68.7 (±21.4), and 69.2 (±20.8) in Wave 1 (p<0.001), and 76.4 (±18.6), 68.2 (±20.3), and 67.8 (±21.7) in Wave 2 (p<0.001). Between waves, only the VAS scores in the US were significantly different (p<0.001). Mean (SD) utility scores were 0.822 (±0.222), 0.768 (±0.260), and 0.808 (±0.248) in wave 1 (p<0.001), and 0.823 (±0.221), 0.783 (±0.237), and 0.777 (±0.271) in wave 2 (p<0.001); there were no significant differences between waves for all three countries. Anxiety/depression was consistently the most problematic EQ-5D-5L subdomain among Swedish and Americans (>50%), followed by pain/discomfort. >45% Norwegians also reported problems in anxiety/depression subdomain in both waves. The proportions reporting problems in anxiety/depression increased in wave 2 for Sweden and Norway, but decreased for the US. Population HRQoL in Sweden and Norway has been similar throughout the pandemic, while a rebound in population mean VAS was observed in the US. However, the large proportions reporting problems in anxiety/depression across waves in all 3 countries indicates that mental health issues resulting from the pandemic are a major concern.

ObjectivesThroughout the coronavirus disease 2019 pandemic, susceptible-infectious-recovered (SIR) modeling has been the preeminent modeling method to inform policy making worldwide. Nevertheless, the usefulness of such models has been subject to controversy. An evolution in the epidemiological modeling field is urgently needed, beginning with an agreed-upon set of modeling standards for policy recommendations. The objective of this article is to propose a set of modeling standards to support policy decision making.MethodsWe identify and describe 5 broad standards: transparency, heterogeneity, calibration and validation, cost-benefit analysis, and model obsolescence and recalibration. We give methodological recommendations and provide examples in the literature that employ these standards well. We also develop and demonstrate a modeling practices checklist using existing coronavirus disease 2019 literature that can be employed by readers, authors, and reviewers to evaluate and compare policy modeling literature along our formulated standards.ResultsWe graded 16 articles using our checklist. On average, the articles met 6.81 of our 19 categories (36.7%). No articles contained any cost-benefit analyses and few were adequately transparent.ConclusionsThere is significant room for improvement in modeling pandemic policy. Issues often arise from a lack of transparency, poor modeling assumptions, lack of a system-wide perspective in modeling, and lack of flexibility in the academic system to rapidly iterate modeling as new information becomes available. In anticipation of future challenges, we encourage the modeling community at large to contribute toward the refinement and consensus of a shared set of standards for infectious disease policy modeling.

OBJECTIVES:Treatment of Clostridioides difficile infection (CDI) has undergone significant change in recent years with the introduction of fidaxomicin and bezlotoxumab. This study evaluated the cost-effectiveness of fidaxomicin and bezlotoxumab for initial CDI compared with standard therapy with oral vancomycin.METHODS:A Markov model with eight health states was built based on transition probabilities, costs and health utilities derived from literature to evaluate the cost-effectiveness of standard fidaxomicin, bezlotoxumab plus vancomycin, and extended-pulsed fidaxomicin versus standard oral vancomycin over a lifetime horizon from the US societal perspective.RESULTS:For overall CDI treatment, oral vancomycin had a cost of $39 178 and was associated with a gain of 11.64 quality-adjusted life-years (QALYs). Extended-pulsed fidaxomicin had a higher QALY gain of 11.65 at a lower cost of $37 613, and therefore was dominant over vancomycin. Standard fidaxomicin had a QALY gain of 11.94 versus vancomycin at an incremental cost of $495 per QALY. Bezlotoxumab plus vancomycin led to a QALY gain of 11.77 at an incremental cost of $17 746 per QALY. At the willingness-to-pay (WTP) threshold of $150 000 per QALY, extended-pulsed fidaxomicin, bezlotoxumab plus vancomycin and standard fidaxomicin were more cost-effective compared with vancomycin alone, yielding incremental net monetary benefits of $3248, $17 011 and $44 308, respectively. One-way sensitivity analysis suggested that the probabilities of sustained cure from the initial episode were the most sensitive inputs, and results were overall not particularly sensitive to any drug costs.CONCLUSIONS:Based on a WTP threshold of $150 000, standard fidaxomicin was estimated to be the most cost-effective treatment. Standard-of-care vancomycin was dominated by extended-pulsed fidaxomicin for treating an episode of CDI and preventing further recurrence, and the addition of bezlotoxumab to vancomycin was dominated by standard fidaxomicin.

Purpose To determine the value of early exome sequencing (eES) relative to the current typical care (TC) in the diagnosis of newborns with suspected severe mitochondrial disorders (MitD). Methods We used a decision tree-Markov hybrid to model neonatal intensive care unit (NICU)-related outcomes and costs, lifetime costs and quality-adjusted life-years among patients with MitD. Probabilities, costs, and utilities were populated using published literature, expert opinion, and the Pediatric Health Information System database. Incremental cost-effectiveness ratios (ICER) and net monetary benefits (NMB) were calculated from lifetime costs and quality-adjusted life-years for singleton and trio eES, and TC. Robustness was assessed using univariate and probabilistic sensitivity analyses (PSA). Scenario analyses were also conducted. Results Findings indicate trio eES is a cost-minimizing and cost-effective alternative to current TC. Diagnostic probabilities and NICU length-of-stay were the most sensitive model parameters. Base case analysis demonstrates trio eES has the highest incremental NMB, and PSA demonstrates trio eES had the highest likelihood of being cost-effective at a willingness-to-pay (WTP) of $200,000 relative to TC, singleton eES, and no ES. Conclusion Trio and singleton eES are cost-effective and cost-minimizing alternatives to current TC in diagnosing newborns suspected of having a severe MitD.

Our website uses cookies to enhance your experience. By continuing to use our site, or clicking "Continue," you are agreeing to our Cookie Policy | Continue

The outbreak of the pandemic COVID-19 (Coronavirus) has resulted in various international and national strategies, including non-pharmaceutical interventions (NPIs) such as social distancing and travel bans, which have purportedly mitigated the health loss due to the pandemic but also given rise to a severe economic crisis. Both factors, the pandemic and the NPIs, can be expected to have an impact on the Health-Related Quality-of-Life (HRQoL) of the population. The objective of this study was to estimate the impact on HRQoL of the Swedish adult population during the outbreak of the COVID-19 pandemic. A web-based survey was sent to randomised samples of the adult Swedish population before the outbreak of the pandemic in Sweden in February 2020 (n=1,016) and during the outbreak of the pandemic. The first wave pandemic data was collected in April 2020 (n=1,003), one-month after the outbreak and, the second wave data was collected in January 2021 (n=1,013), after 10-months living under the pandemic. HRQoL was measured using the EQ-5D-5L in the pandemic surveys, whereas the Visual Analogue Scale (VAS) was used in all surveys. The results suggested a reduction in average HRQoL as measured by VAS in the adult Swedish population, with 0.059 points reduction in VAS in April 2020 and 0.074 points reduction in January 2021, compared to the pre-pandemic measurement in February 2020. The loss in HRQoL was significant among respondents in the working age population (<65 years), suggesting that the social and economic impact of NPIs were the primary drivers for this specific cohort. Findings of this study supports a wide public health perspective and future HRQoL measurements at the population level throughout the pandemic. Published: Online September 2021

BACKGROUND:The COVID-19 pandemic has resulted in negative impacts on the economy, population health, and health-related quality-of-life (HRQoL).OBJECTIVE:To assess the impact of COVID-19 on US population HRQoL using the EQ-5D-5L.DESIGN:We surveyed respondents on physical and mental health, demographics, socioeconomics, brief medical history, current COVID-19 status, sleep, dietary, financial, and spending changes. Results were compared to online and face-to-face US population norms. Predictors of EQ-5D-5L utility were analyzed using both standard and post-lasso OLS regressions. Robustness of regression coefficients against unmeasured confounding was analyzed using the E-Value sensitivity analysis.SUBJECTS:Amazon MTurk workers (n=2776) in the USA.MAIN MEASURES:EQ-5D-5L utility and VAS scores by age group.KEY RESULTS:We received n=2746 responses. Subjects 18-24 years reported lower mean (SD) health utility (0.752 (0.281)) compared with both online (0.844 (0.184), p=0.001) and face-to-face norms (0.919 (0.127), p<0.001). Among ages 25-34, utility was worse compared to face-to-face norms only (0.825 (0.235) vs. 0.911 (0.111), p<0.001). For ages 35-64, utility was better during pandemic compared to online norms (0.845 (0.195) vs. 0.794 (0.247), p<0.001). At age 65+, utility values (0.827 (0.213)) were similar across all samples. VAS scores were worse for all age groups (p<0.005) except ages 45-54. Increasing age and income were correlated with increased utility, while being Asian, American Indian or Alaska Native, Hispanic, married, living alone, having history of chronic illness or self-reported depression, experiencing COVID-19-like symptoms, having a family member diagnosed with COVID-19, fear of COVID-19, being underweight, and living in California were associated with worse utility scores. Results were robust to unmeasured confounding.CONCLUSIONS:HRQoL decreased during the pandemic compared to US population norms, especially for ages 18-24. The mental health impact of COVID-19 is significant and falls primarily on younger adults whose health outcomes may have been overlooked based on policy initiatives to date.

Objective To determine the most cost-effective reconstruction method after salvage total laryngectomy. Study Design Cost-effectiveness analysis Setting Tertiary care hospitals with head and neck oncologic and reconstructive surgeons. Subjects and Methods We constructed a Markov-based decision model to compare reconstruction by primary closure to pectoralis flap and free flap after salvage total laryngectomy. The model simulated disease with transition probabilities and health utilities found in primary literature and estimated the average overall cost of each reconstructive method using Medicare billing codes. Effectiveness was compared using quality-adjusted life years (QALYs). One-way and probabilistic sensitivity analyses were performed to scrutinize the conclusions of our model. Reconstruction methods were compared using incremental cost-effectiveness ratios (ICERs). In the United States, less than $150,000 per QALY gained is considered cost-effective (2019 US dollars). Results Our base case analysis revealed that primary closure was less expensive ($44,370) and yielded more QALYs (0.91) than both pectoralis ($45,163, 0.81 QALYs) and free flap ($46,244, 0.85 QALYs), making it the most cost-effective option. Between flaps, free flap was cost-effective over pectoralis flap (ICER = $27,025/QALY gained). Sensitivity analyses showed primary closure as cost-effective 69% of the time over either flap. These conclusions were sensitive to the health utilities (quality of life) of each method of reconstruction. Conclusion Tissue flaps to augment closure after salvage total laryngectomy are not always the most cost-effective reconstructive option. The long-term morbidity of flap surgery oftentimes outweighs the benefit of lowering fistula rates after surgery. Careful consideration must be taken when advising patients of their reconstructive options.

Treatment of Clostridium difficile infection (CDI) has undergone significant change in recent years with the introduction of fidaxomicin and bezlotoxumab. This study evaluated the cost-effectiveness of fidaxomicin and bezlotoxumab for initial and recurrent CDI compared to standard therapy with oral vancomycin.

To evaluate the cost-effectiveness of additional weeks on extracorporeal membrane oxygenation (ECMO) beyond two weeks for newborns with severe congenital diaphragmatic hernia (CDH) who may only receive a maximum of 2 weeks in some centers. A decision tree was used to model out the lifetime outcomes from the US societal perspective. Probability of survival and long-term sequelae, direct medical costs, indirect costs, and quality-adjusted life years (QALYs) for long-term disability were considered. Costs and QALYs were discounted at an annual rate of 3%. One-way sensitivity analyses, threshold analyses, and probabilistic sensitivity analyses were conducted to test the robustness of the model. Costs are reported in 2018 USD. The lifetime costs per CDH infant generated from staying on ECMO for < 2 weeks, 2-3 weeks, and > 3 weeks are $473,334, $654,771, $1,007,476, and the total lifetime QALYs gained are 1.83, 3.6, and 5.05. At a willingness-to-pay (WTP) threshold of $200,000, the incremental net monetary benefits (INMB) per patient are -$108,034 for ECMO < 2 weeks, $64,258 for ECMO of 2-3 weeks, and $2,955 for ECMO > 3 weeks. One-way sensitivity analyses showed that the model is the most sensitive to the probability of survival at discharge, followed by the initial encounter costs of mortality, and the probability of getting severe neuro impairment. ECMO < 2 weeks is dominated by ECMO of 2-3 weeks in 65.3% of cases and dominated by over 3 weeks in 60.2% of cases. ECMO of 2-3 weeks is more cost-effective than over 3 weeks in 68.6% of simulations. Our model finds that ECMO duration of 2-3 weeks may be the most cost-effective option for infants with severe CDH requiring ECMO. There may be some benefit in extending ECMO beyond 2 weeks, but not longer than 3 weeks.

Purpose: Previous SEER (Surveillance, Epidemiology, and End Results)-Medicare analyses have shown no definitive survival benefit for adjuvant chemotherapy (AC) with fluoropyrimidines. Impact of oxaliplatin-containing regimens for elderly stage II patients in real-world setting is unknown. We explored the utilization and outcome of AC after the Food and Drug Administration (FDA) approval of oxaliplatin. Patients and Methods: Patients with stage II colon cancer (2004-2011) who underwent resection were selected for this analysis. Medicare claims data were used to ascertain the administration of AC within 120 days after surgery. The primary endpoint of the analysis was overall survival. We used the Cox proportional hazards model to estimate the effect of AC while adjusting for clinical and sociodemographic variables available in SEER. To adjust for referral pattern, a source of selection bias, we conducted an instrumental variable analysis using the surgeon of record and health service area. Results: A total of 16,468 patients were identified and 12.1% received AC. AC recipients were significantly younger, more likely to be male, nonwhite, married, and had lower comorbidity index. Their tumors had a more advanced stage, more likely to be left sided, and were less differentiated. The hazard ratio (HR) from the Cox model showed a statistically significant survival advantage for AC (HR=0.847, 95% confidence interval: 0.782-0.916). However, results from the instrumental variable analysis indicated that there was no definitive benefit of survival in AC recipients (HR=1.779, 95% confidence interval: 0.927-3.415). AC use decreased over time. Conclusions: After controlling for referral patterns, administration of AC provided no definitive survival benefit. Future studies may elucidate the elderly population who may benefit from AC.

Pembrolizumab/axitinib and nivolumab/ipilimumab have shown improvement in overall survival as compared to sunitinib in randomized Phase III trials. Based on these data, both of these regimes were FDA-approved as first-line treatment for advanced renal cell carcinoma (RCC), and have become the standard of care. Given the high price point of immune checkpoint inhibitors, it is important to understand and compare the economic value of these two regimens. From a US societal perspective, a Markov model compared pembrolizumab/axitinib and nivolumab/ipilimumab to sunitinib over a lifetime horizon among treatment-naïve patients with advanced RCC. Under a cohort model design, a single cohort was modeled in each of the 3 treatment arms in parallel, and patients could transition between the health states of progression-free, progression, and death. Patients were allowed to discontinue treatment to second-line therapy or to no therapy. Model inputs were obtained from the KEYNOTE-426 and CheckMate 214 clinical trials, scientific literature, and governmental data sources. Incremental cost-effectiveness ratios (ICERs) were evaluated in terms of cost per quality-adjusted life-year (QALY) gained and sensitivity analyses assessed robustness of model results. The ICER for pembrolizumab/axitinib versus sunitinib was $850,672 per QALY gained, whereas the ICER for nivolumab/ipilimumab versus sunitinib was $122,480 per QALY gained. One-way sensitivity analyses showed results to be robust to reasonable variation in model inputs, with ICERs most sensitive to drug costs, probability of discontinuing first-line therapy, and disease state-specific health utilities. According to probabilistic sensitivity analyses, pembrolizumab/axitinib and nivolumab/ipilimumab are cost-effective at a willingness-to-pay (WTP) of $150,000 per QALY gained in 2% and 83% of simulations, respectively. Based on an accepted WTP of $150,000 per QALY gained, pembrolizumab/axitinib is a low-value therapy compared to sunitinib in patients with advanced RCC. However, at this threshold, nivolumab/ipilimumab represents a cost-effective alternative to sunitinib.

Our website uses cookies to enhance your experience. By continuing to use our site, or clicking "Continue," you are agreeing to our Cookie Policy | Continue JAMA Pediatrics HomeNew OnlineCurrent IssueFor Authors Podcast Publications JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry (1919-1959) JN Learning / CMESubscribeJobsInstitutions / LibrariansReprints & Permissions Terms of Use | Privacy Policy | Accessibility Statement 2023 American Medical Association. All Rights Reserved Search All JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Forum Archive JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry Input Search Term Sign In Individual Sign In Sign inCreate an Account Access through your institution Sign In Purchase Options: Buy this article Rent this article Subscribe to the JAMA Pediatrics journal

Of the estimated 2.1 million individuals in the US with a diagnosis of opioid use disorder (OUD), only 20% receive treatment. More evidence on the cost-effectiveness of medication-assisted treatments (MAT) is critical to increasing access to and use of MAT. We sought to assess the cost-per-quality-adjusted life years (QALY) gained from four long-term MATs for OUD in the US. We conducted a cost-utility analysis using a Markov model simulation of OUD treatments from the U.S. societal perspective. The hypothetical cohort consisted of OUD patients with a mean age of 41 years, 50% of whom used prescription opioids and 50% of whom used heroin. Treatments included take-home buprenorphine-naloxone (BUP/NX), clinic-based methadone maintenance (MMT), extended-release naltrexone injection (XR-NTX), and long-lasting buprenorphine injection (XR-BUP). Costs (2019 USD) per QALY were estimated in 1-month cycles over a 10-year time horizon with a 3% discount rate. The impact of model uncertainty was assessed via deterministic and probabilistic sensitivity analyses (PSA). Incremental cost-effectiveness ratios (ICERs) and incremental net monetary benefits (iNMBs) were estimated for each pairwise comparison. In the reference case, BUP/NX is a dominant treatment strategy compared to MMT and XR-BUP. XR-NTX is cost-effective at a willingness-to-pay (WTP) threshold of $175,000 per QALY. All four treatments yield similar QALY gains (∼5.0) but vary substantially in cost per patient ($1.7 - $1.85 million). In a PSA of 5,000 simulations, BUP/NX is cost-effective in 23% of 5,000 simulations while XR-BUP is cost-effective in 66% of simulations and dominates the other three treatments. This model demonstrates that buprenorphine, in take-home or injectable form, is the most cost-effective long-term treatment for OUD. MMT, the most commonly used MAT, is not cost-effective compared to any treatments included. This model can be used to simulate how MAT costs and outcomes will change in response to interventions aimed at reducing OUD.