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1.
Article in English | MEDLINE | ID: mdl-38570896

ABSTRACT

BACKGROUND: The role of altruism in the acceptance of novel preventive healthcare technologies like vaccines has not been thoroughly elucidated. METHODS: We 1:1 randomized n = 2004 Amazon Mechanical Turk (MTurk) participants residing in the USA into a control or treatment arm with vaccination decisions framed altruistically, to elicit their preferences for COVID-19 vaccination using web-based discrete choice experiments. We used conditional and mixed logit models to estimate the impact of framing decisions in terms of altruism on vaccination acceptance. RESULTS: Valid responses were provided by 1674 participants (control, n = 848; treatment, n = 826). Framing vaccination decisions altruistically had no significant effect on vaccination acceptance. Further, respondents' degree of altruism had no association with vaccination acceptance. LIMITATIONS: The MTurk sample may not be representative of the American population. We were unable to ascertain concordance between stated and revealed preferences. CONCLUSIONS AND IMPLICATIONS: Framing vaccination decisions in terms of altruism does not appear to significantly influence vaccination acceptance and may not be an effective nudging mechanism to increase the uptake of novel vaccines. Instead, a favorable vaccination profile appears to be the primary driver of uptake.

2.
JAMA Netw Open ; 7(3): e240555, 2024 Mar 04.
Article in English | MEDLINE | ID: mdl-38470421

ABSTRACT

Importance: High-risk infants, defined as newborns with substantial neonatal-perinatal morbidities, often undergo multiple procedures and require prolonged intubation, resulting in extended opioid exposure that is associated with poor outcomes. Understanding variation in opioid prescribing can inform quality improvement and best-practice initiatives. Objective: To examine regional and institutional variation in opioid prescribing, including short- and long-acting agents, in high-risk hospitalized infants. Design, Setting, and Participants: This retrospective cohort study assessed high-risk infants younger than 1 year from January 1, 2016, to December 31, 2022, at 47 children's hospitals participating in the Pediatric Health Information System (PHIS). The cohort was stratified by US Census region (Northeast, South, Midwest, and West). Variation in cumulative days of opioid exposure and methadone treatment was examined among institutions using a hierarchical generalized linear model. High-risk infants were identified by International Statistical Classification of Diseases and Related Health Problems, Tenth Revision codes for congenital heart disease surgery, medical and surgical necrotizing enterocolitis, extremely low birth weight, very low birth weight, hypoxemic ischemic encephalopathy, extracorporeal membrane oxygenation, and other abdominal surgery. Infants with neonatal opioid withdrawal syndrome, in utero substance exposure, or malignant tumors were excluded. Exposure: Any opioid exposure and methadone treatment. Main Outcomes and Measures: Regional and institutional variations in opioid exposure. Results: Overall, 132 658 high-risk infants were identified (median [IQR] gestational age, 34 [28-38] weeks; 54.5% male). Prematurity occurred in 30.3%, and 55.3% underwent surgery. During hospitalization, 76.5% of high-risk infants were exposed to opioids and 7.9% received methadone. Median (IQR) length of any opioid exposure was 5 (2-12) cumulative days, and median (IQR) length of methadone treatment was 19 (7-46) cumulative days. There was significant hospital-level variation in opioid and methadone exposure and cumulative days of exposure within each US region. The computed intraclass correlation coefficient estimated that 16% of the variability in overall opioid prescribing and 20% of the variability in methadone treatment was attributed to the individual hospital. Conclusions and Relevance: In this retrospective cohort study of high-risk hospitalized infants, institution-level variation in overall opioid exposure and methadone treatment persisted across the US. These findings highlight the need for standardization of opioid prescribing in this vulnerable population.


Subject(s)
Analgesics, Opioid , Practice Patterns, Physicians' , Infant , Female , Pregnancy , Humans , Infant, Newborn , Male , Child , Adult , Analgesics, Opioid/therapeutic use , Retrospective Studies , Methadone , Hospitals, Pediatric , Infant, Extremely Low Birth Weight
4.
Pediatr Neonatol ; 65(2): 152-158, 2024 Mar.
Article in English | MEDLINE | ID: mdl-37758594

ABSTRACT

BACKGROUND: Respiratory syncytial virus (RSV) hospitalizations have increased since the 2014 guideline update recommended against the use of palivizumab for preterm infants born ≥29 0/7 weeks' gestational age (GA) without additional risk factors. A novel drug candidate, nirsevimab, has been developed for this population. We analyzed the cost-effectiveness of palivizumab/nirsevimab vs. no prophylaxis in this population. METHODS: A hybrid-Markov model predicted the RSV clinical course in the first year of life and sequelae in the subsequent four years for preterm infants from the healthcare and societal perspectives. Model parameters were derived from the literature. We calculated costs and quality-adjusted life-years (QALYs) to produce an incremental cost-effectiveness ratio (ICER) evaluated at a willingness-to-pay threshold of $150,000/QALY. Sensitivity analyses assessed model robustness. A threshold analysis examined nirsevimab pricing uncertainty. RESULTS: Compared to no prophylaxis, palivizumab costs $9572 and $9584 more from the healthcare and societal perspectives, respectively, with 0.0019 QALYs gained per patient over five years, resulting in ICERs >$5 million per QALY from each perspective. Results were robust to parameter uncertainties; probabilistic sensitivity analysis revealed that no prophylaxis had a 100% probability of being cost-effective. The threshold analysis suggested that nirsevimab is not cost-effective when compared to no prophylaxis if the price exceeds $1962 from a societal perspective. CONCLUSION: Palivizumab is dominated by no prophylaxis for preterm infants 29 0/7-34 6/7 weeks' GA with no additional risk factors. Relevant stakeholders should consider alternatives to palivizumab for this population that are both effective and economical.


Subject(s)
Antibodies, Monoclonal, Humanized , Infant, Premature , Respiratory Syncytial Virus Infections , Infant , Infant, Newborn , Humans , United States , Pregnancy , Female , Palivizumab/therapeutic use , Cost-Benefit Analysis , Gestational Age , Antiviral Agents/therapeutic use , Respiratory Syncytial Virus Infections/prevention & control , Respiratory Syncytial Viruses , Hospitalization
5.
J Craniofac Surg ; 35(1): 129-132, 2024.
Article in English | MEDLINE | ID: mdl-38011624

ABSTRACT

Class III malocclusion for individuals with cleft lip and palate has historically been managed with surgery. Orthodontic protraction is a noninvasive alternative that may be associated with lower costs. This analysis investigated the budget impact of protraction versus surgery from an institutional perspective. Using a decision tree, analysis was conducted using costs derived from Medicaid reimbursement codes and using actual institutional reimbursement. Probabilities of success, failure, and complications were based on a clinical trial comparing the 2 treatment modalities. One-way and probabilistic sensitivity analyses tested the robustness of results to model parameters. Based on Medicaid fee schedules and failure rates requiring additional surgery, the total cost of protraction was $79,506 versus $172,807 for surgery, resulting in $93,302 cost-savings per patient. The cost and probability of surgery success, as well as the cost of surgery failure and repeat surgery, had the largest impact on these cost-savings. Probabilistic sensitivity analysis showed cost-savings of nearly $92,000 or higher in >50% of simulations. This study showed that protraction is associated with lower costs than surgery and may present a cost-effective alternative to surgery in eligible, appropriate patients.


Subject(s)
Cleft Lip , Cleft Palate , Malocclusion, Angle Class III , Humans , Cleft Lip/surgery , Cleft Palate/surgery
6.
Infect Control Hosp Epidemiol ; 44(11): 1850-1853, 2023 Nov.
Article in English | MEDLINE | ID: mdl-37424225

ABSTRACT

Inappropriate dental antibiotic prescriptions to prevent infective endocarditis in the United States results in ∼$31 million in excess costs to the healthcare system and patients. This includes out-of-pocket costs ($20.5 million), drug costs ($2.69 million) and adverse event costs (eg, Clostridioides difficile and hypersensitivity) of $5.82 million (amoxicillin), $1.99 million (clindamycin), and $380,849 (cephalexin).


Subject(s)
Endocarditis, Bacterial , Endocarditis , Humans , United States , Antibiotic Prophylaxis/adverse effects , Anti-Bacterial Agents/therapeutic use , Amoxicillin , Endocarditis/etiology , Endocarditis/prevention & control , Dentistry
7.
JAMA Netw Open ; 6(6): e2316346, 2023 06 01.
Article in English | MEDLINE | ID: mdl-37266941

ABSTRACT

Importance: Autistic children have poorer oral health and greater oral care challenges, which are often associated with sensory overresponsivity, than neurotypical peers. It is important to identify innovative solutions enabling dentists to successfully perform standard clinic-based procedures for this population. Objective: To determine whether a sensory-adapted dental environment (SADE) reduces physiological and behavioral distress in autistic children undergoing dental cleanings, compared with a regular dental environment (RDE). Design, Setting, and Participants: This randomized crossover trial was conducted at a pediatric dentistry clinic in a large urban children's hospital between May 2016 and April 2022. Coders were blinded to study condition for physiological but not behavioral measurements. Autistic children aged 6 to 12 years were identified and invited to participate. Interested families were enrolled consecutively; after confirmation of autism diagnosis, children were randomized. Analysis for this per-protocol study were conducted from April to October 2022. Intervention: Each child underwent 1 RDE and 1 SADE dental cleaning, administered in randomized and counterbalanced order approximately 6 months apart. SADE included modified visual, auditory, and tactile stimuli. Main Outcomes and Measures: The primary outcome was physiological stress, assessed by electrodermal activity. The secondary outcome was behavioral distress measured from video recordings. Results: Among 452 families invited to participate, 220 children were enrolled, and 162 children (mean [SD] age, 9.16 [1.99] years; 136 [84.0%] male) with confirmed autism were randomized, with 83 children receiving RDE first and 80 children receiving SADE first. Most children (94 children [58.0%]) had moderate autism severity. Children had significantly lower physiological stress during dental care in SADE compared with RDE (mean difference in skin conductance level, -1.22 [95% CI, -2.17 to -0.27] µS), suggesting decreased sympathetic activity and increased relaxation during SADE dental care. No significant differences were found in nonspecific skin conductance responses (mean difference, -0.30 [95% CI, -0.86 to 0.25] per min). Video-coded frequency and duration of behavioral distress (but not questionnaire) measures were significantly lower in SADE vs RDE (Cohen d = -0.84 to -1.19). Physiological stress was associated with behavioral distress during the dental cleaning (eg, nonspecific skin conductance responses associated with the Frankl Scale: ß = -0.29; 95% CI, -0.39 to -0.19); age, IQ, and expressive communication moderated the intervention's success. No participants withdrew due to adverse effects. Conclusions and Relevance: In this randomized crossover trial of autistic children, using SADE was safe and efficacious in decreasing physiological and behavioral distress during dental care. This is important because enhancing oral care is critical for autistic children; this intervention may also be beneficial for populations beyond autism. Trial Registration: ClinicalTrials.gov Identifier: NCT02430051.


Subject(s)
Autistic Disorder , Humans , Male , Child , Female , Cross-Over Studies
8.
Arch Public Health ; 81(1): 58, 2023 Apr 20.
Article in English | MEDLINE | ID: mdl-37081573

ABSTRACT

BACKGROUND: Limited studies have directly compared health-related quality of life (HRQoL) in different countries during the COVID-19 global pandemic. The objective of this study was to evaluate the HRQoL outcomes in the US, Sweden, and Norway during the first year under the pandemic. METHODS: In April 2020, during early phase of the pandemic, separately in the US, Sweden, and Norway, we surveyed 2,734, 1,003 and 1,020 respondents, then again in January 2021, we collected 2,252, 1,013 and 1,011 respondents. The survey was first developed in English and translated into Swedish and Norwegian. Selected variables were used for the current study. We collected respondents' HRQoL using the EQ-5D-5L. Respondents' background information included their sociodemographic data, medical history, and COVID-19 status. We reported the EQ-5D-5L utility, EQ-VAS, and the proportion of problems with each of the EQ-5D-5L health subdomains. Population quality-adjusted life year (QALY) changes based on EQ-5D-5L utility scores were also calculated. Outcomes were stratified by age. One-way ANOVA test was used to detect significant differences between countries and Student's t-tests were used to assess the differences between waves. RESULTS: Respectively for the US, Sweden, and Norway, mean EQ-5D-5L utilities were 0.822, 0.768, and 0.808 in April 2020 (p < 0.001); 0.823, 0.783, and 0.777 in January 2021 (p < 0.001); mean EQ-VAS scores were 0.746, 0.687, and 0.692 in April 2020 (p < 0.001), 0.764, 0.682, and 0.678 in January 2021 (p < 0.001). For both waves, EQ-5D-5L utilities and EQ-VAS scores in the US remained higher than both Sweden and Norway (p < 0.001). Norwegians reported considerably lowered HRQoL over time (p < 0.01). Self-reported problems with anxiety/depression were highest for the US and Sweden, while Norwegians reported most problems with pain/discomfort, followed by anxiety/depression. The population QALYs increased in the US and Sweden, but decreased in Norway. CONCLUSIONS: In the first year of the pandemic, a rebound in HRQoL was observed in the US, but not in Sweden or Norway. Mental health issues during the pandemic warrant a major public health concern across all 3 countries.

10.
Health Qual Life Outcomes ; 20(1): 104, 2022 Jul 06.
Article in English | MEDLINE | ID: mdl-35794553

ABSTRACT

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.


Subject(s)
COVID-19 , Quality of Life , COVID-19/epidemiology , Humans , Longitudinal Studies , Pandemics , Surveys and Questionnaires
11.
J Perinatol ; 42(1): 103-109, 2022 Jan.
Article in English | MEDLINE | ID: mdl-34657144

ABSTRACT

OBJECTIVES: Examine: (1) Prevalence of diagnosed venous thromboembolism (VTE) in infants <6 months discharged from U.S. NICUs; (2) Associations between sociodemographic and clinical factors and VTE; (3) Secondary outcomes related to VTE. STUDY DESIGN: Multivariable logistic regressions examined associations between VTE and sociodemographic and clinical factors among infants <6 months discharged from Pediatric Health Information System (PHIS) NICUs between 2016 and 2019. RESULTS: Of 201,033 infants, 2720 (1.35%) had diagnosed VTE. Birthweight 300-1000 g (aOR 3.14, 95% CI 2.54-3.88), 1000-1500 g (aOR 1.77, 95% CI 1.40-2.42) versus 2500-3999 g, and public (aOR 1.18, 95% CI 1.02-1.37) versus private insurance were associated with increased odds of VTE, as were CVC, TPN, mechanical ventilation, infection, ECMO, and surgery. All types of central lines (non-tunneled and tunneled CVCs, PICCs, and umbilical catheters) had higher odds of VTE than not having that type of line. CVCs in upper versus lower extremities had higher odds of VTE. CONCLUSION: Infants with risk factors may require monitoring for VTE. Results may inform VTE prevention.


Subject(s)
Catheterization, Peripheral , Central Venous Catheters , Venous Thromboembolism , Catheterization, Peripheral/adverse effects , Central Venous Catheters/adverse effects , Child , Hospitals, Pediatric , Humans , Infant , Retrospective Studies , Risk Factors , United States/epidemiology , Venous Thromboembolism/epidemiology , Venous Thromboembolism/etiology
12.
J Perinatol ; 42(2): 223-230, 2022 02.
Article in English | MEDLINE | ID: mdl-34561556

ABSTRACT

BACKGROUND AND OBJECTIVES: Describe the financial burden and worry that families of preterm infants experience after discharge from the neonatal intensive care unit (NICU). METHODS: We surveyed 365 parents of preterm infants in a cross-sectional study regarding socio-demographics, supplemental security income (SSI), and financial worry. We completed a multivariable logistic regression model to examine the adjusted association of financial worry with modifiable factors. RESULTS: We found that 53% of participants worried about healthcare costs after NICU discharge. After adjusting for socio-demographic and infant characteristics, we identified that, aOR (95% CI), out-of-pocket costs from the NICU index hospitalization, 3.51 (1.7, 7.26) and durable medical equipment use, 2.41 (1.11, 5.23) was associated with increased financial worry while enrollment in SSI, 0.38 (0.19, 0.76) was associated with decreased financial worry. CONCLUSIONS: We identified factors that could contribute to financial burden after NICU discharge that may advise future work to target financial support systems.


Subject(s)
Infant, Premature , Intensive Care Units, Neonatal , Cross-Sectional Studies , Financial Stress , Humans , Infant , Infant, Newborn , Patient Discharge
13.
Arch Dis Child Fetal Neonatal Ed ; 107(3): 324-328, 2022 May.
Article in English | MEDLINE | ID: mdl-34462319

ABSTRACT

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.


Subject(s)
Hypothermia, Induced , Hypoxia-Ischemia, Brain , Infant, Newborn, Diseases , Female , Gestational Age , Humans , Hypothermia, Induced/adverse effects , Hypoxia-Ischemia, Brain/complications , Infant , Infant, Newborn , Infant, Newborn, Diseases/therapy , Pregnancy , Retrospective Studies
14.
JHEP Rep ; 3(6): 100367, 2021 Dec.
Article in English | MEDLINE | ID: mdl-34825154

ABSTRACT

BACKGROUND & AIMS: Uncertainties exist surrounding the timing of liver transplantation (LT) among patients with acute-on-chronic liver failure grade 3 (ACLF-3), regarding whether to accept a marginal quality donor organ to allow for earlier LT or wait for either an optimal organ offer or improvement in the number of organ failures, in order to increase post-LT survival. METHODS: We created a Markov decision process model to determine the optimal timing of LT among patients with ACLF-3 within 7 days of listing, to maximize overall 1-year survival probability. RESULTS: We analyzed 6 groups of candidates with ACLF-3: patients age ≤60 or >60 years, patients with 3 organ failures alone or 4-6 organ failures, and hepatic or extrahepatic ACLF-3. Among all groups, LT yielded significantly greater overall survival probability vs. remaining on the waiting list for even 1 additional day (p <0.001), regardless of organ quality. Creation of 2-way sensitivity analyses, with variation in the probability of receiving an optimal organ and expected post-transplant mortality, indicated that overall survival is maximized by earlier LT, particularly among candidates >60 years old or with 4-6 organ failures. The probability of improvement from ACLF-3 to ACLF-2 does not influence these recommendations, as the likelihood of organ recovery was less than 10%. CONCLUSION: During the first week after listing for patients with ACLF-3, earlier LT in general is favored over waiting for an optimal quality donor organ or for recovery of organ failures, with the understanding that the analysis is limited to consideration of only these 3 variables. LAY SUMMARY: In the setting of grade 3 acute-on-chronic liver failure (ACLF-3), questions remain regarding the timing of transplantation in terms of whether to proceed with liver transplantation with a marginal donor organ or to wait for an optimal liver, and whether to transplant a patient with ACLF-3 or wait until improvement to ACLF-2. In this study, we used a Markov decision process model to demonstrate that earlier transplantation of patients listed with ACLF-3 maximizes overall survival, as opposed to waiting for an optimal donor organ or for improvement in the number of organ failures.

15.
Value Health ; 24(7): 917-924, 2021 07.
Article in English | MEDLINE | ID: mdl-34243834

ABSTRACT

OBJECTIVES: Throughout 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. METHODS: We 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. RESULTS: We 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. CONCLUSIONS: There 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.


Subject(s)
Communicable Diseases, Emerging/drug therapy , Communicable Diseases, Emerging/prevention & control , Epidemiologic Methods , Cost-Benefit Analysis , Disease Outbreaks/prevention & control , Disease Outbreaks/statistics & numerical data , Forecasting/methods , Humans , Policy Making , Reference Standards
16.
Genet Med ; 23(10): 1854-1863, 2021 10.
Article in English | MEDLINE | ID: mdl-34040192

ABSTRACT

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.


Subject(s)
Exome , Mitochondrial Diseases , Child , Cost-Benefit Analysis , Exome/genetics , Humans , Infant, Newborn , Mitochondrial Diseases/diagnosis , Mitochondrial Diseases/genetics , Quality-Adjusted Life Years
18.
Clin Microbiol Infect ; 27(10): 1448-1454, 2021 Oct.
Article in English | MEDLINE | ID: mdl-33878506

ABSTRACT

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.


Subject(s)
Anti-Bacterial Agents , Antibodies, Monoclonal , Broadly Neutralizing Antibodies , Clostridium Infections , Fidaxomicin , Vancomycin , Anti-Bacterial Agents/economics , Anti-Bacterial Agents/therapeutic use , Antibodies, Monoclonal/economics , Antibodies, Monoclonal/therapeutic use , Broadly Neutralizing Antibodies/economics , Broadly Neutralizing Antibodies/therapeutic use , Clostridioides difficile , Clostridium Infections/drug therapy , Cost-Benefit Analysis , Fidaxomicin/economics , Fidaxomicin/therapeutic use , Humans , United States , Vancomycin/economics , Vancomycin/therapeutic use
19.
J Pediatr Surg ; 56(12): 2311-2317, 2021 Dec.
Article in English | MEDLINE | ID: mdl-33743989

ABSTRACT

BACKGROUND: Many studies have established that extracorporeal membrane oxygenation (ECMO) can be a cost-effective treatment in some populations, but limited data exist on which factors are associated with length of stay (LOS) and total hospital costs. This study aimed to determine if inborn (i.e., cared for in their birth hospitals) neonates who receive ECMO have different resource utilization and outcomes compared to outborn (i.e., not cared for in their birth hospitals) neonates who receive ECMO. METHODS: A retrospective cohort study was conducted using the Healthcare Cost and Utilization Project's Kids' Inpatient Database from 1997-2012. Neonates (infants, <28 days) placed on ECMO were categorized as either inborn or outborn. Salient clinical characteristics were compared between groups. A multivariable analysis was performed to identify the factors associated with length of stay (LOS), total hospital costs, and mortality in these two patient populations. RESULTS: Of 5,152 neonates receiving ECMO, 800 were inborn and 4,352 were outborn. Inborn neonates were more frequently diagnosed with cardiac-related diagnoses (70.5% vs 62.1%, p < 0.001). After adjusting for demographics and hospital-level factors, inborn neonates had longer hospital LOS (13.2 days, 95% CI, 8.7-18.7; p < 0.001), higher total encounter costs ($62,000, 95% CI, 40,000-85,000; p < 0.001) and higher mortality (OR 2.4, 95% CI 1.9-2.9; p < 0.001) compared to outborn neonates. CONCLUSIONS: Inborn neonates placed on ECMO were more frequently diagnosed with cardiac-related diseases or congenital diaphragmatic hernia, had longer LOS, higher total encounter costs, and higher mortality rates relative to their outborn counterparts, and likely represent a higher risk population. These two populations of infants may be inherently different and their differences should be further explored to inform decision making about optimal site of delivery.


Subject(s)
Extracorporeal Membrane Oxygenation , Hernias, Diaphragmatic, Congenital , Hospital Costs , Humans , Infant , Infant, Newborn , Retrospective Studies , Treatment Outcome
20.
J Gen Intern Med ; 36(5): 1292-1301, 2021 05.
Article in English | MEDLINE | ID: mdl-33686587

ABSTRACT

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.


Subject(s)
COVID-19 , Population Health , Adolescent , Adult , Aged , Health Status , Humans , Middle Aged , Pandemics , Quality of Life , SARS-CoV-2 , Surveys and Questionnaires , Young Adult
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