Cost-effectiveness Evaluation Research Articles

Mitraclip Versus Medical Therapy or Surgery in Patients With Mitral Regurgitation: Long-Term Outcomes Determined by the Reconstruction of Individual Patient Data.

Although MitraClip has been studied in numerous trials, its evidence in the long term is basedon a few original studies. We used an original technique of evidence synthesis to review long-termcomparative trialsevaluating MitraClip. We searched the PubMed database to select long-term comparative trials of MitraClip. The endpoint was all-cause mortality (minimum follow-up, one year). Included trials were analyzed using theIPDfromKM (reconstruct Individual Patient Data from published Kaplan-Meier survival curves)method to reconstruct individual patient data from Kaplan-Meier curves. Standard survivalstatistics were used to interpret these long-term efficacy data. The survival benefit per patient was estimatedfrom the restricted mean survival time (RMST). Six comparative studies of MitraClip were included; 973 patients were treated with MitraClip (six arms), 717 with medical therapy (five arms), and 80 with surgical repair or replacement (one arm). In our mainanalysis, the outcomes observed in patients treated with MitraClip were significantly better than thoseof medical therapy (hazard ratio for all-cause mortality, 0.5276; 95% confidence interval, 0.4412 to 0.6309;p < 0.001); the number of patients treated with surgery was too small to make reliable comparisons. Mediansurvival was 30.4 months for medical therapy versus not reached for the other two groups. RMST was 43.931 and33.756 monthsfor MitraClip and controls, respectively, yielding a gain per patient of 10.17 months (95%confidence interval, 7.47 to 12.88). In our simplified cost-effectiveness evaluation, a gain of approximately10 months per patient compared favorably with the device cost.Our analysis provided an original interpretation of the long-termevidence available on MitraClip.

Effectiveness and cost-effectiveness of a 12-month automated text message intervention for weight management in postpartum women with overweight or obesity: protocol for the Supporting MumS (SMS) multisite, parallel-group, randomised controlled trial

IntroductionThe reproductive years can increase women’s weight-related risk. Evidence for effective postpartum weight management interventions is lacking and engaging women during this life stage is challenging. Following a promising pilot...

Abstract PO2-03-09: Cost-effectiveness analysis of RAD51 functional biomarker for platinum sensitivity in the GeparSixto trial

Abstract Background: Homologous recombination repair deficiency (HRD) has long been recognized as a predictive biomarker for platinum sensitivity and is present in up to 60% of early triple negative breast cancers (TNBC). Different commercial and lab-developed assays to measure HRD are available, but they do not necessarily reflect homologous recombination functionality. Functional HRD, defined by a low score in RAD51 foci, is emerging as an accurate and dynamic biomarker to predict DNA damaging agents’ response. We performed a cost-effectiveness evaluation of the RAD51 test compared to tumor BRCA1/2 mutation (tBRCA1/2) or genomic HRD by Myriad Mychoice® to identify patients sensitive to platinum-based chemotherapy within the GeparSixto trial. Methods: The effectiveness measure was the primary outcome of the GeparSixto study: rate of pathological complete response (pCR), defined as ypT0 ypN0. The pharmacological costs were calculated following treatment protocol of GeparSixto where TNBC patients received neoadjuvant paclitaxel plus Myocet®-nonpegylated liposomal doxorubicin (PM) or PM plus carboplatin (PMCb), both arms including bevacizumab. Bevacizumab was excluded from the cost analysis as it is not administered by standard practice. The cost of a session/stay at the day hospital was added, as well as the cost of the test used, comparing 4 scenarios: All comers (assuming all patients receiving PMCb); RAD51 test (assuming RAD51 ≤10% received PMCb and RAD51 &amp;gt;10% received PM); tBRCA1/2 test (assuming tBRCA1/2 mutated received PMCb and tBRCA1/2 non-mutated received PM) and genomic HRD by Myriad Mychoice® (HRD score ≥42 received PMCb, and HRD score &amp;lt;42 received PM). The available commercial costs were considered for tBRCA1/2 and Myriad Mychoice®, and the VHIO performance cost was considered for the RAD51 test. Parameter uncertainties were assessed by means of one-way and probabilistic sensitivity analyses. Results: All comers total cost was 20,530€ per patient, with associated pCR rate of 54.5%. Functional RAD51 test was associated with a pCR probability of 55.1% with a Δ cost of 198€, as opposed to tBRCA1/2 which presented a pCR rate of 40.1% with a Δ cost of 814€, and genomic HRD with 47% pCR rate and the higher Δ cost of 1,023€ per patient, when compared to all comers. The incremental cost-effectiveness ratio (Δ cost/Δ pCR) was 33,000 for RAD51 test and dominated in both tBRCA1/2 and genomic HRD tests. These results were robust on one way and probabilistic sensitivity analyses. Conclusion: The most efficient scenarios are the functional RAD51 test and all comers. Both tBRCA1/2 and genomic HRD by Myriad Mychoice® scenarios provide worse health outcomes at a higher cost, based on the data of the GeparSixto trial. This study highlights the potential overall benefit of functional HRD biomarkers to predict PARPi sensitivity. Citation Format: Isabel Pimentel, Carles Forné, Alba Llop-Guevara, Misericòrdia Carles-Lavila, Violeta Serra, Montserrat Rué, Judith Balmaña. Cost-effectiveness analysis of RAD51 functional biomarker for platinum sensitivity in the GeparSixto trial [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO2-03-09.

Fully Automated Quantitative Coronary Angiography Versus Optical Coherence Tomography Guidance for Coronary Stent Implantation (FLASH): Study Protocol for a Randomized Controlled Non-Inferiority Trial

BackgroundArtificial intelligence-based quantitative coronary angiography (AI-QCA) has been developed to provide a more objective and reproducible data about the severity of coronary artery stenosis and the dimensions of the vessel for intervention in real-time, overcoming the limitations of significant inter- and intra-observer variability, and time-consuming nature of on-site QCA, without requiring extra time and effort. Compared with the subjective nature of visually estimated conventional CAG guidance, AI-QCA guidance provides a more practical and standardized angiography-based approach. Although the advantage of intravascular imaging-guided PCI is increasingly recognized, their broader adoption is limited by clinical and economic barriers in many catheterization laboratories. MethodsThe FLASH (Fully automated quantitative coronary angiography versus optical coherence tomography guidance for coronary stent implantation) trial is a randomized, investigator-initiated, multicenter, open-label, non-inferiority trial comparing the AI-QCA-assisted PCI strategy with optical coherence tomography-guided PCI strategy in patients with significant coronary artery disease. All operators will utilize a novel, standardized AI-QCA software and PCI protocol in the AI-QCA-assisted group. A total of 400 patients will be randomized to either group at a 1:1 ratio. The primary endpoint is the minimal stent area (mm2), determined by the final OCT run after completion of PCI. Clinical follow-up and cost-effectiveness evaluations are planned at 1 month and 6 months for all patients enrolled in the study. ResultsEnrollment of a total of 400 patients from the 13 participating centers in South Korea will be completed in February 2024. Follow-up of the last enrolled patients will be completed in August 2024, and primary results will be available by late 2024. ConclusionThe FLASH is the first clinical trial to evaluate the feasibility of AI-QCA-assisted PCI, and will provide the clinical evidence on AI-QCA assistance in the field of coronary intervention.

Cost-effectiveness analysis of transcatheter aortic valve implantation in aortic stenosis patients at low- and intermediate-surgical risk in Japan

Objective To analyze the cost-effectiveness of transcatheter aortic valve implantation (TAVI) using the SAPIEN 3 (Edwards Lifesciences, Irvine, CA) compared to surgical aortic valve replacement (SAVR) in low- and intermediate-risk patients from a Japanese public healthcare payer perspective. Methods A Markov model cost-effectiveness analysis was developed. Clinical and utility data were extracted from a systematic literature review. Cost inputs were obtained from analysis of the Medical Data Vision claims database and supplemented with a targeted literature search. The robustness of the results was assessed using sensitivity analyses. Scenario analyses were performed to determine the impact of lower mean age (77.5 years) and the effect of two different long-term mortality hazard ratios (TAVI versus SAVR: 0.9–1.09) on both risk-level populations. This analysis was conducted according to the guidelines for cost-effectiveness evaluation in Japan from Core 2 Health. Results In intermediate-risk patients, TAVI was a dominant procedure (TAVI had lower cost and higher effectiveness). In low-risk patients, the incremental cost effectiveness ratio (ICER) for TAVI was ¥750,417/quality-adjusted-life-years (QALY), which was below the cost-effectiveness threshold of ¥5 million/QALY. The ICER for TAVI was robust to all tested sensitivity and scenario analyses. Conclusions TAVI was dominant and cost-effective compared to SAVR in intermediate- and low-risk patients, respectively. These results suggest that TAVI can provide meaningful value to Japanese patients relative to SAVR, at a reasonable incremental cost for patients at low surgical risk and potentially resulting in cost-savings in patients at intermediate surgical risk.

Cost-effectiveness and economic growth potential evaluation of olive pomace gasification process to sustainable fuel: Comparison study of different gasifying agent

This research addresses the growing global demand for sustainable and renewable energy solutions, focusing on biomass gasification (BG) as a promising avenue. Olive pomace is explored as a primary feedstock, assessing its potential as a plentiful and renewable energy source. The study aims to bridge existing gaps in the literature, particularly in the simultaneous and comparative examination of CO2, steam, and air as traditional gasifying agents. Utilizing Aspen Plus, a validated simulation tool, the research models the gasification process (GP) while considering key parameters such as gasifying agent, feed composition, and temperature. Through meticulous simulation and validation against experimental data, the goal is to develop a robust model tailored for gasification. A thorough sensitivity analysis evaluates critical parameters' impact on exergy efficiency, levelized cost of hydrogen (LCOH), and molar flow of hydrogen (H2). Parameters include temperature variations (600°C to 1000°C), equivalence ratio (ER) for the air agent (0.15–0.3), C/CO2 ratio for the CO2 agent (1–2.5), and SBR for the steam agent (0.5–2). The aim is to provide a nuanced understanding of these parameters and their influence on system performance, offering valuable insights for process optimization. The response surface methodology is employed to optimize critical parameters for distinct gasification agents, aiming to identify the most economically efficient agent. The study's findings are crucial for enhancing BG efficiency and developing sustainable energy systems. An economic evaluation underscores the cost-effectiveness of the GP, emphasizing its potential for economic growth in the renewable energy sector and long-term savings. In the concluding phase, optimization of the GP aimed at minimizing LCOH and maximizing molar flow of H2 and exergy efficiency was conducted. Upon scrutinizing the optimization results, a compelling conclusion arose: The steam agent emerged as the most suitable, with 48% exergy efficiency, 1.77 €/kg LCOH, and an optimal temperature of 884°C. Notably, steam showcased remarkable exergy efficiency, even when subjected to lower temperatures, positioning it as a superior choice.

Cost-effective evaluation of Aptamer candidates in SELEX-based Aptamer isolation

Aptamers are short, single-stranded nucleic acids with high affinity and specificity for various targets, making them valuable in diagnostics and therapeutics. Their isolation traditionally involves a time-consuming and costly process called SELEX. While SELEX methods have evolved to improve binding and amplification, the crucial step of aptamer identification from sequencing data remains expensive and often overlooked. Common identification methods require modification of aptamer candidates with labels like biotin or fluorescent dyes, which becomes costly and cumbersome for high-throughput sequencing data. This paper presents an efficient and cost-effective approach to streamline aptamer identification. It employs asymmetric polymerase chain reaction (PCR) to generate modified single-stranded DNA copies of aptamer candidates, simplifying the modification process. By using excess modified forward primers and limited reverse primers, this method reduces costs since only unmodified candidates need to be synthesized initially. The approach was demonstrated with an IgE protein aptamer and successfully applied to identify aptamers from a pool of 12 candidates against a monoclonal antibody. The validity of the results was further confirmed through the direct synthesis of fluorophore-conjugated aptamer candidates, yielding consistent outcomes while reducing the cost by threefold. This approach addresses a critical bottleneck in aptamer discovery by significantly reducing the time and cost associated with aptamer identification, facilitating aptamer-based research and making aptamers more accessible for various applications in diagnostics and therapeutics.

Economic analysis of a health system strengthening program in the Democratic Republic of the Congo

Due to the Democratic Republic of the Congo’s (DRC) precarious health system that provides only limited access to health care, the European Union, via Memisa Belgium, implemented a program to strengthen provision of and access to health care (known as PRO DS) in the provinces of Kongo Central and Ituri. This program took a holistic approach, seeking to improve equitable access and combat malnutrition. To measure the program’s social return on investment and to estimate the cost per capita and effectiveness per euro invested (efficiency), a 61-month (1 July 2017 to 31 July 2022) cost-effectiveness evaluation with a societal perspective was carried out. The double-difference method was used to compare the results of PRO DS and non-PRO DS zones. The social return on investment was assessed via the ratio of effectiveness to costs. Analyses revealed the program cost between 3.72 and 3.96 euros per capita per year (2022) in Kongo Central, and between 3.12 and 3.36 euros in Ituri. Importantly, it was cost-effective in the areas of reproductive health, nutrition, and the use of health and nutrition services. The program’s strong nutritional component and overall holistic vision may explain why it was so efficient. PRO DS stands out from other programs that focus solely on one specific problem or population. Although the program has some limitations, it would be worthwhile for the government to invest in it.

Measuring refugees' capabilities: translation, adaptation, and valuation of the OxCAP-MH into Juba Arabic for use among South Sudanese male refugees in Uganda.

Forcibly displaced populations are highly vulnerable to psychosocial distress and mental disorders, including alcohol misuse. In an ongoing trial that seeks to develop a transdiagnostic intervention addressing psychological distress and alcohol use disorders among conflict-affected populations, we will carry out a cost-effectiveness evaluation using a capability-based Oxford Capabilities Mental Health (OxCAP-MH) measure. The OxCAP-MH is a 16-item questionnaire developed from the Capability Approach, that covers multiple domains of functioning and welfare. The aim of the current paper is to present the results of the translation, cultural adaptation and valuation of the OxCAP-MH into Juba Arabic for South Sudanese refugees living in Uganda. We adhered to the official Translation and Linguistic Validation process of the OxCAP-MH. To carry out the translation, the Concept Elaboration document, official English version of the OxCAP-MH, and the Back-Translation Review Template were used. Four independent translators were used for forward and back translations. The reconciled translated version was then piloted in two focus group discussions (N = 16) in Rhino refugee settlement. A most important to least important valuation of the sixteen capability domains covered in the OxCAP-MH was also done. The Juba Arabic version of the OxCAP-MH was finalized following a systematic iterative process. The content of the Juba Arabic version remained unchanged, but key concepts were adapted to ensure cultural acceptability, feasibility, and comprehension of the measure in the local context of Rhino refugee settlement. Most participants had low levels of literacy and required support with filling in the tool. Participants suggested an additional capability that is currently not reflected in the OxCAP-MH, namely access to food. Furthermore, discussions around the valuation exercise of the sixteen domains led to two separate importance scales, which showed relevant differences. In this context, the OxCAP-MH was considered culturally acceptable. The valuation exercise proved cognitively demanding. Participants voiced confusion over how to answer the questions on the OxCAP-MH instrument due to low levels of literacy. These concerns invite consideration for future research to consider how measures such as the OxCAP-MH can be made more accessible to individuals with low literacy rates in resource poor settings.

Challenges, opportunities, and priorities for tier-1 emergency medical services (EMS) development in low- and middle-income countries: A modified Delphi-based consensus study among the global prehospital consortium

IntroductionThough the disease burden addressable by prehospital and out-of-hospital emergency care(OHEC) spans communicable diseases, maternal conditions, chronic conditions and injury, the single largest disability-adjusted life year burden contributor is injury, primarily driven by road traffic injuries(RTIs). Establishing OHEC for RTIs and other common emergencies in low- and middle-income countries(LMICs) where the injury burden is disproportionately greatest is a logical first step toward more comprehensive emergency medical services(EMS). However, with limited efforts to formalize and expand existing informal bystander care networks, there is a lack of consensus on how to develop and maintain bystander-driven Tier-1 EMS systems in LMICs. Resultantly, Tier-1 EMS development is fragmented among non-governmental organizations and the public sector globally. MethodsA steering committee coordinated a 9-round, modified Delphi-based expert discussion to identify current challenges, opportunities, and priorities in Tier-1 EMS development globally. 11 panelists represented seven Global Prehospital Consortium(GPC) member organizations with a mean 9.57 years of organizational Tier-1 EMS development/implementation experience(median = 9 years). The consortium represents the largest collaboration between organizations directing Tier-1 EMS programs globally across 12 countries on 3 continents(Americas, sub-Saharan Africa, and South Asia) with 22,000 first responders. ResultsThe GPC identified seven priority areas for Tier-1 EMS development: infrastructure/operations, communication, education/training, impact evaluation, financing, governance/legal, and transportation/equipment. A high level of consensus exists regarding priorities for investigation, including Tier-1 responder density/distribution, Tier-1 patient data variable standardization for trauma registries/quality improvement, dispatch technologies/protocols, modular curricula, broader cost-effectiveness and impact evaluation indices capturing secondary impacts of EMS, standardizing legal protections for first responders, and transportation/equipment standards. DiscussionConsensus is necessary to avoid duplicative and disorganized efforts due to the fragmented nature of parallel Tier-1 EMS efforts globally. A Delphi-like multi-round expert discussion among the members of the largest collaboration between organizations directing Tier-1 EMS programs globally generated relevant priorities to direct future efforts.

An economic evaluation of chronic obstructive pulmonary disease clinical pathway in Saskatchewan, Canada: Data-driven techniques to identify cost-effectiveness among patient subgroups.

Saskatchewan has implemented care pathways for several common health conditions. To date, there has not been any cost-effectiveness evaluation of care pathways in the province. The objective of this study was to evaluate the real-world cost-effectiveness of a chronic obstructive pulmonary disease (COPD) care pathway program in Saskatchewan. Using patient-level administrative health data, we identified adults (35+ years) with COPD diagnosis recruited into the care pathway program in Regina between April 1, 2018, and March 31, 2019 (N = 759). The control group comprised adults (35+ years) with COPD who lived in Saskatoon during the same period (N = 759). The control group was matched to the intervention group using propensity scores. Costs were calculated at the patient level. The outcome measure was the number of days patients remained without experiencing COPD exacerbation within 1-year follow-up. Both manual and data-driven policy learning approaches were used to assess heterogeneity in the cost-effectiveness by patient demographic and disease characteristics. Bootstrapping was used to quantify uncertainty in the results. In the overall sample, the estimates indicate that the COPD care pathway was not cost-effective using the willingness to pay (WTP) threshold values in the range of $1,000 and $5,000/exacerbation day averted. The manual subgroup analyses show the COPD care pathway was dominant among patients with comorbidities and among patients aged 65 years or younger at the WTP threshold of $2000/exacerbation day averted. Although similar profiles as those identified in the manual subgroup analyses were confirmed, the data-driven policy learning approach suggests more nuanced demographic and disease profiles that the care pathway would be most appropriate for. Both manual subgroup analysis and data-driven policy learning approach showed that the COPD care pathway consistently produced cost savings and better health outcomes among patients with comorbidities or among those relatively younger. The care pathway was not cost-effective in the entire sample.

Exploring Tourists’ Behavioral Patterns in Bali’s Top-Rated Destinations: Perception and Mobility

The tourism sector plays a crucial role in the global economy, encompassing both physical infrastructure and cultural engagement. Indonesia has a wide range of attractions and has experienced remarkable growth, with Bali as a notable example of this. With the rapid advancements in technology, travelers now have the freedom to explore independently, while online travel agencies (OTAs) serve as important resources. Reviews from tourists significantly impact the service quality and perception of destinations, and text mining is a valuable tool for extracting insights from unstructured review data. This research integrates multiclass text classification and a network analysis to uncover tourists’ behavioral patterns through their perceptions and movement. This study innovates beyond conventional sentiment and cognitive image analysis to the tourists’ perceptions of cognitive dimensions and explores the sentiment correlation between different cognitive dimensions. We find that destinations generally receive positive feedback, with 80.36% positive reviews, with natural attractions being the most positive aspect while infrastructure is the least positive aspect. We highlight that qualitative experiences do not always align with quantitative cost-effectiveness evaluations. Through a network analysis, we identify patterns in tourist mobility, highlighting three clusters of attractions that cater to diverse preferences. This research underscores the need for tourism destinations to strategically adapt to tourists’ varied expectations, enhancing their appeal and aligning their services with preferences to elevate destination competitiveness and increase tourist satisfaction.

Quality assurance of 3D-printed patient specific anatomical models: a systematic review

BackgroundThe responsible use of 3D-printing in medicine includes a context-based quality assurance. Considerable literature has been published in this field, yet the quality of assessment varies widely. The limited discriminatory power of some assessment methods challenges the comparison of results. The total error for patient specific anatomical models comprises relevant partial errors of the production process: segmentation error (SegE), digital editing error (DEE), printing error (PrE). The present review provides an overview to improve the general understanding of the process specific errors, quantitative analysis, and standardized terminology.MethodsThis review focuses on literature on quality assurance of patient-specific anatomical models in terms of geometric accuracy published before December 4th, 2022 (n = 139). In an attempt to organize the literature, the publications are assigned to comparable categories and the absolute values of the maximum mean deviation (AMMD) per publication are determined therein.ResultsThe three major examined types of original structures are teeth or jaw (n = 52), skull bones without jaw (n = 17) and heart with coronary arteries (n = 16). VPP (vat photopolymerization) is the most frequently employed basic 3D-printing technology (n = 112 experiments). The median values of AMMD (AMMD: The metric AMMD is defined as the largest linear deviation, based on an average value from at least two individual measurements.) are 0.8 mm for the SegE, 0.26 mm for the PrE and 0.825 mm for the total error. No average values are found for the DEE.ConclusionThe total error is not significantly higher than the partial errors which may compensate each other. Consequently SegE, DEE and PrE should be analyzed individually to describe the result quality as their sum according to rules of error propagation. Current methods for quality assurance of the segmentation are often either realistic and accurate or resource efficient. Future research should focus on implementing models for cost effective evaluations with high accuracy and realism. Our system of categorization may be enhancing the understanding of the overall process and a valuable contribution to the structural design and reporting of future experiments. It can be used to educate specialists for risk assessment and process validation within the additive manufacturing industry.Graphical Context of the figures in this review. Center: Fig. 5+ 7; top (blue): Fig. 8; right (green): Fig. 9; bottom (yellow): Fig. 10; left (red): Fig. 11. A version in high resolution can be found online in the supplementary material.

Recommended Ten Years' Follow-Up Strategy for Small Hepatocellular Carcinoma after Radiofrequency Ablation: A Cost-Effectiveness Evaluation.

An optimal follow-up schedule for small (≤3 cm) hepatocellular carcinoma (HCC) after radiofrequency ablation (RFA) remains unclear in clinical guidelines. We aimed to assess the cost-effectiveness of follow-up strategies in patients with small HCC after RFA. In total, 11,243 patients were collected from global institutions to calculate recurrence rates. Subsequently, a Markov model covering a 10-year period was developed to compare 25 surveillance strategies involving different surveillance techniques (computed tomography [CT], magnetic resonance imaging or ultrasonography [US] and alpha-fetoprotein [AFP]) and intervals (3 or 6 months). The study endpoint was incremental cost-effectiveness ratio (ICER), which represented additional cost per incremental quality-adjusted life-year (QALY). Sensitivity analysis was conducted by varying the values of input parameters to observe the ICER. In a base case analysis, the dominant strategy was CT every three months during an initial two years, followed by semi-annual CT, and then switch to biannual the combination of US screening and AFP testing after five years (m3_CT-m6_CT-m6_USAFP), with an ICER of $68,570.92 compared to the "not followed" strategy. One-way sensitivity analysis showed the ICER consistently remained below the willingness-to-pay (WTP) threshold of $100,000.00. In a probabilistic sensitivity analysis, m3_CT-m6_CT-m6_USAFP was the most cost-effective approach in 95.6% of simulated scenarios at a WTP threshold. For small HCC after RFA, the recommended follow-up strategy is CT, with scans scheduled every three months for the first two years, every six months thereafter, and transition to biannual the combination of US screening and AFP testing after five years.

Cost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura

AbstractWhile awaiting confirmatory results, empiric therapy for patients suspected to have immune thrombotic thrombocytopenic purpura (iTTP) provides benefits and also accrues risks and costs. Rapid assays for ADAMTS13 may be able to avoid the cost and risk exposure associated with empiric treatment. We conducted, to our knowledge, the first cost-effectiveness evaluation of testing strategies with rapid vs traditional ADAMTS13 assays in patients with intermediate- to high-risk PLASMIC scores, with and without caplacizumab use. We built a Markov cohort simulation with 4 clinical base-case analyses: (1) intermediate-risk PLASMIC score with caplacizumab; (2) intermediate-risk PLASMIC score without caplacizumab; (3) high-risk PLASMIC score with caplacizumab; and (4) high-risk PLASMIC score without caplacizumab. Each of these evaluated 3 testing strategies: (1) rapid assay (<1-hour turnaround); (2) in-house fluorescence resonance energy transfer (FRET)–based assay (24-hour turnaround); and (3) send-out FRET-based assay (72-hour turnaround). The primary outcome was the incremental net monetary benefit reported over a 3-day time horizon and across accepted willingness-to-pay thresholds in US dollars per quality-adjusted life-year (QALY). While accruing the same amount of QALYs, the rapid assay strategy saved up to $46 820 (95% CI, $41 961-$52 486) per patient tested. No parameter variation changed the outcome. In probabilistic sensitivity analyses, the rapid assay strategy was favored in 100% (3 base cases and scenario analyses) and 99% (1 base-case and scenario analysis) across 100 000 Monte Carlo iterations within each. Rapid ADAMTS13 testing for patients with intermediate- or high-risk PLASMIC scores yields significant per patient cost savings, achieved by reducing the costs associated with unnecessary therapeutic plasma exchange and caplacizumab therapy in patients without iTTP.

Ultrasound-Derived Fat Fraction for Hepatic Steatosis Assessment: Prospective Study of Agreement With MRI PDFF and Sources of Variability in a Heterogeneous Population.

Background: Metabolic dysfunction-associated steatotic liver disease is a growing global public health concern. Quantitative ultrasound measurements, such as ultrasound-derived fat fraction (UDFF), could provide noninvasive, cost-effective, and portable steatosis evaluation. Objective: To evaluate utility of UDFF for steatosis assessment using PDFF as reference in patients undergoing liver MRI for heterogeneous indications, and to assess UDFF variability. Methods: This prospective study included a primary analysis of 187 patients (mean age, 53.8 years; 112 men, 75 women) who underwent 3-T liver MRI for any clinical indication from December 2020 to July 2021. Patients underwent investigational proton-density fat fraction (PDFF) measurement using MR spectroscopy, and investigational ultrasound with UDFF calculation (mean of 5 intercostal measurements) within 1 hour after MRI. In a subanalysis, 21 of these patients underwent additional UDFF measurements 1, 3, and 5 hours after meal consumption. The study also included repeatability and reproducibility (R&R) analysis of 30 patients (mean age, 26.3 years; 11 men, 20 women) who underwent clinical abdominal ultrasound between November 2022 and January 2023; in these patients, three operators sequentially performed UDFF measurements. Results: In primary analysis, UDFF and PDFF showed intraclass correlation coefficient (ICC) of 0.79. In Bland-Altmananalysis, UDFF and PDFF showed mean difference of 1.5% (95% CI, 0.6-2.4%), with 95% limits of agreement from -11.0% to 14.0%. UDFF exhibited AUC for detecting PDFF at historic thresholds of ≥6.5%, ≥17.4%, and ≥22.1% of 0.90, 0.95, and 0.95, respectively. In subanalysis, mean UDFF was not significantly different across time points with respect to meal consumption (p=.21). In R&R analysis, ICC for intraoperator repeatability ranged from 0.98 to 0.99, and for interoperator reproducibility from 0.90 to 0.96. Visual assessment of patient-level data plots indicated increasing variability of mean UDFF measurements across operators, and of intercostal measurements within individual patients, with increasing steatosis. Conclusion: UDFF showed robust agreement with PDFF, diagnostic performance for steatosis grades, as well as intraoperator repeatability and interoperator reproducibility. Nonetheless, UDFF exhibited bias toward slightly larger values versus PDFF, intraoperator and interoperator variation increased with increasing steatosis. Clinical Impact: UDFF shows promise for steatosis assessment across diverse populations, although continued optimization remains warranted.

Regional Forest Structure Evaluation Model Based on Remote Sensing and Field Survey Data

The assessment of a forest’s structure is pivotal in guiding effective forest management, conservation efforts, and ensuring sustainable development. However, traditional evaluation methods often focus on isolated forest parameters and incur substantial data acquisition costs. To address these limitations, this study introduces a cost-effective and innovative evaluation model that incorporates remote sensing imagery and machine learning algorithms. This model holistically considers the forest composition, the tree age structure, and spatial configuration. Using a comprehensive approach, the forest structure in Longquan City was evaluated at the stand level and categorized into three distinct categories: good, moderate, and poor. The construction of this evaluation model drew upon multiple data sources, namely Sentinel-2 imagery, digital elevation models (DEMs), and forest resource planning and design survey data. The model employed the Recursive Feature Elimination with Cross-Validation (RFECV) method for feature selection, alongside various machine learning algorithms. The key findings from this research are summarized as follows: The application of the RFECV method proved effective in eliminating irrelevant factors, reducing data dimensionality and, subsequently, enhancing the model’s generalizability; among the tested machine learning algorithms, the CatBoost model emerged as the most accurate and stable across all the datasets; specifically, the CatBoost model achieved an impressive overall accuracy of 88.07%, a kappa coefficient of 0.6833, and a recall rate of 76.86%. These results significantly surpass the classification precision of previous methods. The forest structure assessment of Longquan City revealed notable variations in the forest quality distribution. Notably, forests classified as “good” quality comprised 11.18% of the total, while “medium” quality forests constituted the majority at 76.77%. In contrast, “poor” quality forests accounted for a relatively minor proportion of the total, at 12.05%. The distribution findings provide valuable insights for targeted forest management and conservation strategies.

Evaluation of the Cost-Effectiveness of Evidence-Based Interventions to Increase Female Breast and Cervical Cancer Screens: A Systematic Review.

To systematically review published cost-effectiveness analyses of Evidence-Based Interventions (EBIs) recommended by the United States Community Preventive Services Task Force (CPSTF) to increase breast and cervical cancer screening. We searched PubMed and Embase for prospective cost-effectiveness evaluations of EBIs for breast and cervical cancer screening since 1999. We reviewed studies according to the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and compared the incremental cost-effectiveness ratio (ICERs), defined as cost per additional woman screened, adjusted to 2021 USD, within and across EBIs by cancer type. We identified eleven studies meeting our review criteria: nine were breast cancer-focused, one breast and cervical cancer combined, and one cervical only, which together reported twenty-four cost-effectiveness assessments of outreach programs spanning eight EBIs. One-on-one education programs were the most common EBI evaluated. The average ICER across breast cancer studies was USD 545 (standard deviation [SD] = USD 729.3), while that for cervical cancer studies was USD 197 (SD = 186.6. Provider reminder/recall systems for women already linked to formal care were the most cost-effective, with an average ICERs of USD 41.3 and USD 10.6 for breast and cervical cancer, respectively. Variability in ICERs across and within EBIs reflect the population studied, the specific EBI, and study settings, and was relatively high. ICER estimate uncertainty and the potential for program replicability in other settings and with other populations were not addressed. Given these limitations, using existing cost-effectiveness estimates to inform program funding allocations is not warranted at this time. Additional research is needed on outreach programs for cervical cancer and those which serve minority populations for either of the female cancer screens.

Personalized, interdisciplinary patient pathway for cross-sector care of multimorbid patients (eliPfad trial): study protocol for a randomized controlled trial

BackgroundMultimorbid and frail elderly patients often carry a high burden of treatment. Hospitalization due to the onset of an acute illness can disrupt the fragile balance, resulting in further readmissions after hospital discharge. Current models of care in Germany do not meet the needs of this patient group. Rather lack of coordination and integration of care combined with a lack of interdisciplinary approaches result in fragmented and inadequate care and increase the burden of treatment even more.MethodseliPfad is a randomized controlled trial conducted in 6 hospitals in Germany. Multimorbid elderly patients aged 55 or older are randomly assigned to the intervention or control group. Patients in the intervention group receive the eliPfad intervention additional to standard care. The core components of eliPfad are:Early assessment of patients’ individual treatment burden and support through a specially trained case managerInvolvement of the patient’s general practitioner (GP) right from the beginning of the hospital stayPreparation of an individual, cross-sectoral treatment plan through the interdisciplinary hospital team with the involvement of the patient’s GPEstablishment of a cross-sectoral electronic patient record (e-ePA) for documentation and cross-sectoral exchangeSupport/Promote patient adherenceTailored early rehabilitation during the hospital stay, which is continued at homeClose-tele-monitoring of medically meaningful vital parameters through the use of tablets, digital devices, and personal contacts in the home environmentThe intervention period begins in the hospital and continues 6 weeks after discharge. Patients in the control group will be treated according to standard clinical care and discharged according to current discharge management. The primary aim is the prevention/reduction of readmissions in the first 6 months after discharge. In addition, the impact on health-related quality of life, the burden of treatment, survival, self-management, medication prescription, health literacy, patient-centered care, cost-effectiveness, and process evaluation will be examined. Nine hundred forty-eight patients will be randomized 1:1 to intervention and control group.DiscussionIf eliPfad leads to fewer readmissions, proves (cost-)effective, and lowers the treatment burden, it should be introduced as a new standard of care in the German healthcare system.Trial registrationThe trial was registered in the German Clinical Trials Registry (Deutsches Register Klinischer Studien (DRKS)) on 08/14/2023 under the ID DRKS00031500.

Cost-effective prognostic evaluation of breast cancer: using a STAR nomogram model based on routine blood tests.

Breast cancer (BC) is the most common and prominent deadly disease among women. Predicting BC survival mainly relies on TNM staging, molecular profiling and imaging, hampered by subjectivity and expenses. This study aimed to establish an economical and reliable model using the most common preoperative routine blood tests (RT) data for survival and surveillance strategy management. We examined 2863 BC patients, dividing them into training and validation cohorts (7:3). We collected demographic features, pathomics characteristics and preoperative 24-item RT data. BC risk factors were identified through Cox regression, and a predictive nomogram was established. Its performance was assessed using C-index, area under curves (AUC), calibration curve and decision curve analysis. Kaplan-Meier curves stratified patients into different risk groups. We further compared the STAR model (utilizing HE and RT methodologies) with alternative nomograms grounded in molecular profiling (employing second-generation short-read sequencing methodologies) and imaging (utilizing PET-CT methodologies). The STAR nomogram, incorporating subtype, TNM stage, age and preoperative RT data (LYM, LYM%, EOSO%, RDW-SD, P-LCR), achieved a C-index of 0.828 in the training cohort and impressive AUCs (0.847, 0.823 and 0.780) for 3-, 5- and 7-year OS rates, outperforming other nomograms. The validation cohort showed similar impressive results. The nomogram calculates a patient's total score by assigning values to each risk factor, higher scores indicating a poor prognosis. STAR promises potential cost savings by enabling less intensive surveillance in around 90% of BC patients. Compared to nomograms based on molecular profiling and imaging, STAR presents a more cost-effective, with potential savings of approximately $700-800 per breast cancer patient. Combining appropriate RT parameters, STAR nomogram could help in the detection of patient anemia, coagulation function, inflammation and immune status. Practical implementation of the STAR nomogram in a clinical setting is feasible, and its potential clinical impact lies in its ability to provide an early, economical and reliable tool for survival prediction and surveillance strategy management. However, our model still has limitations and requires external data validation. In subsequent studies, we plan to mitigate the potential impact on model robustness by further updating and adjusting the data and model.