Search PubMedSearch

SEARCH · Search PubMed

Results for “Healthcare access”

Search indexed PubMed citations on genomics, clinical trials, systematic reviews and public health. Explore titles, authors and supplied subject terms, then open the PubMed record.

Quote a phrase for an exact phrase match. Source license links do not imply unrestricted reuse.

269 records · Page 14Linked to original sources

Integrated salivary proteomic and metabolomic analyses reveal molecular characterization and novel biomarker panels of chronic obstructive pulmonary disease.

Chronic obstructive pulmonary disease (COPD) is a respiratory disorder characterized by chronic inflammation, oxidative stress, and metabolic dysregulation. The lack of convenient and easily-accessible non-invasive diagnostic approaches remains a major clinical challenge. This study applied an integrated saliva-based proteomic and untargeted metabolomic strategy to identify potential biomarkers for COPD classification. Comprehensive multi-omics analyses identified 225 differentially abundant proteins and 60 differentially abundant metabolites between patients with COPD and healthy controls, including 24 biologically relevant endogenous metabolites. Functional enrichment analyses revealed pronounced dysregulation of mitochondrial energy metabolism, redox homeostasis, lipid remodeling, and inflammatory-related pathways in COPD. By integrating salivary proteomic and metabolomic biomarkers, a stepwise feature selection combined with LASSO logistic regression was used to construct diagnostic models, yielding an optimized biomarker panel consisting of 11 proteins and 2 endogenous metabolites. This integrated model achieved excellent diagnostic performance, with an area under the ROC curve of 0.96. Collectively, these findings demonstrate that integrated salivary proteomic and metabolomic profiling provides a robust, non-invasive approach for COPD classification and offers a promising foundation for the development of biosensor-based diagnostic platforms and early disease detection. SIGNIFICANCE: Chronic obstructive pulmonary disease (COPD) remains a major global health burden. Current diagnostic approaches rely largely on spirometry and clinical assessment, which are limited in sensitivity for early-stage disease and unsuitable for large-scale screening. This study employs an integrated saliva-based proteomic and metabolomic strategy to identify non-invasive biomarkers for COPD classification. Our findings reveal coordinated dysregulation of mitochondrial energy metabolism, redox homeostasis, and lipid remodeling in COPD, highlighting the interconnected roles of metabolic reprogramming, oxidative stress, and inflammation in disease pathophysiology. Notably, a robust diagnostic panel comprising 11 proteins and 2 endogenous metabolites was established, achieving excellent classification performance (AUC of 0.96). To our knowledge, the integrated application of salivary proteomics and metabolomics for COPD diagnosis remains largely unexplored, underscoring the significance and translational potential of our findings.

Humans

Multicenter randomized effectiveness/implementation trial of a digital self-management support tool to improve the quality of life during adjuvant hormonal therapy for patients with early breast cancer: The HOPE trial.

BACKGROUND: For patients with hormone receptor (HR) positive early breast cancer (BC), adjuvant endocrine therapy (ET) represents the cornerstone of treatment. However, 75% of patients experience ET-related symptoms that negatively affect their quality of life (QOL). Despite their high prevalence, these symptoms are often underestimated and under-addressed during consultations. As a result, non-adherence to ET is common and remains a major barrier for optimal disease and survival outcomes. METHODS: National, prospective, randomized, open-label hybrid type 1 effectiveness/implementation trial conducted in France comparing a personalized digital health pathway plus standard of care (SoC) vs. SoC alone in patients with HR+ early BC reporting ET-related symptoms. 180 patients will be randomized 1:1 to receive either 12 weeks of the digital health pathway or 12 weeks of SoC. The intervention is anchored by the Resilience© digital companion including remote symptom and needs assessment, an introductory nurse-navigator phone call, and access to personalized, symptom-specific online educational and self-management programs (physical activity, yoga, meditation or cognitive behavioral therapy). In both arms, patients will be invited to wear a wearable device to objectively monitor behavioral parameters. The primary endpoint is the ET symptoms scale of the European Organization for Research and Treatment of Cancer (EORTC) QLQ-BR45 over 12-weeks. Secondary endpoints include other QOL domains, self-reported ET adherence, eHealth literacy, self-efficacy, and evaluation of the implementation process. DISCUSSION: This study should provide evidence on the effectiveness and real-world implementation of a personalized digital health pathway to improve QOL in patients experiencing ET-related symptoms. TRIAL REGISTRATION: ClinicalTrials.gov NCT06781996; Protocol version 3.0.

Humans

Molecular Landscape and Advanced Diagnostic Technologies for BRAF Mutations in Cancer: From Quantitative PCR and ddPCR to CRISPR-Based Platforms.

BRAF mutations are key oncogenic alterations across multiple malignancies, including melanoma, thyroid carcinoma, colorectal cancer, non-small cell lung cancer, glioma, and hairy cell leukemia. The most prevalent variant, BRAF-V600E, induces constitutive activation of the MAPK signaling pathway, promoting tumor progression and influencing therapeutic responsiveness. Accurate detection of BRAF alterations is therefore essential for molecular classification, prognostic assessment, treatment selection, and resistance surveillance. This review summarizes the molecular heterogeneity of BRAF mutations and critically evaluates current diagnostic methodologies. Conventional approaches such as allele-specific PCR and Sanger sequencing are compared with advanced quantitative platforms, including high-resolution melting analysis, droplet digital PCR, and next-generation sequencing, with emphasis on analytical sensitivity, mutation coverage, and clinical applicability. Emerging technologies such as CRISPR-based assays, rolling circle amplification systems, and nanoparticle-based biosensors and point-of-care diagnostic platforms are also discussed for their potential to enhance ultra-sensitive detection, particularly in liquid biopsy settings. These emerging tools are highlighted for their potential to enable ultra-sensitive, rapid, and decentralized mutation detection, particularly in liquid biopsy settings. Key challenges, including intratumoral heterogeneity, low allele-frequency variants, FFPE-associated artifacts, and clonal evolution under therapeutic pressure, are examined within a translational framework. In addition, we examine critical barriers to clinical implementation, including standardization, cost, and global accessibility of molecular diagnostics, and outline potential solutions through scalable technologies and decentralized testing strategies. We propose that optimal BRAF testing requires a mutation subclass-informed and clinically integrated strategy combining comprehensive baseline profiling with longitudinal molecular monitoring. Future diagnostic paradigms will likely integrate multi-omics data and artificial intelligence (AI)-assisted interpretation to refine precision oncology implementation. Looking forward, we propose that optimal BRAF testing will require integration of multi-omics profiling with AI-assisted interpretation, enabling automated variant classification, real-time clinical decision support, and improved prediction of therapeutic response and resistance.

Humans

A pragmatic randomized controlled trial of self-directed online writing interventions for posttraumatic stress symptoms in a real-world digital setting.

Background: Public health and other large-scale crises, such as the COVID-19 pandemic, have intensified the global mental health burden, creating unprecedented demand for accessible interventions for posttraumatic stress symptoms (PTSS).Objective: We evaluated the feasibility and effectiveness of two self-directed online writing interventions embedded within China's WeChat ecosystem during the COVID-19 pandemic through a pragmatic randomised controlled trial.Methods: Between December 2021 and August 2022, 1,526 adults were screened for PTSS via a Tencent Medinfo Mini-Program. Eligible participants (n = 211) were randomised to Guided Narrative Technique-Writing (GNT-W, n = 100) or Expressive Writing (EW, n = 111). Both interventions comprised three self-directed daily writing sessions delivered entirely online without human support. Primary outcome was PTSD symptom severity (PTSD Checklist-Short), assessed at baseline, post-intervention, 2-week, and 1-month follow-ups.Results: While initial engagement followed typical digital health patterns (64.5% overall attrition), participants who initiated treatment showed strong adherence (77% completion). Both interventions were associated with significant within-group reductions in PTSS severity (GNT-W: b = -0.43, p = .023, d = -0.43; EW: b = -0.60, p = .001, d = -0.58), with no significant between-group difference (group × time: b = 0.18, p = .48). GNT-W did not confer additional benefit over EW protocol on PTSS severity.Conclusions: Both self-directed writing interventions were associated with within-group reductions in PTSS; without an inactive control condition, however, these changes cannot be firmly attributed to the interventions. GNT-W showed no advantage over the simpler EW protocol. These findings offer preliminary support for embedding scalable, low-barrier writing interventions in widely used digital platforms.Chinese Clinical Trial Registry: ChiCTR2000034836.

Humans

Effectiveness of tobacco cessation interventions delivered in clinical settings in South Asia: a systematic review and meta-analysis.

BACKGROUND: Despite the burden of tobacco use, access to cessation support in South Asia remains scarce. OBJECTIVE: This review evaluates the effectiveness of tobacco cessation interventions delivered in clinical settings in South Asia. METHODS: Five relevant databases were searched from inception to February 2025. Eligibility criteria included randomized and non-randomized studies evaluating behavioral, pharmacotherapy, and multicomponent interventions delivered in clinical settings in South Asia. Data on study setting and design, participant information, intervention, comparator, and outcomes were extracted. Meta-analyses using random-effect models were conducted where possible. Certainty of evidence was assessed using GRADE. RESULTS: Thirty-seven studies were included (22 randomized and 15 non-randomized). Interventions involved pharmacotherapy (n = 6; 16.2%), nicotine replacement therapy (n = 7; 18.9%), behavioral counseling (n = 12; 32.4%), or combined/multicomponent interventions (n = 12; 32.4%). Most studies were conducted in India (n = 26; 70.3%), followed by Pakistan (n = 6; 16.2%), Nepal (n = 3; 8.1%), and two studies (5.4%) were multi-country in India, Pakistan, and Bangladesh. Pooled analyses demonstrated higher quit rates among intervention versus control for continuous abstinence at 0-3 months (RR: 1.21, 95%CI: 1.06 to 1.37) and >3 months (RR: 1.68, 95%CI: 1.1.4 to 2.47), and for point abstinence at >3 months post-intervention (RR: 2.03, 95%CI:1.35 to 3.08). Heterogeneity was high for all analyses (I2 range: 94% to 97%). Combined behavioral and pharmacotherapy interventions were most effective (RR: 1.70, 95%CI: 0.98 to 2.92), although not statistically significant (p = 0.06). CONCLUSION: Tobacco cessation interventions delivered in clinical settings in South Asia are effective, particularly when combining behavioral support with pharmacotherapy. However, evidence is limited by methodological weaknesses.

Humans

Beyond survival: Testicular tissue cryopreservation for fertility preservation during childhood cancer, experiences from a single center.

INTRODUCTION: Approximately 30% of all male childhood cancer survivors receive gonadotoxic therapy increasing their risk for infertility. The only current option for potential fertility preservation in prepubertal males is through testicular tissue cryopreservation (TTC), available through clinical trials. Our aim was to evaluate our experience and identify barriers to enrollment in the TTC study. METHODS: A retrospective review was conducted to describe a single center experience with TTC, including an assessment of factors impacting the decision to enroll or not enroll in the TTC trial. RESULTS: A total of 61 patients were found to be eligible for enrollment in the TTC trial, with 23 patients (34%) choosing to enroll and undergo testicular tissue cryopreservation including 19 (83%) oncology patients. In patients who underwent TTC there were no unexpected delays in starting disease directed therapy. For families that declined enrollment, many did not see fertility preservation as a priority at the time of counseling. (n = 17, 44%). Other barriers included concerns about post-operative complications and a feeling of being overwhelmed at the time of counseling. DISCUSSION: Despite being the only preservation option for pre-pubertal boys facing risk of infertility, TTC enrollment was not pursued by a majority of eligible patients and families. Identifying potential barriers or misconceptions, particularly in regard to post operative complication risks, offer opportunities for improved counseling and education. Early introduction of the Fertility Navigation team allows for procedural coordination success and access for patients that are interested. Success is not measured in how many patients proceed with TTC, rather that all patients have the opportunity for counseling and discussion. CONCLUSION: TTC can be offered and completed safely without delays in disease-directed treatment timelines. A Fertility Navigation Program allows for equal opportunity for education and enrollment on TTC protocol which should be the goal for pediatric institutions, though is not pursued by many patients. A majority of those who declined the study did not see fertility as a priority at the time of treatment while others were concerned regarding peri-operative risks.

Humans

Satisfaction with clinical practice environments among early-career health professionals in South Africa: Findings from the WiSDOM cohort study.

BACKGROUND: The work or practice environments of health professionals play a central role in their retention in the healthcare system and their ability to provide quality patient care. The aim of the study was to examine and compare the satisfaction of early-career health professionals in the WiSDOM (Wits longitudinal Study to Determine the Operation of the labour Market among its health professional graduates) study with their clinical practice environments (CPEs) in South Africa, and the factors influencing their satisfaction. METHODS: WiSDOM, a prospective longitudinal cohort study, consists of eight health professions: clinical associates, dentists, doctors, nurses, occupational therapists, oral hygienists, pharmacists, and physiotherapists. Every year we collect information on the cohort's involvement in direct patient care, their perceived workload, availability of medicines and equipment for patients in their care, and their satisfaction with the clinical practice environment (CPE).We used Stata&#xae;19 for analysis. We used panel linear regression to investigate factors associated with the cohort's satisfaction with their clinical practice environments from 2018 to 2024, and logistic regression to evaluate the association between CPE and intention to leave in 2024. RESULTS: In 2024, the mean age of the cohort was 30.9 (&#xb1; 2.0), the majority were female (74.4%) and working in urban areas (92.7%). In 2024, 59.7% of the overall cohort reported a heavy workload compared to 69.2% in 2018. Over the follow-up period, reported problems with the availability of medicines or equipment were worse in the public sector and in rural areas, compared to the private sector and urban areas respectively.The cohort's satisfaction score with the CPE was 6.7 out of 10 in 2018 and 6.9 in 2024. The predictors of CPE were year of follow-up, health profession, employment sector, and geographic location. Nurses (&#x3b2;=-1.2; 95% CI -1.7, -0.7; p&#x2009;<&#x2009;0.001), and pharmacists (&#x3b2;=-0.7; 95% CI -1.1, -0.4; p&#x2009;<&#x2009;0.001) scored their CPE significantly lower compared to the other professional groups. Health professionals in the public sector (&#x3b2;=-1.1; 95% CI -1.3, -0.9; p&#x2009;<&#x2009;0.001) and in rural areas (&#x3b2;=-0.6; 95% CI -0.9, -0.3; p&#x2009;<&#x2009;0.001) were less satisfied with their CPE compared to those in the private sector and urban areas respectively. Dissatisfaction with the CPE was significantly associated with intention to leave the workplace and the profession. CONCLUSION: The study findings underscore the need for positive clinical practice environments for early-career health professionals in South Africa both as a health workforce and patient safety imperative.

South Africa

Survival by Race and Ethnicity in Children and Adolescents/Young Adults With Relapsed/Refractory Hodgkin Lymphoma: A Pooled Analysis of Children's Oncology Group Trials.

PURPOSE: Despite 5-year survival rates of over 90% among children and adolescents/young adults (CAYAs) with classic Hodgkin lymphoma (cHL), 15%-20% relapse after frontline therapy. Prior analysis of frontline Children's Oncology Group (COG) clinical trials demonstrated that, despite similar rates of relapse, non-Hispanic Black (NHB) and Hispanic (vs. non-Hispanic White [NHW]) patients experienced higher post-relapse mortality. It is unknown whether post-relapse disparities persist when second-line treatment is delivered in a cooperative group trial setting. We examined overall survival (OS) by race and ethnicity in CAYAs enrolled in COG trials for relapsed/refractory (r/r) cHL. METHODS: A pooled analysis of individual-level data from CAYAs (&#x2264; 29 years) receiving therapy for r/r cHL on COG clinical trials (2001-2016) was conducted. The Kaplan-Meier method estimated 3-year OS by racial and ethnic groups. Cox regression models examined associations of race and ethnicity and OS, adjusted for age, insurance, first versus &#x2265;2 relapse, and time from initial diagnosis to relapse trial enrollment. RESULTS: Among 175 CAYAs treated on COG trials for r/r cHL (5.7% Asian or Pacific Islander, 14.9% Hispanic, 14.3% NHB, 61.7% NHW, 3.4% other), at median follow-up of 4.9 years, 3-year OS was 82.1% (95% confidence interval [CI], 75.3%-87.1%) and did not differ by race and ethnicity (p = 0.36). In multivariable analyses, shorter time from diagnosis to relapse trial enrollment (p = 0.01) and &#x2265;2 relapses (vs. first, p = 0.004) conferred worse OS, with no significant effect of race and ethnicity (p = 0.43). CONCLUSION: Post-relapse survival did not differ by race and ethnicity among CAYAs enrolled in COG trials for r/r cHL, suggesting access to clinical trials may mitigate OS disparities.

Humans

Two-Year Outcomes of a 211 Care Coordination Trial.

BACKGROUND AND OBJECTIVES: Early screening for developmental concerns enables timely diagnosis and referral, yet many families face barriers accessing services. Prior work showed that early childhood care coordination could improve timely service connection. This study assessed developmental outcomes among children participating in a randomized controlled trial of Information and Referral Federation of Los Angeles County (211LA). METHODS: Participants, aged 21-42&#xa0;months, were randomized to usual care or the 211LA intervention. Developmental and behavioral measures, including the Parental Evaluation of Developmental Status Developmental Milestones Assessment Level (PEDS-DM-AL) and the Child Behavior Checklist (CBCL), were collected at baseline and 24&#xa0;months later. The sample included 499 participants, 250 in the 211LA intervention and 249 in usual care. Primary analyses examined changes in PEDS-DM-AL and CBCL scores over the 24-month period by study arm. Post hoc analyses compared family characteristics between intervention and control families who enrolled in services. RESULTS: Developmental and behavioral measures showed some clinically insignificant change over time, but these changes did not differ by condition (expressive/receptive language skills mastered: P&#x2009;>&#x2009;.9; autism, attention, aggression, and externalizing behavior T scores: P&#x2009;>&#x2009;.4). Post hoc analyses identified potentially relevant imbalances between the treatment arms at baseline as well as in the subgroup that enrolled in services, with families assigned to the 211LA intervention being more likely to have a non-US born parent and a parent with limited English proficiency compared with families assigned to usual care. Intervention families enrolled in services also used telehealth more frequently and received a lower duration of services than those receiving usual care. CONCLUSIONS: This study measured the indirect influence of service enrollment through 211LA care coordination on developmental outcomes. Although increased service enrollment through 211LA did not affect developmental outcomes, we hypothesize this may be because of several factors, including overrepresentation of a subset of historically underrepresented families in the 211LA intervention, suboptimal performance of our developmental assessment tool, and complexity of conducting a trial of this magnitude during the COVID-19 pandemic, which may have diminished the ability of this trial to demonstrate developmental benefits despite demonstrated service enrollment gains.

Humans

Early mobilization within 24 to 48&#xa0;h improves postoperative clinical outcomes in older adults with hip fracture: A systematic review and meta-analysis.

BACKGROUND: Hip fracture is a major public health concern among older adults, often resulting in prolonged disability, institutionalization, and increased healthcare burden. Early mobilization has been widely recommended to enhance postoperative recovery; however, there is a lack of consolidated evidence quantifying its impact on clinical and functional outcomes. This study aimed to synthesize and evaluate the impact of early mobilization following hip fracture surgery in older adults and to explore potential sources of heterogeneity to better inform clinical and nursing practice. METHODS: A comprehensive literature search was conducted across seven databases (PubMed, Embase, Scopus, Web of Science, Cumulative Index to Nursing and Allied Health Literature, Cochrane Library, and Emcare) from inception to June 15, 2025. Eligible studies included randomized controlled trials and observational cohort studies comparing early mobilization (defined as ambulation within 24 to 48&#xa0;h postoperatively) to delayed or usual mobilization in patients undergoing hip fracture surgery. Primary outcomes included mortality, discharge destination, and length of hospital stay. Secondary outcomes included postoperative complications, functional recovery, and readmission. Risk of bias was assessed using funnel plots and Egger's test. RESULTS: Twenty-six studies involving 297,435 patients were included. Compared with delayed mobilization, early mobilization significantly reduced 30-day mortality (relative risk&#xa0;=&#xa0;0.40, 95% confidence interval: 0.25-0.64) and 1-year mortality (relative risk&#xa0;=&#xa0;0.57, 95% confidence interval: 0.40-0.80) (both p&#xa0;<&#xa0;0.05). In regional analyses of pooled mortality, similar reductions were observed across Asia-Pacific, North America, and Europe. Patients receiving early mobilization were more likely to be discharged home and had shorter hospital stays. Early mobilization also resulted in a reduced risk of postoperative complications (relative risk&#xa0;=&#xa0;0.79, 95% confidence interval: 0.74-0.84, p&#xa0;<&#xa0;0.05), with specific improvements in pneumonia and thromboembolism rates. Functional independence was significantly improved, as shown by higher Barthel Index scores and increased odds of achieving Functional Independence Measure &#x2265;5 at discharge. No significant difference was observed in readmission rates. CONCLUSIONS: lization within 24 to 48&#xa0;h following hip fracture surgery was associated with favorable outcomes, including reduced mortality, improved functional independence, higher rates of discharge to home, shorter hospital length of stay, and fewer postoperative complications. Although heterogeneity across studies and the predominance of observational evidence warrant cautious interpretation, these findings support current recommendations for early mobilization and highight the potential value of structured and standardized mobilization protocols in routine postoperative hip fracture care.

Humans

Single-organ proteomics in Drosophila melanogaster larva.

The combination of genetic accessibility, organ complexity, evolutionary conservation, and cost-efficiency makes Drosophila melanogaster (Dm) a well-known model system for biomedical and fundamental biological research. Proteomic analysis of single organs enables the identification and quantification of proteins expressed in specific organs. This will help to uncover specific biological functions and unique protein profiles that are not detectable in whole-organism analyses. In this study we have isolated single organs form Dm larvae, and we have performed a deep proteomics mapping by following a minimal manipulation preparation procedure. The combined dataset across all organs comprised 9132 identified proteins. As anticipated, principal component analysis (PCA) revealed clear separation between the proteomes of most organs, confirming distinct protein profiles. These findings demonstrate the applicability of the sample preparation strategy for high-resolution proteomic characterization of individual organs in Drosophila. Given the extensive genetic tools available for this model organism, our approach has the potential to open new avenues for proteomic studies in Drosophila melanogaster and any other biological systems where the sample amount is limiting. SIGNIFICANCE STATEMENT: Drosophila melanogaster is a well-known model system for biomedical and fundamental biological research that serves as a valuable in vivo model organism due to its high degree of evolutionary conservation with higher vertebrates, tractable genetics, and logistical efficiency. However, the proteome of Drosophila at single organ level has been elusive to date, due to several factors like low sensitivity of previous generation mass spectrometers and sample preparation procedures, difficult isolation of some organs. In this study we have applied a compilation of advanced methods including minimal sample manipulation together with simple, straightforward and efficient protein extraction and digestion methods. Obtained peptides were minimally handled to be analyzed by applying specific and sensitive nLC methods coupled on-line to state-of-the-art MS/MS system. Altogether, the applied strategy allowed us to get the first single organ study to date for this animal. These datasets represent a significative resource for future genomic, transcriptomic and proteomic studies in Drosophila, as multi-omic integration requires deep proteomics to translate data into functional biochemistry, and serves as a critical bridge and an indispensable standalone resource across the genomic, transcriptomic, and proteomic landscapes.

Animals

Effect of demographic characteristics on the outcome of prostate cancer salvage radiotherapy: Analysis from a randomized controlled trial.

BACKGROUND: This study investigated the impact of advanced molecular imaging, race, socioeconomic status, and metabolic dysregulation on the outcome of salvage radiotherapy (sRT) for prostate cancer recurrence in a clinical trial setting. METHODS: The authors randomized post-prostatectomy men with detectable prostate-specific antigen to sRT guided by conventional imaging (arm A) or 18F-fluciclovine-positron emission tomography/computed tomography (arm B) and followed them up for up to 48 months to determine failure-free survival (FFS). The authors computed socioeconomic status (SES) and allostatic load (AL) scores to quantify socioeconomic status and level of metabolic dysregulation. They stratified patients by race as African American men (AAM) versus men of other races (MOR) and compared FFS between them using the z-test. RESULTS: Eighty-one (AAM&#xa0;=&#xa0;29, MOR&#xa0;=&#xa0;52) and 76 (AAM&#xa0;=&#xa0;26, MOR&#xa0;=&#xa0;50) men completed per-protocol sRT in arms A and B, respectively. Across study arms, AAM showed a higher FFS rate than MOR (72.8% [95% CI, 53.8%-85.0%] vs. 58.7% [95% CI, 46.6%-68.9%]; p&#xa0;=&#xa0;.002). In arm A, FFS rate was better for AAM than MOR, (64.0% [95% CI, 34.4%-82.9%] vs. 45.3% [95% CI, 28.8%-60.4%]; p&#xa0;=&#xa0;.008). In arm B, FFS improved for both groups but less so for AAM, (81.5% [95% CI, 57.2%-92.7%] vs. 73.0% [95% CI, 56.3%-84.1%]; p&#xa0;=&#xa0;.131). The authors found lower SES scores and higher AL scores for AAM in both study arms than MOR. CONCLUSION: Despite lower socioeconomic status and higher burden of metabolic dysregulation, in a clinical trial setting that controls for disparities in health care access, AAM have a more favorable sRT outcome than MOR.

Humans

Pricing Combination Therapies: A Systematic Review of Value Attribution, Cost-Sharing Mechanisms and Policy Frameworks.

BACKGROUND: Combination therapies are increasingly central to modern pharmacotherapy, particularly in oncology and other high-burden diseases. However, pharmaceutical pricing and reimbursement systems remain largely designed for single-product-single-indication interventions. When multiple patented medicines are used together, especially when owned by different manufacturers, conventional pricing frameworks may struggle to align prices with the value of the combination while preserving incentives for innovation and timely patient access. OBJECTIVE: To identify, describe, and critically assess the methods, models, and policy frameworks proposed in the literature to establish prices for combination therapies, with particular attention to value attribution mechanisms, cost-sharing arrangements between manufacturers, and budget impact considerations. METHODS: A systematic literature review was conducted in accordance with PRISMA guidelines and a pre-registered Open Science Framework protocol. Searches were performed in MEDLINE, Scopus, Web of Science, EconLit, CRD databases, and grey literature sources for publications up to July 2025. Eligible studies analysed pricing approaches, economic models, reimbursement mechanisms, or policy frameworks relevant to combination therapies, including more recent multi-indication pricing literature. Given the heterogeneity of the literature, findings were synthesized using a structured narrative and thematic approach. RESULTS: Sixty-nine studies met the inclusion criteria. The literature was dominated by conceptual and policy analyses, with relatively few empirical or implementation-oriented studies. Value attribution emerged as the central methodological challenge in pricing combination therapies. Several complementary approaches were proposed to operationalise value attribution, including adaptations of indication- or pathway-based pricing, manufacturer cost-sharing arrangements, managed entry agreements, and outcome-based reimbursement mechanisms. Empirical evidence suggests that health systems continue to rely primarily on pragmatic and often partial solutions rather than fully specified pricing frameworks. A complementary review of the multi-indication pricing literature indicates that, although the two fields address different pricing problems, they share important methodological and institutional lessons that can inform the development of pricing frameworks for combination therapies. CONCLUSIONS: The literature provides a growing repertoire of conceptual approaches for pricing combination therapies but limited empirical evidence on implementation. Pricing frameworks should place value attribution at their core while combining complementary policy mechanisms adapted to national pricing and reimbursement systems. Lessons from multi-indication pricing provide a valuable foundation but require additional governance mechanisms to address value attribution, multi-manufacturer negotiation, and implementation challenges specific to combination therapies.

Journal Article

Feasibility and effectiveness of the Bergen 4-Day Treatment for obsessive-compulsive disorder in Australia: A pilot comparison with 3-week inpatient obsessive-compulsive disorder treatment.

OBJECTIVES: Obsessive-compulsive disorder is a debilitating and chronic condition that, when untreated or unresponsive to treatment, imposes a significant health and economic burden on individuals and families. This prospective non-randomised inpatient study compared the acceptability and clinical outcomes of the Bergen 4-Day Treatment programme with those of a standard 3-week specialised treatment programme for obsessive-compulsive disorder in Australia. METHOD: Twenty-five participants diagnosed with obsessive-compulsive disorder were non-randomly assigned to Bergen 4-Day Treatment (n&#x2009;=&#x2009;12) or a 3-week standard (n&#x2009;=&#x2009;13) inpatient programme. Independent assessments were completed at pre-treatment, 10&#x2009;days post treatment and at 3-month follow-up. The Yale-Brown Obsessive-Compulsive Scale was rated to assess obsessive-compulsive disorder severity, while secondary measures of depression, anxiety, obsessive beliefs and wellbeing were self-rated by participants. RESULTS: Baseline characteristics of both groups were comparable, with obsessive-compulsive disorder symptom severity within the moderate to severe range. After treatment, obsessive-compulsive disorder symptoms as well as secondary depression and anxiety symptoms were reduced in both treatment groups. Participants receiving Bergen 4-Day Treatment had significantly lower Yale-Brown Obsessive-Compulsive Scale scores at 10&#x2009;days (M&#x2009;=&#x2009;13.46) and 3&#x2009;months (M&#x2009;=&#x2009;11.84), compared to standard treatment (M&#x2009;=&#x2009;19.04 and M&#x2009;=&#x2009;19.15, respectively). Response (91.9%) and remission (45.8%) rates for the Bergen 4-Day Treatment group were significantly higher at both post-treatment timepoints, compared to the standard treatment group. No dropouts occurred in the Bergen 4-Day Treatment group, and participant satisfaction was high. CONCLUSION: The findings of this pilot open-label study suggest that Bergen 4-Day Treatment shows promise as an acceptable, efficient and effective treatment for obsessive-compulsive disorder, warranting further investigation as a scalable alternative for improving access to specialised obsessive-compulsive disorder treatment in Australia.

Humans

Cochlear Implantation in Sickle Cell Disease: A Systematic Review of Feasibility and Outcomes.

INTRODUCTION: Sickle cell disease (SCD) is associated with systemic complications, including sensorineural hearing loss (SNHL) from microvascular occlusion and chronic inflammation. Although reports link SCD to higher rates of SNHL, current evidence is limited by small sample size, varied audiologic methods, and lack of standardized screening. This systematic review summarizes available literature on SNHL and cochlear implantation (CI) in SCD. METHODS: A literature search of PubMed MEDLINE, Embase, Scopus, Web of Science, and CINAHL identified 79 citations. After removal of duplicates, 35 records were screened in Rayyan. Studies published between January 1, 2000, and June 30, 2025, were eligible if they reported patients with SCD who developed hearing loss and underwent CI. Nine full texts were reviewed, and 4 met the inclusion criteria. Screening and review were performed independently by 2 authors per PRISMA guidelines. RESULTS: Across 4 case reports, a total of 5 patients with SCD underwent CI, ranging in age from 2 to 42 years. Four presented with bilateral severe-to-profound SNHL and one with unilateral loss. Implantation was technically feasible in all cases, including patients with cochlear fibrosis or ossification requiring modified insertion techniques. Postoperative outcomes were favorable: all patients demonstrated reliable device function and low impedances. Only 60% showed meaningful auditory benefit, characterized by improved functional speech perception in 2 patients (40%) and access to the speech frequency range with hearing testing going from moderate/profound hearing loss to mild hearing loss in 3 patients (60%). Complications occurred in 2 patients (40%): one developed unilateral middle ear infection leading to meningitis, and another experienced a postoperative pulmonary embolism requiring anticoagulation. The remaining 3 patients (60%) had uncomplicated recoveries with reported improved hearing from moderate/profound hearing loss to mild hearing loss post implantation. CONCLUSION: While CI appears feasible in SCD, our findings suggest that additional data are needed to assess its effectiveness in this patient population. However, evidence is limited to case reports, and complications such as thromboembolism and rapid cochlear fibrosis highlight the need for close perioperative management. More comprehensive studies with larger sample size are required to define surgical risk, optimize management, and establish best practices for timely implantation in this population.

Humans

Behaviourally informed text message reminders to increase cervical screening attendance in people with severe mental illness: the OPTIMISE pilot randomised controlled trial.

OBJECTIVES: This study assessed the feasibility of both the delivery and evaluation of 'enhanced' (behaviourally informed) text message reminders containing links to existing co-designed resources supporting decision-making for people with severe mental illness (SMI) regarding attendance of cervical screening. DESIGN: A pilot randomised controlled trial (RCT). SETTING: 13 General Practice (GP) practices in London were recruited. PARTICIPANTS: GP practices identified people with SMI aged 24-64 years who were overdue cervical screening. Target sample size was 120 participants (60 per arm) based on existing guidance for pilot trials. INTERVENTION: In March 2025, participants were randomised (1:1) to receive either the enhanced (intervention) or the standard (control) SMS reminder. PRIMARY AND SECONDARY OUTCOME MEASURES: 18 weeks later, feasibility outcomes were collected (primary outcomes) and data analysis for a definitive RCT was rehearsed (secondary outcome). RESULTS: Of the 150 participants across 13 GP practices that were randomised (n=75 per arm), 132 (88%) texts delivered (intervention n=64/75 (85%), control n=68/75 (91%)). 10 practices (76.9%) provided follow-up data for 102 participants (intervention n=50, control n=52). Five participants (intervention n=4, control n=1) attended screening within the trial period. Participant survey response rate was low (9/132 (7%), intervention n=5, control n=4). Both SMS messages were low cost, with the intervention SMS a 50% higher cost to deliver (7.5p vs 5p per SMS). Primary feasibility measures of recruitment rate of GP practices (27%), retention of GP practices (77%) and participants (100%), SMS delivery (88%) and data completeness (64%) indicated viability, although survey response rate (7%) did not. CONCLUSIONS: Achieving adequate recruitment and retention, data completeness and comparable groups is viable with some amendments, although an alternative method is required to assess fidelity. Behaviourally informed SMS reminders are feasible to deliver to people with SMI, although it is uncertain if the extra resources are accessed and used. With changes to data collection, a definitive trial could be feasible. Given the low observed cervical screening attendance, additional intervention is needed for this group. TRIAL REGISTRATION NUMBER: ISRCTN12558681.

Humans

Evaluation of three Aspergillus antibody assays for screening of chronic pulmonary aspergillosis: prospective diagnostic accuracy study.

OBJECTIVES: Chronic pulmonary aspergillosis (CPA) is a frequent complication of pulmonary tuberculosis (PTB), particularly in high-burden settings where access to reliable serological diagnostics remains limited. We evaluated the diagnostic performance of two immunochromatographic technology (ICT) lateral flow assays (LFAs) and an ELISA for CPA screening among patients with active or previously treated PTB. METHODS: In this two-year prospective multicentre diagnostic evaluation, serum from adults with prior or active PTB was tested using the Era Biology Aspergillus IgG ICT LFA, LDBio Aspergillus IgG/IgM ICT LFA, and Bordier Aspergillus fumigatus IgG ELISA. CPA diagnosis was established using a consensus composite reference standard incorporating clinical, immunological, radiological, and microbiological criteria. The Bordier ELISA was used as part of the immunological component of the consensus CPA diagnosis, with a cutoff optical density of &#x2265;1.0. Diagnostic accuracy, agreement statistics, receiver operating characteristic analysis, and latent class analysis (LCA) were performed. RESULTS: Among 340 participants, 24 (7.06%) had CPA. Proportion of participants with positive antibody tests among all tested individuals were 6.76% for LDBio ICT LFA, 20.0% for Era Biology ICT LFA, and 11.47% for Bordier ELISA. Against consensus CPA diagnosis, Bordier ELISA showed 87.50% sensitivity and 94.30% specificity, LDBio ICT LFA 58.33% sensitivity and 97.15% specificity, and Era Biology LFA 66.67% sensitivity and 83.54% specificity. LCA estimated CPA prevalence at 7.72%. LCA-derived sensitivities and specificities were 86.58% and 99.92% for LDBio ICT LFA, 83.39% and 85.31% for Era Biology LFA, and 79.10% and 94.19% for Bordier ELISA. CONCLUSIONS: The Bordier ELISA showed high sensitivity and specificity, while the LDBio ICT LFA demonstrated very high specificity with strong LCA-derived performance. These findings support the use of ELISA for laboratory diagnosis and ICT as a point-of-care screening tool for CPA in resource-limited settings. Era Biology Aspergillus IgG LFA demonstrated moderate sensitivity and acceptable diagnostic performance, indicating its potential utility as a supplementary screening assay for CPA in settings where rapid, point-of-care testing is required.

Humans

Effectiveness of Multidomain Cardiac Rehabilitation After Myocardial Infarction by Patient Frailty: Prespecified Subgroup Analysis of the PIpELINe Trial.

BACKGROUND: Frailty is common among older patients surviving myocardial infarction, is associated with adverse outcomes, and is often perceived as a barrier to cardiac rehabilitation (CR). The aim of this study is to determine whether frailty influences prognosis after myocardial infarction, and whether frailty modifies the clinical benefit of multidomain CR. METHODS: We performed a prespecified subgroup analysis of the PIpELINe (Physical Activity Intervention in Elderly Patients With Myocardial Infarction) randomized clinical trial conducted in Italy, which enrolled 512 patients aged &#x2265;65 years recovering from myocardial infarction and randomized them in a 2:1 ratio to CR or usual care. Frailty was assessed using the Fried Frailty Phenotype, and patients were categorized as nonfrail (robust) or prefrail/frail. Time-to-event outcomes were analyzed using Kaplan-Meier estimates and Cox proportional hazards models, including treatment-by-frailty interaction terms to evaluate effect modification of the multidomain CR. The primary outcome was a composite of cardiovascular death or unplanned hospitalization for cardiovascular causes within 1 year after randomization. RESULTS: Overall, 350 patients (68.4%) were classified as prefrail/frail, of whom 232 were randomized to intervention arm (66%). Frail patients were older (median age, 80 [75-85] years) and more frequently female (41.7% versus 24.7%). Compared with robust patients, prefrail/frail patients had a higher risk of the primary outcome (16 [9.9%] versus 62 [17.7%]; hazard ratio, 1.59 [95% CI, 0.89-2.82]; adjusted P=0.117). Among prefrail/frail patients, assignment to multidomain CR was associated with a lower risk of the primary outcome compared with usual care (hazard ratio, 0.57 [95% CI, 0.34-0.94]; P=0.028), with no statistically significant interaction in the treatment effect on the primary end point (P=0.57). CONCLUSIONS: Among older patients recovering from myocardial infarction, frailty is associated with worse prognosis but does not diminish the benefit of multidomain CR. These findings support the use of frailty assessment to guide rather than limit access to CR. REGISTRATION: ClinicalTrials.gov; Unique identifier: NCT04183465.

Humans