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Nevin Manimala Statistics

Screening for Osteoporosis to Prevent Fractures: A Systematic Evidence Review for the US Preventive Services Task Force

JAMA. 2025 Jan 14. doi: 10.1001/jama.2024.21653. Online ahead of print.

ABSTRACT

IMPORTANCE: Fragility fractures result in significant morbidity.

OBJECTIVE: To review evidence on osteoporosis screening to inform the US Preventive Services Task Force.

DATA SOURCES: PubMed, Embase, Cochrane Library, and trial registries through January 9, 2024; references, experts, and literature surveillance through July 31, 2024.

STUDY SELECTION: Randomized clinical trials (RCTs) and systematic reviews of screening; pharmacotherapy studies for primary osteoporosis; predictive and diagnostic accuracy studies.

DATA EXTRACTION AND SYNTHESIS: Two reviewers assessed titles/abstracts, full-text articles, study quality, and extracted data; when at least 2 similar studies were available, meta-analyses were conducted.

MAIN OUTCOMES AND MEASURES: Hip, clinical vertebral, major osteoporotic, and total fractures; mortality; harms; accuracy.

RESULTS: Three RCTs and 3 systematic reviews reported benefits of screening in older, higher-risk women. Two RCTs used 2-stage screening: Fracture Risk Assessment Tool estimate with bone mineral density (BMD) testing if risk threshold exceeded. One RCT used BMD plus additional tests. Screening was associated with reduced hip (pooled relative risk [RR], 0.83 [95% CI, 0.73-0.93]; 3 RCTs; 42 009 participants) and major osteoporotic fracture (pooled RR, 0.94 [95% CI, 0.88-0.99]; 3 RCTs; 42 009 participants) compared with usual care. Corresponding absolute risk differences were 5 to 6 fewer fractures per 1000 participants screened. The discriminative accuracy of risk assessment instruments to predict fracture or identify osteoporosis varied by instrument and fracture type; most had an area under the curve between 0.60 and 0.80 to predict major osteoporotic fracture, hip fracture, or both. Calibration outcomes were limited. Compared with placebo, bisphosphonates (pooled RR, 0.67 [95% CI, 0.45-1.00]; 6 RCTs; 12 055 participants) and denosumab (RR, 0.60 [95% CI, 0.37-0.97] from the largest RCT [7808 participants]) were associated with reduced hip fractures. Compared with placebo, no statistically significant associations were observed for adverse events.

CONCLUSIONS AND RELEVANCE: Screening in higher-risk women 65 years or older was associated with a small absolute risk reduction in hip and major fractures compared with usual care. No evidence evaluated screening with BMD alone or screening in men or younger women. Risk assessment instruments, BMD alone, or both have poor to modest discrimination for predicting fracture. Osteoporosis treatment with bisphosphonates or denosumab over several years was associated with fracture reductions and no meaningful increase in adverse events.

PMID:39808441 | DOI:10.1001/jama.2024.21653

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Nevin Manimala Statistics

Preferred and Actual Location of Death in Adolescents and Young Adults With Cancer

JAMA Netw Open. 2025 Jan 2;8(1):e2454000. doi: 10.1001/jamanetworkopen.2024.54000.

ABSTRACT

IMPORTANCE: Adolescent and young adult (AYA) patients with advanced cancer often die in hospital settings. Data characterizing the degree to which this pattern of care is concordant with patient goals are sparse.

OBJECTIVE: To evaluate the extent of concordance between the preferred and actual location of death among AYA patients with cancer.

DESIGN, SETTING, AND PARTICIPANTS: This multicenter retrospective cohort study included AYA patients (aged 12-39 years) with cancer who died between January 1, 2003, and December 31, 2019, after receiving care at Dana-Farber Cancer Institute and Kaiser Permanente Northern California or who died between January 1, 2009, and December 31, 2019, after receiving care at Kaiser Permanente Southern California. Data were analyzed from January 12 to July 1, 2024.

EXPOSURE: Death due to cancer.

MAIN OUTCOMES AND MEASURES: Medical record documentation of discussions about preferred location of death, actual location of death, and concordance between preferred and actual location of death.

RESULTS: The analytic population included 1929 AYA decedents, of whom 1049 (54.4%) were female; 227 (11.8%), Asian; 157 (8.1%), Black; 514 (26.6%), Hispanic; and 1184 (61.4%), White. Median age at death was 32 (IQR, 25-37) years. A total of 1226 AYA patients (63.6%) had a documented discussion about preferred location of death. Among those with a documented discussion, 594 (48.5%) did not have a documented preference, 402 (32.8%) wanted to die at home, 177 (14.4%) preferred a hospital death, and 48 (3.9%) desired inpatient hospice. Eight hundred and thirty patients (43.0%) died in acute care settings (256 [13.3%] intensive care unit [ICU], 548 [28.4%] hospital [non-ICU], and 26 [1.3%] emergency department), while 643 (33.3%) died at home and 47 (2.4%) in an inpatient hospice. Among the 528 patients with both a documented preferred death location of home, hospital, or inpatient hospice and documented death in one of these locations, the concordance between preferred and actual location of death was 401 (75.9%). One hundred and sixty-four of 172 patients (95.3%) who preferred a hospital death died there; 224 of 317 (70.7%) who preferred a home death died at home, and 13 of 39 (33.3%) who desired to die in inpatient hospice did so.

CONCLUSIONS AND RELEVANCE: Although many AYA patients with cancer died in their preferred location, over one-quarter of those who desired to die at home did not realize this goal. These findings highlight the need for effective solutions to enable goal-concordant care for this population.

PMID:39808433 | DOI:10.1001/jamanetworkopen.2024.54000

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Electronic Patient-Reported Outcome System Implementation in Outpatient Cardiovascular Care: A Randomized Clinical Trial

JAMA Netw Open. 2025 Jan 2;8(1):e2454084. doi: 10.1001/jamanetworkopen.2024.54084.

ABSTRACT

IMPORTANCE: The integration of patient-reported outcome (PRO) assessments in cardiovascular care has encountered considerable obstacles despite their established clinical relevance.

OBJECTIVE: To assess the impact of a physician- and patient-friendly electronic PRO (ePRO) monitoring system on the quality of cardiovascular care in clinical practice.

DESIGN, SETTING, AND PARTICIPANTS: This open-label, multicenter, pilot randomized clinical trial was phase 2 of a multiphase study that was conducted from October 2022 to October 2023 and focused on the implementation and evaluation of an ePRO monitoring system in outpatient clinics in Japan. During phase 1 (conducted from December 2021 to March 2022), this ePRO monitoring system was developed at a single outpatient center using feedback from patients and physicians. Eligible patients were older than 18 years with a clinical diagnosis of heart failure (HF), atrial fibrillation (AF), or coronary artery disease (CAD). Participants were randomly assigned 1:1 to either the ePRO or control group. Statistical analysis was based on the intention-to-treat approach.

INTERVENTION: Patients in the ePRO group were asked to complete the 12-item Kansas City Cardiomyopathy Questionnaire (KCCQ-12), Atrial Fibrillation Effect on Quality-of-Life Questionnaire (AFEQT), or Seattle Angina Questionnaire (SAQ) on smart tablets before their clinical examination. Results were reviewed by physicians at each outpatient visit. The control group received usual care consisting of medication management, lifestyle modification, and invasive procedures in accordance with clinical guidelines. Both groups had 5 monthly outpatient visits.

MAIN OUTCOMES AND MEASURES: Patient satisfaction, quality of information (QOI) provided by physicians, and disease knowledge were assessed using questionnaires (eg, Patient Satisfaction Questionnaire [PSQ]; score range: 5-25, with higher scores indicating greater patient satisfaction) at baseline and the fifth visit, over approximately 4 months.

RESULTS: Of the 50 patients included, 48 (median [SD] age, 71.0 [62.3-75.0] years; 28 males [58.3%]) completed follow-up. The prevalence was 56.2% (n = 27) for HF, 75.0% (n = 36) for AF, and 10.4% (n = 5) for CAD. The distribution of PRO measures used was 21 patients (43.8%) for KCCQ-12, 24 (50.0%) for AFEQT, and 3 (6.2%) for SAQ. Compared with the control group, the ePRO group showed significant improvements in mean (SD) PSQ score (0.16 [2.06] vs 1.61 [1.75]; P = .01) and QOI score regarding treatment (-0.12 [0.53] vs 0.35 [0.71]; P = .01). The ePRO group exhibited a greater increase in the PSQ score regarding communication (-0.12 [0.53] vs 0.43 [0.90]; P = .01).

CONCLUSIONS AND RELEVANCE: In this randomized clinical trial, implementation of the ePRO monitoring system significantly enhanced patient-physician communication and the clarity of physicians’ explanations about treatment. These findings suggest that the ePRO monitoring system is capable of supporting patient-centered cardiovascular care.

TRIAL REGISTRATION: University Hospital Medical Information Network Identifier: UMIN000049251.

PMID:39808432 | DOI:10.1001/jamanetworkopen.2024.54084

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Knowledge and Use of Menthol-Mimicking Cigarettes Among Adults in the US

JAMA Netw Open. 2025 Jan 2;8(1):e2454608. doi: 10.1001/jamanetworkopen.2024.54608.

ABSTRACT

IMPORTANCE: Cigarette companies have been introducing synthetic cooling agent menthol-mimicking cigarettes into the US marketplace as menthol cigarette bans are implemented. These cigarettes may reduce the public health benefits of menthol cigarette bans.

OBJECTIVE: To examine the epidemiology of the use of synthetic cooling agent menthol-mimicking cigarettes among adults in the US.

DESIGN, SETTING, AND PARTICIPANTS: This population-based cross-sectional survey study of adults in the US (≥21 years) with oversampling of Asian, American Indian or Alaska Native, Black or African American, and Hispanic or Latino peoples was conducted from March to May 2024. Participants were recruited from an online panel using a sample-matching approach to achieve national representation. Data were weighted to be nationally representative. Descriptive statistics were used to summarize the population characteristics. Weighted prevalence of awareness of, ever use (among those aware), current use (among ever used), susceptibility (among noncurrent use), and likely to switch to synthetic cooling agent menthol-mimicking cigarettes were estimated overall and by covariate. Sequential weighted logistic regression models were used to estimate the associations between covariates and synthetic cooling agent menthol-mimicking-related outcomes (awareness, ever use, current use, susceptibility). Statistical analysis was performed from July to September 2024.

EXPOSURES: Demographics, state menthol cigarette ban, cigarette smoking, and disclosure of synthetic cooling agent.

MAIN OUTCOMES AND MEASURES: Awareness of, susceptibility to, ever and currently using synthetic cooling agent menthol-mimicking cigarettes and intention to switch to these cigarettes if a nationwide menthol cigarette ban were enacted.

RESULTS: The study population included 3200 US adults (47.4% men; 0.8% American Indian or Alaska Native, 5.9% Asian, 12.0% Black or African American, 16.0% Hispanic or Latino, 63.6% White, and 1.9% other race; 34.8% with a college degree; and 36.7% with annual household income of ≥$75 000). Overall, 29.1% (95% CI, 27.0%-31.1%) of US adults were aware of synthetic cooling agent menthol-mimicking cigarettes, 36.9% (95% CI, 33.1%-40.8%) of those aware ever used these cigarettes, 24.7% (95% CI, 20.1%-29.3%) of those who ever used reported currently using these cigarettes, and 37.5% (95% CI, 33.4%-41.6%) of those not currently using were susceptible to using them. Black or African American (vs White), men (vs women), adults aged 60 years or younger (vs adults aged ≥61 years), adults who used menthol and nonmenthol cigarettes (vs adults who did not smoke cigarettes), and those who lived in states with a menthol cigarette ban (vs those who lived in states with no ban) had higher odds of awareness, ever use, current use, and/or susceptibility to these cigarettes. Considering a national menthol cigarette ban, 50.8% (95% CI, 42.8%-58.7%) of adults who smoked menthol cigarettes and were aware of menthol-mimicking cigarettes with a synthetic cooling agent reported likely to switch to these cigarettes. Ever using these cigarettes was associated with the likelihood of switching (adjusted odds ratio, 2.61 [95% CI, 1.20-5.68]).

CONCLUSIONS AND RELEVANCE: In this survey study of US adults, a substantial proportion were aware of and had already experimented with synthetic cooling agent menthol-mimicking cigarettes. These products may serve as a substitute for menthol cigarettes and reduce the public health benefits of a menthol cigarette ban in promoting smoking cessation.

PMID:39808430 | DOI:10.1001/jamanetworkopen.2024.54608

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Use and Costs of Supplemental Benefits in Medicare Advantage, 2017-2021

JAMA Netw Open. 2025 Jan 2;8(1):e2454699. doi: 10.1001/jamanetworkopen.2024.54699.

ABSTRACT

IMPORTANCE: Nearly all Medicare Advantage (MA) plans offer dental, vision, and hearing benefits not covered by traditional Medicare (TM). However, little is known about MA enrollees’ use of those benefits or how much they cost MA insurers or enrollees.

OBJECTIVE: To estimate use, out-of-pocket (OOP) spending, and insurer payments for dental, hearing, and vision services among Medicare beneficiaries.

DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional analysis used pooled 2017-2021 Medical Expenditure Panel Survey (MEPS) and Medicare Current Beneficiary Survey (MCBS) data for MA and TM beneficiaries (excluding those also covered by Medicaid). The analysis was performed from September 10, 2023, to June 30, 2024.

EXPOSURES: MA compared with TM coverage.

MAIN OUTCOMES AND MEASURES: The main outcome was receipt of eye examinations, corrective lenses, hearing aids, optometry and dental visits, and MA and TM enrollees’ and insurers’ spending for such services. MEPS and MCBS data were weighted to be nationally representative.

RESULTS: We included 76 557 non-dually eligible Medicare beneficiaries, including 23 404 from the MEPS and 53 153 from the MCBS. Weighted demographic characteristics of MA and TM beneficiaries were similar (54.7% and 51.9% female; 39.8% and 35.2% older than 75 years, respectively). Only 54.2% (95% CI, 52.4%-55.9%) and 54.3% (95% CI 52.2%-56.3%) of MA beneficiaries were aware of having MA dental and vision coverage, respectively. MA enrollees were no more likely to receive eye examinations, hearing aids, or eyeglasses than TM enrollees. After adjustment for demographic differences, MA and TM enrollees paid OOP $205.86 (95% CI, $192.44-$219.27) and $226.12 (95% CI, $212.02-$240.23), respectively, for eyeglasses (MA – TM difference, -$20.27 [95% CI, -$33.77 to -$6.77] or -9.0% [95% CI, -14.9% to -3.0%]); $226.82 (95% CI, $202.24-$251.40) and $249.98 (95% CI, $226.22-$273.74) for dental visits, respectively (MA – TM difference, -$23.16 [95% CI, -$43.15 to -$3.17] or -9.3% [95% CI, -17.3% to -1.3%]); and no less for optometry visits or durable medical equipment (a proxy for hearing aids). Nationwide, MA plans’ annual spending on vision, dental services, and durable medical equipment totaled $3.9 billion (95% CI, $3.3-$4.4 billion), while enrollees spent OOP $9.2 billion (95% CI, $8.2-$10.2 billion) annually for these services and other private insurers covered $2.8 billion (95% CI, $2.7-$3.0 billion).

CONCLUSIONS AND RELEVANCE: In this cross-sectional study of 2 nationally representative surveys, MA beneficiaries did not receive more supplemental services than TM beneficiaries, possibly because of cost-sharing and limited awareness of benefit coverage.

PMID:39808428 | DOI:10.1001/jamanetworkopen.2024.54699

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High-dimensional partially linear functional Cox models

Biometrics. 2025 Jan 7;81(1):ujae164. doi: 10.1093/biomtc/ujae164.

ABSTRACT

As a commonly employed method for analyzing time-to-event data involving functional predictors, the functional Cox model assumes a linear relationship between the functional principal component (FPC) scores of the functional predictors and the hazard rates. However, in practical scenarios, such as our study on the survival time of kidney transplant recipients, this assumption often fails to hold. To address this limitation, we introduce a class of high-dimensional partially linear functional Cox models, which accommodates the non-linear effects of functional predictors on the response and allows for diverging numbers of scalar predictors and FPCs as the sample size increases. We employ the group smoothly clipped absolute deviation method to select relevant scalar predictors and FPCs, and use B-splines to obtain a smoothed estimate of the non-linear effect. The finite sample performance of the estimates is evaluated through simulation studies. The model is also applied to a kidney transplant dataset, allowing us to make inferences about the non-linear effects of functional predictors on patients’ hazard rates, as well as to identify significant scalar predictors for long-term survival time.

PMID:39808421 | DOI:10.1093/biomtc/ujae164

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Predictive Optimization of Patient No-Show Management in Primary Healthcare Using Machine Learning

J Med Syst. 2025 Jan 14;49(1):7. doi: 10.1007/s10916-025-02143-w.

ABSTRACT

The “no-show” problem in healthcare refers to the prevalent phenomenon where patients schedule appointments with healthcare providers but fail to attend them without prior cancellation or rescheduling. In addressing this issue, our study delves into a multivariate analysis over a five-year period involving 21,969 patients. Our study introduces a predictive model framework that offers a holistic approach to managing the no-show problem in healthcare, incorporating elements into the objective function that address not only the accurate prediction of no-shows but also the management of service capacity, overbooking, and idle resource allocation resulting from mispredictions. Our approach simplifies preprocessing and eliminates the need for expert judgment in variable selection, thereby enhancing the model’s usability in routine healthcare operations. Our research revealed that key predictors of no-shows are consistent across various studies. We employed semi-automatic feature selection techniques, achieving results comparable to state-of-the-art approaches but with significantly reduced complexity in their selection. This method not only streamlines the feature selection process but also enhances the overall efficiency and scalability of our predictive models, making them more adaptable to diverse healthcare settings. This comprehensive strategy enables healthcare providers to optimize resource allocation and improve service delivery, making our findings relevant for healthcare systems globally facing similar challenges. Future work aims to expand the analysis by incorporating additional third-party data sources, such as weather and commuting activities, to explore the broader impacts of external factors on patient no-show behavior. To the best of our knowledge, this innovative approach is expected to provide deeper insights and further enhance the predictability and effectiveness of no-show mitigation strategies in healthcare systems.

PMID:39808378 | DOI:10.1007/s10916-025-02143-w

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Romosozumab adverse event profile: a pharmacovigilance analysis based on the FDA Adverse Event Reporting System (FAERS) from 2019 to 2023

Aging Clin Exp Res. 2025 Jan 14;37(1):23. doi: 10.1007/s40520-024-02921-5.

ABSTRACT

OBJECTIVE: This study aims to analyze adverse drug events (ADE) related to romosozumab from the second quarter of 2019 to the third quarter of 2023 from FAERS database.

METHODS: The ADE data related to romosozumab from 2019 Q2 to 2023 Q3 were collected. After data normalization, four signal strength quantification algorithms were used: ROR (Reporting Odds Ratios), PRR (Proportional Reporting Ratios), BCPNN (Bayesian Confidence Propagation Neural Network), and EBGM (Empirical Bayesian Geometric Mean).

RESULTS: Screening for romosozumab-related AEs (adverse events) included 23 system organ categories (SOCs). PT (preferred terms) levels were screened for adverse drug reaction (ADR) signals. A total of 7055 reports with romosozumab as the primary suspect (PS) and 14,041 PTs induced by romosozumab as PS were identified. Common significant signals of general disorders and administration site conditions, musculoskeletal and connective tissue disorders have emerged. Specifically, unexpected AEs such as gastrointestinal disorder, respiratory, thoracic and mediastinal disorders also occur. Notably, fracture (n = 503, ROR = 107.8, PRR = 103.83, IC = 6.6, EBGM = 97.02) and bone density abnormal (n = 429, ROR = 343.65, PRR = 332.77, IC = 8.08, EBGM = 271.34) exhibited relatively high occurrence rates and signal strengths.

CONCLUSION: Our study identifies potential new AE signals and provides broader data support for the safety of romosozumab. In clinical application, doctors are provided with a warning to closely monitor adverse reactions to support their rational use in diseases such as osteoporosis.

PMID:39808360 | DOI:10.1007/s40520-024-02921-5

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Analysis of macular retinal thickness in polyarteritis nodosa using spectral domain optical coherence tomography

J Ophthalmic Inflamm Infect. 2025 Jan 14;15(1):6. doi: 10.1186/s12348-025-00453-1.

ABSTRACT

PURPOSE: To identify the macular retinal layer thickness changes in polyarteritis nodosa (PAN) patients without pathological findings appearing in color fundus photography (CFP), and to investigate the correlations with disease durations.

METHODS: A total of 24 PAN patients who had been for 3 years or more and underwent SD-OCT were recruited from the UK Biobank, with exclusions for diabetes, eye disease, or abnormal CFP findings. Only the right eyes were included, with each PAN patient paired one-to-one with a control matched for age, sex, and ethnicity. Paired t-tests or Wilcoxon Signed-Rank tests were used to assess the differences in thickness of different retinal layers between groups, followed by linear regression analysis to evaluate the correlations with disease durations.

RESULTS: PAN patients had significantly thinner retinal nerve fiber layer (RNFL) by 12.27% (mean ± standard deviation = 27.39 ± 8.94 μm for PAN patients and 31.22 ± 5.57 μm for controls, p = 0.048) and thinner outer plexiform and outer nuclear layers (OPL-ONL complex) by 10.67% (44.93 ± 6.59 μm for PAN patients and 50.31 ± 7.60 μm for controls, p = 0.032). Visual acuity and the whole macular thickness showed no statistical difference. The RNFL was thinned by 1.22 μm per year of disease progression (95% confidence interval: 0.12, 2.32, p = 0.042).

CONCLUSIONS: PAN patients without visual impairments or abnormal CFP findings may exhibit significant thinning in RNFL and OPL-ONL complex. SD-OCT may serve as a useful tool for early screening of ophthalmic changes in PAN.

PMID:39808358 | DOI:10.1186/s12348-025-00453-1

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Comparison of opportunities and perceptions towards neurosurgery between the public and private sector in Pakistan: a national survey of medical students and recent graduates

Neurosurg Rev. 2025 Jan 14;48(1):46. doi: 10.1007/s10143-025-03198-6.

ABSTRACT

Public and private medical institutes must adhere to the same standards of quality set by the Pakistan Medical & Dental Council (PMDC). However, studies have noted varied learning environments. The current study aims to assess opportunities and compare the differences in perceptions between the two sectors. An online survey was conducted among medical students, interns, and medical officers across Pakistan. Respondents were divided into two groups: private and public sector and the survey responses of the two groups were compared. Descriptive statistical analyses of responses were performed, and a chi-square test was used to obtain the p-values. 2,481 responses were collected (48.6% public vs 51.3% private sector). A higher proportion of public sector respondents reported neurosurgery within their curriculum (71.2% public, 58.9% private) ( p < 0.001), while a greater proportion of private sector respondents had less exposure to neurosurgery clerkships (58.5% public, 71.9% private). A higher proportion from the private sector reported that sufficient mentorship opportunities (38.3% private, 29.5% public) (p < 0.001) were provided by their medical college. More private sector respondents reported lack of work-life balance (39.5% public, 42.4% private), competitive work environment (55.9% public, 61.3% private) (p < 0.001) and financial burden (67.3% private, 60.8% public) (p < 0.001) as major barriers towards pursuing neurosurgery. Our study outlines key areas for improvement from the perspective of the learners themselves. These include increasing exposure to neurosurgery in medical colleges and affiliated hospitals and improving post-graduate training among all sectors. Addressing these concerns of the medical students, interns, recent graduates and medical officers can foster the pursuit of neurosurgery as a career in Pakistan. It is crucial to further investigate medical education in Pakistan and study differences in the public and private education sector globally.

PMID:39808338 | DOI:10.1007/s10143-025-03198-6