Category: Statistics

JAMA Netw Open. 2024 Mar 4;7(3):e244113. doi: 10.1001/jamanetworkopen.2024.4113.

ABSTRACT

IMPORTANCE: The reported phenotypes of men with 47,XXY and 47,XYY syndromes include tall stature, multisystem comorbidities, and poor health-related quality of life (HRQOL). However, knowledge about these sex chromosome aneuploidy (SCA) conditions has been derived from studies in the less than 15% of patients who are clinically diagnosed and also lack diversity in age and genetic ancestry.

OBJECTIVES: To determine the prevalence of clinically diagnosed and undiagnosed X or Y chromosome aneuploidy among men enrolled in the Million Veteran Program (MVP); to describe military service metrics of men with SCAs; and to compare morbidity and mortality outcomes between men with SCA with and without a clinical diagnosis vs matched controls.

DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study used a case-control recruitment design to select biological males enrolled in the MVP biobank in the US Veterans Administration health care system from 2011 to 2022. Cases were participants with 47,XXY syndrome or 47,XYY syndrome, matched 1:5 with controls based on sex, age, and genetic ancestry. Data were analyzed from January 2022 to December 2023.

EXPOSURE: Genomic identification of an additional X or Y chromosome.

MAIN OUTCOMES AND MEASURES: Outcomes of interest included prevalence of men with SCAs from genomic analysis; clinical SCA diagnosis; Charlson Comorbidity Index; rates of outpatient, inpatient, and emergency encounters per year; self-reported health outcomes; and standardized mortality ratio.

RESULTS: Of 595 612 genotyped males in the MVP, 862 had an additional X chromosome (47,XXY) and 747 had an extra Y chromosome (47,XYY), with the highest prevalence among men with East Asian (47,XXY: 10 of 7313 participants; 47,XYY: 14 of 7313 participants) and European (47,XXY: 725 of 427 143 participants; 47,XYY: 625 of 427 143 participants) ancestry. Mean (SD) age at assessment was 61 (12) years, at which point 636 veterans (74.X%) with 47,XXY and 745 veterans (99%) with 47,XYY remained undiagnosed. Individuals with 47,XXY and 47,XYY had similar military service history, all-cause standardized mortality ratio, and age of death compared with matched controls. Individuals with SCA, compared with controls, had higher Charlson Comorbidity Index scores (47,XXY: mean [SD], 4.30 [2.72] vs controls: mean [SD], 3.90 [2.47]; 47,XYY: mean [SD], 4.45 [2.90] vs controls: mean [SD], 3.82 [2.50]) and health care utilization (eg, median [IQR] outpatient encounters per year: 47,XXY, 22.6 [11.8-37.8] vs controls, 16.8 [9.4-28]; 47,XYY: 21.4 [12.4-33.8] vs controls: 17.0 [9.4-28.2]), while several measures of HRQOL were lower (eg, mean [SD] self-reported physical function: 47,XXY: 34.2 [12] vs control mean [SD] 37.8 [12.8]; 47,XYY: 36.3 [11.6] vs control 37.9 [12.8]). Men with a clinical diagnosis of 47,XXY, compared with individuals without a clinical diagnosis, had higher health care utilization (eg, median [IQR] encounters per year: 26.6 [14.9-43.2] vs 22.2 [11.3-36.0]) but lower Charlson Comorbidity Index scores (mean [SD]: 3.7 [2.7] vs 4.5 [4.1]).

CONCLUSION AND RELEVANCE: In this case-control study of men with 47,XXY and 47,XYY syndromes, prevalence of SCA was comparable with estimates in the general population. While these men had successfully served in the military, they had higher morbidity and reported poorer HRQOL with aging. Longer longitudinal follow-up of this sample will be informative for clinical and patient-reported outcomes, the role of ancestry, and mortality statistics.

PMID:38551561 | DOI:10.1001/jamanetworkopen.2024.4113

JAMA Netw Open. 2024 Mar 4;7(3):e244427. doi: 10.1001/jamanetworkopen.2024.4427.

ABSTRACT

IMPORTANCE: Adolescent suicide in the US is a major public health problem, yet temporal trends in suicide methods by demographics are understudied.

OBJECTIVE: To examine national trends in suicide mortality by method (firearm, poisoning, hanging and asphyxiation, and all other means) from 1999 to 2020 by demographic characteristics.

DESIGN, SETTING, AND PARTICIPANTS: This serial cross-sectional study used national death certificate data of adolescent (aged 10-19 years) suicide decedents compiled by the National Center for Health Statistics from January 1, 1999, to December 31, 2020. Data analysis was performed from April 1, 2023, to July 9, 2023.

EXPOSURES: Age, sex, and race and ethnicity.

MAIN OUTCOMES AND MEASURES: Trends in age-standardized mortality rates and average annual percent change (AAPC) in rates were estimated by age, sex, and race and ethnicity for each suicide method.

RESULTS: This study assessed data from 47 217 adolescent suicide decedents. From 1999 to 2020, suicide by firearm (AAPC, 1.0; 95% CI, 0.1-1.9), poisoning (AAPC, 2.7; 95% CI, 1.0-4.4), hanging and asphyxiation (AAPC, 2.4; 95% CI, 0.2-4.6), and other means (AAPC, 2.9; 95% CI, 1.2-4.6) increased. Rapidly increasing rates were observed among female adolescents for poisoning (AAPC, 4.5; 95% CI, 2.3-6.7) and hanging and asphyxiation (AAPC, 5.9; 95% CI, 5.0-6.8) suicides. From 2007 to 2020, firearm suicides sharply increased among female (annual percent change [APC], 7.8; 95% CI, 6.0-9.5) and male (APC, 5.3; 95% CI, 4.3-6.3) adolescents. Firearm suicide rates increased among Black adolescents from 2012 to 2020 (APC, 14.5; 95% CI, 9.7-19.5), Asian and Pacific Islander adolescents from 2008 to 2020 (APC, 12.0; 95% CI, 9.7-14.5), American Indian and Alaska Native adolescents from 2014 to 2020 (APC, 10.6; 95% CI, 2.6-19.3), and Hispanic or Latino adolescents from 2011 to 2020 (APC, 10.2; 95% CI, 6.3-13.8). During the study period, Black adolescents had the highest average increase in hanging and asphyxiation suicides (AAPC, 4.2; 95% CI, 3.2-5.2). From 2011 to 2020, poisoning suicide deaths increased (APC, 12.6; 95% CI, 8.5-16.7) among female adolescents.

CONCLUSIONS AND RELEVANCE: Suicide rates increased across all methods from 1999 to 2020. Differences were noted by sex, age, and race and ethnicity. Increasing suicide rates among racial and ethnic minoritized youth are especially concerning, and effective prevention strategies are urgently needed.

PMID:38551558 | DOI:10.1001/jamanetworkopen.2024.4427

J Perianesth Nurs. 2024 Mar 26:S1089-9472(23)01102-4. doi: 10.1016/j.jopan.2023.12.015. Online ahead of print.

ABSTRACT

PURPOSE: The study was conducted to determine the effect of the therapeutic play method implemented on the pre- and postcircumcision pain and anxiety levels of children and mothers’ anxiety levels in the pediatric surgery clinic.

DESIGN: Prospective, randomized clinical trial.

METHODS: The study consisted of children between the ages of 3 to 6 admitted to the pediatric surgery clinic and their mothers (N = 120; Therapeutic playgroup = 60, Control group = 60). As a therapeutic play method the children and their mothers used play dough during the structured conversation for about 10 minutes before and after the operation. A questionnaire, the Face, Legs, Activity, Cry, Consolability (FLACC) pain scale, the Children’s Emotional Manifestation Scale (CEMS) anxiety scale, and State-Trait Anxiety Inventory-S (STAI-S) anxiety scale for parents were used to collect data.

FINDINGS: In the pre- and postcircumcision period, the pain and anxiety levels of children and mothers’ anxiety levels in the therapeutic playgroup were statistically lower compared to the control group (P < .05). In the postoperative period, a positive and significance correlation was found between anxiety levels of the mothers and pain and anxiety levels of children in the control group (P < .05).

CONCLUSIONS: The therapeutic play method was effective in reducing the pain and anxiety levels of children and mothers’ anxiety in the pre- and postcircumcision period.

PMID:38551545 | DOI:10.1016/j.jopan.2023.12.015

Mayo Clin Proc. 2024 Mar 28:S0025-6196(24)00035-1. doi: 10.1016/j.mayocp.2023.12.025. Online ahead of print.

ABSTRACT

OBJECTIVE: To quantify the burden of metabolic dysfunction-associated steatotic liver disease (MASLD) and related metabolic disorders in premenopausal women.

PATIENTS AND METHODS: Between 2010 and 2019, global evaluations of prevalence, mortality, disability-adjusted life years (DALYs), and their age-standardized rate (ASR) were conducted for metabolic conditions such as MASLD, type 2 diabetes mellitus, dyslipidemia, hypertension (HTN), obesity, and polycystic ovarian syndrome. Subgroup assessments were conducted according to geographical regions and the sociodemographic index. The predictive models were established to estimate mortality and DALYs through 2040.

RESULTS: In 2019, the most significant ASR of deaths was found in HTN (11.37; 9.52 to 13.45), followed by obesity (10.49; 7.57 to 13.64). In contrast, the greatest ASR of DALYs was attributed to obesity (816.13; 581.41 to 1073.32), followed by HTN (634.73; 536.75 to 744.77). The mortality rates for dyslipidemia (^–0.55%) and HTN (^–0.72%) have been decreasing over time, but there has been an increase in obesity (⁺0.58%), type 2 diabetes mellitus (⁺0.85%), and MASLD (⁺0.51%). Lower sociodemographic index countries exhibit a higher disability-to-prevalence ratio. In 2040, obesity is predicted to cause the most deaths (⁺41.59% from 2019).

CONCLUSION: The escalating impact of metabolic syndrome, the rising trends in death rates linked to obesity, and the disparities based on region and socioeconomic status in premenopausal women underscore the alarming increase in the global burden of metabolic syndrome.

PMID:38551541 | DOI:10.1016/j.mayocp.2023.12.025

Am J Kidney Dis. 2024 Mar 15:S0272-6386(24)00620-6. doi: 10.1053/j.ajkd.2023.12.018. Online ahead of print.

ABSTRACT

RATIONALE & OBJECTIVE: Evidence has demonstrated that albuminuria is a key diagnostic and prognostic marker of diabetic chronic kidney disease, but the impact of its day-to-day variability has not been adequately considered. This study quantified within-individual variability of albuminuria in people with type 2 diabetes to inform clinical albuminuria monitoring.

STUDY DESIGN: Descriptive cross-sectional analysis.

SETTING & PARTICIPANTS: People with type 2 diabetes (n=826, 67.1 [IQR, 60.3-72.4] years, 64.9% male) participating in the Progression of Diabetic Complications (PREDICT) cohort study.

EXPOSURE: Four spot urine collections for measurement of urinary albumin-creatinine ratio (UACR) within 4 weeks.

OUTCOME: Variability of UACR.

ANALYTICAL APPROACH: We characterized within-individual variability (coefficient of variation [CV], 95% limits of random variation, intraclass correlation coefficient), developed a calculator displaying probabilities that any observed difference between a pair of UACR values truly exceeded a 30% difference, and estimated the ranges of diagnostic uncertainty to inform a need for additional UACR collections to exclude or confirm albuminuria. Multiple linear regression examined factors influencing UACR variability.

RESULTS: We observed high within-individual variability (CV 48.8%; 95% limits of random variation showed a repeated UACR to be as high/low as 3.78/0.26 times the first). If a single-collection UACR increased from 2 to 5mg/mmol, the probability that UACR actually increased by at least 30% was only 50%, rising to 97% when 2 collections were obtained at each time point. The ranges of diagnostic uncertainty were 2.0-4.0mg/mmol after an initial UACR test, narrowing to 2.4-3.2 and 2.7-2.9mg/mmol for the mean of 2 and 3 collections, respectively. Some factors correlated with higher (female sex; moderately increased albuminuria) or lower (reduced estimated glomerular filtration rate and sodium-glucose cotransporter 2 inhibitor/angiotensin-converting enzyme inhibitor/angiotensin receptor blocker treatment) within-individual UACR variability.

LIMITATIONS: Reliance on the mean of 4 UACR collections as the reference standard for albuminuria.

CONCLUSIONS: UACR demonstrates a high degree of within-individual variability among individuals with type 2 diabetes. Multiple urine collections for UACR may improve capacity to monitor changes over time in clinical and research settings but may not be necessary for the diagnosis of albuminuria.

PLAIN-LANGUAGE SUMMARY: Albuminuria (albumin in urine) is a diagnostic and prognostic marker of diabetic chronic kidney disease. However, albuminuria can vary within an individual from day to day. We compared 4 random spot urinary albumin-creatinine ratio (UACR) samples from 826 participants. We found that a second UACR collection may be as small as a fourth or as large as almost 4 times the first sample’s UACR level. This high degree of variability presents a challenge to our ability to interpret changes in albuminuria. Multiple collections have been suggested as a solution. We have constructed tools that may aid clinicians in deciding how many urine collections are required to monitor and diagnose albuminuria. Multiple urine collections may be required for individual monitoring but not necessarily for diagnosis.

PMID:38551531 | DOI:10.1053/j.ajkd.2023.12.018

Eur J Phys Rehabil Med. 2024 Mar 29. doi: 10.23736/S1973-9087.24.08232-7. Online ahead of print.

ABSTRACT

BACKGROUND: Although loss of muscle mass may be associated with general weakness, intolerance to physical activity and fatigue, it is underestimated and poorly understood in patients with sarcoidosis.

AIM: To compare the quadriceps femoris muscle (QFM) thickness measured by ultrasonography (US) between the female patients with sarcoidosis and controls, secondly to assess the correlation between the muscle strength, fatigue and QFM thickness.

DESIGN: Observational, case-control study.

SETTING: Physical Medicine and Rehabilitation Department of a University Hospital.

POPULATION: Thirty-one women with sarcoidosis and 27 healthy volunteers were included in the study.

METHODS: The participants were evaluated for the following outcomes: 1) handgrip strength; 2) QFM thickness measured using US; and 3) sonographic thigh adjustment ratio (STAR). The sarcoidosis group was also evaluated with the 30-second chair stand test (30s-CST) and Fatigue Severity Scale (FSS).

RESULTS: The QFM thickness and STAR values of the patients with sarcoidosis were significantly lower than those of the controls (P=0.0001). However, no statistically significant difference was observed between the handgrip strengths of the groups (P=0.581). There was no statistically significant correlation between the STAR values and handgrip strength in the sarcoidosis group; however, there was a significant positive correlation between the STAR values and 30s-CST (r=0.467, P=0.008).

CONCLUSIONS: Loss of muscle mass is one of the musculoskeletal conditions in patients with sarcoidosis that may be associated with nonspecific symptoms, such as general debility, intolerance to physical activity, and fatigue. In the present study, no difference was observed in hand grip strength between the groups, while we found that QFM thickness was affected in patients with sarcoidosis when compared to the controls. The ultrasonographic QFM evaluation seems to be an innovative tool which may be used at all stages of sarcoidosis patient follow-up.

CLINICAL REHABILITATION IMPACT: The grip strength is a commonly used test to detect muscle weakness, but onset of a decrease in muscle mass in the lower extremities may occur earlier. Considering the increased burden of musculoskeletal problems in this population, performing 30s-CST and sonographic QFM thickness is practical methods to identify risky patients.

PMID:38551519 | DOI:10.23736/S1973-9087.24.08232-7

Res Psychother. 2024 Mar 21. doi: 10.4081/ripppo.2024.736. Online ahead of print.

ABSTRACT

The present study reports on the development and validation of the clinician affective response (CARE) scale. The CARE scale was designed as a self-report measure of therapists’ patterns of thoughts, feelings, and behaviors toward the patient during an individual psychotherapy session. An initial pool of 116 items was generated, and its quality was evaluated by subject matter experts. Validation data were gathered from licensed psychotherapists (n=554). We used exploratory factor analysis and item response theory-graded response modeling to select items, confirmatory factor analysis to test how well the factor structure fit the data, and k-fold cross-validation to ascertain the robustness of the model. Criterion validity was evaluated by correlating the scores of the scale with the characteristics of therapists, patients, and treatment. The selected model consists of 15 items and a 3-factor structure, which showed excellent model fit, good internal consistency, and evidence of criterion validity. The CARE scale, short and quick to complete, enables therapists to reflect on and recognize their inner experiences and quantify these experiences in ways conducive to statistical analysis and research. Furthermore, the monitoring of these affective reactions toward their patients can guide therapeutic interventions and inform clinical supervisors.

PMID:38551502 | DOI:10.4081/ripppo.2024.736

Facts Views Vis Obgyn. 2024 Mar;16(1):47-58. doi: 10.52054/FVVO.16.1.008.

ABSTRACT

BACKGROUND: The #Enzian classification represents a system to describe endometriotic lesions during surgery. Its use is well established in correlating ultrasound and surgical findings.

OBJECTIVES: To describe interobserver reproducibility of ultrasound use and symptom correlation with compartments involved using #Enzian classification.

MATERIALS AND METHODS: Two experienced operators performed transvaginal sonography (TVS) in 52 patients affected by pelvic endometriosis. A rate agreement was determined. A further 200 women with endometriotic TVS signs, with no previous surgery and not taking any hormonal therapy, were staged by one of three different operators according to the #Enzian (compartments A, B, C, O, T, FA, FB, FI, FU, FO). Statistical analysis compared all the compartments, as single or associated, with single or combined symptoms (dysmenorrhea, dyspareunia, heavy menstrual bleeding – HMB, bowel symptoms).

MAIN OUTCOME MEASURES: Evaluation of the reproducibility of #Enzian classification in assessing pelvic endometriosis among different operators using TVS, and of possible associations between symptoms and specific #Enzian compartments.

RESULTS: Excellent agreement between the two operators in evaluating almost all the compartments (k >0.8) was observed. Dysmenorrhea did not correlate with any specific compartment. We observed a significant association between dyspareunia and B compartment (p=0.02). HMB is associated with FA (p=0.02). Bowel symptoms were associated with B (p=0.02). Combining more symptoms, we observed more significant associations with different compartments.

CONCLUSIONS: #ENZIAN classification is reproducible in the evaluation of pelvic endometriosis. Some symptoms are correlated to specific ultrasound signs of the disease.

WHAT IS NEW?: An accurate evaluation of symptoms could guide TVS examination to detect specific endometriotic lesions and establish the best management for the patients.

PMID:38551474 | DOI:10.52054/FVVO.16.1.008

Int J Biostat. 2024 Mar 29. doi: 10.1515/ijb-2023-0051. Online ahead of print.

ABSTRACT

The mean residual lifetime (MRL) of a unit in a population at a given time t, is the average remaining lifetime among those population units still alive at the time t. In some applications, it is reasonable to assume that MRL function is a decreasing function over time. Thus, one natural way to improve the estimation of MRL function is to use this assumption in estimation process. In this paper, we develop an MRL estimator in ranked set sampling (RSS) which, enjoys the monotonicity property. We prove that it is a strongly uniformly consistent estimator of true MRL function. We also show that the asymptotic distribution of the introduced estimator is the same as the empirical one, and therefore the novel estimator is obtained “free of charge”, at least in an asymptotic sense. We then compare the proposed estimator with its competitors in RSS and simple random sampling (SRS) using Monte Carlo simulation. Our simulation results confirm the superiority of the proposed procedure for finite sample sizes. Finally, a real dataset from the Surveillance, Epidemiology and End Results (SEER) program of the US National Cancer Institute (NCI) is used to show that the introduced technique can provide more accurate estimates for the average remaining lifetime of patients with breast cancer.

PMID:38551082 | DOI:10.1515/ijb-2023-0051

Health Soc Care Deliv Res. 2024 Mar;12(6):1-143. doi: 10.3310/HYTR4612.

ABSTRACT

BACKGROUND: The frequency at which patients should have their vital signs (e.g. blood pressure, pulse, oxygen saturation) measured on hospital wards is currently unknown. Current National Health Service monitoring protocols are based on expert opinion but supported by little empirical evidence. The challenge is finding the balance between insufficient monitoring (risking missing early signs of deterioration and delays in treatment) and over-observation of stable patients (wasting resources needed in other aspects of care).

OBJECTIVE: Provide an evidence-based approach to creating monitoring protocols based on a patient’s risk of deterioration and link these to nursing workload and economic impact.

DESIGN: Our study consisted of two parts: (1) an observational study of nursing staff to ascertain the time to perform vital sign observations; and (2) a retrospective study of historic data on patient admissions exploring the relationships between National Early Warning Score and risk of outcome over time. These were underpinned by opinions and experiences from stakeholders.

SETTING AND PARTICIPANTS: Observational study: observed nursing staff on 16 randomly selected adult general wards at four acute National Health Service hospitals. Retrospective study: extracted, linked and analysed routinely collected data from two large National Health Service acute trusts; data from over 400,000 patient admissions and 9,000,000 vital sign observations.

RESULTS: Observational study found a variety of practices, with two hospitals having registered nurses take the majority of vital sign observations and two favouring healthcare assistants or student nurses. However, whoever took the observations spent roughly the same length of time. The average was 5:01 minutes per observation over a ’round’, including time to locate and prepare the equipment and travel to the patient area. Retrospective study created survival models predicting the risk of outcomes over time since the patient was last observed. For low-risk patients, there was little difference in risk between 4 hours and 24 hours post observation.

CONCLUSIONS: We explored several different scenarios with our stakeholders (clinicians and patients), based on how ‘risk’ could be managed in different ways. Vital sign observations are often done more frequently than necessary from a bald assessment of the patient’s risk, and we show that a maximum threshold of risk could theoretically be achieved with less resource. Existing resources could therefore be redeployed within a changed protocol to achieve better outcomes for some patients without compromising the safety of the rest. Our work supports the approach of the current monitoring protocol, whereby patients’ National Early Warning Score 2 guides observation frequency. Existing practice is to observe higher-risk patients more frequently and our findings have shown that this is objectively justified. It is worth noting that important nurse-patient interactions take place during vital sign monitoring and should not be eliminated under new monitoring processes. Our study contributes to the existing evidence on how vital sign observations should be scheduled. However, ultimately, it is for the relevant professionals to decide how our work should be used.

STUDY REGISTRATION: This study is registered as ISRCTN10863045.

FUNDING: This award was funded by the National Institute for Health and Care Research (NIHR) Health and Social Care Delivery Research programme (NIHR award ref: 17/05/03) and is published in full in Health and Social Care Delivery Research; Vol. 12, No. 6. See the NIHR Funding and Awards website for further award information.

PMID:38551079 | DOI:10.3310/HYTR4612