Tag: statistics

Br J Radiol. 2022 Oct 19:20220634. doi: 10.1259/bjr.20220634. Online ahead of print.

ABSTRACT

OBJECTIVES: To provide ultrasound baselines for spleen length in homozygous sickle cell disease (HbSS) and in normal controls with a HbAA genotype.

METHODS: The Jamaican cohort study identified 311 babies with HbSS and 246 matched HbAA controls during the screening of 100,000 consecutive deliveries in Kingston, Jamaica from 1973 to 1981. Ultrasonography commenced in 1988 when the youngest patients were aged 6 years at which time deaths, emigrations and default had reduced the numbers to 206 HbSS and 89 controls. It continued annually until 2000.

RESULTS: The spleen was visualized in all HbAA controls but in only 1103/2138 (52%) scans in HbSS. Where available, mean splenic lengths were significantly lower in HbSS (77-103 mm in males, 70-83 mm in females) compared to normal controls (89-101 mm in males, 86-95 mm in females). Assessed by statistical modelling after adjusting for body height, the splenic ratio (splenic length/body height) declined over the age range 12-20 years in HbSS, consistent with progressive splenic fibrosis. Genetic factors known to inhibit sickling, α thalassaemia and fetal haemoglobin level (HbF) significantly reduced the decline in splenic ratio. Clinical splenomegaly was an insensitive measure of splenic enlargement as only 50% of patients aged 18 years and above with spleens measuring ≥150 mm on ultrasonography had palpable spleens.

CONCLUSIONS: An age-related decline in splenic length occurred in HbSS and occurred more slowly with genetic factors known to inhibit sickling. The standards provided may be of value in assessing minor degrees of subclinical acute splenic sequestration. (241 words).

ADVANCES IN KNOWLEDGE: These are the first standards available for splenic length in HbSS. They may be useful in detecting red cell sequestration, not apparent from clinical splenomegaly and also provide a model for identifying factors inhibiting vaso-occlusion.

PMID:36259419 | DOI:10.1259/bjr.20220634

Vox Sang. 2022 Oct 19. doi: 10.1111/vox.13370. Online ahead of print.

ABSTRACT

BACKGROUND AND OBJECTIVES: Donor criteria for diabetes vary significantly. We describe our evolving policies for donors with diabetes, their contribution to the Canadian blood supply and their rate of syncopal reactions compared to other donors.

MATERIALS AND METHODS: All donors are asked if they have diabetes and have taken medications in the last 3 days. We assessed donors with diabetes on various medications, the number deferred over time, and syncopal reactions in donors with diabetes and other donors in our donor reaction database.

RESULTS: Policy changes allowing type 2 diabetic donors on oral hypoglycaemics alone, type 2 diabetic donors on oral medications and insulin and type 1 diabetic donors (all on insulin) to donate resulted in a decrease in deferrals from 450 to 22 donors annually. Of donors being treated with medication for diabetes, 11% are receiving insulin as part of their treatment. Syncopal reaction rates were low and not statistically different between diabetic and non-diabetic donors, although confidence intervals (CIs) are large.

CONCLUSION: Policies decreased deferrals while maintaining safety. A longer observation period would strengthen these observations.

PMID:36259374 | DOI:10.1111/vox.13370

Forum Health Econ Policy. 2022 Oct 19. doi: 10.1515/fhep-2021-0035. Online ahead of print.

ABSTRACT

This study aims to investigate the association between gross domestic product (GDP), mortality rate (MR) and current healthcare expenditure (CHE) in 31 high-income countries. We used panel data from 2000 to 2017 collected from WHO and OECD databases. The association between CHE, GDP and MR was investigated through a random-effects model. To control for reverse causality, we adopted a test of Granger causality. The model shows that the MR has a statistically significant and negative effect on CHE and that an increase in GDP is associated with an increase of CHE (p < 0.001). The Granger causality analysis shows that all the variables exhibit a bidirectional causality. We found a two-way relationship between GDP and CHE. Our analysis highlights the economic multiplier effect of CHE. In the debate on the optimal allocation of resources, this evidence should be taken into due consideration.

PMID:36259392 | DOI:10.1515/fhep-2021-0035

Med Decis Making. 2022 Oct 19:272989X221132257. doi: 10.1177/0272989X221132257. Online ahead of print.

ABSTRACT

OBJECTIVES: Immuno-oncology (IO) therapies are often associated with delayed responses that are deep and durable, manifesting as long-term survival benefits in patients with metastatic cancer. Complex hazard functions arising from IO treatments may limit the accuracy of extrapolations from standard parametric models (SPMs). We evaluated the ability of flexible parametric models (FPMs) to improve survival extrapolations using data from 2 trials involving patients with non-small-cell lung cancer (NSCLC).

METHODS: Our analyses used consecutive database locks (DBLs) at 2-, 3-, and 5-y minimum follow-up from trials evaluating nivolumab versus docetaxel in patients with pretreated metastatic squamous (CheckMate-017) and nonsquamous (CheckMate-057) NSCLC. For each DBL, SPMs, as well as 3 FPMs-landmark response models (LRMs), mixture cure models (MCMs), and Bayesian multiparameter evidence synthesis (B-MPES)-were estimated on nivolumab overall survival (OS). The performance of each parametric model was assessed by comparing milestone restricted mean survival times (RMSTs) and survival probabilities with results obtained from externally validated SPMs.

RESULTS: For the 2- and 3-y DBLs of both trials, all models tended to underestimate 5-y OS. Predictions from nonvalidated SPMs fitted to the 2-y DBLs were highly unreliable, whereas extrapolations from FPMs were much more consistent between models fitted to successive DBLs. For CheckMate-017, in which an apparent survival plateau emerges in the 3-y DBL, MCMs fitted to this DBL estimated 5-y OS most accurately (11.6% v. 12.3% observed), and long-term predictions were similar to those from the 5-y validated SPM (20-y RMST: 30.2 v. 30.5 mo). For CheckMate-057, where there is no clear evidence of a survival plateau in the early DBLs, only B-MPES was able to accurately predict 5-y OS (14.1% v. 14.0% observed [3-y DBL]).

CONCLUSIONS: We demonstrate that the use of FPMs for modeling OS in NSCLC patients from early follow-up data can yield accurate estimates for RMST observed with longer follow-up and provide similar long-term extrapolations to externally validated SPMs based on later data cuts. B-MPES generated reasonable predictions even when fitted to the 2-y DBLs of the studies, whereas MCMs were more reliant on longer-term data to estimate a plateau and therefore performed better from 3 y. Generally, LRM extrapolations were less reliable than those from alternative FPMs and validated SPMs but remained superior to nonvalidated SPMs. Our work demonstrates the potential benefits of using advanced parametric models that incorporate external data sources, such as B-MPES and MCMs, to allow for accurate evaluation of treatment clinical and cost-effectiveness from trial data with limited follow-up.

HIGHLIGHTS: Flexible advanced parametric modeling methods can provide improved survival extrapolations for immuno-oncology cost-effectiveness in health technology assessments from early clinical trial data that better anticipate extended follow-up.Advantages include leveraging additional observable trial data, the systematic integration of external data, and more detailed modeling of underlying processes.Bayesian multiparameter evidence synthesis performed particularly well, with well-matched external data.Mixture cure models also performed well but may require relatively longer follow-up to identify an emergent plateau, depending on the specific setting.Landmark response models offered marginal benefits in this scenario and may require greater numbers in each response group and/or increased follow-up to support improved extrapolation within each subgroup.

PMID:36259353 | DOI:10.1177/0272989X221132257

J Magn Reson Imaging. 2022 Oct 19. doi: 10.1002/jmri.28492. Online ahead of print.

ABSTRACT

BACKGROUND: Radiomics-based analyses have demonstrated impact on studies of endometrial cancer (EC). However, there have been no radiomics studies investigating preoperative assessment of MRI-invisible EC to date.

PURPOSE: To develop and validate radiomics models based on sagittal T2-weighted images (T2WI) and T1-weighted contrast-enhanced images (T1CE) for the preoperative assessment of MRI-invisible early-stage EC and myometrial invasion (MI).

STUDY TYPE: Retrospective.

POPULATION: One hundred fifty-eight consecutive patients (mean age 50.7 years) with MRI-invisible endometrial lesions were enrolled from June 2016 to March 2022 and randomly divided into the training (n = 110) and validation cohort (n = 48) using a ratio of 7:3.

FIELD STRENGTH/SEQUENCE: 3-T, T2WI, and T1CE sequences, turbo spin echo.

ASSESSMENT: Two radiologists performed image segmentation and extracted features. Endometrial lesions were histopathologically classified as benign, dysplasia, and EC with or without MI. In the training cohort, 28 and 20 radiomics features were selected to build Model 1 and Model 2, respectively, generating rad-score 1 (RS1) and rad-score 2 (RS2) for evaluating MRI-invisible EC and MI.

STATISTICAL TESTS: The least absolute shrinkage and selection operator logistic regression method was used to select radiomics features. Mann-Whitney U tests and Chi-square test were used to analyze continuous and categorical variables. Receiver operating characteristic curve (ROC) and decision curve analysis were used for performance evaluation. The area under the ROC curve (AUC), accuracy, sensitivity, specificity, positive predictive value, and negative predictive value were calculated. A P-value <0.05 was considered statistically significant.

RESULTS: Model 1 had good performance for preoperative detecting of MRI-invisible early-stage EC in the training and validation cohorts (AUC: 0.873 and 0.918). In addition, Model 2 had good performance in assessment of MI of MRI-invisible endometrial lesions in the training and validation cohorts (AUC: 0.854 and 0.834).

DATA CONCLUSION: MRI-based radiomics models may provide good performance for detecting MRI-invisible EC and MI.

EVIDENCE LEVEL: 3 TECHNICAL EFFICACY: Stage 2.

PMID:36259352 | DOI:10.1002/jmri.28492

Physiother Theory Pract. 2022 Oct 19:1-12. doi: 10.1080/09593985.2022.2135979. Online ahead of print.

ABSTRACT

BACKGROUND: Musculoskeletal ultrasound (MSK-US) use for diagnostic purposes is expanding in physical therapy practice. Identifying and describing physical therapy-specific approaches to incorporating MSK-US into the evaluation process is needed. Musculoskeletal ultrasound extends the physical exam to allow clinicians to visualize anatomy and pathophysiology both statically and dynamically. Purpose: To document 1) weekly use of diagnostic MSK-US; and 2) clinical reasoning approach used in challenging patient cases by physical therapists (PTs) registered by Inteleos in musculoskeletal sonography (RMSK-certified).

METHODS: Longitudinal, observational, cohort study using mixed methods for data collection and analysis. All 23 currently RMSK-certified PTs using MSK-US in clinical practice across the United States were contacted, and 16 participated. Data were collected using an online survey created with the Research Electronic Data Capture System. Participants documented MSK-US clinical use and significant cases using weekly, reflective, online journals for three months. Demographic data were summarized using descriptive statistics. Case data were analyzed thematically.

RESULTS: Participating RMSK-certified PTs performed 1110 MSK-US examinations over 110 weeks. Clinicians averaged 7 (range 1-25) MSK-US examinations weekly, representing 28% of an average caseload. Examinations contributed significant anatomical/ pathological information 100% of the time. The most common joints scanned were the knee (n = 281), shoulder (n = 254), and wrist (n = 228). Case data revealed three themes: 1) augmenting the clinical evaluation to extend or narrow a diagnosis; 2) outcomes guiding action; and 3) lessons learned from clinical findings.

CONCLUSION: RMSK-certified PTs regularly used MSK-US to validate and refine their clinical diagnoses and treatment. Ultrasound imaging directly influenced patient care by informing the diagnostic process, guiding treatment, and appropriately identifying referrals.

PMID:36259351 | DOI:10.1080/09593985.2022.2135979

J Magn Reson Imaging. 2022 Oct 19. doi: 10.1002/jmri.28488. Online ahead of print.

ABSTRACT

BACKGROUND: Vessels encapsulating tumor clusters (VETC) pattern is a novel microvascular pattern associated with poor outcomes of hepatocellular carcinoma (HCC). Preoperative estimation of VETC has potential to improve treatment decisions.

PURPOSE: To develop and validate a nomogram based on gadoxetate disodium-enhanced MRI for estimating VETC in HCC and to evaluate whether the estimations are associated with recurrence after hepatic resection.

STUDY TYPE: Retrospective.

POPULATION: A total of 320 patients with HCC and histopathologic VETC pattern assessment from three centers (development cohort:validation cohort = 173:147).

FIELD STRENGTH/SEQUENCE: A3.0 T/turbo spin-echo T2-weighted, spin-echo echo-planar diffusion-weighted, and 3D T1-weighted gradient-echo sequences.

ASSESSMENT: A set of previously reported VETC- and/or prognosis-correlated qualitative and quantitative imaging features were assessed. Clinical and imaging variables were compared based on histopathologic VETC status to investigate factors indicating VETC pattern. A regression-based nomogram was then constructed using the significant factors for VETC pattern. The nomogram-estimated VETC stratification was assessed for its association with recurrence.

STATISTICAL TESTS: Fisher exact test, t-test or Mann-Whitney test, logistic regression analyses, Harrell’s concordance index (C-index), nomogram, Kaplan-Meier curves and log-rank tests. P value < 0.05 was considered statistically significant.

RESULTS: Pathological VETC pattern presence was identified in 156 patients (development cohort:validation cohort = 83:73). Tumor size, presence of heterogeneous enhancement with septations or with irregular ring-like structures, and necrosis were significant factors for estimating VETC pattern. The nomogram incorporating these indicators showed good discrimination with a C-index of 0.870 (development cohort) and 0.862 (validation cohort). Significant differences in recurrence rates between the nomogram-estimated high-risk VETC group and low-risk VETC group were found (2-year recurrence rates, 50.7% vs. 30.3% and 49.6% vs. 31.8% in the development and validation cohorts, respectively).

DATA CONCLUSION: The nomogram integrating gadoxetate disodium-enhanced MRI features was associated with VETC pattern preoperatively and with postoperative recurrence in patients with HCC.

EVIDENCE LEVEL: 4 TECHNICAL EFFICACY: Stage 2.

PMID:36259347 | DOI:10.1002/jmri.28488

Ann Med. 2022 Dec;54(1):2816-2827. doi: 10.1080/07853890.2022.2131326.

ABSTRACT

BACKGROUND/OBJECTIVE(S)/INTRODUCTION: In clinical practice, treatment goals are often set up without exploring what patients really want. We, therefore, collected individualised treatment goals of patients with osteoarthritis (OA), categorised and mapped them to the World Health Organisation International Classification for Functioning, Disability and Health (ICF).

PATIENTS/MATERIALS AND METHODS: A longitudinal prospective cohort study was conducted (2019-2021). We used descriptive statistics and Chi²/Fisher’s Exact Tests, where appropriate, as well as Kruskal-Wallis-Tests for the mean score ranks of the patients’ goals.

RESULTS: In total, 305 goals reported by 132 participants were analysed (267 women vs. 38 men). The top 3 ICF categories were sensation of pain (ICF:b280), mobility of joint (ICF:b710) and muscle power functions (ICF:b730). Overall, 51% of all individually reported functional goals were achieved after 3 months. Men were more likely to achieve their goals than women (p = 0.009). The majority of the “very important” goals (51%) and “very difficult” goals (57%) was not improved. Goals’ mean score ranks significantly differed between baseline and follow-up.

CONCLUSION(S): As the human lifespan as well as the number of people affected by OA worldwide increase, there is a growing need to identify and evaluate rehabilitation outcomes that are relevant to people with OA.Key MessagesTreat-to-target agreements between patients and health care providers present a step towards more personalised precision medicine, which will eventually lead to better reported functional and health outcomes.In patients with osteoarthritis, the Goal Attainment Scale instrument can be used to measure health outcomes at different time points and its content may be linked to ICF providing a unified language and conceptual scientific basis.

PMID:36259346 | DOI:10.1080/07853890.2022.2131326

Epidemiol Prev. 2022 Jul-Aug;46(4):268-272. doi: 10.19191/EP22.4.A372.075.

ABSTRACT

OBJECTIVES: to assess the occurrence of potentially inappropriate prescribing (PIP) in residents of Tuscany nursing homes (NHs) and its variation before and after NH entry.

DESIGN: retrospective observational study using data from the Regional Administrative Database of Tuscany.

SETTING AND PARTICIPANTS: the study involved residents of 67 Tuscan NHs identified between 2011 and 2012. To estimate PIP prevalence before and after NH, a subset of 10 indicators of the Screening Tool of Older Person’s Prescriptions (STOPP) criteria were selected.

MAIN OUTCOME MEASURES: prevalence of PIP. RESULTS: considering 2,801 NH residents, the proportion of PIP ranged from 0.0% to 55.2% and from 0.0% to 33.9% before and after the NH admission, respectively. Overall, this study showed a decrease in the occurrence of PIP after the NH admission for most of the indicators, reaching statistical significance for indicator 3 (tricyclic antidepressants in combination with an opiate or calcium channel blockers), 7 (prescription of NSAIDs in heart failure patients), and 9 (warfarin in combination with NSAIDs).

CONCLUSIONS: although the reduction of PIP after NH admission may suggest greater awareness about the appropriateness of drug use, more efforts still need to be made.

PMID:36259343 | DOI:10.19191/EP22.4.A372.075

Epidemiol Prev. 2022 Jul-Aug;46(4):259-267. doi: 10.19191/EP22.4.A372.074.

ABSTRACT

BACKGROUND: immigrant women diagnosed with gestational diabetes mellitus (GDM) have additional language and cultural obstacles in following lifestyle and dietary recommendations within a Western health care setting.

OBJECTIVES: to describe: • sociodemographic characteristics and dietary and lifestyle behaviours in Italian and immigrant pregnant women who underwent a GDM screening; • any differences in these aspects among GDM Italian and immigrant women; • any differences in terms of primary maternal-neonatal outcomes among GDM and normal glucose tolerance (NGT) cohorts.

DESIGN: survey.

SETTING AND PARTICIPANTS: this survey was conducted in three hospitals located in Tuscany Region (Central Italy). According to a convenience sampling, an ad hoc questionnaire was administered both to Italian and immigrant women who underwent an oral glucose tolerance test.

MAIN OUTCOME MEASURES: information about nutrition and lifestyle, medical and obstetric history, clinical and therapy data, maternal and neonatal primary outcomes was analysed. Eating habits have been investigated referring to the prudent diet, in order to also include those foods specifically consumed by the immigrant women, in addition to the Mediterranean Diet which is more widespread among Italians.

RESULTS: a total of 117 Italian women (42 diagnosed with GDM) and 95 immigrant women (36 with GDM) were enrolled. Immigrant women showed lower adherence to the prudent diet compared to Italian women (p<0.05) and a broader use of unhealthy food preparation (e.g., frying; p<0.05). Primary maternal and neonatal outcomes (preterm birth, caesarean section, macrosomia) showed no statistical differences among GDM and NGT cohorts.

CONCLUSIONS: even if immigrant and Italian women gained similar pregnancy outcomes, immigrant women showed lower adherence to the prudent diet at the time of GDM screening. An ethnic and tailored meal plan is needed to overcome cultural barriers in dietary recommendations during pregnancy in immigrant women.

PMID:36259342 | DOI:10.19191/EP22.4.A372.074