Category: Statistics

Trials. 2023 Jul 27;24(1):479. doi: 10.1186/s13063-023-07525-4.

NO ABSTRACT

PMID:37501161 | DOI:10.1186/s13063-023-07525-4

BMC Med. 2023 Jul 27;21(1):276. doi: 10.1186/s12916-023-02969-6.

ABSTRACT

BACKGROUND: The estimand for a clinical trial is a precise definition of the treatment effect to be estimated. Traditionally, estimates of treatment effects are based on either an ITT analysis or a per-protocol analysis. However, there are important clinical questions which are not addressed by either of these analyses. For example, consider a trial where patients take a rescue medication. The ITT analysis includes data after use of rescue, while the per-protocol analysis excludes these patients altogether. Neither of these analyses addresses the important question of what the treatment effect would have been if patients did not take rescue medication.

MAIN TEXT: Trial estimands provide a broader perspective compared to the limitations of ITT and per-protocol analysis. Trial treatment effects depend on how events occurring after treatment initiation such as use of alternative medication or discontinuation of the intervention are included in the definition. These events can be accounted for in different ways, depending on the clinical question of interest.

CONCLUSION: The estimand framework is an important step forward in improving the clarity and transparency of clinical trials. The centrality of estimands to clinical trials is currently not reflected in methods recommended by the Cochrane group or the CONSORT statement, the current standard for reporting clinical trials in medical journals. We encourage revisions to these guidelines.

PMID:37501156 | DOI:10.1186/s12916-023-02969-6

Orphanet J Rare Dis. 2023 Jul 27;18(1):217. doi: 10.1186/s13023-023-02826-y.

ABSTRACT

INTRODUCTION: SAPHO syndrome is a group of special syndromes characterized by synovitis, acne, pustulosis, hyperostosis and osteitis. Skin lesions and joint damage are the main clinical manifestations. Among them, females mostly present with palm toe pustulosis, while males have severe acne as the main external manifestation. The bone and joint damage characterized by bone hypertrophy and osteitis is the core manifestation of SAPHO and affects all parts of the body. SAPHO syndrome causes great physical and mental suffering to patients, and it also brings a huge financial burden to the family. The purpose of this study is to explore the impact of SAPHO on the quality of sexual life of patients.

METHODS: We screened and included 249 SAPHO patients (169 women and 80 men) from Peking Union Medical College Hospital (Beijing, China). First, we recorded the basic situation of the patient through questionnaires (including gender, age, SAPHO duration, BMI, smoking, drinking, marital status, educational level, occupational status and work status.). Then, the patient needed to fill in the Short Form-36 quality of life questionnaire (SF-36 QoL) to record the quality of life. For Sexual dysfunction (SD), female patients needed to fill in the Female Sexual Function Index (FSFI) to assess the quality of sexual life; while the International Index of Erectile Function (IIEF) was used to assess the SD of male patients. At the same time, we used self-esteem and relationship questionnaire (SEAR) to analyze the psychological state of SAPHO patients. Finally, we performed statistical analysis on the data obtained, and then explored the connection between SAPHO and SD.

RESULTS: In this cross-sectional study, a total of 249 patients completed the questionnaire and constituted the study population. We found that among 169 female patients, 124 patients had FSD (73.4%); while 45 patients did not have FSD (26.6%); and among 80 male patients, 45 (56.3%) had ED; However, 35 patients did not have ED (43.7%). The results of the quality of life and mental state assessment showed that female patients with SD showed lower scores in terms of mental state. Among all male participants, we found no significant difference in quality of life and mental state among participants with or without SD. In addition, there was no significant difference in the duration of SAPHO between female and male participants with or without SD.

CONCLUSION: This study is the first to evaluate the SD of SAPHO patients. The incidence of SD in female SAPHO patients is higher than that in male patients; the cause of female SD may be mainly psychological factors. These results prove that it is particularly important to focus on regulating their psychological state while diagnosing and treating SAPHO patients in clinical practice.

PMID:37501151 | DOI:10.1186/s13023-023-02826-y

J Ovarian Res. 2023 Jul 27;16(1):148. doi: 10.1186/s13048-023-01226-x.

ABSTRACT

BACKGROUND: Over the past two decades, increasing number of people with cystic fibrosis (CF) survive into adulthood. Compared to the general population, sub-fertility is an obstacle for many women with CF (wwCF). Decreased ovarian reserve has been proposed as a possible cause, but limited data is available to support this. The aim of this study was to evaluate the ovarian reserve in wwCF and to correlate this with patients’ demographic and clinical data.

METHODS: Reproductive-aged wwCF were enrolled during their routine medical appointments. Assessment included Anti-Mullerian hormone (AMH) levels, routine blood tests and antral follicular count (AFC) evaluation. Additionally, demographic, and clinical information were collected.

RESULTS: A total of wenty-three wwCF were enrolled, with ages ranging from 19 to 40 years (median 27 years). Among the fourteen wwCF who were considering pregnancy, five (35.7%) disclosed undergoing an infertility assessment and receiving fertility treatments. All but one patient had an Anti-Mullerian hormone (AMH) level between the 5th and 95th % for age. Measurement of the antral follicular count (AFC) was possible in 12 of the 23 patients and was ranging 8-40 with a median of 17. The proportion of wwCF presenting below median AMH values was not different in sub-fertile as compared to fertile wwCF (P value 0.54). There were no correlations between AMH levels and disease severity parameters. AMH seemed to be relatively higher in wwCF with mild class mutations, but this was not shown to have statistical significance.

CONCLUSIONS: Our results, in contrast with the limited available published data, do not support the hypothesis that decreased ovarian reserve plays a major role in infertility in wwCF.

PMID:37501150 | DOI:10.1186/s13048-023-01226-x

BMC Med Educ. 2023 Jul 27;23(1):536. doi: 10.1186/s12909-023-04470-3.

ABSTRACT

BACKGROUND: Arts-based pedagogical tools have been increasingly incorporated into medical education. Visual Thinking Strategies (VTS) is a research-based, constructivist teaching methodology that aims to improve visual literacy, critical thinking, and communication skills through the process of investigating works of art. Harvard Medical School pioneered the application of VTS within medical education in 2004. While there are several studies investigating the use of VTS, there is a need to systematically assess the different programs that exist for medical education and their efficacy in improving relevant clinical skills. This systematic review aims to critically analyse the available evidence of the effectiveness of VTS in medical education to guide future research and provide a framework to adapt medical curricula.

METHODS: A systematic search of PubMed, PsycINFO, and Cochrane CENTRAL databases (through November 2022) was conducted to identify studies of VTS-based interventions in undergraduate and postgraduate medical education. Two reviewers independently screened citations for inclusion criteria, extracted data, and assessed risk of bias. The extracted data was then narratively synthesized.

RESULTS: Of 5759 unique citations, 10 studies met the inclusion criteria. After reference review, one additional study was included. Therefore, 11 studies were included in our review. Of these, eight reported VTS-based interventions for undergraduate medical students and three reported interventions in residency training, specifically in dermatology and ophthalmology. The main goal of most studies was to increase observational or visual diagnostic skills. Three of the studies in undergraduate medical education and two in postgraduate achieved a statistically significant improvement in observational skills in post-course evaluations. Some studies reported increased tolerance for ambiguity and empathy.

CONCLUSIONS: Although the studies varied considerably in study design, learning objectives, and outcomes, findings consistently indicate that the VTS approach can serve as a vehicle to develop crucial clinical competencies, encouraging more in-depth visual analysis that could be applied when observing a patient. Despite some limitations of the included studies (lack of control groups, self-selection bias, or non-standard outcome measures), the results of this review provide support for greater inclusion of VTS training in the medical curriculum.

PMID:37501147 | DOI:10.1186/s12909-023-04470-3

Chin Med. 2023 Jul 28;18(1):89. doi: 10.1186/s13020-023-00782-0.

ABSTRACT

BACKGROUND: Evaluating traditional Chinese medicine (TCM) quality is a powerful method to ensure TCM safety. TCM quality evaluation methods primarily include characterization evaluations and separate physical, chemical, and biological evaluations; however, these approaches have limitations. Nevertheless, researchers have recently integrated evaluation methods, advancing the emergence of frontier research tools, such as TCM quality markers (Q-markers). These studies are largely based on biological activity, with weak correlations between the quality indices and quality. However, these TCM quality indices focus on the individual efficacies of single bioactive components and, therefore, do not accurately represent the TCM quality. Conventionally, provenance, place of origin, preparation, and processing are the key attributes influencing TCM quality. In this study, we identified TCM-attribute-based quality indices and developed a comprehensive multiweighted multi-index-based TCM quality composite evaluation index (QCEI) for grading TCM quality.

METHODS: The area of origin, number of growth years, and harvest season are considered key TCM quality attributes. In this study, licorice was the model TCM to investigate the quality indicators associated with key factors that are considered to influence TCM quality using multivariate statistical analysis, identify biological-evaluation-based pharmacological activity indicators by network pharmacology, establish real quality indicators, and develop a QCEI-based model for grading TCM quality using a machine learning model. Finally, to determine whether different licorice quality grades differently reduced the inflammatory response, TNF-α and IL-1β levels were measured in RAW 264.7 cells using ELISA analysis.

RESULTS: The 21 quality indices are suitable candidates for establishing a method for grading licorice quality. A computer model was established using SVM analysis to predict the TCM quality composite evaluation index (TCM QCEI). The tenfold cross validation accuracy was 90.26%. Licorice diameter; total flavonoid content; similarities of HPLC chromatogram fingerprints recorded at 250 and 330 nm; contents of liquiritin apioside, liquiritin, glycyrrhizic acid, and liquiritigenin; and pharmacological activity quality index were identified as the key indices for constructing the model for evaluating licorice quality and determining which model contribution rates were proportionally weighted in the model. The ELISA analysis results preliminarily suggest that the inflammatory responses were likely better reduced by premium-grade than by first-class licorice.

CONCLUSIONS: In the present study, traditional sensory characterization and modern standardized processes based on production process and pharmacological efficacy evaluation were integrated for use in the assessment of TCM quality. Multidimensional quality evaluation indices were integrated with a machine learning model to identify key quality indices and their corresponding weight coefficients, to establish a multiweighted multi-index and comprehensive quality index, and to construct a QCEI-based model for grading TCM quality. Our results could facilitate and guide the development of TCM quality control research.

PMID:37501143 | DOI:10.1186/s13020-023-00782-0

Malar J. 2023 Jul 27;22(1):218. doi: 10.1186/s12936-023-04649-5.

ABSTRACT

BACKGROUND: Malaria, transmitted by the bite of infective female Anopheles mosquitoes, remains a global public health problem. The presence of invasive Anopheles stephensi, capable of transmitting Plasmodium vivax and Plasmodium falciparum, was first reported in Ethiopia in 2016. The ecology of this mosquito species differs from that of Anopheles arabiensis, the primary malaria vector in Ethiopia. This study aimed to evaluate the efficacy of selected insecticides, which are used in indoor residual spraying (IRS) and selected long-lasting insecticidal nets (LLINs) for malaria vector control against adult An. stephensi.

METHODS: Anopheles stephensi mosquitoes were collected as larvae and pupae from Awash Subah Kilo Town and Haro Adi village, Ethiopia. Adult female An. stephensi, reared from larvae and pupae collected from the field, aged 3-5 days were exposed to impregnated papers of IRS insecticides (propoxur 0.1%, bendiocarb 0.1%, pirimiphos-methyl 0.25%), and insecticides used in LLINs (alpha-cypermethrin 0.05%, deltamethrin 0.05% and permethrin 0.75%), using diagnostic doses and WHO test tubes in a bio-secure insectary at Aklilu Lemma Institute of Pathobiology, Addis Ababa University. For each test and control tube, batches of 25 female An. stephensi were used to test each insecticide used in IRS. Additionally, cone bioassay tests were conducted to expose An. stephensi from the reared population to four brands of LLINs, MAGNet™ (alpha-cypermethrin), PermaNet^® 2.0 (deltamethrin), DuraNet^© (alpha-cypermethrin) and SafeNet^® (alpha-cypermethrin). A batch of ten sugar-fed female mosquitoes aged 2-5 days was exposed to samples taken from five positions/sides of a net. The data from all replicates were pooled and descriptive statistics were used to describe features of the data.

RESULTS: All An. stephensi collected from Awash Subah Kilo Town and Haro Adi village (around Metehara) were resistant to all tested insecticides used in both IRS and LLINs. Of the tested LLINs, only MAGNet™ (alpha-cypermethrin active ingredient) caused 100% knockdown and mortality to An. stephensi at 60 min and 24 h post exposure, while all other net brands caused mortality below the WHO cut-off points (< 90%). All these nets, except SafeNet^®, were collected during LLIN distribution for community members through the National Malaria Programme, in December 2020.

CONCLUSIONS: Anopheles stephensi is resistant to all tested insecticides used in IRS and in the tested LLIN brands did not cause mosquito mortality as expected, except MAGNet. This suggests that control of this invasive vector using existing adult malaria vector control methods will likely be inadequate and that alternative strategies may be necessary.

PMID:37501142 | DOI:10.1186/s12936-023-04649-5

BMC Public Health. 2023 Jul 27;23(1):1439. doi: 10.1186/s12889-023-16282-7.

ABSTRACT

BACKGROUND: Although evidence on healthcare utilization avoidance during COVID-19 pandemic is emerging, such knowledge is limited in rural settings. An effective policy to the COVID-19 shocks and stresses in rural settings require empirical evidence to inform the design of health policies and programmes. To help overcome this evidence gap and also contribute to policy decisions, this study aimed at examining COVID-19-induced healthcare utilization avoidance and associated factors in rural India.

METHODS: This study used the third-round data from the COVID-19-Related Shocks in Rural India survey conducted between 20-24 September, 2020 across six states. The outcome variable considered in this study was COVID-19-induced healthcare utilization avoidance. Multivariable Binary Logistic Regression Model via Multiple Imputation was used to assess the factors influencing COVID-19-induced healthcare utilization avoidance.

RESULTS: Data on 4,682 respondents were used in the study. Of this, the prevalence of COVID-19-induced healthcare utilization avoidance was 15.5% in rural India across the six states. After adjusting for relevant covariates, participants from the Bihar State have significantly higher likelihood of COVID-19-induced healthcare utilization avoidance compared to those from the Andhra Pradesh. Also, participants whose educational level exceeds high school, those who use government hospital/clinic, engage in daily wage labour in agriculture have significantly higher odds of COVID-19-induced healthcare utilization avoidance compared to their counterparts.

CONCLUSION: Our study revealed that state of residence, type of health facility used, primary work activity and educational level were associated with COVID-19-induced healthcare utilization avoidance in rural India. The findings suggest that policy makers and public health authorities need to formulate policies and design interventions that acknowledge socioeconomic and demographic factors that influence healthcare use avoidance.

PMID:37501140 | DOI:10.1186/s12889-023-16282-7

BMC Med. 2023 Jul 27;21(1):274. doi: 10.1186/s12916-023-02965-w.

ABSTRACT

BACKGROUND: Persistent infection by oncogenic human papillomavirus (HPV) is necessary although not sufficient for development of cervical cancer. Behavioural, environmental, or comorbid exposures may promote or protect against malignant transformation. Randomised evidence is limited and the validity of observational studies describing these associations remains unclear.

METHODS: In this umbrella review, we searched electronic databases to identify meta-analyses of observational studies that evaluated risk or protective factors and the incidence of HPV infection, cervical intra-epithelial neoplasia (CIN), cervical cancer incidence and mortality. Following re-analysis, evidence was classified and graded based on a pre-defined set of statistical criteria. Quality was assessed with AMSTAR-2. For all associations graded as weak evidence or above, with available genetic instruments, we also performed Mendelian randomisation to examine the potential causal effect of modifiable exposures with risk of cervical cancer. The protocol for this study was registered on PROSPERO (CRD42020189995).

RESULTS: We included 171 meta-analyses of different exposure contrasts from 50 studies. Systemic immunosuppression including HIV infection (RR = 2.20 (95% CI = 1.89-2.54)) and immunosuppressive medications for inflammatory bowel disease (RR = 1.33 (95% CI = 1.27-1.39)), as well as an altered vaginal microbiome (RR = 1.59 (95% CI = 1.40-1.81)), were supported by strong and highly suggestive evidence for an association with HPV persistence, CIN or cervical cancer. Smoking, number of sexual partners and young age at first pregnancy were supported by highly suggestive evidence and confirmed by Mendelian randomisation.

CONCLUSIONS: Our main analysis supported the association of systemic (HIV infection, immunosuppressive medications) and local immunosuppression (altered vaginal microbiota) with increased risk for worse HPV and cervical disease outcomes. Mendelian randomisation confirmed the link for genetically predicted lifetime smoking index, and young age at first pregnancy with cervical cancer, highlighting also that observational evidence can hide different inherent biases. This evidence strengthens the need for more frequent HPV screening in people with immunosuppression, further investigation of the vaginal microbiome and access to sexual health services.

PMID:37501128 | DOI:10.1186/s12916-023-02965-w

BMC Geriatr. 2023 Jul 27;23(1):459. doi: 10.1186/s12877-023-04038-2.

ABSTRACT

BACKGROUND: Hip fractures are devastating injuries causing disability, dependence, and institutionalisation, yet hospital care is highly variable. This study aimed to determine hospital organisational factors associated with recovery of mobility and change in patient residence after hip fracture.

METHODS: A cohort of patients aged 60 + years in England and Wales, who sustained a hip fracture from 2016 to 2019 was examined. Patient-level Hospital Episodes Statistics, National Hip Fracture Database, and mortality records were linked to 101 factors derived from 18 hospital-level organisational metrics. After adjustment for patient case-mix, multilevel models were used to identify organisational factors associated with patient residence at discharge, and mobility and residence at 120 days after hip fracture.

RESULTS: Across 172 hospitals, 165,350 patients survived to discharge, of whom 163,230 (99%) had post-hospital discharge destination recorded. 18,323 (11%) died within 120 days. Among 147,027 survivors, 58,344 (40%) across 143 hospitals had their residence recorded, and 56,959 (39%) across 140 hospitals had their mobility recorded, at 120 days. Nineteen organisational factors independently predicted residence on hospital discharge e.g., return to original residence was 31% (95% confidence interval, CI:17-43%) more likely if the anaesthetic lead for hip fracture had time allocated in their job plan, and 8-13% more likely if hip fracture service clinical governance meetings were attended by an orthopaedic surgeon, physiotherapist or anaesthetist. Seven organisational factors independently predicted residence at 120 days. Patients returning to their pre-fracture residence was 26% (95%CI:4-42%) more likely if hospitals had a dedicated hip fracture ward, and 20% (95%CI:8-30%) more likely if treatment plans were proactively discussed with patients and families on admission. Seventeen organisational factors predicted mobility at 120 days. More patients re-attained their pre-fracture mobility in hospitals where (i) care involved an orthogeriatrician (15% [95%CI:1-28%] improvement), (ii) general anaesthesia was usually accompanied by a nerve block (7% [95%CI:1-12%], and (iii) bedside haemoglobin testing was routine in theatre recovery (13% [95%CI:6-20%]).

CONCLUSIONS: Multiple, potentially modifiable, organisational factors are associated with patient outcomes up to 120 days after a hip fracture, these factors if causal should be targeted by service improvement initiatives to reduce variability, improve hospital hip fracture care, and maximise patient independence.

PMID:37501122 | DOI:10.1186/s12877-023-04038-2