Category: Statistics

BMJ Open. 2021 Apr 7;11(4):e044854. doi: 10.1136/bmjopen-2020-044854.

ABSTRACT

OBJECTIVE: Survival gains in teenagers and young adults (TYA) are reported to be lower than children and adults for some cancers. Place of care is implicated, influencing access to specialist TYA professionals and research.Consequently, age-appropriate specialist cancer care is advocated for TYA although systematic investigation of associated outcomes is lacking. In England, age-appropriate care is delivered through 13 Principal Treatment Centres (TYA-PTC). BRIGHTLIGHT is the national evaluation of TYA cancer services to examine outcomes associated with differing places and levels of care. We aimed to examine the association between exposure to TYA-PTC care, survival and documentation of clinical processes of care.

DESIGN: Prospective cohort study.

SETTING: 109 National Health Service (NHS) hospitals across England.

PARTICIPANTS: 1114 TYA, aged 13-24, newly diagnosed with cancer between 2012 and 2014.

INTERVENTION: Participants were assigned a TYA-PTC category dependent on the proportion of care delivered in a TYA-PTC in the first year after diagnosis: all care in a TYA-PTC (ALL-TYA-PTC, n=270), no care in a TYA-PTC (NO-TYA-PTC, n=359), and some care in a TYA-PTC with additional care in a children’s/adult unit (SOME-TYA-PTC, n=419).

PRIMARY OUTCOME: Data were collected on documented processes indicative of age-appropriate care using clinical report forms, and survival through linkage to NHS databases.

RESULTS: TYA receiving NO-TYA-PTC care were less likely to have documentation of molecular diagnosis, be reviewed by a children’s or TYA multidisciplinary team, be assessed by supportive care services or have a fertility discussion. There was no significant difference in survival according to category of care. There was weak evidence that the association between care category and survival differed by age (p=0.08) with higher HRs for those over 19 receiving ALL or SOME-TYA-PTC compared with NO-TYA-PTC.

CONCLUSION: TYA-PTC care was associated with better documentation of clinical processes associated with age-appropriate care but not improved survival.

PMID:33827838 | DOI:10.1136/bmjopen-2020-044854

BMJ Open. 2021 Apr 7;11(4):e044669. doi: 10.1136/bmjopen-2020-044669.

ABSTRACT

INTRODUCTION: Intervention in the preschool period is currently recommended for autism spectrum disorder. Therapies delivered by parents are particularly suitable for young children. Preschool Autism Communication Trial (PACT) is a parent-mediated therapy that has shown a significant and sustained impact on autism symptom reduction. However, access to such evidence-based therapies for families is limited due to autism centres located in large urban areas. Using videoconferencing to deliver PACT training to parents may improve accessibility for families living in underserved areas.

METHODS AND ANALYSIS: This single-blind randomised controlled trial, involving six sites in France, will investigate the efficacy of a telehealth, videoconferencing-based, parent-mediated PACT therapy on autism symptoms, over a 12-month period. It will compare PACT plus treatment as usual (TAU) against TAU only in a cohort of 238 toddlers (119 per group) aged 18-36 months at inclusion and living with their families more than 40 min away from the specialist centres for autism. Primary outcome will include change of overall autism score on the Autism Diagnostic Observation Scale (ADOS) at 12 months. Secondary outcomes will measure change in child skills, child functioning, impact on parents (stress, health, priorities) and implementation characteristics. Repeated measures analyses will be used to test the effect of PACT intervention on the overall ADOS module 1 score over the 12-month study period. Linear mixed models will be used with time, treatment allocation and the interaction between treatment and time as fixed effects and individual variation as random effect.

ETHICS AND DISSEMINATION: This protocol (V.5, date: 25 October 2019) is approved by the French National Review Board (reference no 2018-A02516-49). The results will be disseminated via peer-reviewed journals TRIAL REGISTRATION NUMBER: NCT04244721.

PMID:33827837 | DOI:10.1136/bmjopen-2020-044669

BMJ Open. 2021 Apr 7;11(4):e041742. doi: 10.1136/bmjopen-2020-041742.

ABSTRACT

INTRODUCTION: This double-blind, randomised controlled trial (RCT) aims to estimate the effect of a physiotherapist-led intervention with targeted strengthening compared with a physiotherapist-led intervention with standardised stretching, on hip-related quality of life (QOL) or perceived improvement at 6 months in people with femoroacetabular impingement (FAI) syndrome. We hypothesise that at 6 months, targeted strengthening physiotherapist-led treatment will be associated with greater improvements in hip-related QOL or greater patient-perceived global improvement when compared with standardised stretching physiotherapist-led treatment.

METHODS AND ANALYSIS: We will recruit 164 participants with FAI syndrome who will be randomised into one of the two intervention groups, both receiving one-on-one treatment with the physiotherapist over 6 months. The targeted strengthening physiotherapist-led treatment group will receive a personalised exercise therapy and education programme. The standardised stretching physiotherapist-led treatment group will receive standardised stretching and personalised education programme. Primary outcomes are change in hip-related QOL using International Hip Outcome Tool-33 and patient-perceived global improvement. Secondary outcomes include cost-effectiveness, muscle strength, range of motion, functional task performance, biomechanics, hip cartilage structure and physical activity levels. Statistical analyses will make comparisons between both treatment groups by intention to treat, with all randomised participants included in analyses, regardless of protocol adherence. Linear mixed models (with baseline value as a covariate and treatment condition as a fixed factor) will be used to evaluate the treatment effect and 95% CI at primary end-point (6 months).

ETHICS AND DISSEMINATION: The study protocol was approved (La Trobe University Human Ethics Committee (HEC17-080)) and prospectively registered with the Australian New Zealand Clinical Trials Registry. The findings of this RCT will be disseminated through peer reviewed scientific journals and conferences. Patients were involved in study development and will receive a short summary following the completion of the RCT.

TRIAL REGISTRATION NUMBER: ACTRN12617001350314.

PMID:33827828 | DOI:10.1136/bmjopen-2020-041742

BMJ Open. 2021 Apr 7;11(4):e041917. doi: 10.1136/bmjopen-2020-041917.

ABSTRACT

OBJECTIVE: The purpose of this study was to assess the trends in outcomes of out-of-hospital cardiac arrest (OHCA) in Beijing over 5 years.

DESIGN: Cross-sectional study.

METHODS: Adult patients with OHCA of all aetiologies who were treated by the Beijing emergency medical service (EMS) between January 2013 and December 2017 were analysed. Data were collected using the Utstein Style. Cases were followed up for 1 year. Descriptive statistics were used to characterise the sample and logistic regression was performed.

RESULTS: Overall, 5016 patients with OHCA underwent attempted resuscitation by the EMS in urban areas of Beijing during the study period. Survival to hospital discharge was 1.2% in 2013 and 1.6% in 2017 (adjusted rate ratio=1.0, p for trend=0.60). Survival to admission and neurological outcome at discharge did not significantly improve from 2013 to 2017. Patient characteristics and the aetiology and location of cardiac arrest were consistent, but there was a decrease in the initial shockable rhythm (from 6.5% to 5.6%) over the 5 years. The rate of bystander cardiopulmonary resuscitation (CPR) increased steadily over the years (from 10.4% to 19.4%).

CONCLUSION: Survival after OHCA in urban areas of Beijing did not improve significantly over 5 years, with long-term survival being unchanged, although the rate of bystander CPR increased steadily, which enhanced the outcomes of patients who underwent bystander CPR.

PMID:33827829 | DOI:10.1136/bmjopen-2020-041917

BMJ Open. 2021 Apr 7;11(4):e043364. doi: 10.1136/bmjopen-2020-043364.

ABSTRACT

OBJECTIVE: To establish the acceptability and feasibility of delivering the Active Communication Education (ACE) programme to increase quality of life through improving communication and hearing aid use in the UK National Health Service.

DESIGN: Randomised controlled, open feasibility trial with embedded economic and process evaluations.

SETTING: Audiology departments in two hospitals in two UK cities.

PARTICIPANTS: Twelve hearing aid users aged 18 years or over who reported moderate or less than moderate benefit from their new hearing aid.

INTERVENTIONS: Consenting participants (along with a significant other) were to be randomised by a remote, centralised randomisation service in groups to ACE plus treatment-as-usual (intervention group) or treatment-as-usual only (control group).

PRIMARY OUTCOME MEASURES: The primary outcomes were related to feasibility: recruitment, retention, treatment adherence and acceptability to participants and fidelity of treatment delivery.

SECONDARY OUTCOME MEASURES: International Outcomes Inventory for Hearing Aids, Self-Assessment of Communication, EQ-5D-5L and Short-Form 36. Blinding of the participants and facilitator was not possible.

RESULTS: Twelve hearing aid users and six significant others consented to take part. Eight hearing aid users were randomised: four to the intervention group; and four to treatment-as-usual only. Four significant others participated alongside the randomised participants. Recruitment to the study was very low and centres only screened 466 hearing aid users over the 15-month recruitment period, compared with the approximately 3500 anticipated. Only one ACE group and one control group were formed. ACE could be delivered and appeared acceptable to participants. We were unable to robustly assess attrition and attendance rates due to the low sample size.

CONCLUSIONS: While ACE appeared acceptable to hearing aid users and feasible to deliver, it was not feasible to identify and recruit participants struggling with their hearing aids at the 3-month posthearing aid fitting point.

TRIAL REGISTRATION NUMBER: ISRCTN28090877.

PMID:33827834 | DOI:10.1136/bmjopen-2020-043364

Fertil Steril. 2021 Apr 4:S0015-0282(21)00147-3. doi: 10.1016/j.fertnstert.2021.02.029. Online ahead of print.

ABSTRACT

OBJECTIVE: To assess the association between body mass index (BMI) and embryo aneuploidy and mosaicism in a cohort of patients undergoing in vitro fertilization (IVF) with trophectoderm biopsy for preimplantation genetic testing for aneuploidy (PGT-A) using next-generation sequencing technology.

DESIGN: Retrospective cohort study.

SETTING: Academic center.

PATIENTS: Patients undergoing their first IVF cycle with trophectoderm biopsy and PGT-A at our center between January 1, 2017, and August 31, 2020. Patients classified as underweight on the basis of BMI (BMI <18.5 kg/m²) and patients who underwent fresh embryo transfers were excluded.

INTERVENTION: None.

MAIN OUTCOME MEASURES: Number and proportion of aneuploid, mosaic, and euploid embryos.

RESULTS: The patients were stratified according to the World Health Organization’s BMI classification: normal weight (18.5-24.9 kg/m², n = 1,254), overweight (25-29.9 kg/m², n = 351), and obese (≥30 kg/m², n = 145). Age-adjusted regression models showed no relationship between BMI classification and the number or proportion of aneuploid embryos. There were no statistically significant associations between BMI classifications and the number or proportion of mosaic or euploid embryos. A subgroup analysis of patients classified into age groups of <35, 35-40, and >40 years similarly showed no relationships between BMI and embryo ploidy outcomes.

CONCLUSION: Body mass index was not associated with the number or proportion of aneuploid, mosaic, or euploid embryos in this large cohort of patients undergoing IVF with PGT-A, suggesting that the negative effect of excess weight on reproductive outcomes was independent of the ploidy status of the embryo cohort.

PMID:33827765 | DOI:10.1016/j.fertnstert.2021.02.029

Injury. 2021 Mar 26:S0020-1383(21)00267-9. doi: 10.1016/j.injury.2021.03.049. Online ahead of print.

ABSTRACT

INTRODUCTION: Amongst critically ill trauma patients admitted to ICU and still alive and in ICU after 24 hours, it is unclear which trauma scoring system offers the best performance in predicting in-hospital mortality.

METHODS: The Australia and New Zealand Intensive Care Society Adult Patient Database and Victorian State Trauma Registry were linked using a unique patient identification number. Six scoring systems were evaluated: the Australian and New Zealand Risk of Death (ANZROD), Acute Physiology and Chronic Health Evaluation III (APACHE III) score and associated APACHE III Risk of Death (ROD), Trauma and Injury Severity Score (TRISS), Injury Severity Score (ISS), New Injury Severity Score (NISS) and the Revised Trauma Score (RTS). Patients who were admitted to ICU for longer than 24 hours were analysed. Performance of each scoring system was assessed primarily by examining the area under the receiver operating characteristic curve (AUROC) and in addition using standardised mortality ratios, Brier score and Hosmer-Lemeshow C statistics where appropriate. Subgroup assessments were made for patients aged 65 years and older, patients between 18 and 40 years of age, major trauma centre and head injury.

RESULTS: Overall, 5,237 major trauma patients who were still alive and in ICU after 24 hours were studied from 25 ICUs in Victoria, Australia between July 2008 and January 2018. Hospital mortality was 10.7%. ANZROD (AUROC 0.91; 95% CI 0.90-0.92), APACHE III ROD (AUROC 0.88; 95% CI 0.87-0.90), and APACHE III (AUROC 0.88; 95% CI 0.87-0.89) were the best performing tools for predicting hospital mortality. TRISS had acceptable overall performance (AUROC 0.78; 95% CI 0.76-0.80) while ISS (AUROC 0.61; 95% CI 0.59-0.64), NISS (AUROC 0.68; 95% CI 0.65-0.70) and RTS (AUROC 0.69; 95% CI 0.67-0.72) performed poorly. The performance of each scoring system was highest in younger adults and poorest in older adults.

CONCLUSION: In ICU patients admitted with a trauma diagnosis and still alive and in ICU after 24 hours, ANZROD and APACHE III had a superior performance when compared with traditional trauma-specific scoring systems in predicting hospital mortality. This was observed both overall and in each of the subgroup analyses. The anatomical scoring systems all performed poorly in the ICU population of Victoria, Australia.

PMID:33827776 | DOI:10.1016/j.injury.2021.03.049

J Pediatr Urol. 2021 Jan 21:S1477-5131(20)30746-4. doi: 10.1016/j.jpurol.2020.12.024. Online ahead of print.

ABSTRACT

OBJECTIVES: Hypospadias is a congenital birth defect affecting around one in every 300 male infants, characterized by incomplete fusion of the urethralfold.Our objective was to identify drug and chemical exposure in pregnancy associated with increased risk for hypospadias.

METHODS: We conducted a scoping review of studies of drugs and chemicals associated with hypospadias by searching Medline, EMBASE, Cochrane, Google and Google Scholar from inception to October 9, 2020 in any language. We searched for the terms; drugs, medications, chemicals, environmental agents, hypospadias, humans, In these papers we evaluated the existence of statistical evidence for an association between exposure to drugs and chemicals, and the occurrence of hypospadias. In examining the statistical evidence, we noted whether an association was made between a drug or chemical and hypospadias at p < 0.05, or Odds Ratio or Relative risk above 1, where the lower 95% confidence interval is also above 1. We also noted whether the study’s statistical method adjusted for co-variates or confounders.

RESULTS: Significant statistical evidence for association between hypospadias and the following drugs and chemicals has been verified: Progestins, dydrogesterone, clomiphene, ibuprofen, venlafaxine, endocrine disruptors, valproic acid and polybrominated diphenyl esters flame retardants.

CONCLUSIONS: There is increasing accumulation and quality of evidence of drugs and chemicals associated with hypospadias in humans. These may partially explain the rise in the prevalence of male reproductive health disorders in the last fifty years. Advising women of reproductive age about these risks should be an important component of prenatal counseling.

PMID:33827778 | DOI:10.1016/j.jpurol.2020.12.024

Zygote. 2021 Apr 8:1-7. doi: 10.1017/S096719942100006X. Online ahead of print.

ABSTRACT

This study aimed to describe the viability of domestic feline spermatozoa after epididymal tail vitrification. For this, 10 pairs of testis-epididymis complexes were used. The epididymal tails were vitrified using the solid-surface vitrification (SSV) method, in which two vitrification media containing ethylene glycol (EG) 40% or glycerol (GLY) 40% were tested. Vitrification with the presence of EG resulted in better results for all sperm motility parameters (motility, vigour and CASA) compared with GLY (P < 0.05). There were no statistical differences for sperm viability and acrosome integrity, plasma membrane integrity, or overall health of morphologically normal sperm before or after vitrification among experimental groups. In conclusion, epididymal tail vitrification appears to be a suitable method for long-term storage of cat sperm, especially if the procedure is performed with EG as the cryoprotectant.

PMID:33827737 | DOI:10.1017/S096719942100006X

J Perinat Med. 2021 Apr 7. doi: 10.1515/jpm-2020-0505. Online ahead of print.

ABSTRACT

OBJECTIVES: In Germany, cesarean section (CS) rates more than doubled within the past two decades. For analysis, auditing and inter-hospital comparison, the 10-Group Classification System (TGCS) is recommended. We used the TGCS to analyze CS rates in two German hospitals of different levels of care.

METHODS: From October 2017 to September 2018, data were prospectively collected. Unit A is a level three university hospital, unit B a level one district hospital. The German birth registry was used for comparison with national data. We performed two-sample Z tests and bootstrapping to compare aggregated (unit A + B) with national data and unit A with unit B.

RESULTS: In both datasets (national data and aggregated data unit A + B), Robson group (RG) 5 was the largest contributor to the overall CS rate. Compared to national data, group sizes in RG 1 and 3 were significantly smaller in the units under investigation, RG 8 and 10 significantly larger. Total CS rates between the two units differed (40.7 vs. 28.4%, p<0.001). The CS rate in RG 5 and RG 10 was different (p<0.01 for both). The most relative frequent RG in both units consisted of group 5, followed by group 10 and 2a.

CONCLUSIONS: The analysis allowed us to explain different CS rates with differences in the study population and with differences in the clinical practice. These results serve as a starting point for audits, inter-hospital comparisons and for interventions aiming to reduce CS rates.

PMID:33827151 | DOI:10.1515/jpm-2020-0505