Current medical research and opinion
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Objective: To describe the timeline to diagnosis for children with central precocious puberty (CPP) and evaluate their psychosocial and health-related quality of life (HRQoL). Methods: A cross-sectional survey was used to prospectively collect data from caregivers, recruited via the MAGIC Foundation, of children with CPP. The control (non-CPP) group was recruited from a national panel of parents/caregivers. ⋯ PROMIS peer relationship T score (± standard error) was numerically lower for the CPP versus non-CPP group (45.4 ± 1.0 versus 47.4 ± 0.7, p = .11). Conclusions: In clinical practice, there is a longer than expected delay between CPP symptom onset and referral to an endocrinologist and ultimate treatment. Children with CPP experience a substantial disease burden with a significant impact on emotional, social, and physical functioning compared with children without CPP.
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Objective: ASTRIS is a large real-world, open-label, multinational clinical study of osimertinib in patients with epidermal growth factor receptor (EGFR) T790M mutation-positive advanced non-small cell lung cancer (NSCLC) who have previously received a tyrosine kinase inhibitor (TKI). We report data from the Korean ASTRIS subgroup. Methods: Adult patients with locally advanced or metastatic NSCLC with a confirmed T790M mutation, WHO performance status of 0-2 and prior EGFR-TKI therapy, received osimertinib 80 mg once daily. ⋯ Overall, 31.1% of patients experienced ≥1 adverse event (AE), leading to dose modification (12.0%), discontinuation (5.2%) or death (2.8%). Serious AEs (24.9%) included pulmonary embolism (1.7%), pleural effusion (1.7%), and pneumonia (1.5%). Conclusion: In this real-world subgroup analysis of Korean patients in the ASTRIS study, osimertinib demonstrated comparable clinical efficacy to that attained in the global ASTRIS study and other clinical trials, with no new safety concerns.
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Observational Study
Observational study of the efficacy of prolonged-release metformin in people with prediabetes.
Objectives: Prediabetes is characterized by elevation of indices of blood glucose that is insufficient to provoke a diagnosis of type 2 diabetes, but markedly increases the risk of developing type 2 diabetes in the future. Lifestyle interventions are the main therapeutic intervention for the management of prediabetes. Current guidelines also support treatment of prediabetes with metformin for selected subgroups of patients, and metformin has a therapeutic indication for this use in a number of countries. ⋯ Metformin was well tolerated, with most side effects occurring in the gastrointestinal system, as expected. Conclusions: Metformin XR normalized FPG in about two-fifths of subjects with prediabetes. These real-world data add further support a role for metformin in the management of prediabetes, in line with current guidelines in this area.
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Background: Right but not left ventricular hemodynamic parameters have been found to be independently associated with adverse renal outcomes in patients with acute decompensated heart failure (HF). Aim: To investigate the hemodynamic profile of patients without acute decompensated heart failure and left ventricular ejection fraction >50% referred for elective left and right heart catheterization and to correlate left and right filling pressures, stroke volume and arterial blood pressure to renal function parameters. Subsequently, we tested the hypothesis that right ventricle and left ventricle hemodynamic parameters can predict all-cause mortality in our non-HF subjects. ⋯ Increased RAP (HR = 2.03; 95% [CI]: 1.05 to 3.9; p = .025) and age (HR = 1.08, 95% [CI] 1.04-1.12, p < .001) independently predicted all-cause mortality during follow up. Conclusions: Our study demonstrates that right ventricular preload affects renal function in patients with preserved systolic function and that neither aortic systolic pressure nor left ventricle pressure indices were related to estimated glomerular filtration rate. Furthermore, we demonstrate for the first time that an increased RAP is able to predict a worse prognosis in patients with preserved ejection fraction independently of well-established risk factors, such as blood pressure and SVI.
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Critical thinking is crucially important in both research and practice. This article demonstrates that a lack of critical thinking in two meta-analyses resulted in a conclusion that contradicts another meta-analysis and popular opinions. Kahwati et al. and Zhao et al. drew a conclusion that "Vitamin D supplementation alone or with calcium was not associated with reduced fracture incidence among community-dwelling adults without known vitamin D deficiency, osteoporosis, or prior fracture", which apparently contradicted that of Tang et al. ⋯ Subsequently, their conclusion was misleadingly interpreted by the public media as "Vitamin D and Calcium Don't Prevent Bone Fractures" and "Vitamin D Does Not Prevent Falls, Calcium Does Not Prevent Fractures-A $2 Billion Waste of Money". If study conclusions do not specify the applicable conditions, guidelines on medications, including supplements, are clinically unacceptable. Researchers must critically think about every step of their studies, including the way their conclusions are presented.