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Being a chronic condition, dRTA also deserves attention in the transition between pediatric and adult nephrology care, and as a rare disease it has a place in the European and Italian rare nephrological diseases network.Vericiguat (VERQUVO™; Merck & Co, Bayer AG) is a soluble guanylate cyclase (sGC) stimulator being developed for the treatment of chronic heart failure. Vericiguat stimulates sGC and cGMP production independent of nitric oxide (NO) and enhances the effects of NO by stabilizing the NO-sGC binding. Based on the results of the phase III VICTORIA trial vericiguat was recently approved in the USA for risk reduction in patients with heart failure and ejection fraction less then 45%. This article summarizes the milestones in the development of vericiguat leading to this first approval.
ATTR-ACT (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial) demonstrated the efficacy and safety of tafamidis in transthyretin amyloid cardiomyopathy (ATTR-CM). Model-based analyses from ATTR-ACT can examine predictor effects on dose-response/exposure-response relationships.
Parametric hazard distributions were developed for all-cause mortality and frequency of cardiovascular-related hospitalization. Time-to-event models were fitted to survival data, and repeated time-to-event models were fitted to hospitalization data. Disease-specific characteristics were assessed as baseline predictors of event hazards.
There were 441 patients in this analysis. At month 30, 70.5% (tafamidis) and 57.1% (placebo) of patients were alive, with 154/441 deaths reported; 495 cardiovascular-related hospitalizations occurred. The cumulative risk of death was 42.1% (95% confidence interval [CI] 24.2-58.0) lower with tafamidis than with placebo, regardless of New York Heart Association (NYHA) class; significant predictors of decreased risk were genotype (wild-type), greater 6-Minute Walk Test (6MWT) distance, higher left ventricular ejection fraction (LVEF), and lower blood urea nitrogen (BUN) and N-terminal pro-B-type natriuretic peptide concentrations. The average cumulative risk of cardiovascular-related hospitalization up to 30 months was 40.8% (95% CI 31.0-49.7) lower with tafamidis in NYHA class I/II patients. Significant predictors of reduced risk were greater 6MWT distance, higher LVEF, and lower BUN and troponin I concentrations.
Tafamidis reduced cumulative mortality and hospitalization risk versus placebo in patients with ATTR-CM. Baseline predictors of outcome were consistent with the cardiovascular nature of the disease and suggested that earlier treatment may improve outcomes. CLINICAL TRIALS.
NCT01994889 (date of registration November 26, 2013).
NCT01994889 (date of registration November 26, 2013).
To investigate the long-term changes in posterior corneal elevation in moderate and high myopia after small incision lenticule extraction (SMILE).
Eye & ENT Hospital, Fudan University.
Prospective case series.
Thirty-three eyes of 20 patients (30.1+/-9.5 years, 7 male and 13 female, spherical equivalent [SE] range -4.00 to -8.75 D, average SE -6.25+/-1.29 D) who underwent SMILE were included. Changes in the posterior corneal elevation at central points (PCE), the thinnest point (PTE), the maximal point (PME), and an additional 20 points within the 4-mm area of the best-fit sphere were evaluated with a Pentacam (Oculus Optikgerate GmbH, Wetzlar, Germany) during a 7-year follow-up period. Mixed linear models were used to evaluate changes with P-values <0.05. Correlations of elevation changes and residual bed thickness (RBT) were also evaluated.
No ectasia was observed among the 33 eyes. The safety index was 1.08, and the efficacy index was 1.03. The mean change in PCE at 1, 3, 5, and 7 years was -1.42+/-0.95, -2.67+/-0.98, -2.44+/-1.06, and -1.91+/-0.92 [micro]m, respectively. Significant differences were found at 3, 5, and 7 years (P=0.007, 0.023, and 0.040, respectively). PTE was significantly reduced at each time point compared to baseline. The mean change was -2.82+/-1.19, -3.55+/-1.22, -3.47+/-1.32, and -2.39+/-1.15 [micro]m, respectively (P=0.019, 0.004, 0.010, and 0.039, respectively). PME changed 2.45+/-1.02 [micro]m at 7 years compared to baseline (P=0.017). The changes in PCE and PME negatively correlated with the RBT.
Long-term posterior corneal elevation was stable in moderate and high myopia after SMILE.
Long-term posterior corneal elevation was stable in moderate and high myopia after SMILE.In this review, we consider issues relating to the pharmacological treatment of young children with attention deficit hyperactivity disorder (ADHD). ADHD in preschool-age children has a profound impact on psychosocial function and developmental trajectory. Clinical studies on pharmacotherapies for ADHD in young children have expanded rapidly in the past 2 decades, providing some evidence of efficacy for both psychostimulant and non-psychostimulant medications. However, preschool children may be more susceptible to adverse effects of medications, including growth reduction and cardiovascular side effects. Many questions remain regarding the long-term safety and effectiveness of these interventions; thus more research is needed to help clinicians evaluate the risk-benefit ratio for preschoolers with ADHD. As this body of knowledge grows, providers should consider the level of impairment caused by current symptoms in the risk-benefit analysis. Families should be educated not just about potential effects of medication but known complications of untreated ADHD; parents will likely not fully appreciate the long-term psychological effects of chronic behavioral problems and underachievement on a young child. A blanket "wait and see" approach should be avoided, in order to prevent a permanent loss of self-esteem and motivation that may affect some children throughout their lifespan.MPL exon 10 mutations are one of the driver mutations in essential thrombocythemia (ET) or myelofibrosis (MF). We have established an in-house MPL mutation analysis system, covering the entire region of MPL exon 10 by direct sequencing. Since 2009, MPL exon 10 mutation analysis has been performed for diagnosis of myeloproliferative neoplasms (MPN) without JAK2 V617F or CALR exon 9 mutations. So far, 11 cases of MPL exon 10 mutation have been found in 51 patients with suspected MPN. In patients with ET, we detected five non-canonical MPL mutations including one novel mutation, MPL R514_P518delinsK, and one canonical MPL W515L mutation. Notably, three ET patients without canonical MPL mutations had thrombotic events. click here Meanwhile, in primary or secondary MF, only canonical MPL W515L/K mutations were found. Further cases need to be examined to elucidate the full MPL mutation profile in MPN. However, our data indicate that analysis of the whole of MPL exon 10 is warranted for the diagnosis of MPL mutations, especially in ET, and that the use of Japanese commercial laboratory tests that only detect canonical MPL W515L/K mutations may miss a significant percentage of MPL exon 10 mutations, which could delay the administration of anti-thrombotic therapy.
As the incidence of new cases of coronavirus disease increased exponentially, the use of viral swabs to collect nasopharyngeal specimens are increasing drastically. Therefore, healthcare workers military staff and uneducated nonprofessional's were ordered to make this swabs. Subsequently case reports reported about basal skull perforation, cerebrospinal fluid fistula and injury due to an incorrect technique.
Search of the literature.
Only in 44% of the videos (Youtube) nasal swabs were correctly performed. Due to an false technique biological sampling resulted in false-negative COVID-19 tests.
Although professional societies started to report about this unacceptable situation, no publication reported about this health endangerment. In this time of overwhelming information and diversity of opinions, it is necessary to report about this in the hope, all media and TV reports will follow this article to show correctly performed nasal swabs to reduce false-negative COVID-19 tests and injury.
Although professional societies started to report about this unacceptable situation, no publication reported about this health endangerment. In this time of overwhelming information and diversity of opinions, it is necessary to report about this in the hope, all media and TV reports will follow this article to show correctly performed nasal swabs to reduce false-negative COVID-19 tests and injury.
Although there has been a rising emphasis on patient-centered care, limited research has assessed differences in patient experience based on ethnicity and language.
This study examined differences in quality of care (N = 6945) using the Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) survey. Data were collected between January 2014 and April 2019. Bivariate and multivariate analyses assessed relationships between ethnicity/language with individual items capturing specific components of care and global hospital evaluations using regression modeling.
Compared to English-speaking non-Hispanic White patients, Spanish-speaking Hispanic/Latinx patients reported more positive interactions with nurses, physicians, and the hospital environment and reported a better understanding of care after discharge. Findings also indicated that Spanish-speaking Hispanic/Latinx patients were more satisfied with their experience compared to non-Hispanic White patients.
Spanish-speaking Hispanic/Latinx patients were more satisfied with specific components of care and also scored higher in a measure of the global patient experience. Findings suggest the need for setting clear expectations for health care encounters and adapting health system responses to better capture factors driving Hispanic/Latinx patient satisfaction.
Spanish-speaking Hispanic/Latinx patients were more satisfied with specific components of care and also scored higher in a measure of the global patient experience. Findings suggest the need for setting clear expectations for health care encounters and adapting health system responses to better capture factors driving Hispanic/Latinx patient satisfaction.
Alopecia areata (AA) is a chronic, autoimmune disease of hair loss, which can significantly affect the emotional and psychological well-being of patients. A systematic literature review was conducted to better understand the burden of AA from the patient perspective.
Embase, MEDLINE and Cochrane databases were searched for published studies (2008-2018) reporting on assessments of health-related quality of life (HRQoL) for patients with AA. Qualitative, and quantitative data were collected.
The review included 37 studies encompassing a range of clinical outcome assessment (COA) tools. None of the COA tools were specific for AA, and only one study used the Hairdex scale, which was designed to evaluate HRQoL in patients with disorders of the hair and scalp. All studies reported substantial impact on HRQoL due to AA, both overall and in domains related to personality (i.e. temperament and character), emotions and social functioning. Acute stress was also noted, and several studies identified lack of emotional awareness (alexithymia) in 23-50% of the patients with AA.
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