Search results for "OUTCOME"

Acquired IFNγ resistance impairs anti-tumor immunity and gives rise to T-cell-resistant melanoma lesions

2016

Melanoma treatment has been revolutionized by antibody-based immunotherapies. IFNγ secretion by CD8+ T cells is critical for therapy efficacy having anti-proliferative and pro-apoptotic effects on tumour cells. Our study demonstrates a genetic evolution of IFNγ resistance in different melanoma patient models. Chromosomal alterations and subsequent inactivating mutations in genes of the IFNγ signalling cascade, most often JAK1 or JAK2, protect melanoma cells from anti-tumour IFNγ activity. JAK1/2 mutants further evolve into T-cell-resistant HLA class I-negative lesions with genes involved in antigen presentation silenced and no longer inducible by IFNγ. Allelic JAK1/2 losses predisposing to …

Late Cardiac Events after Childhood Cancer: Methodological Aspects of the Pan-European Study PanCareSurFup.

2016

Background and AimChildhood cancer survivors are at high risk of long-term adverse effects of cancer and its treatment, including cardiac events. The pan-European PanCareSurFup study determined the incidence and risk factors for cardiac events among childhood cancer survivors. The aim of this article is to describe the methodology of the cardiac cohort and nested case-control study within PanCareSurFup.MethodsEight data providers in Europe participating in PanCareSurFup identified and validated symptomatic cardiac events in their cohorts of childhood cancer survivors. Data on symptomatic heart failure, ischemia, pericarditis, valvular disease and arrhythmia were collected and graded accordi…

Use of asthma medication during pregnancy and risk of specific congenital anomalies: A European case-malformed control study.

2015

Background: Pregnant women with asthma need to take medication during pregnancy.Objective: We sought to identify whether there is an increased risk of specific congenital anomalies after exposure to antiasthma medication in the first trimester of pregnancy.Methods: We performed a population-based case-malformed control study testing signals identified in a literature review. Odds ratios (ORs) of exposure to the main groups of asthma medication were calculated for each of the 10 signal anomalies compared with registrations with nonchromosomal, nonsignal anomalies as control registrations. In addition, exploratory analyses were done for each nonsignal anomaly. The data set included 76,249 reg…

Correlation between neonatal outcomes of twins depends on the outcome : Secondary analysis of twelve randomised controlled trials

2018

ObjectiveTo estimate the magnitude of the correlation between neonatal outcomes of twins and demonstrate how this information can be used in the design of randomised controlled trials (RCTs) in women with twin pregnancies.DesignSecondary analysis of data from 12 RCTs.SettingObstetric care in multiple countries, 2004-2012.Population or sample4504 twin pairs born to women who participated in RCTs to assess treatments given during pregnancy.MethodsIntraclass correlation coefficients (ICCs) were estimated using log-binomial and linear models.Main outcome measuresPerinatal death, respiratory distress syndrome, bronchopulmonary dysplasia, intraventricular haemorrhage, necrotising enterocolitis, s…

Atypical presentation of anti-N-methyl-D-aspartate receptor encephalitis: two case reports

2017

Abstract Background Anti-N-methyl-D-aspartate receptor encephalitis is a rare autoimmune disease characterized by severe neurological and psychiatric symptoms and a difficult diagnosis. The disease is often secondary to a neoplastic lesion, seldom diagnosed years later. Psychiatric symptoms are prevalent in adults; neurologic symptoms are more evident in children, who typically present primarily with neurological symptoms. To the best of our knowledge, the association with juvenile idiopathic arthritis has not been described. Case presentation We report the cases of two caucasian girls with an atypical presentation. The first patient was an 8-year-old girl with normal psychomotor developmen…

Efficacy of Guanfacine Extended Release in Children and Adolescents with Attention-Deficit/Hyperactivity Disorder and Comorbid Oppositional Defiant D…

2020

OBJECTIVE To assess the efficacy of the non-stimulant guanfacine extended release (GXR) on attention-deficit/hyperactivity disorder (ADHD) symptoms in children and adolescents, with and without comorbid oppositional defiant disorder (ODD). METHODS Data were derived from 4 phase 3, randomized, placebo-controlled trials of dose-optimized GXR monotherapy, in which at least 10% of participants had a diagnosis of comorbid ODD. SPD503-312 and SPD503-316 were 10- to 13-week studies of GXR (1-7 mg/d). SPD503-314 and SPD503-307 were 8-week studies of GXR (1-4 mg/d). Efficacy was assessed using the ADHD Rating Scale IV (ADHD-RS-IV) total scores. RESULTS In total, 1,084 participants were included (SPD…

Differences in Symptomatology and Clinical Course of Acute Coronary Syndromes in Women ≤45 Years of Age Compared to Older Women

2021

Acute coronary syndromes (ACS) in young people are rare. The data regarding differences in symptoms in relation to age are scarce, which may have an influence on outcomes. The aim of this study was to evaluate the differences in the clinical course of ACS between younger women (≤45 years old) and older women (63-64 years old). We compared 7481 women with ACS from the Polish Registry of ACS between 2007 and 2014 (1834 women aged ≤45 years and 5647 women aged 63-64 years). The predominant symptom of ACS in both groups was chest pain, with a higher incidence occurring in younger women (90.4% vs 88.5%, P = 0.025). Prehospital cardiac arrest occurred more often in younger women (2.1% vs 0.8%, P0…

Ophthalmological manifestations in Fabry disease children in the Fabry Outcome Survey

2015

Van Ewijk et al. respond to "Ramadan prenatal fasting and adult health outcomes"

2013

Autosomal recessive polycystic kidney disease: case report of a newborn with rare PKHD1 mutation, rapid renal enlargement and early fatal outcome

2020

Abstract Introduction Autosomal recessive polycystic kidney disease (ARPKD; MIM#263200) is one of the most frequent pediatric renal cystic diseases, with an incidence of 1:20,000. It is caused by mutations of the PKHD1 gene, on chromosome 6p12. The clinical spectrum is highly variable, ranging from late-onset milder forms to severe perinatal manifestations. The management of newborns with severe pulmonary insufficiency is challenging, and causes of early death are sepsis or respiratory failure. In cases of massive renal enlargement, early bilateral nephrectomy and peritoneal dialysis may reduce infant mortality. However, there is no conclusive data on the role of surgery, and decision-makin…