123 research outputs found
Association between Technical Performance Scores and neurodevelopmental outcomes after congenital cardiac surgery
ObjectivesTechnical Performance Score (TPS) has been shown to have a strong association with early and late outcomes after congenital cardiac surgery, with greater morbidity and reintervention in children with major residual lesions (TPS class 3). We sought to explore the effect of TPS on the neurodevelopmental outcomes.MethodsAll infants undergoing cardiac surgery, excluding those with trisomy 21, were offered neurodevelopmental testing at 1 year of age using the Bayley Scales of Infant Development, 3rd edition. TPSs from the discharge echocardiograms were graded as class 1 (optimal), class 2 (minor residual), or class 3 (major residual). Multivariate regression analysis was performed using patient characteristics and preoperative variables.ResultsNeurodevelopmental testing was performed in 140 patients at a median age of 16 months. Of these, 28 (20%) had single ventricle palliation; 39 (28%) were in Risk Adjustment for Congenital Heart Surgery category 4 to 6. Significant differences between the groups were found in the cognitive (PÂ =Â .01) and motor (PÂ =Â .05) domains, with subjects in TPS class 3 having significantly lower cognitive and motor composite scores. The scores did not vary significantly according to single ventricle versus biventricular repair or Risk Adjustment for Congenital Heart Surgery categorization. In multivariate modeling, class 3 TPS remained significantly associated with a lower Bayley cognitive score (PÂ =Â .02), with a trend toward a lower Bayley motor score (PÂ =Â .08).ConclusionsWe found that TPS is an independent predictor of neurodevelopmental outcomes after infant heart surgery. Future research should explore whether a structured program of intraoperative recognition and intervention on residual lesions can improve the TPS and neurodevelopmental outcomes
Validation of the Italian version of the cystic fibrosis quality of life questionnaire (CFQoL), a disease specific measure for adults and adolescents with cystic fibrosis.
Background
Disease specific, health-related quality of life (HRQoL) measurement is important in cystic fibrosis (CF). This work aimed to translate the original English Cystic Fibrosis Quality of Life Questionnaire (CFQoL) into Italian, evaluate the linguistic translation and to psychometrically evaluate the Italian version of the CFQoL.
Methods
The linguistic translation followed the international guidelines of forward and backward translation. Psychometric evaluation of the Italian CFQoL involved the assessment of construct validity, internal reliability, concurrent validity, known groups validity and testâretest reliability.
Results
The instrument was acceptable to adolescents and adults with CF and demonstrated robust psychometric properties. Principle components analysis indicated that the factorial structure was essentially similar to the original, and the internal reliability of each domain was good (Cronbach alpha coefficients 0.73 to 0.91). Appropriate domains of the CFQoL and SF-36 correlated well indicating good concurrent validity (r=0.68â0.80). Consistent with theoretical expectations some domains were able to discriminate between disease severity groups. Testâretest reliability, assessed by intraclass correlation coefficients, was found to be excellent (ICC 0.83 to 0.98).
Conclusions
The Italian CFQoL is a valid and reliable measure. Its use in individual patient monitoring and research should complement traditional clinical outcome measures
Mapping the disease-specific LupusQoL to the SF-6D
Purpose
To derive a mapping algorithm to predict SF-6D utility scores from the non-preference-based LupusQoL and test the performance of the developed algorithm on a separate independent validation data set.
Method
LupusQoL and SF-6D data were collected from 320 patients with systemic lupus erythematosus (SLE) attending routine rheumatology outpatient appointments at seven centres in the UK. Ordinary least squares (OLS) regression was used to estimate models of increasing complexity in order to predict individualsâ SF-6D utility scores from their responses to the LupusQoL questionnaire. Model performance was judged on predictive ability through the size and pattern of prediction errors generated. The performance of the selected model was externally validated on an independent data set containing 113 female SLE patients who had again completed both the LupusQoL and SF-36 questionnaires.
Results
Four of the eight LupusQoL domains (physical health, pain, emotional health, and fatigue) were selected as dependent variables in the final model. Overall model fit was good, with R2 0.7219, MAE 0.0557, and RMSE 0.0706 when applied to the estimation data set, and R2 0.7431, MAE 0.0528, and RMSE 0.0663 when applied to the validation sample.
Conclusion
This study provides a method by which health state utility values can be estimated from patient responses to the non-preference-based LupusQoL, generalisable beyond the data set upon which it was estimated. Despite concerns over the use of OLS to develop mapping algorithms, we find this method to be suitable in this case due to the normality of the SF-6D data
Design and Rationale of Safe Pediatric Euglycemia After Cardiac Surgery (SPECS): A Randomized Controlled Trial of Tight Glycemic Control After Pediatric Cardiac Surgery
Objectives: To describe the design of a clinical trial testing the hypothesis that children randomized to tight glycemic control with intensive insulin therapy after cardiac surgery will have improved clinical outcomes compared to children randomized to conventional blood glucose management. Design: Two-center, randomized controlled trial. Setting: Cardiac ICUs at two large academic pediatric centers. Patients: Children from birth to those aged 36 months recovering in the cardiac ICU after surgery with cardiopulmonary bypass. Interventions: Subjects in the tight glycemic control (intervention) group receive an intravenous insulin infusion titrated to achieve normoglycemia (target blood glucose range of 80â110 mg/dL; 4.4â6.1 mmol/L). The intervention begins at admission to the cardiac ICU from the operating room and terminates when the patient is ready for discharge from the ICU. Continuous glucose monitoring is performed during insulin infusion to minimize the risks of hypoglycemia. The standard care group has no target blood glucose range. Measurements and Main Results: The primary outcome is the development of any nosocomial infection (bloodstream, urinary tract, and surgical site infection or nosocomial pneumonia). Secondary outcomes include mortality, measures of cardiorespiratory function and recovery, laboratory indices of nutritional balance, immunologic, endocrinologic, and neurologic function, cardiac ICU and hospital length of stay, and neurodevelopmental outcome at 1 and 3 yrs of age. A total of 980 subjects will be enrolled (490 in each treatment arm) for sufficient power to show a 50% reduction in the prevalence of the primary outcome. Conclusions: Pediatric cardiac surgery patients may recognize great benefit from tight glycemic control in the postoperative period, particularly with regard to reduction of nosocomial infections. The Safe Pediatric Euglycemia after Cardiac Surgery trial is designed to provide an unbiased answer to the question of whether this therapy is indeed beneficial and to define the associated risks of therapy
Girls and Boys Born before 28Â Weeks Gestation: Risks of Cognitive, Behavioral, and Neurologic Outcomes at Age 10Â Years
To compare the prevalence of cognitive, neurological, and behavioral outcomes at 10 years of age in 428 girls and 446 boys who were born extremely preterm (EP)
The Relationship of Maternal Prepregnancy Body Mass Index and Pregnancy Weight Gain to Neurocognitive Function at Age 10 Years among Children Born Extremely Preterm
OBJECTIVE: To assess the association between maternal prepregnancy body mass index and adequacy of pregnancy weight gain in relation to neurocognitive function in school-aged children born extremely preterm.
STUDY DESIGN: Study participants were 535 ten-year-old children enrolled previously in the prospective multicenter Extremely Low Gestational Age Newborns cohort study who were products of singleton pregnancies. Soon after delivery, mothers provided information about prepregnancy weight. Prepregnancy body mass index and adequacy of weight gain were characterized based on this information. Children underwent a neurocognitive evaluation at 10 years of age.
RESULTS: Maternal prepregnancy obesity was associated with increased odds of a lower score for Differential Ability Scales-II Verbal IQ, for Developmental Neuropsychological Assessment-II measures of processing speed and visual fine motor control, and for Wechsler Individual Achievement Test-III Spelling. Children born to mothers who gained an excessive amount of weight were at increased odds of a low score on the Oral and Written Language Scales Oral Expression assessment. Conversely, children whose mother did not gain an adequate amount of weight were at increased odds of a lower score on the Oral and Written Language Scales Oral Expression and Wechsler Individual Achievement Test-III Word Reading assessments.
CONCLUSION: In this cohort of infants born extremely preterm, maternal obesity was associated with poorer performance on some assessments of neurocognitive function. Our findings are consistent with the observational and experimental literature and suggest that opportunities may exist to mitigate risk through education and behavioral intervention before pregnancy
Informing research priorities for immature sea turtles through expert elicitation
Although sea turtles have received substantial focus worldwide, research on the immature life stages is still relatively limited. The latter is of particular importance, given that a large proportion of sea turtle populations comprises immature individuals. We set out to identify knowledge gaps and identify the main barriers hindering research in this field. We analyzed the perceptions of sea turtle experts through an online survey which gathered their opinions on the current state of affairs on immature sea turtle research, including species and regions in need of further study, priority research questions, and barriers that have interfered with the advancement of research. Our gap analysis indicates that studies on immature leatherback Dermochelys coriacea and hawksbill Eretmochelys imbricata turtles are lacking, as are studies on all species based in the Indian, South Pacific, and South Atlantic Oceans. Experts also perceived that studies in population ecology, namely on survivorship and demography, and habitat use/behavior, are needed to advance the state of knowledge on immature sea turtles. Our survey findings indicate the need for more inter-disciplinary research, collaborative efforts (eg data-sharing, joint field activities), and improved communication among researchers, funding bodies, stakeholders, and decision-makers
Culture Adaptation Alters Transcriptional Hierarchies among Single Human Embryonic Stem Cells Reflecting Altered Patterns of Differentiation
We have used single cell transcriptome analysis to re-examine the substates of early passage, karyotypically Normal, and late passage, karyotypically Abnormal (âCulture Adaptedâ) human embryonic stem cells characterized by differential expression of the cell surface marker antigen, SSEA3. The results confirmed that culture adaptation is associated with alterations to the dynamics of the SSEA3(+) and SSEA3(-) substates of these cells, with SSEA3(-) Adapted cells remaining within the stem cell compartment whereas the SSEA3(-) Normal cells appear to have differentiated. However, the single cell data reveal that these substates are characterized by further heterogeneity that changes on culture adaptation. Notably the Adapted population includes cells with a transcriptome substate suggestive of a shift to a more naĂŻve-like phenotype in contrast to the cells of the Normal population. Further, a subset of the Normal SSEA3(+) cells expresses genes typical of endoderm differentiation, despite also expressing the undifferentiated stem cell genes, POU5F1 (OCT4) and NANOG, whereas such apparently lineage-primed cells are absent from the Adapted population. These results suggest that the selective growth advantage gained by genetically variant, culture adapted human embryonic stem cells may derive in part from a changed substate structure that influences their propensity for differentiation
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Low-intensity cognitive-behaviour therapy interventions for obsessive-compulsive disorder compared to waiting list for therapist-led cognitive-behaviour therapy: 3-arm randomised controlled trial of clinical effectiveness
Background Obsessive-compulsive disorder (OCD) is prevalent and without adequate treatment usually follows a chronic course. âHigh-intensityâ cognitive-behaviour therapy (CBT) from a specialist therapist is current âbest practice.â However, access is difficult because of limited numbers of therapists and because of the disabling effects of OCD symptoms. There is a potential role for âlow-intensityâ interventions as part of a stepped care model. Low-intensity interventions (written or web-based materials with limited therapist support) can be provided remotely, which has the potential to increase access. However, current evidence concerning low-intensity interventions is insufficient. We aimed to determine the clinical effectiveness of 2 forms of low-intensity CBT prior to high-intensity CBT, in adults meeting the Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition (DSM-IV) criteria for OCD. Methods and findings This study was approved by the National Research Ethics Service Committee North WestâLancaster (reference number 11/NW/0276). All participants provided informed consent to take part in the trial. We conducted a 3-arm, multicentre randomised controlled trial in primary- and secondary-care United Kingdom mental health services. All patients were on a waiting list for therapist-led CBT (treatment as usual). Four hundred and seventy-three eligible patients were recruited and randomised. Patients had a median age of 33 years, and 60% were female. The majority were experiencing severe OCD. Patients received 1 of 2 low-intensity interventions: computerised CBT (cCBT; web-based CBT materials and limited telephone support) through âOCFighterâ or guided self-help (written CBT materials with limited telephone or face-to-face support). Primary comparisons concerned OCD symptoms, measured using the Yale-Brown Obsessive Compulsive ScaleâObserver-Rated (Y-BOCS-OR) at 3, 6, and 12 months. Secondary outcomes included health-related quality of life, depression, anxiety, and functioning. At 3 months, guided self-help demonstrated modest benefits over the waiting list in reducing OCD symptoms (adjusted mean difference = â1.91, 95% CI â3.27 to â0.55). These effects did not reach a prespecified level of âclinically significant benefit.â cCBT did not demonstrate significant benefit (adjusted mean difference = â0.71, 95% CI â2.12 to 0.70). At 12 months, neither guided self-help nor cCBT led to differences in OCD symptoms. Early access to low-intensity interventions led to significant reductions in uptake of high-intensity CBT over 12 months; 86% of the patients allocated to the waiting list for high-intensity CBT started treatment by the end of the trial, compared to 62% in supported cCBT and 57% in guided self-help. These reductions did not compromise longer-term patient outcomes. Data suggested small differences in satisfaction at 3 months, with patients more satisfied with guided self-help than supported cCBT. A significant issue in the interpretation of the results concerns the level of access to high-intensity CBT before the primary outcome assessment. Conclusions We have demonstrated that providing low-intensity interventions does not lead to clinically significant benefits but may reduce uptake of therapist-led CBT
International collaborative study to assess cardiovascular risk and evaluate long-term health in cats with preclinical hypertrophic cardiomyopathy and apparently healthy cats:The REVEAL Study
Background: Hypertrophic cardiomyopathy is the most prevalent heart disorder in cats and principal cause of cardiovascular morbidity and mortality. Yet, the impact of preclinical disease is unresolved. Hypothesis/Objectives: Observational study to characterize cardiovascular morbidity and survival in cats with preclinical nonobstructive (HCM) and obstructive (HOCM) hypertrophic cardiomyopathy and in apparently healthy cats (AH). Animals: One thousand seven hundred and thirty client-owned cats (430 preclinical HCM; 578 preclinical HOCM; 722 AH). Methods: Retrospective multicenter, longitudinal, cohort study. Cats from 21 countries were followed through medical record review and owner or referring veterinarian interviews. Data were analyzed to compare long-term outcomes, incidence, and risk for congestive heart failure (CHF), arterial thromboembolism (ATE), and cardiovascular death. Results: During the study period, CHF, ATE, or both occurred in 30.5% and cardiovascular death in 27.9% of 1008 HCM/HOCM cats. Risk assessed at 1, 5, and 10 years after study entry was 7.0%/3.5%, 19.9%/9.7%, and 23.9%/11.3% for CHF/ATE, and 6.7%, 22.8%, and 28.3% for cardiovascular death, respectively. There were no statistically significant differences between HOCM compared with HCM for cardiovascular morbidity or mortality, time from diagnosis to development of morbidity, or cardiovascular survival. Cats that developed cardiovascular morbidity had short survival (mean \ub1 standard deviation, 1.3 \ub1 1.7 years). Overall, prolonged longevity was recorded in a minority of preclinical HCM/HOCM cats with 10% reaching 9-15 years. Conclusions and Clinical Importance: Preclinical HCM/HOCM is a global health problem of cats that carries substantial risk for CHF, ATE, and cardiovascular death. This finding underscores the need to identify therapies and monitoring strategies that decrease morbidity and mortality
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