Background Studies of cardiomyopathy in children with Noonan syndrome (NS) have been primarily small case series or cross-sectional studies with small or no comparison groups. Methods We used the ...Pediatric Cardiomyopathy Registry database to compare the survival experience of children with NS and hypertrophic cardiomyopathy (HCM) with children with idiopathic or familial HCM and to identify clinical and echocardiographic predictors of clinical outcomes. Results Longitudinal data in 74 children with NS and HCM and 792 children with idiopathic or familial isolated HCM were compared. Children with NS were diagnosed with HCM before 6 months old more often (51%) than children with HCM (28%) and were more likely to present with congestive heart failure (CHF) (24% vs 9%). The NS cohort had lower crude survival than the group with other HCM ( P = .03), but survival did not differ after adjustment for CHF and age at diagnosis. Within the NS cohort (1-year survival 78%), a diagnosis of HCM before age 6 months with CHF resulted in 31% 1-year survival. Lower height-for-age z score (hazard ratio 0.26, P = .005) in place of CHF and lower left ventricular fractional shortening z score (hazard ratio 0.79, P = .04) also independently predicted mortality. Conclusions Patients with NS with HCM have a worse risk profile at presentation compared with other children with HCM, resulting in significant early mortality (22% at 1 year). Decreased height-for-age and lower, although still supranormal, left ventricular fractional shortening z score are independent predictors of mortality in patients with NS with HCM. Such patients should have an aggressive therapeutic approach including potential listing for cardiac transplantation.
Fontan Physiology Revisited Jolley, Matthew; Colan, Steven D; Rhodes, Jonathan ...
Anesthesia and analgesia,
2015-July, Letnik:
121, Številka:
1
Journal Article
Recenzirano
Odprti dostop
The Fontan operation places the systemic and pulmonary circulations in series, driven by a single ventricular chamber. It has become the treatment strategy of choice for palliating single-ventricle ...congenital heart disease. This anatomy engenders profound changes in physiology, affecting the cardiovascular and respiratory systems with direct implications for anesthetic and intensive care. The physical basis of these changes and their sequelae are reviewed.
BACKGROUNDUnplanned catheter-based or surgical reinterventions after congenital heart operations are independently associated with operative mortality and increased postoperative length of stay. ...OBJECTIVESThis study assessed the long-term outcomes of transplant-free survivors of hospital discharge requiring predischarge reinterventions after congenital cardiac surgery. METHODSData from patients who required predischarge reinterventions in the anatomic area of repair after congenital cardiac surgery and survived to hospital discharge at a quaternary referral center from January 2011 to December 2019 were retrospectively reviewed. Previously published echocardiographic criteria were used to assess the severity of persistent residual lesions at discharge (Grade 1, no residua; Grade 2, minor residua; and Grade 3, major residua). Outcomes included postdischarge (late) mortality or transplant and unplanned reintervention. Associations between predischarge residual lesion severity and outcomes were assessed by using Cox or competing risk models, adjusting for baseline patient characteristics, case complexity, and preoperative risk factors. RESULTSAmong the 408 patients who met entry criteria, there were 58 (14.2%) postdischarge deaths or transplants and 208 (51.0%) late reinterventions at a median follow-up of 3.0 years (IQR: 1.1-6.8 years). Greater predischarge residual lesion severity was associated with worse transplant-free survival and freedom from reintervention (both, P < 0.05). On multivariable analyses, Grade 3 patients had an increased risk of postdischarge mortality or transplant (HR: 4.8; 95% CI: 2.0-11; P < 0.001) and late reintervention (subdistribution HR: 2.1; 95% CI: 1.4-3.1; P < 0.001) vs Grade 1 patients. CONCLUSIONSAmong transplant-free survivors requiring predischarge reinterventions after congenital cardiac surgery, those with persistent major residua have significantly worse long-term outcomes. These high-risk patients warrant closer surveillance.
Summary Background Treatment of children with hypertrophic cardiomyopathy might be improved if the risk of death or heart transplantation could be predicted by risk factors present at the time of ...diagnosis. Methods We analysed data from the Pediatric Cardiomyopathy Registry, which collected longitudinal data for 1085 children with hypertrophic cardiomyopathy from 1990 to 2009. Our goal was to understand how patient factors measured at diagnosis predicted the subsequent risk of the primary outcome of death or heart transplantation. The Kaplan-Meier method was used to calculate time-to-event rates from time of diagnosis to the earlier of heart transplantation or death for children in each subgroup. Cox proportional-hazards regression was used to identify univariable and multivariable predictors of death or heart transplantation within each causal subgroup. Findings The poorest outcomes were recorded for the 69 children with pure hypertrophic cardiomyopathy with inborn errors of metabolism, for whom the estimated rate of death or heart transplantation was 57% (95% CI 44–69) at 2 years. Children with mixed functional phenotypes also did poorly, with rates of death or heart transplantation of 45% (95% CI 32–58) at 2 years for the 69 children with mixed hypertrophic and dilated cardiomyopathy and 38% (95% CI 25–51) at 2 years for the 58 children with mixed hypertrophic and restrictive cardiomyopathy. For children diagnosed with hypertrophic cardiomyopathy at younger than 1 year, the rate of death or transplantation was 21% (95% CI 16–27) at 2 years. For children diagnosed with hypertrophic cardiomyopathy and a malformation syndrome, the rate of death or transplantation was 23% (95% CI 12–34) at 2 years. Excellent outcomes were reported for the 407 children who were diagnosed with idiopathic hypertrophic cardiomyopathy at age 1 year or older, with a rate of death or heart transplantation of 3% (95% CI 1–5) at 2 years. The risk factors for poor outcomes varied according to hypertrophic cardiomyopathy subgroup, but they generally included young age, low weight, presence of congestive heart failure, lower left ventricular fractional shortening, or higher left ventricular end-diastolic posterior wall thickness or end-diastolic ventricular septal thickness at the time of cardiomyopathy diagnosis. For all hypertrophic cardiomyopathy subgroups, the risk of death or heart transplantation was significantly increased when two or more risk factors were present and also as the number of risk factors increased. Interpretation In children with hypertrophic cardiomyopathy, the risk of death or heart transplantation was greatest for those who presented as infants or with inborn errors of metabolism or with mixed hypertrophic and dilated or restrictive cardiomyopathy. Risk stratification by subgroup of cardiomyopathy, by characteristics such as low weight, congestive heart failure, or abnormal echocardiographic findings, and by the presence of multiple risk factors allows for more informed clinical decision making and prognosis at the time of diagnosis. Funding US National Institutes of Health and Children's Cardiomyopathy Foundation.
Many factors affect outcomes after congenital cardiac surgery.
The RLS (Residual Lesion Score) study explored the impact of severity of residual lesions on post-operative outcomes across operations ...of varying complexity.
In a prospective, multicenter, observational study, 17 sites enrolled 1,149 infants undergoing 5 common operations: tetralogy of Fallot repair (n = 250), complete atrioventricular septal defect repair (n = 249), arterial switch operation (n = 251), coarctation or interrupted arch with ventricular septal defect (VSD) repair (n = 150), and Norwood operation (n = 249). The RLS was assigned based on post-operative echocardiography and clinical events: RLS 1 (trivial or no residual lesions), RLS 2 (minor residual lesions), or RLS 3 (reintervention for or major residual lesions before discharge). The primary outcome was days alive and out of hospital within 30 post-operative days (60 for Norwood). Secondary outcomes assessed post-operative course, including major medical events and days in hospital.
RLS 3 (vs. RLS 1) was an independent risk factor for fewer days alive and out of hospital (p ≤ 0.008) and longer post-operative hospital stay (p ≤ 0.02) for all 5 operations, and for all secondary outcomes after coarctation or interrupted arch with VSD repair and Norwood (p ≤ 0.03). Outcomes for RLS 1 versus 2 did not differ consistently. RLS alone explained 5% (tetralogy of Fallot repair) to 20% (Norwood) of variation in the primary outcome.
Adjusting for pre-operative factors, residual lesions after congenital cardiac surgery impacted in-hospital outcomes across operative complexity with greatest impact following complex operations. Minor residual lesions had minimal impact. These findings may provide guidance for surgeons when considering short-term risks and benefits of returning to bypass to repair residual lesions.
Repair of partial or transitional atrioventricular septal defects (P/TAVSDs) has excellent outcomes; however, late reinterventions remain a concern. Technical performance score (TPS) measures residua ...after repair and has been associated with early/mid-term outcomes after congenital cardiac operation. Our study investigates TPS as a predictor of outcomes after P/TAVSD repair.
This was a single-center retrospective review of P/TAVSD repair from July 2000 to November 2015. Intraoperative and discharge TPS were assigned based on echocardiographic criteria: class 1, no residua; class 2, minor residua; and class 3, major residua or reintervention for major residua. Intensive care unit (ICU) hospital length of stay and reintervention after discharge were analyzed with Cox regression.
In our cohort, 124 partial (68%) and 59 transitional (32%) AVSDs underwent repair. Median age was 1.5 years (interquartile range IQR: 0.6 to 3.8 years), median weight was 9.7 kg (IQR: 6.6 to 14.1 kg), and 96 (52%) were female. Twenty patients (11%) required reintervention after discharge. On multivariable modeling, patients with TPS class 3 spent more days in the ICU (hazard ratio HR 0.33, 95% confidence interval CI: 0.19 to 0.58, p < 0.001) and hospital (HR 0.33, 95% CI: 0.19 to 0.57, p < 0.001) and had shorter time to reintervention after discharge (HR 8.76, 95% CI: 1.03 to 74.7, p = 0.047).
Major residua, that is TPS class 3, were a predictor of in-hospital outcomes and unplanned reinterventions after discharge, with left atrioventricular valve regurgitation being the strongest predictor. Use of TPS as a tool for residual lesions may identify patients predisposed to prolonged ICU hospital stay and reinterventions after discharge, providing feedback on areas in need of improvement in technique and identification of patients who warrant closer follow-up.
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Objectives To test prospectively the reproducibility and feasibility of endothelial pulse amplitude testing (Endo-PAT), a novel Food and Drug Administration-approved technology, in healthy ...adolescents. Study design We performed Endo-PAT testing on 2 different days separated by no more than 7 days in 30 healthy fasting adolescents, ages 13 to 19 years, to assess reproducibility and feasibility. The reported level of discomfort, as measured on a pain scale of 1 to 5, was documented. Results The mean difference in paired Endo-PAT indices was 0.12 (95% CI, –0.09-0.33; P = .24; intraclass correlation coefficient, 0.78), and the within-subject variation of Endo-PAT index was 0.16. The Endo-PAT index on test days 1 and 2 were 1.91 ± 0.57 and 1.78 ± 0.51 (mean plus or minus SD), respectively. All attempted studies (100%) were completed (95% CI, 88%-100%), and all completed studies (100%) could be analyzed (95% CI, 88%-100%). The median pain score was 1 on both days. Conclusion In healthy adolescents, Endo-PAT is feasible and has excellent reproducibility. This technology may provide an easy and reliable means of assessing endothelial function in the pediatric population.
The Residual Lesion Score (RLS) was developed as a novel tool for assessing residual lesions after congenital heart operations based on widely available clinical and echocardiographic ...characteristics. The RLS ranks postoperative findings as follows: Class 1 (no/trivial residua), Class 2 (minor residua), or Class 3 (major residua or reintervention before discharge for residua). The multicenter prospective RLS study aims to analyze the influence of residual lesions on outcomes in common congenital cardiac operations. We hypothesize that RLS will predict postoperative adverse events, resource utilization, mortality, and reinterventions by 1 year postoperatively.
The study cohort consisted of infants aged ≤12 months undergoing definitive surgery for complete atrioventricular septal defect, tetralogy of Fallot, dextro-transposition of the great arteries with or without intact ventricular septum, single ventricle (Norwood procedure), and coarctation or interrupted/hypoplastic arch with ventricular septal defect. Children with major congenital or acquired extracardiac anomalies that could independently affect the primary end point, which was number of days alive and out of the hospital within 30 days of surgery (60 days for Norwood procedure), were excluded. Secondary outcomes included ≥1 early major postoperative adverse event; days of intensive care unit and hospital stay, and initial and total ventilator time; mortality/transplant after discharge; unplanned reinterventions after discharge; and cost. All analyses will be performed separately by surgical operation.
This is the first multicenter prospective validation of a tool for surgical outcome assessment and quality improvement specific to congenital heart surgery.
BACKGROUND:Hypertrophic cardiomyopathy is the leading cause of sudden cardiac death (SCD) in children and young adults. Our objective was to develop and validate a SCD risk prediction model in ...pediatric hypertrophic cardiomyopathy to guide SCD prevention strategies.
METHODS:In an international multicenter observational cohort study, phenotype-positive patients with isolated hypertrophic cardiomyopathy <18 years of age at diagnosis were eligible. The primary outcome variable was the time from diagnosis to a composite of SCD events at 5-year follow-upSCD, resuscitated sudden cardiac arrest, and aborted SCD, that is, appropriate shock following primary prevention implantable cardioverter defibrillators. Competing risk models with cause-specific hazard regression were used to identify and quantify clinical and genetic factors associated with SCD. The cause-specific regression model was implemented using boosting, and tuned with 10 repeated 4-fold cross-validations. The final model was fitted using all data with the tuned hyperparameter value that maximizes the c-statistic, and its performance was characterized by using the c-statistic for competing risk models. The final model was validated in an independent external cohort (SHaRe Sarcomeric Human Cardiomyopathy Registry, n=285).
RESULTS:Overall, 572 patients met eligibility criteria with 2855 patient-years of follow-up. The 5-year cumulative proportion of SCD events was 9.1% (14 SCD, 25 resuscitated sudden cardiac arrests, and 14 aborted SCD). Risk predictors included age at diagnosis, documented nonsustained ventricular tachycardia, unexplained syncope, septal diameter z-score, left ventricular posterior wall diameter z score, left atrial diameter z score, peak left ventricular outflow tract gradient, and presence of a pathogenic variant. Unlike in adults, left ventricular outflow tract gradient had an inverse association, and family history of SCD had no association with SCD. Clinical and clinical/genetic models were developed to predict 5-year freedom from SCD. Both models adequately discriminated between patients with and without SCD events with a c-statistic of 0.75 and 0.76, respectively, and demonstrated good agreement between predicted and observed events in the primary and validation cohorts (validation c-statistic 0.71 and 0.72, respectively).
CONCLUSION:Our study provides a validated SCD risk prediction model with >70% prediction accuracy and incorporates risk factors that are unique to pediatric hypertrophic cardiomyopathy. An individualized risk prediction model has the potential to improve the application of clinical practice guidelines and shared decision making for implantable cardioverter defibrillator insertion.
REGISTRATION:URLhttps://www.clinicaltrials.gov; Unique identifierNCT0403679.
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