Outcomes in Patients with Persistent Ventricular Dysfunction After Stage I Palliation for Hypoplastic Left Heart Syndrome

We sought to describe the clinical course for patients with hypoplastic left heart syndrome and persistent ventricular dysfunction and identify risk factors for death or transplantation before stage II palliation. 138 children undergoing stage I palliation from 2004 to 2011 were reviewed. Twenty-two (16 %) patients (seven Hybrid, 15 Norwood) with two consecutive echocardiograms reporting at least moderate dysfunction were included and compared to case-matched controls. Eleven of the 22 patients with dysfunction (50 %) underwent stage II, seven (32 %) were transplanted, and four (18 %) died prior to stage II. Of the patients who survived to hospital discharge (n = 17) following stage 1, 14 (82 %) required readmission for heart failure (HF) compared to only two (10 %) for controls (p < 0.001). Among patients with ventricular dysfunction, there was an increased use of ACE inhibitors or beta-blockers (82 vs. 25 %; p = 0.001), inotropes (71 vs. 15 %; p = 0.001), ventilation (58 vs. 10 %; p = 0.001), and ECMO (29 vs. 0 %; p = 0.014) for HF management post-discharge when compared to controls. There was a lower heart transplant-free survival at 7 months in patients with dysfunction compared to controls (50.6 vs. 90.9 %; p = 0.040). ECMO support (p = 0.001) and duration of inotropic support (p = 0.04) were significantly associated with death or transplantation before stage II palliation. Patients with ventricular dysfunction received more HF management and related admissions. Longer inotropic support should prompt discussion regarding alternative treatment strategies given its association with death or transplant.     

Medical Therapy Leads to Favorable Remodeling in Left Ventricular Non-compaction Cardiomyopathy: Dilated Phenotype

Left ventricular non-compaction cardiomyopathy (LVNC) is a distinct and heterogeneous entity that can lead to progressive cardiac dysfunction and heart failure. LVNC with dilation and/or dysfunction is associated with a greater mortality risk. We hypothesized that initiation of heart failure medications in patients with LVNC and ventricular dysfunction or dilation would improve systolic function and result in favorable left ventricular remodeling. The study was a retrospective chart review. Inclusion criteria included: presence of LVNC, reduced systolic function or ventricular dilation, therapy with at least one medication (beta blocker, angiotensin-converting-enzyme inhibitor, angiotensin II receptor blocker), imaging pre- and post-initiation of therapy. Fifty-one patients met inclusion criteria. Mean age at initiation of medication was 11.5 ± 11.8 years. Follow-up was 2.4 ± 2.3 years. Three patients (6 %) were solely on a beta blocker, 15 (29 %) on ACE/ARB monotherapy, and 33 (65 %) on dual therapy. At follow-up 45/51 patients (88 %) had improvement in ejection fraction/shortening and 6/51 (12 %) had no change. Ejection fraction, shortening fraction, and left ventricular end-diastolic dimension in the cohort before and after therapeutic intervention demonstrated a 16 ± 12 % improvement in ejection fraction (p < 0.0001), an 8 ± 9 % improvement in shortening fraction (p < 0.0001), and a 0.83 ± 1.93 (p < 0.05) decrease in left ventricular end-diastolic z-score. Early diagnosis and medical treatment of LVNC with reduced systolic function and/or dilation leads to favorable remodeling evident by an improvement in ventricular systolic function and reduction of ventricular end-diastolic dimensions.     

Radiofrequency Ablation in the Sinus of Valsalva for Ventricular Arrhythmia in Pediatric Patients

The need to perform catheter ablation of ventricular arrhythmia from within the sinuses of Valsalva in a pediatric patient is uncommon. This has been reported in adults, but there are little data about the feasibility, safety or efficacy of catheter ablation in the sinuses of Valsalva in the pediatric patients. This is a retrospective review of all patients aged 18 years or less, at two separate institutions with no structural heart disease that underwent an ablation procedure for ventricular arrhythmia mapped to the sinus of Valsalva from 2010 to 2015. We identified 8 total patients meeting inclusion criteria. Median age was 16 years and the median weight was 61 kg. All patients were symptomatic or had developed arrhythmia-induced ventricular dysfunction. Ablation was performed in the left sinus in 4 patients and the right sinus in 4 patients. No ablations were required in the non-coronary sinus. All 8 patients had an acutely successful ablation using radiofrequency energy. There were no complications. At a mean follow-up of 7 months (4–15 months), all patients were known to be living. Follow-up data regarding arrhythmia were available in 6 of the 8 patients, and none had recurrence of their ventricular arrhythmia off of all antiarrhythmic medications. Radiofrequency catheter ablation of ventricular arrhythmia in the sinus of Valsalva can be done safely and effectively in pediatric patients.     

Clinical and Echocardiographic Outcome in Patients Receiving Carvedilol for Treatment of Dilated Cardiomyopathy

Objective

To determine outcome of children receiving carvedilol in addition to other standard drug therapy for treatment of dilated cardiomyopathy.

Methods

Children receiving carvedilol for treatment of dilated cardiomyopathy with moderate to severe ventricular dysfunction were included into the study. Data on history, clinical examination and investigations were obtained and detailed echocardiography findings were recorded for the initial and all subsequent visits.

Results

Thirty-three children, mean age 26?±?30 mo (range 7 mo to 138 mo) were enrolled. Carvedilol was initiated at a mean dose of 0.14?±?0.03 mg/kg/d and the maintenance dose was 0.46?±?0.14 mg/kg/d. At a follow up of 6–90 mo (mean of 28?±?23 mo), functional class using Ross classification for pediatric heart failure improved from 2.7 to 1.3. The left ventricular ejection fraction rose from a basal value of 22 %?±?7 % (10–40 %) to 42 %?±?15 % (15–65 %) (p?<?0.0001). Similarly, left ventricular fractional shortening increased significantly from 16?±?6 % (8–34 %) to 21?±?7 % (10–44 %) (p?<?0.0001). One patient deteriorated and died of refractory heart failure. Carvedilol was discontinued in two more patients temporarily due to bronchospasm during respiratory infection.

Conclusions

The present study suggests that improvement in ventricular function and clinical symptoms is seen on oral carvedilol added to standard drug therapy in pediatric patients with dilated cardiomyopathy and moderate to severe ventricular dysfunction. The drug is well tolerated with minimal side effects but close monitoring is required as it may worsen heart failure and bronchospasm.     

The effect of treatment with angiotensin-converting enzyme inhibitors on survival of pediatric patients with dilated cardiomyopathy

Summary Outcome in 81 pediatric patients with dilated cardiomyopathy was reviewed to assess whether treatment with angiotensin-converting enzyme (ACE) inhibitors affected survival. Age at onset was 3.6±0.6 years. Twenty-seven children (group 1) were treated with ACE inhibitors. Conventional therapy was used in the remaining 54 patients (group 2). There were no significant differences between the two groups in age at onset, left ventricular shortening fraction, left ventricular end-diastolic pressure, or mean pulmonary artery pressure. Patients treated with ACE inhibitors had a significantly better survival during the first year (p<0.05) with continuation of this trend throughout the second year (p=0.06). Beyond 2 years there was a tendency toward better survival in ACE inhibitor-treated patients, but the differences were no longer significant (p=0.14). These data, along with observations in adult patients with chronic cardiac failure, indicate that converting enzyme inhibitors have a beneficial effect on prolonging survival of infants and children with severe left ventricular dysfunction from dilated cardiomyopathy.     

Safety and Tolerability of Targeted Therapies for Pulmonary Hypertension in Children

The objective of this study is to evaluate the safety and tolerability of the pharmacological treatment of pulmonary hypertension in pediatric patients. It is a retrospective, longitudinal, observational study on pediatric patients undergoing treatment with pulmonary targeted therapies. 63 patients were included (51 % male), with a median age of 3.4 years (IQR, 3.6 months–10 years) and a median weight 13 kg (IQR, 6–30 kg). Congenital heart disease was the etiology of pulmonary hypertension in the majority of cases (n = 33) and 28 patients were in NYHA functional class III–IV. The most commonly used drug was sildenafil (n = 79, 56 %), followed by bosentan (n = 27, 23 %), and a combination of both (n = 14, 41 %). 34 patients had adverse reactions (54 %) with an incidence rate of 1.02 per patient per year. The most commonly reported reactions were gastrointestinal symptoms (22 %) and spontaneous erections (22 %) in males. Nine severe adverse reactions (10 %) occurred, requiring eight treatment withdrawal and one hospital admission. Treatment with targeted therapies for pulmonary hypertension is safe in the pediatric population. Severe ADRs were uncommon both in monotherapy and in combination therapy. Combination therapy was associated with a higher rate of ADRs. We observed similar survival rates in children receiving sildenafil doses according to the European Medicines Agency (EMA) recommendations or higher.     

The Effect of Carvedilol Treatment on Chronic Heart Failure in Pediatric Patients With Dilated Cardiomyopathy: A Prospective, Randomized-Controlled Study

This study aimed to evaluate the clinical efficacy and safety of carvedilol for pediatric patients with chronic heart failure caused by dilated cardiomyopathy. Seven pediatric medical centers participated in this prospective study. Pediatric patients (n = 89) were randomly divided into an experimental group (carvedilol treatment) and a control group (conventional treatment). The analysis excluded 12 patients lost during the follow-up period. Carvedilol was added to the therapy of the experimental group after at least 1 month of basic treatment with digoxin, an angiotensin-converting-enzyme inhibitor, and diuretics. The control patients received the same basic treatment but did not receive carvedilol. The initial dose of carvedilol was 0.1 (mg/kg day), and the dose was doubled every 2 weeks until the maximum tolerated dose or 0.8 (mg/kg day) was achieved. The tolerated dose was maintained for 6 months. The Ross scales and echocardiographic parameters including left ventricular diastolic diameter, left ventricular systolic diameter, left ventricular ejection fraction, left ventricular fractional shortening, and serous brain natriuretic peptide (BNP) concentration, as well as clinical progress were compared between the two groups. The Ross scales decreased by 11.94 % in the experimental group, which was more than in the control group (2.81 %). In addition, changes to other echocardiographic parameters in the experimental group also were superior to those in the control group. The serous BNP concentration in the experimental group decreased by 30.1 %, which also was more than the decrease (22.2 %) observed in the control group. Clinical improvement was demonstrated by 40 %, no change by 35 %, and clinical deterioration by 25 % of the patients in the experimental group, and by respectively 37.8, 27, and 35.2 % of the patients in the control group. These differences were not statistically significant. Only one patient demonstrated a severe adverse event, severe pulmonary infection, and complete atrioventricular block. None of the other carvedilol-treated patients experienced drug-related side effects. Carvedilol can be safely used for treatment of chronic heart dysfunction in pediatric patients with dilated cardiomyopathy. The results in this study showed an apparent improvement in the cardiac function of these patients. Additional clinical studies are required to determine the most favorable dosing levels and regimens of carvedilol before its safety and efficacy for the pediatric population can be determined conclusively.     

Behçet disease: treatment of vascular involvement in children

Behçet disease is the only primary vasculitis that affects both arteries and veins of any size. We present our treatment protocol in disease with vascular involvement in seven pediatric patients. All seven patients met the international criteria for the disease before the age of 16 years. Only one was a girl. The vascular involvement was as follows: Two patients had superficial vein thrombosis, two patients had atrial or ventricular thrombosis, one had arterial involvement with pulmonary aneurysms, and two had thrombosis of the venous sinuses in the central nervous system. The median duration of vascular involvement was 4 months (range 3–24 months) after the diagnosis of BD and was concomitant with diagnosis in three patients. All received colchicine and steroids. The ones with thrombosis in the venous system received additional azathioprine, whereas those with pulmonary arterial or cardiac involvement initially received cyclophosphamide for 150–180 mg/kg total dose (IV or oral) and then were switched to azathioprine for a further 6 months. All except the patient with pulmonary arterial involvement received a course of anticoagulation treatment as well. These patients have been followed up for a period of at least 18 months and so far are free of vascular relapses. One has developed a severe uveitis necessitating further therapy. In conclusion, features of vascular involvement should be carefully sought for in patients with Behçet disease. Effective management has enabled disease-free survival in the presented patients.     

Parvovirus B19 Myocarditis Causes Significant Morbidity and Mortality in Children

Although parvovirus B19 (PVB19) currently is the most common cause of viral myocarditis, limited pediatric data exist. Whereas other viruses infect cardiomyocytes, PVB19 targets coronary endothelium, leading to myocardial ischemia and dysfunction. A retrospective review investigated patients with polymerase chain reaction (PCR)-verified PVB19 myocarditis at Texas Children’s Hospital and Arkansas Children’s Hospital (January 2005 to August 2008). The primary end points of the study were transplant-free survival and circulatory collapse (death, mechanical support, or transplantation). For the 19 patients identified (age, 6 months to 15 years), the most common presenting symptoms were respiratory and gastrointestinal. At admission, all the patients demonstrated ventricular dysfunction requiring inotropic support (median ejection fraction, 24 %; median left ventricle end-diastolic diameter [LVEDD] z-score, 4.6). Whereas T-wave abnormalities were common, ST elevation was evident in five patients (two died and three required transplantation). Serum B-type natrietic peptide was elevated in all 12 patients tested (range, 348–8,058 pg/ml), and troponin I was high in 7 of 9 patients (range, 0.04–14.5 ng/ml). Of the 15 patients with circulatory collapse, nine received mechanical support, eight underwent successful transplantation, and five died. Only six patients (32 %) experienced transplant-free survival, and five patients had full recovery of function at discharge. In the transplant-free survival group, ST changes on presenting electrocardiography were less likely (p = 0.03), and the admission LVEDD z-score tended to be lower (3.3 vs 5.6; p = 0.08). In children, PVB19 myocarditis causes significant mortality and morbidity. Although mechanical intervention can support patients in the initial stage of decompensated heart failure, patients with PVB19 myocarditis often demonstrate persistent dysfunction requiring medical therapy and transplantation.     

Antegrade continence enema (ACE): predictors of outcome in 111 patients

Purpose

The antegrade continence enema (ACE) is used as a means of managing faecal incontinence and constipation with varying outcomes. We aim to evaluate our outcomes of ACEs and identify predictors of outcome.

Methods

A retrospective case-note review of patients ≤16 years of age undergoing an ACE (March 2000–September 2013) was carried out. Data collected included: patient demographics, functional outcomes and complications. Data are quoted as median (range) and compared using Mann–Whitney and Fisher’s exact test. Univariate analysis was performed to identify predictors of successful outcomes. P < 0.05 is significant. Successful outcome = total continence/occasional leakage and failed outcome = regular soiling and/or constipation.

Results

111 patients with complete data sets underwent an ACE [59 % male, median age = 9.5 years (3.4–16 years)] and median follow-up = 48 months (4 months–11 years 4 months). Underlying diagnoses were idiopathic constipation (n = 68), anorectal malformation (n = 27), neuropathic bowel (n = 7), Hirschsprung disease (n = 5) and gastrointestinal dysmotility (n = 4). Social continence was achieved in 87/111 (78 %). Fifteen percent of patients underwent reversal of ACE due to resolution of symptoms. There was no difference in outcomes related to diagnosis, gender, age or follow-up duration. Complication rate was 20.7 % (23/111).

Conclusions

The ACE is safe and effective in the management of intractable constipation and soiling. No predictors of outcome were identified.     

Permanent Cardiac Pacing in Pediatric Patients

Pediatric pacemaker (PM) implants comprise less than 1 % of all PM implants. This study aimed to investigate permanent cardiac pacing among the pediatric population, identifying different indications and complications of pediatric cardiac pacing, especially focusing on the effect of the pacing sites, the PM lead type, and the indications for pacing. The current work is a cross-sectional study of 103 procedures for permanent PM insertion in pediatric patients between January 2001 and December 2010. The patients were followed up 1, 3, and 6 months after implantation, then every 6 months or as needed. Evaluation included routine clinical examination, electrocardiography, chest X-ray, echocardiography, and a full analysis of the pacing system measurements. The ages of the patients ranged from 0.09 to 12 years (median, 2.3 years). The most common indication for pacing was postoperative complete heart bock, noted in 54 patients (52.4 %). Transvenous endocardial PM insertion was performed in 92 procedures (89.3 %), whereas transthoracic epicardial insertion was performed in 11 procedures (10.7 %). The most common site of pacing was the right ventricular apex (n = 64, 62 %), followed by the right ventricular outflow tract (n = 25, 24.3 %). Transthoracic epicardial PM insertion was associated with a significantly higher percentage and greater severity of complications. In this study, 65 % of the patients with left ventricle (LV) dilation before pacing showed a significant improvement in LV dimensions and function after pacing. This was noted only in those with endocardially inserted PM leads in both the congenital and the postoperative groups regardless of the pacing site. Endocardial PM insertion in children is a safe procedure with fewer complications and a lower ventricular threshold than the epicardial route. Permanent single-chamber right ventricle pacing is safe and can lead to significant improvement in LV function and dimensions. However, long-term follow-up assessment is needed for further evaluation.     

Ventricular dysfunction clinical research in infants, children and adolescents

These two issues of Progress in Pediatric Cardiology comprehensively illustrate the wealth of currently available information on the pathophysiology of heart failure, age-related myocardial responsiveness, energy metabolism, cardiopulmonary interactions, the pressure-volume relationship, the systemic inflammatory response, the management of heart failure, pediatric pharmacology, the use of heart failure therapies including digoxin, ACE inhibitors, beta-adrenergic blockers, inotropic agents, diuretics, vasodilators, calcium sensitizers, angiotensin and aldosterone receptor blockers, growth hormone, and future gene therapy. The etiology and course of ventricular dysfunction in children is poorly characterized. Furthermore, many changing developmental properties of the pediatric myocardium and differences in the etiologies of ventricular dysfunction in children compared with adults are illustrated in these articles, invalidating the concept that children can safely be considered small adults for the purpose of understanding heart failure pathophysiology and treatment. However, these articles reveal that strikingly little research in children with ventricular dysfunction exists in terms of well-designed large-scale studies of the epidemiology or multicenter controlled clinical therapeutic trials. A future research agenda is proposed to improve understanding etiologies, course and treatment of ventricular dysfunction in children that is based on organized and funded cooperative groups since no one pediatric cardiac center treats enough children with a particular etiology of ventricular dysfunction. In conclusion, significant understanding of basic mechanisms of pediatric ventricular dysfunction and effective therapies for adults with ventricular dysfunction exist. A multicenter pediatric cardiac ventricular dysfunction network would allow improved understanding of diseases and treatments, and result in evidence-based medicine for pediatric patients with ventricular dysfunction.     

Angiotensin-converting enzyme inhibitor therapy for ventricular dysfunction in infants, children and adolescents: a review

Angiotensin-converting enzyme (ACE) inhibitors have become an important part of the pharmacologic armamentarium in the battle against treatment of ventricular dysfunction. There have been a number of large controlled, randomized trials in adults with both asymptomatic and symptomatic ventricular dysfunction, which confirm the safety and efficacy of this category of drugs for the treatment of this potentially lethal condition. ACE inhibitors may be used to treat infants, children and adolescents with asymptomatic and symptomatic ventricular dysfunction as well. The data supporting their use in children is less complete than that concerning the treatment of adults. We review here the various causes of ventricular dysfunction and congestive heart failure (CHF) in infants, children, and adolescents; the data available regarding treatment of these conditions with ACE inhibitors, and the safety and efficacy of these drugs for the various conditions. The pharmacokinetics and proposed mechanisms of action of ACE inhibitors in children are reviewed, as are speculated long-term results of ACE inhibitor use in cohorts of growing children. Recommendations are made for future studies.     

Clinical Outcome,Valve Dysfunction,and Progressive Aortic Dilation in a Pediatric Population With Isolated Bicuspid Aortic Valve

The aim of this study was to explore the medium-term clinical outcome and the risk of progression of aortic valve disease and aortic dilation in pediatric patients with isolated bicuspid aortic valve (BAV). 179 pediatric patients with isolated BAV were prospectively followed from January 1995 to December 2010. Patients with severe valve dysfunction at baseline were excluded. Clinical outcome included cardiac death, infective endocarditis, aortic complications, cardiac surgery and percutaneous valvuloplasty. Echocardiographic endpoints were: progression of aortic stenosis (AS) or regurgitation (AR) and progressive aortic enlargement at different levels of the aortic root, evaluated as z-score. The median age at diagnosis was 7.8 [2.7–12.0] years. After a median followup of 5.4 [2.3–9.2] years, all patients were alive. The clinical endpoint occurred in 4 (2.2 %) patients (0.41 events per 100 patient-years). A progression of AS and AR was observed in 9 (5.0 %) and 29 (16.2 %) patients, respectively. The z-scores at the end of follow-up were not significantly different from baseline at the annulus, Valsalva sinuses and sinotubular junction, whereas a slight increase was observed at the level of the ascending aorta (1.9 vs 1.5, p = 0.046). Significant progressive aortic dilation occurred in a minority of patients (10.6, 5.6, 9.5, and 19.0 % respectively). The clinical outcome in pediatric patients with isolated BAV is favourable and the progression of aortic valve dysfunction and aortic dilation is relatively slow. These findings may be taken into account to better guide risk assessment and clinical follow-up in these patients.     

Percutaneous Closure of Atrial Septal Defects in Spontaneously Breathing Children Under Deep Sedation: A Feasible and Safe Concept

Interventional cardiac catheterization in children and adolescents is traditionally performed with the patient under general anesthesia and endotracheal intubation. However, percutaneous closure of atrial septum defect (ASD) without general anaesthesia is currently being attempted in a growing number of children. The study objective was to evaluate the success and complication rate of percutaneous ASD closure in spontaneously breathing children under deep sedation. Retrospective single centre cohort study of consecutive children undergoing percutaneous ASD closure at a tertiary care pediatric cardiology centre. Transesophageal echocardiography (TEE) and percutaneous ASD closure were performed with the patient under deep sedation with intravenous bolus of midazolam and ketamine for induction and propofol continuous infusion for maintenance of sedation in spontaneously breathing children. One hundred and ninety-seven patients (median age 6.1 years [minimum 0.5; maximum 18.8]) underwent TEE and ASD balloon sizing. Percutaneous ASD closure was attempted in 174 patients (88 %), and device implantation was performed successfully in 92 %. To achieve sufficient deep sedation, patients received a median ketamine dose of 2.7 mg/kg (0.3; 7) followed by a median propofol continuous infusion rate of 5 mg/kg/h (1.1; 10.7). There were no major cardiorespiratory complications associated with deep sedation, and only two patients (1 %) required endotracheal intubation due to bronchial obstruction immediately after induction of sedation. Seventeen patients (8 %) had minor respiratory complications and required frequent oral suctioning or temporary bag-mask ventilation. TEE and percutaneous ASD closure can be performed safely and successfully under deep sedation in spontaneously breathing children of all ages.     

Left Ventricular Tonic Contraction as a Novel Biomarker of Cardiomyopathy in Duchenne Muscular Dystrophy

Dilated cardiomyopathy (DCM) inevitably afflicts patients with Duchenne muscular dystrophy (DMD) as a consequence of cell death induced by unguarded calcium influx into cardiomyocytes. This mechanism may also inhibit muscle relaxation in early stages of cardiomyopathy. ACE inhibition (ACEi) is known to delay the onset and slow the progression of DCM in DMD. The objective of this study is to use echocardiography to assess for preclinical cardiac changes consistent with intracellular calcium dysregulation before the onset of overt ventricular dysfunction, and to evaluate how prophylactic ACEi may alter these pre-cardiomyopathic changes in the pediatric DMD population. We examined 263 echocardiograms from 70 pediatric patients with DMD. We defined abnormal tonic contraction (TC) as left ventricular internal dimension in diastole (LVIDd) Z-score < ?1.5. In our cohort, we found that TC is detectable as early as 8 years of age, and most commonly affects patients between 11 and 15 years. This effect was independent of LV mass and systolic function. Prophylactic ACEi decreased the incidence of TC (p = 0.007) and preserved cardiac function (p < 0.0001). Left ventricular TC often precedes DCM in DMD, most commonly affecting the 11- to 15-year-old age range. TC is not related to ventricular hypertrophy, but rather may be a clinical correlate of the “calcium hypothesis” of DMD pathophysiology. LV TC is thus a promising biomarker for early detection of cardiomyopathy in DMD. ACEi prophylaxis suppresses LV TC and delays the development of DCM in DMD.     

Echocardiographic Evaluation of Ventricular Function in Children With Pulmonary Hypertension

Although described in adults, it remains unclear whether ventricular dysfunction exists in pediatric patients with pulmonary hypertension (PHN). The goal of this study was to identify differences in echocardiographic indices of ventricular function among pediatric PHN patients. From 2009 to 2011, pediatric PHN patients with normal intracardiac anatomy and age-matched controls (1:3 ratio) were enrolled in this retrospective case–control study. Diagnosis of PHN was based on tricuspid regurgitation velocity or septal position estimating right-ventricular (RV) pressure >50 % systemic. Measures of RV and left ventricular systolic and diastolic function, including tissue Doppler imaging (TDI) of the mitral annulus (MA) and tricuspid annulus (TA), were compared. Enrollees included 25 PHN patients and 75 age-matched controls (mean age 7.5 years [range 1 day to 19 years]). Parameters of RV systolic and diastolic function were worse in PHN patients. Compared with controls, PHN patients had significantly decreased tricuspid valve inflow ratio, decreased TA TDI early diastolic velocities, decreased systolic velocities, increased tricuspid E/E’ ratio (all p < 0.01) and increased myocardial performance index. In an age-stratified analysis, TDI measures in PHN patients <1 year of age were similar to controls, whereas differences in TA TDI velocities and MA TDI velocities were noted in patients ≥1 year of age. Abnormalities in Doppler echocardiographic indices of ventricular systolic and diastolic function were identified in pediatric PHN patients and were more prominent with older age. These indices are promising for serial noninvasive monitoring of disease severity, but further correlation with catheterization-derived measures is needed.     

Management of asymptomatic anthracycline-induced cardiac damage after treatment for childhood cancer: a postal survey among Dutch adult and pediatric cardiologists

Asymptomatic anthracycline-induced cardiac damage (A-CD) is a serious problem among young childhood cancer survivors. The aim of this survey was to assess the current treatment policy in these patients in the Netherlands. A questionnaire was sent to all 136 departments of adult or pediatric cardiology in the Netherlands. It was returned by 61% of the departments. Sixty-six percent of the respondents started medical treatment (ie, an ACE inhibitor and/or a beta-blocker) in childhood cancer survivors with asymptomatic A-CD. Fifty-eight percent of the respondents indicated that their treatment decision was based on published findings in the literature, but none of them referred to studies evaluating the treatment of asymptomatic A-CD. A majority of adult and pediatric cardiologists started medical treatment in childhood cancer survivors with asymptomatic A-CD without knowledge of the benefits and risks of treatment in this patient group. Before ACE inhibitors and/or beta-blockers can be recommended as routine practice in childhood cancer survivors with asymptomatic A-CD, randomized controlled trials should be performed. Until then, the authors recommend centralizing the treatment of childhood cancer survivors with asymptomatic A-CD in a specialized center to cluster the available knowledge and experience.     

Oxcarbazepine oral suspension in young pediatric patients with partial seizures and/or generalized tonic–clonic seizures in routine clinical practice in China: a prospective observational study

Background

This study aimed to assess efficacy and safety of oxcarbazepine (OXC) oral suspension in pediatric patients aged 2–5 years with partial seizures (PS) and/or generalized tonic–clonic seizures (GTCS) in real-world clinical practice in China.

Methods

This 26-week, prospective, single-arm, multicenter, observational study recruited pediatric patients aged 2–5 years with PS or GTCS suitable for OXC oral suspension treatment based on physicians’ judgments from 11 medical centers in China. Enrolled subjects started OXC oral suspension treatment as monotherapy or in combination with other antiepileptic drugs. Primary efficacy outcome was the percentage of pediatric subjects achieving ≥ 50% seizure frequency reduction at the end of the 26-week treatment. Secondary efficacy-related parameters and safety parameters such as adverse events (AEs) and serious AEs (SAEs) were also monitored during the 26-week treatment period.

Results

Six hundred and six pediatric patients were enrolled and 531 (87.6%) completed the study. After 26 weeks of treatment, 93.3% subjects achieved ≥ 50% seizure frequency reduction, and 81.8% achieved 100% seizure frequency reduction compared to baseline. Among different seizure types, OXC was effective in all subjects with simple PS and in > 90% of subject with other type of seizure present in the study. AEs were observed in 49 (8.1%) subjects. Only three subjects experienced SAE. Rash (n = 18, 2.97%) was the most common AE. Only 17 subjects discontinued due to AEs.

Conclusion

This study, reporting the real-world data, further confirms the efficacy and good safety profile of OXC oral suspension in Chinese pediatric patients aged 2–5 years with PS and/or GTCS.