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BACKGROUND: Sacubitril/valsartan, an angiotensin receptor-neprilysin inhibitor (ARNI), is an established treatment for heart failure (HF) with reduced left ventricular ejection fraction. It has not been rigorously compared with angiotensin-converting enzyme inhibitors in children. PANORAMA-HF (Prospective Trial to Assess the Angiotensin Receptor Blocker Neprilysin Inhibitor LCZ696 Versus Angiotensin-Converting Enzyme Inhibitor for the Medical Treatment of Pediatric HF) is a randomized, double-blind trial that evaluated the pharmacokinetics and pharmacodynamics (PK/PD), safety, and efficacy of sacubitril/valsartan versus enalapril in children 1 month to <18 years of age with HF attributable to systemic left ventricular systolic dysfunction (LVSD). METHODS: Children with HF attributable to LVSD were randomized to sacubitril/valsartan versus enalapril to assess the efficacy and safety of sacubitril/valsartan at 52 weeks of follow-up. The primary end point of the study was to determine whether sacubitril/valsartan was superior to enalapril for the treatment of pediatric patients with HF attributable to systemic LVSD, assessed using a primary global rank end point consisting of ranking patients from worst to best on the basis of clinical events such as death, listing for urgent heart transplant, mechanical life support requirement, worsening HF, New York Heart Association (NYHA)/Ross class, Patient Global Impression of Severity (PGIS), and Pediatric Quality of Life Inventory physical functioning domain. The change from baseline to 52 weeks in NT-proBNP (N-terminal pro-B-type natriuretic peptide) was an exploratory end point. RESULTS: A total of 375 children (mean age, 8.1±5.6 years; 52% female) were randomized to sacubitril/valsartan (n=187) or enalapril (n=188). At week 52, no significant difference was observed between the 2 treatment arms in the global rank end point (Mann-Whitney probability, 0.52 [95% CI, 0.47-0.58]; Mann-Whitney odds, 0.91 [95% CI, 0.72-1.14]; P=0.42). At week 52, clinically meaningful reductions were observed in both treatment arms in NYHA/Ross, PGIS, Patient Global Impression of Change, and NT-proBNP, without significant differences between groups. Adverse events were similar between treatment arms (incidence: sacubitril/valsartan, 88.8%; enalapril, 87.8%), and the safety profile of sacubitril/valsartan was acceptable in children. CONCLUSIONS: In this study, sacubitril/valsartan did not show superiority over enalapril in the treatment of children with HF attributable to systemic LVSD using the prespecified global rank end point. However, both treatment arms showed clinically meaningful improvements over 52 weeks. REGISTRATION: URL: https://www.clinicaltrials.gov; Unique identifier: NCT02678312.
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The clinical significance of B-type natriuretic peptide (BNP) and N-terminal pro-B-type natriuretic peptide (NT-proBNP) in pediatric patients remains an area of evolving understanding, particularly regarding their utility in the presence or absence of pre-existing heart conditions. While clear cutoff values and established roles in heart failure are understood in adult patients, pediatric norms vary with age, complicating interpretation. Notably, the emergence of multi-system inflammatory syndrome in children (MIS-C) has highlighted the importance of these markers not only in the detection of acute heart failure but also as a marker of disease severity and even as a differential diagnosis tool. This review summarizes current knowledge on the utility of BNP and NT-proBNP in pediatric patients. Their unique physiology, including circulation and compensation mechanisms, likely influence BNP and NT-proBNP release, potentially even in non-heart failure states. Factors such as dynamic volemic changes accompanying inflammatory diseases in children may contribute. Thus, understanding the nuanced roles of BNP and NT-proBNP in pediatric populations is crucial for the accurate diagnosis, management, and differentiation of cardiac and non-cardiac conditions.
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Biomarcadores , Insuficiência Cardíaca , Peptídeo Natriurético Encefálico , Fragmentos de Peptídeos , Humanos , Peptídeo Natriurético Encefálico/sangue , Peptídeo Natriurético Encefálico/metabolismo , Criança , Fragmentos de Peptídeos/sangue , Biomarcadores/sangue , Insuficiência Cardíaca/sangue , Insuficiência Cardíaca/metabolismo , Insuficiência Cardíaca/diagnóstico , Cardiopatias/sangue , Cardiopatias/metabolismo , Cardiopatias/diagnóstico , Síndrome de Resposta Inflamatória Sistêmica/sangue , Síndrome de Resposta Inflamatória Sistêmica/diagnóstico , Relevância ClínicaRESUMO
In September 2023, the European Society of Cardiology (ESC) published new guidelines for the management of endocarditis. Infective endocarditis (IE) remains a significant life-threatening disease, concerning an increasingly younger age group, especially children with congenital heart disease (CHD) and young adults after multiple cardiac surgeries. This study's aim was to alert the medical community to the problem of increasing IE case numbers and IE course complexity. Of the eight patients who suffered from IE treated in 2023 in our department, we describe the four whose course was the most extraordinary. Afterward, we compared the number of IE patients treated over the years in our department. All cases described children with congenital heart disease suffering from IE. The IE clinical presentation in all patients was extremely diverse, necessitating the utilization of all available diagnostic methods. Each child underwent specialized treatment and subsequently qualified for cardiac surgery. While the etiology and treatment of IE are well established, it remains a formidable challenge for physicians. Pediatric patients who have undergone multiple cardiac surgeries constitute a steadily expanding group and are especially susceptible to IE throughout their lives. Currently, no recommendations exist for the management of endocarditis in pediatric patients. This gap compels pediatricians to adapt existing guidelines designed for adult patients and to rely on scientific reports, such as case studies.
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Comunicação Interatrial , Veias Pulmonares , Criança , Humanos , Veias Pulmonares/diagnóstico por imagem , Veias Pulmonares/cirurgia , Veias Pulmonares/anormalidades , Comunicação Interatrial/complicações , Comunicação Interatrial/diagnóstico por imagem , Comunicação Interatrial/cirurgia , DrenagemRESUMO
BACKGROUND: The growing number of adult patients after the Fontan operation requires regular surveillance tests in specialized centers. AIMS: Our study aimed to evaluate the current practice of care for Fontan patients in Poland using a multicenter survey. METHODS: Eight centers were included in the study including 5 adult congenital heart disease (ACHD) and 3 pediatric centers for adolescents. To compare the centers and facilitate interpretation of results, the Fontan Surveillance Score (FSS) was developed. The higher score is consistent with better care, with a maximum of 19 points. RESULTS: We included in the study 398 Fontan patients (243 adults and 155 adolescents [aged 14-18 years]). The median FSS was 13 points with variability between centers (interquartile range 7-14 points). Centers providing continuous care from the pediatric period until 18 years of age achieved a higher FSS compared to ACHD centers (median: 14 points vs. 12 points; P <0.001). Most of the patients, both in the ACHD (82.3%) and pediatric centers (89%), were seen annually and had a physical examination, electrocardiogram, and echocardiogram performed at each visit. However, we observed unsatisfactory utilization of tests identifying early stages of Fontan circulation failure (cardiopulmonary exercise tests, cardiac magnetic resonance, liver biochemistry and imaging, detection of protein-losing enteropathy). CONCLUSIONS: Our results showed that there is no unified surveillance approach for Fontan patients in Poland. The practice of care for adults differs from that of adolescents.
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Técnica de Fontan , Cardiopatias Congênitas , Criança , Humanos , Adulto , Adolescente , Cardiopatias Congênitas/cirurgia , Cardiopatias Congênitas/diagnóstico , Polônia , Teste de Esforço , Eletrocardiografia , Estudos RetrospectivosRESUMO
BACKGROUND: Radiofrequency catheter ablation (RFCA) of the slow pathway in atrioventricular nodal reentry tachycardia (AVNRT) is associated with high efficacy and low risk of total perioperative or late atrioventricular block. This study aimed to evaluate the efficacy, safety, and feasibility of slow-pathway RFCA for AVNRT using a zero-fluoroscopy approach. METHODS: Data were obtained from a prospective multicenter registry of catheter ablation from January 2012 to February 2018. Consecutive unselected patients with the final diagnosis of AVNRT were recruited. Electrophysiological and 3-dimensional (3D) electroanatomical mapping systems were used to create 3D maps and to navigate only 2 catheters from the femoral access. Acute procedural efficacy was evaluated using the isoproterenol and/or atropine test, with 15-min observation after ablation. Each case of recurrence or complication was consulted at an outpatient clinic during long-term follow-up. RESULTS: Of the 1032 procedures, 1007 (97.5%) were completed without fluoroscopy. Conversion to fluoroscopy was required in 25 patients (2.5%), mainly due to an atypical location of the coronary sinus (n = 7) and catheter instability (n = 7). The mean radiation exposure time was 1.95 ± 1.3 min for these cases. The mean fluoroscopy time for the entire study cohort was 0.05 ± 0.4 min. The mean total procedure time was 44.8 ± 18.6 min. There were no significant in-hospital complications. The total success rate was 96.1% (n = 992), and the recurrence rate was 3.9% (n = 40). CONCLUSION: Slow-pathway RFCA can be safely performed without fluoroscopy, with a minimal risk of complications and a high success rate.
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Bloqueio Atrioventricular , Ablação por Cateter , Taquicardia por Reentrada no Nó Atrioventricular , Humanos , Taquicardia por Reentrada no Nó Atrioventricular/diagnóstico , Estudos Prospectivos , Bloqueio Atrioventricular/etiologia , Isoproterenol , Fluoroscopia/métodos , Ablação por Cateter/métodos , Resultado do TratamentoRESUMO
Riociguat, a soluble guanylate cyclase stimulator, is approved for treatment of adults with pulmonary arterial hypertension (PAH). The safety, tolerability, and pharmacokinetics (PK) of oral riociguat in a pediatric population with PAH was assessed in PATENT-CHILD (NCT02562235), a multicenter, single-arm, 24-week, open-label, Phase 3 study. Patients aged 6-17 years in World Health Organization functional class (WHO-FC) I-III treated with stable endothelin receptor antagonists and/or prostacyclin analogs received riociguat equivalent to 0.5-2.5 mg three times daily in adults, as either oral pediatric suspension or tablets, based on bodyweight. Primary outcomes were safety, tolerability, and PK of riociguat. Twenty-four patients (mean age 12.8 years), 18 of whom were in WHO-FC II, were enrolled. Adverse events (AEs), mostly mild or moderate, were reported in 20 patients (83%). Four patients (17%) experienced a serious AE; all resolved by study end and two (8%) were considered study-drug related. Hypotension was reported in three patients and hemoptysis in one (all mild/moderate intensity). Riociguat plasma concentrations in pediatric patients were consistent with those published in adult patients. From baseline to Week 24, mean ± standard deviation increase in 6-minute walking distance was 23 ± 69 m (n = 19), and mean decrease in NT-proBNP was -66 ± 585 pg/ml (n = 14). There was no change in WHO-FC. Two patients experienced clinical worsening events of hospitalization for right heart failure. PK results confirmed a suitable riociguat dosing strategy for pediatric patients with PAH. The data suggest an acceptable safety profile with potential efficacy signals.
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Cases of severe heart damage in patients presenting with multisystem inflammatory syndrome in children are one of the most intriguing phenomena during the coronavirus disease of 2019 pandemic. The pathophysiology of myocardial changes in the course of this syndrome has not been fully understood yet. We present a case of a child with multisystem inflammatory syndrome in children and with cardiac changes corresponding to Takotsubo syndrome.
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Doenças do Tecido Conjuntivo , Infecções por Coronavirus , Pneumonia Viral , Cardiomiopatia de Takotsubo , COVID-19/complicações , Criança , Infecções por Coronavirus/epidemiologia , Humanos , Pandemias , Pneumonia Viral/epidemiologia , Síndrome de Resposta Inflamatória Sistêmica , Cardiomiopatia de Takotsubo/diagnóstico , Cardiomiopatia de Takotsubo/etiologiaRESUMO
During the winter months of 2020/2021 a wave of multisystem inflammatory syndrome in children (MIS-C) emerged in Poland. We present the results of a nationwide register aiming to capture and characterise MIS-C with a focus on severity determinants. The first MIS-C wave in Poland was notably high, hence our analysis involved 274 children. The group was 62.8% boys, with a median age of 8.8 years. Besides one Asian, all were White. Overall, the disease course was not as severe as in previous reports, however. Pediatric intensive care treatment was required for merely 23 (8.4%) of children, who were older and exhibited a distinguished clinical picture at hospital admission. We have also identified sex-dependent differences; teenage boys more often had cardiac involvement (decreased ejection fraction in 25.9% vs. 14.7%) and fulfilled macrophage activation syndrome definition (31.0% vs. 15.2%). Among all boys, those hospitalized in pediatric intensive care unit were significantly older (median 11.2 vs. 9.1 years). Henceforth, while ethnicity and sex may affect MIS-C phenotype, management protocols might be not universally applicable, and should rather be adjusted to the specific population.
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COVID-19/complicações , Síndrome de Resposta Inflamatória Sistêmica/diagnóstico , Síndrome de Resposta Inflamatória Sistêmica/epidemiologia , Adolescente , Fatores Etários , COVID-19/diagnóstico , COVID-19/epidemiologia , Criança , Pré-Escolar , Estudos de Coortes , Monitoramento Epidemiológico , Feminino , Humanos , Incidência , Lactente , Unidades de Terapia Intensiva Pediátrica , Masculino , Síndrome de Linfonodos Mucocutâneos/complicações , Síndrome de Linfonodos Mucocutâneos/diagnóstico , Síndrome de Linfonodos Mucocutâneos/epidemiologia , Polônia/epidemiologia , Prevalência , Sistema de Registros , SARS-CoV-2 , Índice de Gravidade de Doença , Fatores SexuaisAssuntos
Bioprótese , Implante de Prótese de Valva Cardíaca , Próteses Valvulares Cardíacas , Síndrome do Coração Esquerdo Hipoplásico , Insuficiência da Valva Tricúspide , Pré-Escolar , Próteses Valvulares Cardíacas/efeitos adversos , Implante de Prótese de Valva Cardíaca/efeitos adversos , Humanos , Síndrome do Coração Esquerdo Hipoplásico/diagnóstico por imagem , Síndrome do Coração Esquerdo Hipoplásico/cirurgia , Masculino , Desenho de Prótese , Resultado do Tratamento , Insuficiência da Valva Tricúspide/cirurgiaRESUMO
Hypoplastic left heart syndrome (HLHS) was first described by Lev in 1952, but it was not until 1958 that it received a name from Noonan and Nadas. For the next several decades, the defect was considered untreatable. In 1979, William Norwood and his colleagues from Boston initiated a program to evaluate staged surgical management for infants with HLHS. The Norwood operation has became a milestone in the effective palliation for neonates born with HLHS. Today, the Norwood procedure is the first step of a three-stage heart surgery aimed at creating a new circulatory pathway (i.e., the Fontan pathway).
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Procedimentos Cirúrgicos Cardíacos , Síndrome do Coração Esquerdo Hipoplásico , Procedimentos de Norwood , Humanos , Síndrome do Coração Esquerdo Hipoplásico/cirurgia , Lactente , Recém-Nascido , Cuidados Paliativos , Estudos Retrospectivos , Resultado do TratamentoRESUMO
The COVID-19 pandemic has impacted healthcare systems worldwide. Little is known about the impact of the pandemic on medical and psycho-social aspects of children with rare diseases such as pulmonary arterial hypertension and their parents. The study is based on children registered in The Database of Pulmonary Hypertension in the Polish Population and a parent-reported survey deployed during the first 6 months of the pandemic. The questionnaire consisted of six question panels: demographic data, fear of COVID-19, General Anxiety Disorder-7 (GAD-7), social impact of pandemic, patients' medical status, and alarming symptoms (appearance or exacerbation). Out of 80 children registered, we collected 58 responses (72.5% response rate). Responders (parents) were mostly female (n = 55; 94.8%) at a mean age of 40.6 ± 6.9 years. Patients (children) were both females (n = 32; 55%) and males with a mean age of 10.0 ± 5.1 years. Eleven (19%) children had symptoms of potential disease exacerbation. Eight parents (72.7%) decided for watchful waiting while others contacted their GPs or cardiologists (n = 6; 54.5%). Three children had to be hospitalized (27.3%). Most planned hospitalizations (27/48; 56.2%) and out-patient visits (20/35; 57.1%) were cancelled, delayed, or substituted by telehealth services. Among the participating parents, the study shows very high levels of anxiety (n = 20; 34.5%) and concern (n = 55; 94.8%) and the need for detailed information (52; 89.6%) regarding COVID-19 and medical service preparedness during the pandemic. The COVID-19 pandemic has influenced child healthcare and caused high levels of anxiety among parents.
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Angioplastia/história , Cateterismo Cardíaco/história , Cardiologia/história , Cardiopatias Congênitas/cirurgia , Adolescente , Adulto , Angioplastia/métodos , Animais , Cateterismo Cardíaco/métodos , Procedimentos Cirúrgicos Cardíacos/história , Cardiologia/normas , Cardiologia/tendências , Criança , Cães , Feminino , Artéria Femoral/cirurgia , História do Século XVIII , História do Século XIX , História do Século XX , Humanos , Lactente , Recém-Nascido , Masculino , Pediatria/normas , Pediatria/tendências , Valva Pulmonar/cirurgia , Adulto JovemRESUMO
Atrial septal defect is the most common congenital heart lesion in adults. Although atrial septal defect closure is recommended in those with right heart enlargement or paradoxical embolism, data informing such indications in adults are quite limited. This population has many unique characteristics and needs. In recent years, significant progress has been made with regard to diagnostic modalities that facilitate the diagnostic workup of these patients. However, the decisionmaking process, especially in selected adult patients population (advanced age, various concomitant diseases, poor LV function [both systolic and diastolic], pulmonary hypertension, concomitant arrhythmias or multiple defects, or deficient rims) is still not easy. Available data are predominantly focused on imaging endpoints and shortterm morbidity and mortality rather than longterm. The evidence base for outcomes with or without defect closure comes from various studies with different observation periods. Moreover, the clinical experience in diagnosing and treating that subgroup of patients is inhomogeneous between individual physicians (cardiologists, imaging specialists, operators) and between small and large experience centers. In the view of the above, the joint group of experts from the Association of Cardiovascular Interventions and the GrownUp Congenital Heart Disease Section of the Polish Cardiac Society developed the following consensus opinion in order to standardize the principles of diagnosis, indications for treatment, methods of performing procedures, and tenets of postoperative care in Poland.