Pediatric Cardiology最新文献

{"title":"Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy.","authors":"Carol A Wittlieb-Weber, Brian F Birnbaum, Chesney D Castleberry, Tyler W Cunningham, Paul Esteso, Katheryn E Gambetta, Emily A Hayes, Daphne T Hsu, Beth D Kaufman, Benjamin Kroslowitz, Ashwin K Lal, Angela Lorts, Hugo Martinez, Deepa Mokshagundam, Deipanjan Nandi, John J Parent, Frank Raucci, Nelia Soares, Jonathan H Soslow, Renata Shih, Svetlana Shugh, Chet R Villa, Sarah J Wilkens, Bethany L Wisotzkey, Jennifer Conway","doi":"10.1007/s00246-025-03917-2","DOIUrl":"https://doi.org/10.1007/s00246-025-03917-2","url":null,"abstract":"<p><p>This study seeks to understand cardiac medication use in a large cohort of males with Duchenne Muscular Dystrophy (DMD) followed prospectively with focus on current practices and adherence to consensus directed medical therapy (CDMT). DMD patients have been enrolled in the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) Dystrophinopathy Registry since 2021. Cardiac medication use was analyzed at enrollment and most recent follow-up. CDMT was defined as concurrent use of angiotensin-converting-enzyme inhibitor (ACEi)/angiotensin II receptor blocker (ARB) /angiotensin receptor-neprilysin inhibitor (ARNI) plus beta-blocker (BB) plus mineralocorticoid receptor antagonist (MRA). Two hundred sixty-five males with DMD (median age 17.5 (IQR 14.5-21.5) years) were prospectively followed; median follow-up was 11.5 (IQR 6.2-15.6) months. At most recent follow-up, 153 patients (57.7%) had decreased LV systolic function, 67 (25.3% of the cohort) had moderate or severe dysfunction. For patients with moderate or severe dysfunction, CDMT was used for 49/67 (73.1%) at most recent follow-up, similar to 36/51 (70.6%) at enrollment (p = 0.92). Target doses of CDMT were achieved for 27% of males on ACEi/ARB/ARNI, 28% on BB, and 23% on MRA. Initial analysis of a prospective registry of males with DMD showed that ~ 30% of patients with moderate or severe LV dysfunction were not on CDMT at most recent follow-up and the majority did not reach target dosing. Further understanding regarding the optimal combination of cardiac medications for DMD cardiomyopathy is needed, as is a better understanding of the barriers to CDMT optimization given increasing cardiac causes of death for DMD patients.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333729","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Do Hemodynamic and Echocardiographic Derangements After the Fontan Operation Correlate with Severity of Liver Disease on Biopsy?","authors":"Yuen Lo Yau, Dipankar Gupta, Matthew S Purlee, Lindsey M Brinkley, David M Saulino, Dalia Lopez-Colon, John-Anthony Coppola, Himesh Vyas","doi":"10.1007/s00246-025-03930-5","DOIUrl":"https://doi.org/10.1007/s00246-025-03930-5","url":null,"abstract":"<p><p>Liver biopsy remains the gold standard for evaluating Fontan-associated liver disease. Prior studies show that unfavorable Fontan hemodynamics are associated with a higher risk of developing liver disease. We sought to determine whether hemodynamics pre- and post-Fontan, severity of atrioventricular valve regurgitation (AVV), and systemic ventricular dysfunction by echocardiography correlate with the degree of liver disease on biopsy. In this single-center retrospective study, we included patients with Fontan physiology who had a liver biopsy between January 2008 and May 2024. Pre- and post-Fontan catheterization data, as well as post-Fontan echocardiogram data, were reviewed. Severity of liver fibrosis was classified by the congestive hepatic fibrosis score. Data was analyzed using dichotomization (low grade score 0-2B and high grade 3-4) with χ² and Fisher's tests. 154 Post-Fontan liver biopsies corresponding to 120 patients were analyzed. The median time between the Fontan operation and liver biopsy was 23 years [IQR 17, 28]. The median Glenn pressure was 14 mmHg at pre-Fontan catheterization [IQR 11, 15]. On post-Fontan catheterization, median Fontan pressure was 18 mmHg [IQR 16, 22], and median systemic oxygen saturation was 92% [IQR 85, 95]. High-grade hepatic fibrosis did not significantly correlate with time from Fontan, elevated Glenn pressure, post-Fontan systemic venous pressure, or post-Fontan systemic ventricular function. The correlation between moderate or worse systemic AVV regurgitation approached but did not reach statistical significance (p = 0.051). No correlation was found between time from Fontan procedure, pre- or post-Fontan hemodynamics, significant AVV regurgitation, or degree of systemic ventricular dysfunction with high-grade liver fibrosis in patients with Fontan physiology.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333730","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cerebral Autoregulation and Optimal Blood Pressure from Birth to Surgery in Neonates with Critical Congenital Heart Disease.","authors":"Angelica Vasquez, Eliza Gentzler, David Bateman, Diana Vargas Chaves, David Kalfa, Emile Bacha, Ganga Krishnamurthy, Nimrod Goldshtrom","doi":"10.1007/s00246-025-03921-6","DOIUrl":"https://doi.org/10.1007/s00246-025-03921-6","url":null,"abstract":"<p><p>Increased time to surgery has been correlated with increasing cerebral fractional oxygen extraction without compensatory increase in cerebral blood flow in neonates with critical congenital heart disease (cCHD) suggesting an overwhelmed cerebral autoregulation system. Autoregulation analysis can provide precision targets such as optimal blood pressure but it remains unclear how autoregulation evolves in days preceding surgery. Our hypothesis is that longer wait times between birth and surgery are associated with increasing periods of impaired autoregulation. A retrospective observational study evaluating the time spent within optimal blood pressure (MAP_OPT) range identified by cerebral autoregulation analysis in full term newborns with cCHD awaiting neonatal surgery. Time domain autoregulation methods using continuous blood pressure and cerebral near infrared spectroscopy were employed to generate daily MAP_OPT and time with impaired autoregulation. Sixty subjects had a median [IQR (interquartile range)] of 5.2 days [3.9-7.9] of recordings from birth to surgery. Subjects spent a median of 7.6%, [5.3-13%] with impaired autoregulation. Time within MAP_OPT decreases slightly within the first ten days of life when controlling for confounders. Subjects spent a mean (± standard deviation) of 40.8% (± 15.5%) of each day within MAP_OPT (51.3 ± 6.1 mmHg) and a median of 28.1% [13.6-45.5] time below MAP_OPT. Mixed effects regression showed that subjects spent 1.93% less time each day within MAP_OPT range (p = 0.001) over time but there was no difference in time spent below MAP_OPT. Full term neonates with cCHD experience variable periods of impaired autoregulation throughout the pre-operative period and spend less time within MAP_OPT over that time. More work is needed to understand if autoregulation is a potential contributor to previously described association between longer wait time to surgery and increased white matter injury.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144326508","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effect of Transcatheter Closure of the Ductus Arteriosus on Right Ventricular Function in Preterm Neonates.","authors":"Sanjeev Aggarwal, Gilda Kadiu, Girija Natarajan","doi":"10.1007/s00246-025-03894-6","DOIUrl":"https://doi.org/10.1007/s00246-025-03894-6","url":null,"abstract":"<p><strong>Background: </strong>A large non-restricted left-to-right shunt across a patent ductus arteriosus (PDA) with the resultant volume and pressure load may impact right ventricle (RV) function. We aimed to compare RV function on echocardiogram before and soon after transcatheter closure of PDA in preterm neonates.</p><p><strong>Method: </strong>This single-center retrospective study included preterm infants ≤ 2 kg who underwent PDA transcatheter closure. Echocardiograms before and within 24 h after transcatheter closure were analyzed by a single reader for i) Tricuspid Annular Plane systolic excursion (TAPSE), ii) RV Velocity Time Integral (VTI) outflow, iii) RV Fractional area change (FAC), iv) Systolic-to-diastolic time (SD) ratio from the tricuspid valve regurgitation, and v) Stroke distance, the product of VTI and heart rate. Paired t test was used to compare parameters before and after device closure.</p><p><strong>Results: </strong>Our cohort (n = 97), 54.6% of whom were males, had a mean (Standard Deviation) gestational age of 24.9 (1.9) weeks and birth weight of 742 (244) grams. The median (IQR) age at procedure was 29 (21-45) days. We found evidence of RV dysfunction (abnormal TAPSE in 14%, FAC in 66.5% and SD in 100%) at baseline with significant improvement 24 h after device closure in RV VTI, FAC, SD, stroke distance, and myocardial performance index.</p><p><strong>Conclusion: </strong>Among preterm infants with a PDA, transcatheter closure was associated with significant short-term improvement in RV systolic function. These data provide novel intriguing insights into the potential benefit of unloading of the RV through device closure in this population.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Extubation on the Operating Table in Pediatric Cardiac Surgery: A Multicenter Analysis of 986 Patients.","authors":"Mustafa Kemal Avşar, Yasin Güzel, İbrahim Özgür Önsel, Barış Kırat","doi":"10.1007/s00246-025-03920-7","DOIUrl":"https://doi.org/10.1007/s00246-025-03920-7","url":null,"abstract":"<p><p>Extubation on the operating table is increasingly utilized to minimize ventilator-associated complications and promote early recovery in pediatric cardiac surgery. However, its safety across diverse congenital heart disease (CHD) populations remains insufficiently defined. To evaluate the feasibility, safety, and clinical outcomes of on-table extubation across a broad spectrum of corrective and palliative congenital heart surgeries in children. This retrospective multicenter study included 986 pediatric patients (aged 7 days to 16 years) who underwent on-table extubation after CHD surgery between 2019 and 2025. Patients were grouped as corrective (n = 632) or palliative (n = 354) cases. Primary outcomes were reintubation and mortality. Secondary outcomes included ICU and hospital stay durations, and incidence of ventilator-associated pneumonia (VAP). Overall reintubation and mortality rates were 5.78 and 1.22%, respectively. Corrective procedures demonstrated significantly lower reintubation (4.11%) and mortality (0.63%) compared to palliative surgeries (8.76 and 2.26%, respectively; p < 0.01 and p < 0.05). Highest complication rates were observed in HLHS (reintubation and mortality 40%) and aortopulmonary shunt (53.13 and 21.88%). In contrast, Glenn and Fontan procedures showed low reintubation (1.69, 2.91%) and minimal mortality. No cases of VAP were reported. Mean ICU and hospital stays were 3.69 and 9.7 days. Of the 57 reintubation events, 23 (40.4%) occurred within 6 h of extubation, suggesting extubation failure, while 34 (59.6%) occurred between 6 and 24 h, potentially due to secondary complications. Early reintubations (0-6 h) were more common in aortopulmonary shunt (17 cases) and coarctation/IAA repair (3 cases), whereas later reintubations (6-24 h) predominated in ToF (5 cases), truncus arteriosus (4 cases), and TGA (3 cases). A moderate correlation was found between reintubation and mortality (Spearman's r = 0.45, p < 0.01). On-table extubation is a safe and feasible strategy in pediatric cardiac surgery, particularly in corrective procedures and select single-ventricle palliation. However, caution is warranted in high-risk physiologies such as HLHS and shunt-dependent circulation. Careful perioperative evaluation remains essential for optimal outcomes.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144326509","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mid-Term Longitudinal Assessment of Ventricular Function after Transcatheter Pulmonary Valve Replacement in Tetralogy of Fallot with or without Pulmonary Atresia.","authors":"Mohammad Baidoun, James Galas, Ahmad Farooqi, Gautam Singh, Ahmad Charaf Eddine","doi":"10.1007/s00246-025-03915-4","DOIUrl":"https://doi.org/10.1007/s00246-025-03915-4","url":null,"abstract":"<p><p>Tetralogy of Fallot (TOF) is the most common form of cyanotic congenital heart disease. Patients who undergo surgical repair for TOF or TOF with pulmonary atresia (PA) may develop chronic severe pulmonary regurgitation (PR), which leads to right ventricle (RV) dilation and dysfunction. Pulmonary valve replacement is performed to restore pulmonary valve function and reverse the RV remodeling. Traditionally, pulmonary valve replacement has been performed surgically, but over the past two decades, transcatheter pulmonary valve replacement (TPVR) has emerged as an alternative approach. However, the optimal timing for TPVR remains a clinical challenge and a highly debated topic. Our study's primary aim is to evaluate the mid-term effect of TPVR on biventricular systolic function in patients with long-standing PR post-repair of TOF or TOF with PA. This retrospective single-center study included 30 patients with repaired TOF or TOF/PA who underwent first-time TPVR between 2012 and 2022. Echocardiographic assessments of right ventricular (RV) and left ventricular (LV) function were conducted at baseline and up to 3-5 years post-TPVR. Key parameters included RV fractional area change (FAC), RV strain, RV dP/dt, LV ejection fraction (EF), and LV longitudinal strain (LVLS). Statistical comparisons were made between pre- and post-TPVR time points. At baseline, most patients showed reduced RV and LV systolic function. Following TPVR, RV function did not show significant improvement on follow-up. However, LV longitudinal strain gradually improved over time and reached statistical significance at long-term follow-up (p = 0.004). LV EF showed an upward trend but did not reach significance. biventricular systolic function is decreased in many patients with repaired TOF or TOF/PA and long-standing severe PR. Transcatheter pulmonary valve replacement in this cohort resulted in improved LV strain but no improvement in RV function on mid-term follow up. These findings suggest the need to reconsider the timing of TPVR. Future studies should investigate whether earlier valve replacement could lead to better outcome of ventricular function in this patient population.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144317584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Artificial Intelligence-Enabled ECG to Detect Congenitally Corrected Transposition of the Great Arteries.","authors":"Sunil J Ghelani, Nikhil Thatte, William La Cava, John K Triedman, Joshua Mayourian","doi":"10.1007/s00246-025-03916-3","DOIUrl":"https://doi.org/10.1007/s00246-025-03916-3","url":null,"abstract":"<p><p>L-loop congenitally corrected transposition of the great arteries (ccTGA) is a rare congenital heart defect that may remain undiagnosed for decades and lead to significant morbidities, making it of interest for early detection. In this study, we address this gap by developing and internally testing an artificial intelligence-enabled electrocardiogram (AI-ECG) model to diagnose ccTGA from standard 12-lead ECGs. The dataset included the first ECG from 61,482 patients (0.7% with ccTGA), which was partitioned into training (70%) and testing (30%) cohorts. The convolutional neural network model achieved an area under the receiver-operating characteristic curve of 0.95 [95% CI 0.94-0.96] and an area under the precision-recall curve of 0.16 [95% CI 0.12-0.21]. The model performed well across different age groups, with slightly lower performance in patients < 1 month old. Key features identified by the model included widened QRS complexes, negative QRS complexes in leads V1-V2, and the lack of Q waves in lateral precordial leads. This study highlights the potential of AI-ECG to detect subtle patterns in rare congenital heart defects, providing a scalable method for early diagnosis and improving access to care. Future studies may include external validation in diverse clinical settings and multi-modal models to enhance performance and clinical utility.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144310275","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluating Vocal Cord Dysfunction in Infants Following the Norwood Operation: A National Pediatric Cardiology Quality Improvement Collaborative Analysis.","authors":"Liza Hashim, Alicia Chaves, Mark Conaway, Jeffrey Vergales","doi":"10.1007/s00246-025-03919-0","DOIUrl":"https://doi.org/10.1007/s00246-025-03919-0","url":null,"abstract":"<p><p>The Norwood operation is associated with postoperative complications including vocal cord dysfunction (VCD). This multicenter study evaluates VCD prevalence, risk factors, and diagnostic practices in a high-risk population. We performed a retrospective review of data from the National Pediatric Cardiology Quality Improvement Collaborative registry, examining infants who underwent the Norwood operation (S1P) with a surgical shunt and either completed stage 2 palliation or died after S1P discharge. The primary outcome was the endoscopic diagnosis of VCD after Norwood operation. Analysis of patient characteristics, operative details, and postoperative outcomes was performed to identify associations with VCD. Center variation in VCD was also assessed. Among the 2342 infants, VCD was diagnosed in 21.7%. Aortic arch reintervention (AAR) occurred in 19% and was associated with higher odds of VCD (OR 1.525, p = 0.001). Infants with VCD had higher rates of G-tube placement (42% vs. 22%, p < 0.001), though tracheostomy was uncommon (3.3%). Center analysis revealed lower rates of VCD at higher volume centers. Over time, AAR rates declined while VCD diagnoses increased. Our findings suggest that the Norwood operation plays a significant role in the development of VCD. The link between AAR and VCD is complex but may be influenced by institutional differences in evaluation practices. Given its impact on feeding and potential for persistence, VCD represents an important postoperative complication. Comprehensive postoperative care, low threshold to screen for VCD, and a multidisciplinary approach to management is needed to continually improve outcomes in infants undergoing the Norwood operation.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144302690","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Standardized Clinical Assessment and Management Plan Enhances Neonatal Outcomes in Prenatally Diagnosed Congenital Heart Disease.","authors":"Lior Kashani Ligumsky, Angela Desmond, Vanessa Kirschner, Guadalupe Martinez, Joanne Newens, Gary Satou, Kara Calkins, Yalda Afshar","doi":"10.1007/s00246-025-03923-4","DOIUrl":"https://doi.org/10.1007/s00246-025-03923-4","url":null,"abstract":"<p><p>Congenital heart disease (CHD) remains a leading cause of neonatal morbidity and mortality. The University of California Fetal Consortium implemented a Standardized Clinical Assessment and Management Plan (SCAMP) to optimize birth timing and mode of birth in pregnancies complicated by fetal CHD. This study evaluates the impact of SCAMP implementation on neonatal outcomes, specifically survival to hospital discharge, birth weight, and hospital length of stay. A retrospective cohort study was conducted comparing neonates with prenatally diagnosed CHD before (historical cohort) and after (intervention cohort) SCAMP implementation. Neonatal data, including mode of birth, birth weight, survival to discharge, and CHD classification was collected from five UC medical centers. Comparative analyses were performed using t-tests for continuous variables and chi-square tests for categorical variables. A total of 414 neonates met inclusion criteria (167 in the historical cohort, 247 in the intervention cohort). The overall neonatal survival rate to discharge was significantly higher in the intervention cohort (91.1% vs. 83.1%, p = 0.04). Survival following cesarean birth was also improved in the intervention cohort (89.1% vs. 78.1%, p = 0.04), while no significant differences were found for induction of labor or spontaneous vaginal births. Birth weight was significantly higher post-SCAMP (2977 g vs. 2838 g, p = 0.01), and hospital length of stay was significantly shorter (11.5 vs. 26 days, p < 0.01). Survival differences by CHD risk classification were not statistically significant. SCAMP implementation was associated with improved neonatal survival, increased birth weight, and reduced hospitalization duration in CHD-affected pregnancies. These findings underscore the importance of standardized perinatal management in optimizing outcomes for neonates with CHD.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144294766","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Promoting Advancements in Pediatric and Congenital Cardiology Amid Clinical Trial Challenges.","authors":"Nadia Chaudhry-Waterman, Kristin M Burns, Gail D Pearson, Shelley D Miyamoto","doi":"10.1007/s00246-025-03918-1","DOIUrl":"https://doi.org/10.1007/s00246-025-03918-1","url":null,"abstract":"<p><p>Due to improvements in the diagnosis and treatment of pediatric heart disease across the lifespan, an increasing number of these patients are surviving well into adulthood. However, compared to our adult cardiology colleagues, pediatric cardiologists do not have nearly as much evidence to guide the management of their patients. The heterogeneity of diagnoses and the relatively small number of individuals affected by each condition pose challenges in achieving adequate study power. The broad spectrum of potential morbidities and unique pathophysiology associated with the different conditions makes identifying clinically meaningful and achievable study endpoints particularly complex. In addition, recruitment and retention of study participants is difficult due to the fragile nature of patients as well as the regionalization of cardiac care, which results in long distances from study sites for many patients and families. In the past few decades, researchers in the field of pediatric and congenital cardiology have established several multicenter registries, programs, learning networks, and other collaborative efforts to help address these issues. While not a comprehensive review, this paper, which arose in part from the 2023 CardioVascular Clinical Trialists (CVCT) forum, will describe some of the most significant and impactful pediatric cardiology clinical research efforts to date and will emphasize areas that require ongoing focused attention.</p>","PeriodicalId":19814,"journal":{"name":"Pediatric Cardiology","volume":" ","pages":""},"PeriodicalIF":1.5,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144286091","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}