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This article examines gender disparities in leadership positions in pediatric cardiology in Germany, Austria, and Switzerland (DACH). The study found that while 70% of medical students in Germany are female, this representation does not continue in senior clinical positions, including pediatric cardiology. Male doctors reported higher job satisfaction and were more likely to work full-time and aspire to become chief physicians. Female doctors reported feeling inadequate for leadership roles and experienced discrimination, particularly related to gender and motherhood. Both male and female doctors acknowledged gender disparities in training rotations. The article suggests that new professional structures, such as job-sharing, could help address these issues and make leadership positions in pediatric cardiology more attractive and flexible. [Extracted from the article]

This article discusses a novel sedation strategy using isoflurane, a volatile anesthetic, in the pediatric cardiac ICU. The study involved six patients with congenital heart defects and cardiomyopathy who required long-term ventilation after heart surgery. The results showed that isoflurane sedation was effective and safe, with advantages such as shorter ICU stays, predictable awakening, and reduced use of opioids. The authors suggest that further studies are needed to explore the use of volatile sedation in pediatric cardiology patients. [Extracted from the article]

This article discusses the role of pediatric palliative care (PPC) in German-speaking centers for heart transplantation. The study surveyed 12 centers for pediatric cardiology and found that the majority of nurses and physicians reported the availability of palliative care, with some even offering specialized pediatric palliative care. However, the consultation of PPC was mainly for end-of-life matters rather than as part of a holistic treatment plan. Nurses favored the consultation of PPC specialists, possibly due to an ethical dilemma between caring and curing. The study suggests that early integration of PPC in the trajectory of the disease could benefit pediatric cardiac patients with severe defects and compromised organ functions. [Extracted from the article]

This article discusses tachycardia-induced cardiomyopathy (TCM), a relatively uncommon but reversible form of cardiomyopathy in pediatric heart failure patients. The study conducted a comprehensive review of medical records and identified eight cases of TCM characterized by severe heart failure and impaired cardiac function. The cases were treated with antiarrhythmic, anticongestive, and/or inotropic therapy, and successful catheter ablation was performed without recurrence of tachycardia. All cases showed symptom improvement and cardiac function recovery after the management of the tachyarrhythmias. The article emphasizes the importance of recognizing the diverse manifestations of tachycardiomyopathies for accurate diagnosis and treatment. [Extracted from the article]

This article discusses the use of transient elastography (TE) in the follow-up of Fontan patients in Germany. Fontan associated liver disease (FALD) is a concern after Fontan surgery, but there are currently no consensus guidelines for diagnosing and monitoring FALD. The study found that only 20% of centers in Germany offer TE directly, with most transferring patients to another department or not offering TE at all. The study suggests that TE should be systematically introduced as a part of routine follow-up for Fontan patients to effectively monitor and screen for FALD. [Extracted from the article]

This article discusses the case of a 4-year-old girl with dilated cardiomyopathy (DCM) who presented in cardiogenic shock and severe ventricular failure. After initial therapy, she underwent left ventricular assist device (LVAD) placement. The article describes the identification and successful ablation of an arrhythmogenic substrate in the right ventricular outflow tract (RVOT), which resulted in the termination of ventricular tachycardia and subsequent recovery of ventricular function. The patient was successfully weaned off the LVAD and discharged home. This case highlights the importance of investigating and addressing arrhythmias in severe DCM to avoid heart transplantation. [Extracted from the article]

This article discusses two cases of acute yew intoxication in teenagers, which resulted in severe cardiac arrhythmias. In the first case, a boy consumed a smoothie made from yew fruit and leaves, leading to symptoms such as nausea, vomiting, and dizziness. Despite multiple attempts at treatment, the patient's condition worsened until the identification of the yew intoxication and the initiation of digoxin-specific Fab antibody fragments, which stabilized his heart rhythm. The second case involved a boy who experienced a seizure and cardiac arrest, with similar cardiac arrhythmias. The identification and removal of yew leaves, along with gastric charcoal administration, led to the normalization of his heart rhythm. The article emphasizes the importance of promptly identifying yew intoxication and implementing appropriate treatment. [Extracted from the article]

This article discusses the long-term effects of multisystem inflammatory syndrome in children (MIS-C) on microcirculation. The study conducted microcirculatory and endothelial assessments in MIS-C patients during the acute phase and three to six months later. The results showed significant microcirculatory changes associated with MIS-C, including reductions in microvascular flow, vessel density, and the proportion of perfused vessels. These abnormalities persisted even after the inflammation subsided. The findings suggest that MIS-C patients experience acute and persistent microvascular dysfunction, which may have implications for long-term cardiovascular health. [Extracted from the article]

This article discusses the therapy of an enormous extracranial carotid artery aneurysm using a covered coronary stent. The case study involves a 2-year-old boy with chronic cervical lymphadenopathy who developed a giant pseudoaneurysm of the left internal carotid artery. Due to the proximity to the skull base, surgical intervention was not possible, so endovascular stenting was chosen as the treatment option. The implantation of a covered coronary stent was successful, and the patient remains asymptomatic with a mild Horner's syndrome. The article concludes that endovascular treatment with covered coronary stent systems can be a safe and effective approach for pediatric patients with excellent mid-term outcomes. [Extracted from the article]

This article discusses the use of transjugular liver biopsy (TJLB) as a technique for monitoring liver disease in patients with Fontan palliation and congenital heart defects (CHD). The study involved four patients with Fontan circulation and three patients with CHD who underwent TJLB during routine cardiac catheterization. The procedures were successful with no complications, and the samples obtained were suitable for histopathological assessment. The authors conclude that TJLB is a safe and efficient method for obtaining liver specimens in these patient populations and should be considered as a standard approach in their follow-up care. [Extracted from the article]

This article discusses the use of B-type natriuretic peptide (BNP) levels as a prognostic tool in pediatric patients with heart failure who receive left ventricular assist devices (LVADs). The study found that BNP levels decreased in all patients after LVAD implantation, and patients who were successfully weaned from the LVAD had significantly lower BNP levels compared to those who required transplantation. The authors suggest that patients with BNP levels within the upper limit of normal after 2 months of LVAD support have a potential for successful explantation, while patients with BNP levels exceeding 3 should be transferred to transplantation. These findings may help guide clinical decisions regarding transplantation or the chance of recovery. [Extracted from the article]

This article discusses the evaluation of cardiorespiratory fitness (CRF) using a standardized stair climbing test (SCT) in comparison to standard treadmill cardiopulmonary exercise testing (CPET) and the 6-minute walking test (6MWT). The study evaluated CRF in various groups, including healthy individuals, obese adults, Fontan patients, and patients after heart transplantation. The results showed significant correlations between the SCT and CPET, indicating that the SCT can be used as an alternative or additional test to evaluate CRF in different groups. The SCT is described as an easy, cost-effective, and reliable test. [Extracted from the article]

BACKGROUND Adjuvant pembrolizumab therapy after surgery for renal-cell carcinoma was approved on the basis of a significant improvement in disease-free survival in the KEYNOTE-564 trial. Whether the results regarding overall survival from the third prespecified interim analysis of the trial would also favor pembrolizumab was uncertain. METHODS In this phase 3, double-blind, placebo-controlled trial, we randomly assigned (in a 1:1 ratio) participants with clear-cell renal-cell carcinoma who had an increased risk of recurrence after surgery to receive pembrolizumab (at a dose of 200 mg) or placebo every 3 weeks for up to 17 cycles (approximately 1 year) or until recurrence, the occurrence of unacceptable toxic effects, or withdrawal of consent. A significant improvement in disease-free survival according to investigator assessment (the primary end point) was shown previously. Overall survival was the key secondary end point. Safety was a secondary end point. RESULTS A total of 496 participants were assigned to receive pembrolizumab and 498 to receive placebo. As of September 15, 2023, the median follow-up was 57.2 months. The disease-free survival benefit was consistent with that in previous analyses (hazard ratio for recurrence or death, 0.72; 95% confidence interval [CI], 0.59 to 0.87). A significant improvement in overall survival was observed with pembrolizumab as compared with placebo (hazard ratio for death, 0.62; 95% CI, 0.44 to 0.87; P = 0.005). The estimated overall survival at 48 months was 91.2% in the pembrolizumab group, as compared with 86.0% in the placebo group; the benefit was consistent across key subgroups. Pembrolizumab was associated with a higher incidence of serious adverse events of any cause (20.7%, vs. 11.5% with placebo) and of grade 3 or 4 adverse events related to pembrolizumab or placebo (18.6% vs. 1.2%). No deaths were attributed to pembrolizumab therapy. CONCLUSIONS Adjuvant pembrolizumab was associated with a significant and clinically meaningful improvement in overall survival, as compared with placebo, among participants with clear-cell renal-cell carcinoma at increased risk for recurrence after surgery. [ABSTRACT FROM AUTHOR]

Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported. Mesenchymal-epithelial transition (MET) signaling pathway plays a role in the pathogenesis of selected patients with papillary renal cell carcinoma (PRCC). In the phase II PAPMET trial (ClinicalTrials.gov identifier: NCT02761057), cabozantinib significantly prolonged progression-free survival and improved objective response rate compared with sunitinib in patients with advanced PRCC. Here, we present the final overall survival (OS) analysis. In this multicenter, randomized phase II, open-label trial, 147 patients with advanced PRCC who have received up to one previous therapy (excluding vascular endothelial growth factor-directed agents) were assigned to sunitinib, cabozantinib, crizotinib, or savolitinib. Ultimately, savolitinib and crizotinib arms were closed because of futility. With a median follow-up of 17.5 months, the median OS was 21.5 months (95% CI, 12.0 to 28.1) with cabozantinib and 17.3 months (95% CI, 12.8 to 21.8) with sunitinib (hazard ratio, 0.83; 95% CI, 0.51 to 1.36; P = .46). The OS landmark estimates for cabozantinib and sunitinib were 50% versus 39% at 24 months and 32% versus 28% at 36 months. In conclusion, we observed no significant difference in OS across treatment arms. Although cabozantinib represents a well-supported option for advanced PRCC, the lack of survival benefit underscores the need to develop novel therapies for this disease.

Background: Patients with Kawasaki disease (KD) with coronary artery involvement require long-term cardiac care. Although respective evidence-based recommendations are missing, cardiac catheterization is still considered the gold standard for diagnosing detailed coronary pathology. Therefore, to better understand coronary artery pathology development, we conducted a survey to document and evaluate cardiac catheterization data in a European population., Methods and Results: We retrospectively analyzed cardiac catheterization data from KD children from the year 2010 until April 2023. This registry covers basic acute-phase clinical data, and more importantly, detailed information on morphology, distribution, and the development of coronary artery pathologies. A total of 164 mainly White patients (65% boys) were included. A relevant number of patients had no coronary artery aneurysm (CAA) at the cardiac catheterization, indicating that distal CAAs were almost exclusively detected alongside proximal CAAs. Patients with multiple CAAs revealed a significant positive correlation between the number of CAAs and their dimensions in diameter and in length. Location of the CAA within the coronary artery, age at onset of KD, or natal sex did not significantly influence CAA diameters, but CAAs were longer in older children and in boys., Conclusions: That distal CAAs were only present together with proximal ones will hopefully reduce diagnostic CCs in patients with KD without echocardiographically detected proximal CAAs. Furthermore, this study gives valuable insights into dimensional specifics of CAAs in patients with KD. As an ongoing registry, future analyses will further explore long-term outcomes and performed treatments, helping to refine clinical long-term strategies for patients with KD., Registration: URL: https://drks.de/; Unique Identifier: DRKS00031022.

Nonsense variations, characterized by premature termination codons, play a major role in human genetic diseases as well as in cancer susceptibility. Despite their high prevalence, effective therapeutic strategies targeting premature termination codons remain a challenge. To understand and explore the intricate mechanisms involved, we developed StopKB, a comprehensive knowledgebase aggregating data from multiple sources on nonsense variations, associated genes, diseases, and phenotypes. StopKB identifies 637 317 unique nonsense variations, distributed across 18 022 human genes and linked to 3206 diseases and 7765 phenotypes. Notably, ∼32% of these variations are classified as nonsense-mediated mRNA decay-insensitive, potentially representing suitable targets for nonsense suppression therapies. We also provide an interactive web interface to facilitate efficient and intuitive data exploration, enabling researchers and clinicians to navigate the complex landscape of nonsense variations. StopKB represents a valuable resource for advancing research in precision medicine and more specifically, the development of targeted therapeutic interventions for genetic diseases associated with nonsense variations. Database URL: https://lbgi.fr/stopkb/., (© The Author(s) 2024. Published by Oxford University Press.)

Competing Interests: Disclosure The authors report no relevant conflicts, including financial interests, activities, relationships, and affiliations. This study was supported by Grant 129784-IRG-16-188-38-IRG from the American Cancer Society (VN) and Burroughs Wellcome Fund (MG).

The SARS-CoV-2 pandemic has highlighted the importance of contact tracing for epidemiological mitigation. Contact tracing interviews (CTIs) typically rely on episodic memory, which is prone to decline over time. Here, we provide a quantitative estimate of reporting decline for age- and gender-representative samples from the United Kingdom and Germany, emulating >15,000 CTIs. We find that the number of reported contacts declines as a power function of recall delay and is significantly higher for younger subjects and for those who used memory aids, such as a scheduler. We further find that these factors interact with delay: Older subjects and those who made no use of memory aids have steeper decline functions. These findings can inform epidemiological modeling and policies in the context of infectious diseases., (© The Author(s) 2024. Published by Oxford University Press on behalf of National Academy of Sciences.)

Background: Using an iterative user-centered design process, our team developed a patient-centered adaptive supportive care system, PatientCareAnywhere, that provides comprehensive biopsychosocial screening and supportive cancer care to patients across the continuum of care adaptively. The overarching goal of PatientCareAnywhere is to improve health-related quality of life (HRQOL) and self-efficacy of patients with cancer by empowering them with self-management skills and bringing cancer care support directly to them at home. Such support is adaptive to the patient's needs and health status and coordinated across multiple sources in the forms of referrals, education, engagement of community resources, and secure social communication., Objective: This study aims to assess the usability of the new web-based PatientCareAnywhere system and examine the preliminary efficacy of PatientCareAnywhere to improve patient-reported outcomes compared with usual care., Methods: For phase 1, usability testing participants included patients with cancer (n=4) and caregivers (n=7) who evaluated the software prototype and provided qualitative (eg, interviews) and quantitative (eg, System Usability Scale) feedback. For phase 2, participants in the 3-month pilot randomized controlled trial were randomized to receive the PatientCareAnywhere intervention (n=36) or usual care control condition (n=36). HRQOL and cancer-relevant self-efficacy were assessed at baseline (preintervention assessment) and 12 weeks from baseline (postintervention assessment); mean differences between pre- and postintervention scores were compared between the 2 groups., Results: Participants were highly satisfied with the prototype and reported above-average acceptable usability, with a mean System Usability Scale score of 84.09 (SD 10.02). Qualitative data supported the overall usability and perceived usefulness of the intervention, with a few design features (eg, "help request" function) added based on participant feedback. With regard to the randomized controlled trial, patients in the intervention group reported significant improvements in HRQOL from pre- to postintervention scores (mean difference 6.08, SD 15.26) compared with the control group (mean difference -2.95, SD 10.63; P=.01). In contrast, there was no significant between-group difference in self-efficacy (P=.09)., Conclusions: Overall, PatientCareAnywhere represents a user-friendly, functional, and acceptable supportive care intervention with preliminary efficacy to improve HRQOL among patients diagnosed with cancer. Future studies are needed to further establish the efficacy of PatientCareAnywhere as well as explore strategies to enhance user engagement and investigate the optimal intensity, frequency, and use of the intervention to improve patient outcomes., Trial Registration: ClinicalTrials.gov NCT02408406; https://clinicaltrials.gov/study/NCT02408406., (©Sharon H Baik, Karen Clark, Marisol Sanchez, Matthew Loscalzo, Ashley Celis, Marianne Razavi, Dershung Yang, William Dale, Niina Haas. Originally published in JMIR Cancer (https://cancer.jmir.org), 10.07.2024.)

Aim: Optimal selection and allocation of donor hearts is a relevant aspect in transplantation medicine. Donor age and cardiac allograft vasculopathy (CAV) affect post-transplant mortality. To what extent donor age impacts intimal hyperplasia (CAV IH ) in pediatric and adult patients after heart transplantation (HTx) is understudied., Methods: In a cohort of 98 HTx patients, 58 pediatric (24.1% with adult donors) and 40 adult patients, we assessed the effect of donor age and donor-recipient age difference (D-R) on the continuous parameter of maximal intima thickness (mIT) in optical coherence tomography. We evaluated their predictive value regarding higher mIT and the prevalence of CAV IH , defined as mIT > 0.3 mm, and compared it to established CAV risk factors., Results: In the overall population, donor age correlated with mIT (p < 0.001), while in the pediatric subpopulation, both donor age and D-R correlated with mIT (p < 0.001 and p = 0.002, respectively). In the overall population, donor age was a main predictor of higher mIT and CAV IH (p = 0.001 and p = 0.01, respectively) in addition to post-transplant interval, arterial hypertension, and dyslipidemia. In the pediatric patients, dyslipidemia remained a main predictor of both higher mIT and CAV IH (p = 0.004 and p = 0.040, respectively), while donor age and D-R were not., Conclusion: While there was an effect of the non-modifiable parameter of donor age regarding maximal intimal thickness, a stronger association was seen between the modifiable risk factor dyslipidemia and higher maximal intimal thickness and CAV IH in both the overall population and the pediatric subpopulation., (© 2024. The Author(s).)

Background: In patients with renal cell carcinoma (RCC) enrolled in the phase III KEYNOTE-564 trial (NCT03142334), disease-free survival (DFS) following nephrectomy was prolonged with use of adjuvant pembrolizumab therapy versus placebo. Patient-reported outcomes (PROs) provide an important measure of health-related quality of life (HRQoL) and can complement efficacy and safety results., Patients and Methods: In KEYNOTE-564, 994 patients were randomly assigned to receive pembrolizumab 200 mg (n = 496) or placebo (n = 498) intravenously every 3 weeks for ≤17 cycles. Patients who received ≥1 dose of treatment and completed ≥1 HRQoL assessment were included in this analysis. HRQoL end points were assessed using the EORTC QLQ-C30, FKSI-DRS, and EQ VAS. Prespecified and exploratory PRO end points were mean change from baseline in EORTC QLQ-C30 GHS/QoL score, EORTC QLQ-C30 physical function subscale score, and FKSI-DRS score., Results: No clinically meaningful difference in least squares mean scores for pembrolizumab versus placebo were observed at week 52 for EORTC QLQ-C30 GHS/QoL (-2.5; 95% CI -5.2 to 0.1), EORTC QLQ-C30 physical functioning (-0.87; 95% CI -2.7 to 1.0), and FKSI-DRS (-0.7; 95% CI -1.2 to -0.1). Most PRO scores remained stable or improved for the EORTC QLQ-C30 GHS/QoL (pembrolizumab, 54.3%; placebo, 67.5%), EORTC QLQ-C30 physical functioning (pembrolizumab, 64.7%; placebo, 68.8%), and FKSI-DRS (pembrolizumab, 58.2%; placebo, 66.3%)., Conclusions: Adjuvant treatment with pembrolizumab did not result in deterioration of HRQoL. These findings together with the safety and efficacy findings support adjuvant pembrolizumab treatment following nephrectomy., Trial Registration: Clinicaltrials.gov Identifier: NCT03142334., (© The Author(s) 2023. Published by Oxford University Press.)