{"title":"Advances in understanding and managing pediatric heart failure and transplant.","authors":"Wenyuan Xu,Marc Richmond","doi":"10.1097/mop.0000000000001393","DOIUrl":"https://doi.org/10.1097/mop.0000000000001393","url":null,"abstract":"PURPOSE OF REVIEWThis article highlights the most recent advances in a review of the current literature in the field of pediatric heart failure and transplantation.RECENT FINDINGSDiagnostically, the identification of new genetic factors has contributed to a deeper understanding of cardiomyopathy in children. Novel medications like sacubitril/valsartan and Sodium-Glucose cotransporter-2 (SGLT2) inhibitors, which are now standard in the adult population are being studied in pediatric population and offer new promise of pediatric heart failure treatment. Ventricular assist devices are more commonly used in cardiomyopathy patients and single ventricle patients as a bridge to transplant. Recent pediatric heart transplant society (PHTS) data demonstrated that waitlist survival improved significantly over the past decades (i) and new treatments such as daratumumab and eculizumab have been used in high-risk populations and demonstrate promising results. TEAMMATE trial is the first multicenter randomized clinical trial (RCT) in pediatric heart transplant (HT) to evaluate the safety and efficacy of everolimus (EVL) and low-dose tacrolimus (TAC) compared to standard-dose TAC and mycophenolate mofetil (MMF). It will provide valuable information about the safety and efficacy of EVL, TAC, and MMF (ii).Donor cell-free DNA has been used more in pediatric transplant recipients and has significantly decreased invasive EMB (iii).SUMMARYThis past 5 years have witness dramatic progress in the field of pediatric heart failure and transplantation including more use of mechanical support in heart failure patients with various underlying etiology, especially use of mechanical support in single ventricle patients and the use of sacubitril/valsartan and SGLT2 inhibitors in the pediatric population. The problem of the highly sensitized transplant recipient remains, although novel therapeutics have been added to our toolbox of options to maintain healthy allograft function. Ongoing research aims to further enhance our understanding and management of pediatric heart failure, emphasizing the need for continued innovation in this complex field.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"15 1","pages":"489-495"},"PeriodicalIF":3.6,"publicationDate":"2024-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142191100","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Continued contribution to atopic innovation with dupilumab.","authors":"Alex E Wright, Will Wood, Aakash Goyal","doi":"10.1097/MOP.0000000000001390","DOIUrl":"https://doi.org/10.1097/MOP.0000000000001390","url":null,"abstract":"<p><strong>Purpose of review: </strong>Dupilumab is an emerging medication that has shown efficacy for multiple atopic conditions. This review provides insight into the common conditions that are treated, mechanism of action, and continued innovation with the medication.</p><p><strong>Recent findings: </strong>There continues to be applicability of dupilumab in treating a growing number of atopic conditions. Increasingly younger patient populations with eosinophil based conditions are being approved for this therapy. It is also considered as an additional option treatment for patients who cannot tolerate oral therapy or have adverse effects from other agents. Patients with more severe conditions are finally finding symptom stability while also being able to reduce reliance on corticosteroids. Patients as young as 6 months of age have benefited from treatment.</p><p><strong>Summary: </strong>As atopic conditions become more prevalent, dupilumab has been shown to be successful in inducing remission of symptoms. Overall, it continues to be a promising treatment for reducing the burden of disease severity as well as improving the quality of life of affected children.</p>","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281741","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Continued contribution to atopic innovation with dupilumab.","authors":"Alex E Wright,Will Wood,Aakash Goyal","doi":"10.1097/mop.0000000000001390","DOIUrl":"https://doi.org/10.1097/mop.0000000000001390","url":null,"abstract":"PURPOSE OF REVIEWDupilumab is an emerging medication that has shown efficacy for multiple atopic conditions. This review provides insight into the common conditions that are treated, mechanism of action, and continued innovation with the medication.RECENT FINDINGSThere continues to be applicability of dupilumab in treating a growing number of atopic conditions. Increasingly younger patient populations with eosinophil based conditions are being approved for this therapy. It is also considered as an additional option treatment for patients who cannot tolerate oral therapy or have adverse effects from other agents. Patients with more severe conditions are finally finding symptom stability while also being able to reduce reliance on corticosteroids. Patients as young as 6 months of age have benefited from treatment.SUMMARYAs atopic conditions become more prevalent, dupilumab has been shown to be successful in inducing remission of symptoms. Overall, it continues to be a promising treatment for reducing the burden of disease severity as well as improving the quality of life of affected children.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"68 1","pages":"537-541"},"PeriodicalIF":3.6,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142191108","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Genetic disease amongst the Plain community.","authors":"Katherine L Mascia","doi":"10.1097/mop.0000000000001392","DOIUrl":"https://doi.org/10.1097/mop.0000000000001392","url":null,"abstract":"PURPOSE OF REVIEWThe purpose of this review is to highlight recent genetic studies of the Amish and Mennonite (Plain) communities. For decades, the study of genetically isolated populations has improved our understanding and management of genetic diseases that affect all populations.RECENT FINDINGSMajor themes of current genetic research of the Amish and Mennonites include new causative gene and new candidate gene discovery, phenotype expansion of previously identified genetic disease, and a target for AAV9-mediated gene therapy. Additionally, several genome-wide association studies (GWAS) examining complex traits such as dementia, cardiometabolic disease, and age-related macular degeneration have been conducted. Finally, clinically relevant studies of attitudes of the Plain community towards genetic testing and telemedicine, as well as reviews of and management suggestions for the Amish variants of propionic acidemia and APOB-associated familial hypercholesterolemia have been recently published.SUMMARYRecent genetic studies of the Plain community continue to highlight the value of studying isolated populations to propel genetic disease discovery and treatment. Additionally, population-specific polygenic risk scores are needed for underrepresented and minority populations, to avoid exacerbating disparities in medical genetics care. Finally, it is important for clinicians to develop management guidelines for variants common in this rapidly growing population, such as propionic acidemia.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"38 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142191107","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Practical considerations in diagnosing inborn errors of immunity according to the Middle East and North Africa guidelines.","authors":"Ilknur Kulhas Celik,Gholamreza Azizi,Hasibe Artac","doi":"10.1097/mop.0000000000001391","DOIUrl":"https://doi.org/10.1097/mop.0000000000001391","url":null,"abstract":"PURPOSE OF REVIEWThe rate of inborn errors of immunity (IEI) in the Middle East and North Africa (MENA) region is generally higher than in other parts of the world. IEI patients in MENA exhibit more severe disease phenotypes. One of the most important reasons for this is delayed diagnosis. In this review, we examine issues pertinent to primary, secondary, and tertiary physicians in diagnosing IEI in children and discuss the key points for pediatricians according to the MENA guideline.RECENT FINDINGSProtocols and stepwise approaches designed by a panel of clinical immunologists included in the MENA-IEI registry network can help physicians facilitate the diagnosis of patients with IEI by providing recommendations. These recommendations for diagnostic approaches improve the care of patients within the MENA region and can also be applied to IEI patients in other parts of the world other regions.SUMMARYPhysicians in the MENA region should be aware of IEI, obtain a detailed family history, request tests that can be ordered in primary care when IEI is suspected, and refer patients to clinical immunologists without delay. Primary and secondary care physicians should be aware that patients with IEI may present with noninfectious manifestations and increased infection frequency, severity, and atypical infections.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"31 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142224853","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Management of undescended testis.","authors":"Seth Saylors, Tolulope A Oyetunji","doi":"10.1097/MOP.0000000000001387","DOIUrl":"https://doi.org/10.1097/MOP.0000000000001387","url":null,"abstract":"<p><strong>Purpose of review: </strong>Describe why this review is timely and relevant.Undescended testis, or cryptorchidism, is a common diagnosis encountered by pediatricians that requires timely collaboration with pediatric surgical specialists to optimize outcomes for these patients. As this topic continues to be heavily researched, it is imperative to understand current recommendations and emerging management options including new surgical techniques, as well as common pitfalls in care highlighted in the literature.</p><p><strong>Recent findings: </strong>Describe the main themes in the literature covered by the article.This review primarily examines current practice in management including delays in surgical referral, with unnecessary imaging being a key factor that delays time to surgery. This review briefly discusses the diagnosis of undescended testis and the various surgical techniques used including the more recently proposed laparoscopic staged traction orchiopexy (Shehata technique). The ineffectiveness of hormonal therapy is also addressed.</p><p><strong>Summary: </strong>describe the implications of the findings for clinical practice or research.This review emphasizes prompt evaluation and diagnosis of undescended testis to facilitate appropriately timed surgical intervention, which plays a major role in outcomes for these patients. Identifying patients at risk of delayed referral is an area of focus for improvement, along with better resource utilization with fewer imaging. Familiarization of surgical options can also facilitate better patient education and provider understanding of risks/benefits.</p>","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":" ","pages":""},"PeriodicalIF":2.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281742","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Shawheen J Rezaei, Danny Linggonegoro, Shehla Admani
{"title":"Neonatal skin health and associated dermatological conditions.","authors":"Shawheen J Rezaei, Danny Linggonegoro, Shehla Admani","doi":"10.1097/MOP.0000000000001372","DOIUrl":"10.1097/MOP.0000000000001372","url":null,"abstract":"<p><strong>Purpose of review: </strong>This review describes recent developments in neonatal skincare management and situates these findings within the preexisting literature on neonatal dermatology.</p><p><strong>Recent findings: </strong>The studies included in this review expand research methods evaluating skincare management to different contexts across the world. Several studies explore the roles of emollient therapy, disinfection, and skin-to-skin contact on improving neonates' long-term health outcomes. Recent findings also assess the impact of neonatal interventions on atopic dermatitis risk later in life as well as epidemiological and microbiome variables that may predict this risk. Additionally, updates on various dermatological conditions unique to neonates are discussed in further detail.</p><p><strong>Summary: </strong>Neonatal skincare management differs in notable ways from that of other age groups. The presentation of dermatologic diseases as well as the rare conditions that affect neonates make their clinical management unique. The recent literature on neonatal dermatology can help inform clinicians regarding important considerations in treating their neonatal population.</p>","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":" ","pages":"418-424"},"PeriodicalIF":2.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141237504","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Tools for history taking for adolescents.","authors":"Areej Hassan, Carly E Guss","doi":"10.1097/MOP.0000000000001360","DOIUrl":"10.1097/MOP.0000000000001360","url":null,"abstract":"","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"36 4","pages":"349-350"},"PeriodicalIF":2.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141491233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Nevi - when to refer.","authors":"Saloni A Shah, Marla N Jahnke","doi":"10.1097/MOP.0000000000001375","DOIUrl":"10.1097/MOP.0000000000001375","url":null,"abstract":"<p><strong>Purpose of review: </strong>Congenital melanocytic nevi (CMN) and acquired nevi are prevalent in pediatric populations, with distinct characteristics and management considerations. This chapter aims to equip pediatricians with knowledge to discern between benign and high-risk nevi, facilitating appropriate referrals and management within primary care settings. Risk factors associated with malignant melanoma (MM) underscore the importance of vigilant monitoring and early referral to dermatology for suspicious lesions.</p><p><strong>Recent findings: </strong>Recent findings highlight the variability in CMN presentation and the evolving diagnostic strategies, emphasizing the need for multidisciplinary approaches to optimize patient outcomes.</p><p><strong>Summary: </strong>Management of CMN involves tailored surveillance and intervention strategies, with an emphasis on early identification of high-risk features for MM and neurocutaneous melanosis (NCM). Pediatricians play a crucial role in advocating for sun protection practices and facilitating timely referrals, thereby contributing to the overall well being of pediatric patients with nevi.</p>","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"36 4","pages":"411-417"},"PeriodicalIF":2.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141491219","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Linear growth in children and adolescents with congenital adrenal hyperplasia.","authors":"Martin O Savage","doi":"10.1097/MOP.0000000000001361","DOIUrl":"10.1097/MOP.0000000000001361","url":null,"abstract":"<p><strong>Purpose of review: </strong>Congenital adrenal hyperplasia (CAH) is a relatively common disorder and one of the most challenging conditions seen by pediatric endocrinologists. Poor linear growth in CAH has been recognized for many years. There are new insights to explain this abnormality and shed light on strategies to promote normal growth.</p><p><strong>Recent findings: </strong>Published data suggest that the dose of hydrocortisone during two critical periods of rapid growth, namely infancy and at puberty, has a fundamental effect on growth velocity, and by definition adult height. To prevent over-treatment, hydrocortisone dosage should remain within the range of 10-15 mg/m 2 body surface area per day. Precursor steroids such as 17-hydroxy progesterone (17OHP) should not be suppressed to undetectable levels. In fact, 17OHP should always be measurable, as complete suppression suggests over-treatment.</p><p><strong>Summary: </strong>CAH is a challenging disorder. High-quality compliance within the consultation setting, with the patient seeing the same specialist at every visit, will be rewarded by improved long-term growth potential. Quality auxological monitoring can avoid phases of growth suppression. New therapy with CRH receptor antagonists may lead to a more nuanced approach by allowing fine tuning of hydrocortisone replacement without the need to suppress ACTH secretion.</p>","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":" ","pages":"463-466"},"PeriodicalIF":2.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140921184","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}