Pub Date : 2024-09-10DOI: 10.1097/mop.0000000000001398
Ionela Iacobas,Adrienne M Hammill
PURPOSE OF REVIEWHereditary hemorrhagic telangiectasia (HHT) diagnostic and management approach for pediatrics underwent significant advances over the last couple of years.RECENT FINDINGSIn 2020, new guidelines for HHT were published that included a pediatric section thus attracting special focus into the childhood presentation.SUMMARYCuracao criteria are specific, but not sensitive enough in children. Genetic testing is encouraged for all family members even if asymptomatic. Standardized scoring for epistaxis is strongly encouraged, as it allows monitoring and can stratify therapeutic approaches. Early screening for pulmonary and brain visceral arteriovenous malformations (AVMs) in pediatric patients with confirmed genetic alterations of HHT should be instituted. Graded trans-esophageal echocardiogram with agitated saline contrast can be used as screening method for pulmonary AVMs. As pulmonary AVMs can develop throughout lifetime, guidelines recommend repeated screening even in asymptomatic patients at least every 5 years. Signs of stroke in childhood are more subtle than in adults. Cerebral imaging in early childhood can identify brain AVMs that may benefit from early intervention. Embolization of high-risk pulmonary and cerebral AVMs should be performed at specialized centers even at pediatric age. One or two classic HHT telangiectasia can be considered diagnostic in children. Antibiotic prophylaxis with dental procedures continues to be recommended.
{"title":"Hereditary hemorrhagic telangiectasia - pediatric review.","authors":"Ionela Iacobas,Adrienne M Hammill","doi":"10.1097/mop.0000000000001398","DOIUrl":"https://doi.org/10.1097/mop.0000000000001398","url":null,"abstract":"PURPOSE OF REVIEWHereditary hemorrhagic telangiectasia (HHT) diagnostic and management approach for pediatrics underwent significant advances over the last couple of years.RECENT FINDINGSIn 2020, new guidelines for HHT were published that included a pediatric section thus attracting special focus into the childhood presentation.SUMMARYCuracao criteria are specific, but not sensitive enough in children. Genetic testing is encouraged for all family members even if asymptomatic. Standardized scoring for epistaxis is strongly encouraged, as it allows monitoring and can stratify therapeutic approaches. Early screening for pulmonary and brain visceral arteriovenous malformations (AVMs) in pediatric patients with confirmed genetic alterations of HHT should be instituted. Graded trans-esophageal echocardiogram with agitated saline contrast can be used as screening method for pulmonary AVMs. As pulmonary AVMs can develop throughout lifetime, guidelines recommend repeated screening even in asymptomatic patients at least every 5 years. Signs of stroke in childhood are more subtle than in adults. Cerebral imaging in early childhood can identify brain AVMs that may benefit from early intervention. Embolization of high-risk pulmonary and cerebral AVMs should be performed at specialized centers even at pediatric age. One or two classic HHT telangiectasia can be considered diagnostic in children. Antibiotic prophylaxis with dental procedures continues to be recommended.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"21 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142191083","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}
Pub Date : 2024-09-04DOI: 10.1097/mop.0000000000001397
Chaitanya Maddukuri,Navya Kartha,Alexandra E Conway,Marcus S Shaker
PURPOSE OF REVIEWTo share important highlights on the management of anaphylaxis from the latest 2023 practice parameter.RECENT FINDINGSThe 2023 Allergy Immunology Joint Task Force on Practice Parameters (JTFPP) anaphylaxis practice parameter provides updated anaphylaxis guidance. Criteria for the diagnosis of anaphylaxis are reviewed. The parameter highlights that while anaphylaxis is not more severe in younger children, age-specific symptoms can vary. Activation of emergency medical services may not be required in patients who experience prompt resolution of symptoms following epinephrine use and caregivers are comfortable with observation. For children weighing <15 kg, the anaphylaxis parameter suggests the clinician may prescribe either the 0.1 mg or the 0.15 mg epinephrine autoinjector, with the 0.3 mg autoinjector prescribed for those weighing 25 kg or greater. In patients with heart disease, discontinuing or changing beta blockers and/or angiotensin converting enzyme inhibitors may pose a larger risk for worsened cardiovascular disease compared with risk for severe anaphylaxis with medication continuation. Furthermore, in patients with a history of perioperative anaphylaxis, shared decision-making based on diagnostic testing and clinical history is recommended prior to repeat anesthesia use. Beyond the recent parameter update, novel contemporary therapies can decrease risk of community anaphylaxis.SUMMARYThe 2023 JTFPP Anaphylaxis Guidelines offer up-to-date guidance for the diagnosis and management of anaphylaxis in infants, children, and adults.
{"title":"Pearls for practice from the 2023 joint task force anaphylaxis practice parameter.","authors":"Chaitanya Maddukuri,Navya Kartha,Alexandra E Conway,Marcus S Shaker","doi":"10.1097/mop.0000000000001397","DOIUrl":"https://doi.org/10.1097/mop.0000000000001397","url":null,"abstract":"PURPOSE OF REVIEWTo share important highlights on the management of anaphylaxis from the latest 2023 practice parameter.RECENT FINDINGSThe 2023 Allergy Immunology Joint Task Force on Practice Parameters (JTFPP) anaphylaxis practice parameter provides updated anaphylaxis guidance. Criteria for the diagnosis of anaphylaxis are reviewed. The parameter highlights that while anaphylaxis is not more severe in younger children, age-specific symptoms can vary. Activation of emergency medical services may not be required in patients who experience prompt resolution of symptoms following epinephrine use and caregivers are comfortable with observation. For children weighing <15 kg, the anaphylaxis parameter suggests the clinician may prescribe either the 0.1 mg or the 0.15 mg epinephrine autoinjector, with the 0.3 mg autoinjector prescribed for those weighing 25 kg or greater. In patients with heart disease, discontinuing or changing beta blockers and/or angiotensin converting enzyme inhibitors may pose a larger risk for worsened cardiovascular disease compared with risk for severe anaphylaxis with medication continuation. Furthermore, in patients with a history of perioperative anaphylaxis, shared decision-making based on diagnostic testing and clinical history is recommended prior to repeat anesthesia use. Beyond the recent parameter update, novel contemporary therapies can decrease risk of community anaphylaxis.SUMMARYThe 2023 JTFPP Anaphylaxis Guidelines offer up-to-date guidance for the diagnosis and management of anaphylaxis in infants, children, and adults.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"10 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142224851","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}
Pub Date : 2024-09-03DOI: 10.1097/mop.0000000000001396
Daniel DiGiacomo,Sara Barmettler
PURPOSE OF REVIEWSecondary hypogammaglobulinemia, or low serum immunoglobulins, is associated with a variety of medications or medical conditions and may be symptomatic and lead to increased infectious risk. There is limited data regarding the study of acquired, or secondary, hypogammaglobulinemia (SHG) in pediatrics. The data to date has suffered from methodologic issues including retrospective study design, lack of baseline immunoglobulin measurements, and limited longitudinal follow-up.RECENT FINDINGSThere is emerging research on the impact of B-cell depleting therapies, specifically rituximab and chimeric antigen T-cells, along with other autoimmune and malignant disease states, in the development of SHG in pediatric patients. This review will also summarize other relevant pediatric conditions related to SHG.SUMMARYThe clinical relevance of SHG in pediatrics is increasingly appreciated. Improved understanding of the specific etiologies, risk factors, and natural history of SHG have informed screening and management recommendations.
综述目的继发性低丙种球蛋白血症或低血清免疫球蛋白症与多种药物或医疗条件有关，可能会出现症状并导致感染风险增加。有关儿科获得性或继发性低丙种球蛋白血症（SHG）的研究数据十分有限。最新发现：关于 B 细胞耗竭疗法（特别是利妥昔单抗和嵌合抗原 T 细胞）以及其他自身免疫性疾病和恶性疾病对儿科患者 SHG 发病的影响的研究正在兴起。本综述还将总结与 SHG 相关的其他儿科疾病。摘要 SHG 在儿科的临床意义日益受到重视。对SHG的具体病因、风险因素和自然病史的进一步了解为筛查和管理建议提供了依据。
{"title":"Secondary hypogammaglobulinemia: diagnosis and management of a pediatric condition of clinical importance.","authors":"Daniel DiGiacomo,Sara Barmettler","doi":"10.1097/mop.0000000000001396","DOIUrl":"https://doi.org/10.1097/mop.0000000000001396","url":null,"abstract":"PURPOSE OF REVIEWSecondary hypogammaglobulinemia, or low serum immunoglobulins, is associated with a variety of medications or medical conditions and may be symptomatic and lead to increased infectious risk. There is limited data regarding the study of acquired, or secondary, hypogammaglobulinemia (SHG) in pediatrics. The data to date has suffered from methodologic issues including retrospective study design, lack of baseline immunoglobulin measurements, and limited longitudinal follow-up.RECENT FINDINGSThere is emerging research on the impact of B-cell depleting therapies, specifically rituximab and chimeric antigen T-cells, along with other autoimmune and malignant disease states, in the development of SHG in pediatric patients. This review will also summarize other relevant pediatric conditions related to SHG.SUMMARYThe clinical relevance of SHG in pediatrics is increasingly appreciated. Improved understanding of the specific etiologies, risk factors, and natural history of SHG have informed screening and management recommendations.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"40 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142191084","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}
Pub Date : 2024-08-20DOI: 10.1097/mop.0000000000001395
Timothy G Chow,Anum F Muzaffar,Santiago Alvarez-Arango
PURPOSE OF REVIEWDespite their prevalence and potential severity, non-IgE-mediated drug-induced hypersensitivity reactions (DHRs) are under-researched and poorly defined, particularly in children. Presentations range from mild cutaneous reactions to severe systemic diseases, with pathophysiological mechanisms and reliable diagnostic markers not well established. The lack of validated tests often leads to permanent drug restrictions, reliance on second-line drugs, and increased costs. Focusing on recent advancements and areas needing further research, this review aims to enhance children's recognition, diagnosis, and management of non-IgE-mediated DHRs.RECENT FINDINGSRecent studies have enhanced the understanding of immediate and delayed non-IgE-mediated drug reactions. Key findings include the Mas-related G protein-coupled receptor X2 in mast cells and the identification of HLA alleles linked to severe cutaneous adverse reactions, such as Stevens-Johnson syndrome and toxic epidermal necrolysis. Improved diagnostic techniques, including skin testing, show promise in identifying immediate and delayed non-IgE DHRs. Additionally, research highlights the impact of cofactors, drug metabolites, and co-infections on these DHRs and explores potential biomarkers for predicting reaction severity.SUMMARYNon-IgE-mediated DHRs are a significant cause of morbidity and treatment changes in pediatric patients. Recent research underscores their clinical presentations and mechanisms, paving the way for more precise diagnostic and therapeutic strategies to improve patient outcomes.
综述目的尽管非 IgE 介导的药物超敏反应（DHRs）普遍存在且可能很严重，但对其研究不足且定义不清，尤其是在儿童中。表现形式从轻微的皮肤反应到严重的全身性疾病不等，病理生理机制和可靠的诊断指标尚未完全确定。缺乏有效的检测往往导致永久性用药限制、对二线药物的依赖以及成本增加。本综述侧重于最新进展和需要进一步研究的领域，旨在加强儿童对非 IgE 介导的 DHRs 的识别、诊断和管理。主要发现包括肥大细胞中与 Mas 相关的 G 蛋白偶联受体 X2，以及与严重皮肤不良反应（如 Stevens-Johnson 综合征和中毒性表皮坏死）相关的 HLA 等位基因的鉴定。经改进的诊断技术（包括皮肤测试）有望识别即时和延迟的非 IgE DHR。此外，研究还强调了辅因子、药物代谢物和合并感染对这些 DHR 的影响，并探讨了预测反应严重程度的潜在生物标记物。最新研究强调了其临床表现和机制，为制定更精确的诊断和治疗策略以改善患者预后铺平了道路。
{"title":"Non-IgE-mediated drug-induced hypersensitivity reactions in pediatrics.","authors":"Timothy G Chow,Anum F Muzaffar,Santiago Alvarez-Arango","doi":"10.1097/mop.0000000000001395","DOIUrl":"https://doi.org/10.1097/mop.0000000000001395","url":null,"abstract":"PURPOSE OF REVIEWDespite their prevalence and potential severity, non-IgE-mediated drug-induced hypersensitivity reactions (DHRs) are under-researched and poorly defined, particularly in children. Presentations range from mild cutaneous reactions to severe systemic diseases, with pathophysiological mechanisms and reliable diagnostic markers not well established. The lack of validated tests often leads to permanent drug restrictions, reliance on second-line drugs, and increased costs. Focusing on recent advancements and areas needing further research, this review aims to enhance children's recognition, diagnosis, and management of non-IgE-mediated DHRs.RECENT FINDINGSRecent studies have enhanced the understanding of immediate and delayed non-IgE-mediated drug reactions. Key findings include the Mas-related G protein-coupled receptor X2 in mast cells and the identification of HLA alleles linked to severe cutaneous adverse reactions, such as Stevens-Johnson syndrome and toxic epidermal necrolysis. Improved diagnostic techniques, including skin testing, show promise in identifying immediate and delayed non-IgE DHRs. Additionally, research highlights the impact of cofactors, drug metabolites, and co-infections on these DHRs and explores potential biomarkers for predicting reaction severity.SUMMARYNon-IgE-mediated DHRs are a significant cause of morbidity and treatment changes in pediatric patients. Recent research underscores their clinical presentations and mechanisms, paving the way for more precise diagnostic and therapeutic strategies to improve patient outcomes.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"17 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142191097","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}
Pub Date : 2024-08-16DOI: 10.1097/mop.0000000000001394
John P Martinez,Gulnigor Ganieva,Jamie K Harrington
PURPOSE OF REVIEWSpeckle tracking echocardiography (STE)-derived measures of myocardial mechanics, referred to herewithin as strain measurements, directly assess myocardial contractility and provide a nuanced assessment of ventricular function. This review provides an overview of strain measurements and their current clinical value and utility in decision making in pediatric cardiology.RECENT FINDINGSStrain measurements are advancing understanding of how cardiac dysfunction occurs in children with acquired and congenital heart disease (CHD). Global strain measurements can detect early changes in cardiac function and are reliable methods of serially monitoring systolic function in children. Global strain measurements are increasingly reported in echocardiographic assessment of ventricular function alongside ejection fraction. Research is increasingly focused on how strain measurements can help improve clinical management, risk stratification, and prognostic insight. Although more research is needed, preliminary studies provide hope that there will be clinical benefit for strain in pediatric cardiology management.SUMMARYStrain measurements provide a more detailed assessment of ventricular function than conventional measures of echocardiographic functional assessment. Strain measurements are increasingly being used to advance understanding of normal and abnormal myocardial contractility, to increase sensitivity to detect early cardiac dysfunction, and to improve prognostic management in children with acquired and CHD.
{"title":"Echocardiographic strain imaging in the pediatric heart: clinical value and utility in decision making.","authors":"John P Martinez,Gulnigor Ganieva,Jamie K Harrington","doi":"10.1097/mop.0000000000001394","DOIUrl":"https://doi.org/10.1097/mop.0000000000001394","url":null,"abstract":"PURPOSE OF REVIEWSpeckle tracking echocardiography (STE)-derived measures of myocardial mechanics, referred to herewithin as strain measurements, directly assess myocardial contractility and provide a nuanced assessment of ventricular function. This review provides an overview of strain measurements and their current clinical value and utility in decision making in pediatric cardiology.RECENT FINDINGSStrain measurements are advancing understanding of how cardiac dysfunction occurs in children with acquired and congenital heart disease (CHD). Global strain measurements can detect early changes in cardiac function and are reliable methods of serially monitoring systolic function in children. Global strain measurements are increasingly reported in echocardiographic assessment of ventricular function alongside ejection fraction. Research is increasingly focused on how strain measurements can help improve clinical management, risk stratification, and prognostic insight. Although more research is needed, preliminary studies provide hope that there will be clinical benefit for strain in pediatric cardiology management.SUMMARYStrain measurements provide a more detailed assessment of ventricular function than conventional measures of echocardiographic functional assessment. Strain measurements are increasingly being used to advance understanding of normal and abnormal myocardial contractility, to increase sensitivity to detect early cardiac dysfunction, and to improve prognostic management in children with acquired and CHD.","PeriodicalId":10985,"journal":{"name":"Current opinion in pediatrics","volume":"119 1","pages":"512-518"},"PeriodicalIF":3.6,"publicationDate":"2024-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142191085","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}
Pub Date : 2024-08-12DOI: 10.1097/mop.0000000000001393
Wenyuan Xu,Marc Richmond
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.
{"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}
Pub Date : 2024-08-09DOI: 10.1097/MOP.0000000000001390
Alex E Wright, Will Wood, Aakash Goyal
Purpose of review: 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.
Recent findings: 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.
Summary: 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.
{"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}
Pub Date : 2024-08-08DOI: 10.1097/mop.0000000000001390
Alex E Wright,Will Wood,Aakash Goyal
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.
{"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}
Pub Date : 2024-08-07DOI: 10.1097/mop.0000000000001392
Katherine L Mascia
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.
{"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}
Pub Date : 2024-08-05DOI: 10.1097/mop.0000000000001391
Ilknur Kulhas Celik,Gholamreza Azizi,Hasibe Artac
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.
{"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}
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