Pediatric Drugs最新文献

{"title":"Pediatric Myocarditis: Challenges in Diagnosis and Treatment.","authors":"Bibhuti B Das","doi":"10.1007/s40272-026-00739-4","DOIUrl":"10.1007/s40272-026-00739-4","url":null,"abstract":"<p><p>Pediatric myocarditis is a clinically diverse and often under-recognized inflammatory condition of the myocardium, with presentations that range from mild symptoms to acute heart failure. Advances in cardiac imaging, biomarker discovery, and molecular genetics have improved diagnostic precision and individualized care. Genetic studies indicate that 8-10% of pediatric patients with myocarditis carry pathogenic variants in cardiomyopathy-related genes, supporting the \"double-hit\" hypothesis where genetic predisposition interacts with viral or autoimmune triggers. This paradigm shift has underscored the relevance of precision medicine, emphasizing tailored management based on the underlying etiology and genetic background. While supportive care remains the foundation of therapy, including rest, hemodynamic monitoring, and guideline-directed medical therapy for heart failure, pharmacologic strategies are evolving. Corticosteroids are under investigation for modulating inflammation, with mixed results showing improvements in ventricular function but unclear survival benefits. Immunomodulatory agents, such as intravenous immunoglobulin and other immunosuppressive therapies, offer promise in reducing myocardial fibrosis and injury. Virus-specific therapies are increasingly utilized in confirmed viral myocarditis, improving outcomes in selected cases. Recent studies highlight the potential role of biologic agents targeting inflammatory pathways, such as interferons, interleukin modulators, and micro-RNA-based therapy in refractory cases. Despite limited pediatric-specific trial data, extrapolation from adult myocarditis studies continues to inform treatment direction. Continued translational research and pediatric-focused clinical trials are critical to optimizing treatment of myocarditis and improving long-term cardiac outcomes. Future investigations should focus on refining immunomodulatory strategies, identifying novel therapeutic targets, and enhancing risk stratification to improve individualized care.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"249-268"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146137762","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":"Puzolcabtagene Autoleucel: Pediatric First Approval.","authors":"Yahiya Y Syed","doi":"10.1007/s40272-026-00743-8","DOIUrl":"10.1007/s40272-026-00743-8","url":null,"abstract":"<p><p>Puzolcabtagene autoleucel [^®] is an autologous, humanized anti-CD19 chimeric antigen receptor (CAR) T-cell therapy developed by Chongqing Precision Biotech for the treatment of B-cell acute lymphoblastic leukemia (B-ALL), B-cell leukemias and lymphomas, and non-Hodgkin lymphoma. CAR T-cell therapy is a personalized immunotherapy in which a patient's own T cells are genetically engineered to recognize and eliminate malignant cells through direct cytotoxicity, induction of apoptosis, and TNF ligand-mediated signaling pathways. To reduce immunogenicity, puzolcabtagene autoleucel incorporates a humanized CD19-specific single-chain variable fragment. On 4 November 2025, puzolcabtagene autoleucel received its first approval in China for the treatment of CD19⁺ relapsed or refractory (R/R) B-ALL in patients aged 3-21 years. This article summarizes the key developmental milestones leading to this first approval.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"321-324"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146202551","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":"Effect of Lactoferrin in Obese Children and Adolescents with Metabolic Dysfunction-Associated Steatotic Disease: A Randomized Controlled Study.","authors":"Doaa El Amrousy, Dalia El-Afify, Esraa Abd Al-Fattah Sorour, Shymaa Elrifaey","doi":"10.1007/s40272-026-00742-9","DOIUrl":"10.1007/s40272-026-00742-9","url":null,"abstract":"<p><strong>Background and objective: </strong>Lactoferrin is an iron-binding glycoprotein existing in mammalian milk. It has immunomodulatory, antioxidant, and anti-inflammatory properties, and it can also regulate metabolism. The present study investigated the effect of lactoferrin in obese children and adolescents with metabolic dysfunction-associated steatotic liver disease.</p><p><strong>Methods: </strong>This randomized controlled trial was performed on 73 obese children and adolescents with metabolic dysfunction-associated steatotic liver disease. The patients were randomized into two groups: group I, who received lactoferrin 100 mg once daily for 3 months, and group II, who did not receive lactoferrin or placebo as the control group. Both groups were on a hypocaloric diet. Measurements of weight, body mass index, alanine aminotransferase, aspartate aminotransferase, fasting blood glucose, fasting insulin, homeostatic model assessment method of insulin resistance, lipid profile, homocysteine, malondialdehyde, interleukin-6, and interleukin-10 were assessed at baseline and after 3 months of treatment.</p><p><strong>Results: </strong>Seventy patients completed the study. After 3 months of treatment, the lactoferrin group had a significantly lower weight, body mass index (28.7 ± 1.48 vs 30.2 ± 1.45, p < 0.001), alanine aminotransferase (47.7 ± 4.4 vs 56.4 ± 4.3, p < 0.001), homeostatic model assessment method of insulin resistance (2.86 ± 0.43 vs 3.08 ± 0.4, p = 0.03), aspartate aminotransferase, fasting blood glucose, total cholesterol, triglycerides, homocysteine, malondialdehyde, and interleukin-6 compared with the control group and the pre-treatment levels.</p><p><strong>Conclusions: </strong>Lactoferrin may help in weight reduction, improve insulin resistance and lipid profile, and decrease oxidative stress and inflammation in obese children and adolescents with metabolic dysfunction-associated steatotic liver disease.</p><p><strong>Clinical trial registration: </strong>The clinical trial was registered at Pan African Clinical Trial Registry with ID: PACTR202302847529384,T https://pactr.samrc.ac.za/TrialDisplay.aspx?TrialID=24309 .</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"285-294"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13121533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147434510","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ferric Maltol: Pediatric First Approval.","authors":"Susan J Keam","doi":"10.1007/s40272-026-00748-3","DOIUrl":"10.1007/s40272-026-00748-3","url":null,"abstract":"<p><p>Ferric maltol (ACCRUFeR^®), is an oral iron replacement product being developed by Shield Therapeutics plc for the treatment of iron deficiency (ID). Ferric maltol was originally approved for the treatment of iron deficiency anemia (IDA) in adult patients with inflammatory bowel disease in 2016 (with an indication extension for ID/IDA in adults in 2018) in the EU, and in ID in adults in 2019 in the USA. In December 2025 the indication in the USA was extended to include pediatric patients aged ≥ 10 years. A similar indication extension in pediatric patients aged ≥ 12 years is being evaluated in the EU. This article summarizes the milestones in the development of ferric maltol leading to this first pediatric approval for the treatment of ID.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"325-329"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147575152","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":"GLP-1 Receptor Agonist Use Across Preconception, Pregnancy, and the Postpartum Periods.","authors":"Florence Porterfield, Serena Michelle Ogunwole, Jacqueline Maya, Fatima Cody Stanford","doi":"10.1007/s40272-026-00746-5","DOIUrl":"10.1007/s40272-026-00746-5","url":null,"abstract":"<p><p>Obesity in reproductive-age women is associated with increased risk of cardiometabolic disease, infertility, and pregnancy-related complications affecting both the mother and fetus. Recently, the introduction of highly effective glucagon-like peptide-1 (GLP-1) receptor agonists has led to increased utilization of obesity medications in this population. While these agents are not recommended during pregnancy, their use in the preconception, periconception, and postpartum periods is increasing. Yet, data on their safety and clinical utility during these reproductive windows remain limited. This review summarizes the current literature on the risks and benefits of GLP-1 receptor agonist use for both the mother and fetus, highlighting the need for ongoing high-quality research in this population. This review highlights key clinical considerations across reproductive stages, emphasizes the primacy of human exposure data where available, and identifies critical evidence gaps requiring future prospective study.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"239-248"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593881","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":"Advances in Management of Eosinophilic Esophagitis in Pediatrics.","authors":"Julia Sessions, Maureen Bauer, Calies Menard-Katcher, Nathalie Nguyen","doi":"10.1007/s40272-026-00744-7","DOIUrl":"10.1007/s40272-026-00744-7","url":null,"abstract":"<p><p>Eosinophilic esophagitis is a chronic immune-mediated inflammatory disorder of the esophagus comprising symptoms of esophageal dysfunction with eosinophilic inflammation. The diagnosis is based on histopathologic criteria with elevated esophageal eosinophil count and the presence of characteristic symptoms such as dysphagia. The goal of treatment is to improve symptoms, induce histopathologic remission, and prevent progression to fibrostenotic disease. In pediatrics, maintaining growth and development are essential considerations in treatment and play a role in the selection of treatment. Mainstays of pharmacologic treatment include proton pump inhibitors, topical corticosteroids, and dietary therapy, all of which are first-line treatment options for pediatric patients with eosinophilic esophagitis. More recently, a biologic therapy, dupilumab (a monoclonal antibody against interleukin-4⍺ receptor), has been approved for eosinophilic esophagitis in patients aged ≥1 year and ≥15 kg. Esophageal dilations are used as an adjunct to medical or dietary therapy for fibrostenotic eosinophilic esophagitis, but do not treat the underlying inflammation. Several emerging biologics that target pathways implicated in the inflammatory mechanisms of eosinophilic esophagitis are currently under investigation in adults; however, studies are lacking in much of the pediatric population. This review outlines updates in treatment approaches for pediatric eosinophilic esophagitis including the use of proton pump inhibitors, new topical corticosteroid formulations, paradigm shifts in dietary therapy approaches, and use of biologics with a focus on pediatric disease.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"269-283"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147326653","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":"Oral Lamivudine for Extended HIV Postnatal Prophylaxis.","authors":"Philippe Van de Perre, Nicolas Meda, Souleymane Tassembedo, Jean Pierre Moles, Chipepo Kankasa, Anais Mennecier, Mwiya Mwiya, Nicolas Nagot, Thorkild Tylleskär","doi":"10.1007/s40272-026-00745-6","DOIUrl":"10.1007/s40272-026-00745-6","url":null,"abstract":"<p><p>Despite considerable progress in preventing mother-to-child transmission (PMTCT) of human immunodeficiency virus (HIV), eliminating paediatric HIV has not been achieved. Extended postnatal prophylaxis (ePNP), defined as prophylaxis administered to an HIV-exposed child after PMTCT perinatal prophylaxis ends, has been evaluated and is now proposed as an improved approach towards elimination. This approach should be urgently incorporated into international PMTCT recommendations. The antiretroviral drugs most commonly studied as ePNP, either alone or in combination, are lamivudine, nevirapine, lopinavir/ritonavir and zidovudine. In this study, we examined the efficacy, safety, pharmacology, genetic barrier to resistance and practicality of various ePNP regimens. Regimens combining multiple antiretroviral drugs are no more effective than single-drug regimens in terms of protective efficacy but they are associated with increased toxicity. On the basis of these criteria, we recommend lamivudine as the preferred ePNP drug or nevirapine as an alternative. Guided by maternal HIV viral load, ePNP may be particularly indicated, as it could ensure that the prophylaxis provides the greatest benefit/risk to children at highest risk. Long-acting injectable antiretroviral drugs and broadly neutralising antibodies (bNAbs) have yet to be fully evaluated in neonates, infants and children; however, they may offer new alternatives in the future.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"229-238"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13121178/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147343602","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Fixed Dosing of Leuprolide Acetate, a GnRH Agonist, in Children with Central Precocious Puberty: A Population Pharmacokinetic Justification.","authors":"Chay Ngee Lim, Omar N Al Yacoub, Nael M Mostafa, Ahmed Hamed Salem","doi":"10.1007/s40272-025-00733-2","DOIUrl":"10.1007/s40272-025-00733-2","url":null,"abstract":"<p><strong>Background: </strong>Central precocious puberty is the early onset of puberty due to premature activation of the hypothalamic-pituitary-gonadal axis, which can reduce adult height. Leuprolide, a gonadotropin-releasing hormone agonist, reduces gonadotropin secretion and is the standard treatment for central precocious puberty.</p><p><strong>Objective: </strong>This study aimed to build a population model to describe the pharmacokinetics of a 3-month leuprolide acetate depot formulation in pediatric patients with central precocious puberty, evaluate covariate effects (age and weight) on leuprolide pharmacokinetics, and assess flat-dosing feasibility in pediatrics.</p><p><strong>Methods: </strong>Samples from 48 patients (aged 1-10 years) were collected over 24 weeks following the administration of 11.25 and 30 mg of a leuprolide acetate 3-month depot formulation. A population pharmacokinetic model was developed using non-linear mixed-effects modeling (NONMEM). Covariate effects were tested using a forward inclusion and backward elimination approach and exploratory data analysis.</p><p><strong>Results: </strong>A one-compartment model with immediate and delayed first-order absorption and proportional error model best described leuprolide pharmacokinetics in children. A transit compartment model characterized the delayed absorption. Apparent clearance and volume estimates were 181 L/day and 7.11 L, respectively, which were in alignment with those estimated in adult patients with prostate cancer. The immediate and delayed absorption rate constants were 0.441 day^-1 and 0.00879 day^-1, respectively. The number of transit compartments and the mean transit time were 3 and 34.1 days, respectively. No covariates significantly affected leuprolide pharmacokinetics.</p><p><strong>Conclusions: </strong>The developed model adequately characterized leuprolide pharmacokinetics in pediatrics. No significant covariate effects were observed, supporting the use of a fixed leuprolide dose in pediatrics.</p><p><strong>Clinical trial registration: </strong>NCT00635817, registered 13 March, 2008.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"295-305"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13121213/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147459378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clozapine in Child and Adolescent Psychiatry.","authors":"Ahmed Naguy, Bibi Alamiri","doi":"10.1007/s40272-026-00749-2","DOIUrl":"10.1007/s40272-026-00749-2","url":null,"abstract":"","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"225-228"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593807","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":"Long-Term Safety and Efficacy of Dupilumab Treatment in Children Aged 6 Months to 5 Years with Severe Atopic Dermatitis.","authors":"Amy S Paller, Andreas Pinter, Lara Wine Lee, Roland Aschoff, Jacek Zdybski, Christina Schnopp, Faisal A Khokhar, Amy H Praestgaard, Ashish Bansal, Brad Shumel, Mike Bastian","doi":"10.1007/s40272-026-00747-4","DOIUrl":"10.1007/s40272-026-00747-4","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Infants and young children with severe atopic dermatitis (AD) have a high burden of disease with a strong impact on quality of life. Here we assess long-term efficacy and safety of dupilumab in pediatric patients aged 6 months to 5 years with severe AD.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This is a subgroup analysis of patients aged 6 months to 5 years enrolled in the ongoing LIBERTY AD PED open-label extension (OLE) study of dupilumab who had previously participated in the parent study LIBERTY AD PRESCHOOL part B and had severe AD (Investigator's Global Assessment [IGA] = 4) at parent study baseline. Patients received weight-tiered dupilumab every 4 weeks (200 mg for patients weighing 5 to &lt; 15 kg; 300 mg for patients weighing 15 to &lt; 30 kg). Key endpoints included the incidence and rate of treatment-emergent adverse events (TEAEs), the proportion of patients with a ≥ 75% improvement in Eczema Area and Severity Index (EASI-75) from parent study baseline, proportions of patients achieving IGA = 0/1 and IGA ≤ 2, and the proportion of patients with a ≥ 6-point improvement in Children's Dermatology Life Quality Index (CDLQI) for patients aged ≥ 4 years or Infants' Dermatitis Quality of Life (IDQoL) questionnaire for patients aged &lt; 4 years.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;This analysis included 121 patients, of whom 50 completed the week 104 visit. TEAEs were reported in 88% of patients; most TEAEs were mild or moderate and not related to treatment. Common TEAEs included upper respiratory tract infection, nasopharyngitis, and cough. Conjunctivitis events were reported in 19% of patients and were mild or moderate, with a median duration of 8 days. No conjunctivitis event led to treatment discontinuation, and most events resolved during the study. One drug-related event (severe urticaria) led to treatment discontinuation, but it was not serious and resolved over time. Serious TEAEs were reported in 17 patients (14%), including one drug-related pinworm event. No serious TEAE led to treatment discontinuation. By week 4 of the OLE study, patients who had received placebo in the parent study exhibited efficacy improvements comparable to patients who had received dupilumab. By week 104, 96% of patients achieved EASI-75 from parent study baseline, 27% of patients achieved an IGA score of 0/1 (clear/almost clear skin), 92% of patients achieved IGA ≤ 2 (clear skin to mild AD), and the least squares mean percent change in EASI from parent study baseline was - 89%. Additionally, 89% (23/26) of patients achieved a ≥ 6-point (clinically meaningful) improvement in CDLQI, and 100% (3/3) of patients achieved a ≥ 6-point improvement in IDQoL.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;Long-term treatment with dupilumab for up to 2 years showed acceptable safety and sustained efficacy in signs, symptoms, and quality of life in patients aged 6 months to 5 years with severe AD, with a rapid improvement for patients who had received placebo in the par","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"307-320"},"PeriodicalIF":3.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13121186/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}