Pediatric Drugs最新文献

{"title":"Safety and Effectiveness of Pain Medications for Migraine Management During Pregnancy and Lactation.","authors":"Liza Smirnoff, Nicolas Garzon Mancera, Tayina Hyppolite, Daniela Lozano, Alexandra Cocores","doi":"10.1007/s40272-026-00760-7","DOIUrl":"10.1007/s40272-026-00760-7","url":null,"abstract":"<p><p>The management of migraine during pregnancy and lactation poses a major challenge owing to the paucity of safety data. While very little safety data are available for new targeted migraine therapies such as calcitonin gene receptor protein (CGRP) blocking medications, including the CGRP monoclonal antibodies and gepants, well-established migraine therapies such as topiramate, valproate, and dihydroergotamine have known risk for teratogenicity. Likewise, the safety evidence for commonly used therapies for migraine in pregnancy such as propranolol and verapamil is of low quality, and the level of evidence for efficacy of verapamil is limited. Meanwhile women experience migraine at the highest rates during their reproductive years necessitating an effective and balanced approach that considers maternal and fetal risk as well as optimal benefit to the patient to ensure adequate and appropriate treatment during pregnancy and lactation. The purpose of this review is to provide an overview of the available data on the safety of available and efficacious medications in the management of migraine during pregnancy and lactation. Based on more recent studies, there is increased evidence for use of more specific and efficacious medications for migraine, such as onabotulinumtoxinA, local nerve blocks with lidocaine, and triptans during pregnancy and lactation.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"387-403"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379431/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148430902","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":"Molluscum Contagiosum Treatments in Children: A Systematic Review with Network Meta-Analysis.","authors":"Letícia Rocha Campos, Gisella Chiara Carpi, Ana Beatriz Nardelli da Silva, Beatriz Ximenes Mendes, Fabiana Castro Porto Silva Lopes","doi":"10.1007/s40272-026-00758-1","DOIUrl":"10.1007/s40272-026-00758-1","url":null,"abstract":"<p><strong>Background/objectives: </strong>Molluscum contagiosum affects up to 11.5% of children, yet optimal treatment remains uncertain. Previous systematic reviews included mixed adult/pediatric populations, limiting applicability to children. We performed the first pediatric-specific network meta-analysis comparing molluscum contagiosum treatment efficacy and safety.</p><p><strong>Methods: </strong>We searched MEDLINE, Embase, Cochrane Library, and Web of Science through December 2025 for controlled trials evaluating molluscum contagiosum treatments in children. Two reviewers independently selected studies, extracted data, and assessed risk of bias using Cochrane Risk of Bias 2 and ROBINS-I tools. We performed frequentist network meta-analysis with random-effects modeling, calculating risk ratios (RR) with 95% confidence intervals (CI). Treatments were ranked using Surface Under Cumulative Ranking (SUCRA) scores. This systematic review and network meta-analysis was registered in PROSPERO (registration number CRD420251040622).</p><p><strong>Results: </strong>Eighteen controlled trials involving 2377 pediatric participants evaluating 15 treatments were included. The network demonstrated consistency (inconsistency p = 0.77). Among treatments evaluated in multiple studies, salicylic acid + lactic acid 16.7% (RR 3.16, 95% CI 1.81-5.52, SUCRA 86.0%), cantharidin 0.7% (RR 3.25, 95% CI 1.39-7.58, SUCRA 81.2%), and potassium hydroxide 10% (RR 3.05, 95% CI 1.77-5.24, SUCRA 78.6%) demonstrated superior efficacy versus placebo. Single-study treatments including Backhousia citriodora oil and Candida albicans antigen achieved highest rankings but require validation. Potassium hydroxide demonstrated concentration-dependent efficacy and adverse events.</p><p><strong>Conclusions: </strong>Salicylic acid + lactic acid 16.7%, cantharidin 0.7%, and potassium hydroxide 10% demonstrate superior efficacy for pediatric molluscum contagiosum with robust multi-study evidence. Treatment selection should balance efficacy, safety, and patient factors. Top-ranked single-study treatments require validation before clinical recommendations.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"359-372"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148339658","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":"Discontinuing Colchicine in Pediatric Familial Mediterranean Fever: Real-Life Experience from a Long-Term Follow-Up Cohort.","authors":"Selen Duygu Arık, Gülşah Kavrul Kayaalp, Ayşenur Doğru Kılınç, Bengisu Menentoğlu, Büşra Başer Taşkın, Aslı Dudaklı, Ayşe Tanatar, Özlem Akgün, Nuray Aktay Ayaz","doi":"10.1007/s40272-026-00755-4","DOIUrl":"10.1007/s40272-026-00755-4","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Colchicine is the cornerstone of treatment in familial Mediterranean fever and is generally recommended as lifelong therapy. However, a subset of pediatric patients may experience a milder disease course, raising questions about the feasibility and safety of colchicine discontinuation. Evidence guiding this decision remains limited, and no consensus exists on patient selection criteria or monitoring strategies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;The objective of this study was to evaluate long-term outcomes after colchicine discontinuation in a large pediatric familial Mediterranean fever cohort and to identify clinical and genetic factors associated with sustained colchicine-free remission.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We retrospectively analyzed medical records of 2316 pediatric patients diagnosed with familial Mediterranean fever and followed at the Pediatric Rheumatology Unit of Istanbul University, Istanbul Faculty of Medicine, Istanbul, Türkiye. Patients fulfilling at least one of the Tel-Hashomer or Eurofever/Pediatric Rheumatology International Trials Organization (PRINTO) classification criteria and carrying at least one pathogenic or likely pathogenic MEFV (Mediterranean fever) gene mutation were eligible. Colchicine was discontinued in 127 patients (5.48%), who were classified into two groups: Group 1 (n = 31), patients who required colchicine re-initiation because of recurrent attacks, and Group 2 (n = 96), patients who maintained sustained colchicine-free remission during follow-up. A contemporaneous comparator group of 118 patients who continued colchicine therapy without any attempt at discontinuation (Group 3) was included to provide clinical and genetic context. Demographic, clinical, genetic, and disease severity characteristics were compared across groups.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Sustained colchicine-free remission was maintained in 96 of 2316 patients (4.14%; Group 2). Among all patients who discontinued colchicine (Groups 1 and 2 combined), the median duration of colchicine treatment before discontinuation was 3.85 years (interquartile range 1.84-7.10), and the median follow-up after cessation was 4.91 years (interquartile range 1.92-7.23). Baseline clinical, demographic, and genetic characteristics were largely similar between Group 1 and Group 2, and no robust independent predictor of sustained remission was identified. Biallelic pathogenic exon 10 MEFV variants were significantly more prevalent in Group 3 (32.2%) compared with Group 1 (12.9%) and Group 2 (3.1%), reflecting a progressive genetic gradient across the three groups. Patients in Group 3 also exhibited higher frequencies of fever, chest pain, arthritis, arthralgia, erysipelas-like erythema, and exercise-induced leg pain compared with the discontinuation groups. Fulfillment of Eurofever/PRINTO classification criteria was significantly lower in Groups 1 (54.8%) and 2 (54.2%) than in Group 3 (87.3%), consistent with a milder","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"405-415"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379450/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148218993","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":"Copper Histidinate: Pediatric First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40272-026-00750-9","DOIUrl":"10.1007/s40272-026-00750-9","url":null,"abstract":"<p><p>Copper histidinate (ZYCUBO^®) is a bioavailable copper replacement therapy developed by Cyprium Therapeutics and Sentynl Therapeutics for the treatment of Menkes disease, a genetic disorder caused by variants in the copper transporter gene, ATP7A. Because copper cannot be absorbed from the gastrointestinal tract by individuals with Menkes disease, copper histidinate is administered as a daily subcutaneous injection. In January 2026, copper histidinate received its first approval in the USA by the U.S. Food and Drug Administration for the treatment of Menkes disease in pediatric patients. Orphan Designation was granted by The European Commission to copper histidinate for the treatment of Menkes disease. This article summarizes the milestones in the development of copper histidinate leading to this first approval for Menkes disease in pediatric patients.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"441-445"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147778291","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":"Risks of Undertreatment and Overtreatment in Pediatric Inflammatory Bowel Disease: Navigating the Balance.","authors":"Giulia D'Arcangelo, Silvana Ancona, Giulia Naspi Catassi, Francesca Musto, Rosaria Celano, Marina Aloi","doi":"10.1007/s40272-026-00751-8","DOIUrl":"10.1007/s40272-026-00751-8","url":null,"abstract":"<p><p>Despite major therapeutic advances in recent years, and early signs that the overall standard of care we can deliver to children with inflammatory bowel disease (IBD) is improving, the promise of precision medicine remains largely unfulfilled in daily practice. The goal is to match the right therapy to the right patient, in the right time window, so that treatment intensity reflects true disease biology and future risk rather than broad population averages. However, this approach is still an unmet need. Even when management is framed within a risk-based paradigm, clinical decisions are often guided by generalized recommendations and imperfect predictors, with limited capacity to individualize strategies at diagnosis and during follow-up. As a result, two opposite but equally relevant pitfalls remain very real. On one hand, overtreatment can lead to unnecessary exposure to immunosuppressive regimens in children and adolescents with mild, slowly progressive, or self-limited disease, increasing the burden of adverse events, monitoring, and psychosocial impact. On the other hand, undertreatment in truly high-risk patients can delay effective control of inflammation, allowing ongoing tissue damage, disease extension, growth impairment, and avoidable complications, ultimately worsening long-term outcomes. This critical review summarizes and critically appraises current recommendations and available evidence, focusing on the pitfalls of overtreatment and undertreatment in pediatric IBD.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"331-342"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379467/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148219043","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":"Sirolimus Therapy and Dyslipidemia in Neonates and Infants: A Retrospective Cohort and Pharmacovigilance Analysis.","authors":"Akinobu Taniguchi, Basile Chrétien, Takashi Maeda, Kazuto Ueda, Ryosuke Miura, Ryuichi Tanaka, Toshihiko Suzuki, Yukako Muramatsu, Yoshiaki Sato, Kazuki Nishida","doi":"10.1007/s40272-026-00754-5","DOIUrl":"10.1007/s40272-026-00754-5","url":null,"abstract":"<p><strong>Importance: </strong>Sirolimus is increasingly used for complex vascular and lymphatic anomalies in neonates and infants. While dyslipidemia is a known adverse effect in adults, the incidence, timing, and severity in this population remain poorly characterized.</p><p><strong>Objective: </strong>The aim of this study was to evaluate the reporting odds and characteristics of sirolimus-associated dyslipidemia in neonates and infants compared with older populations.</p><p><strong>Design, setting, and participants: </strong>This study combined a single-center retrospective cohort study and global pharmacovigilance analysis. The clinical cohort included neonates and infants treated with sirolimus at Nagoya University Hospital (October 2018-September 2025). The pharmacovigilance analysis utilized VigiBase (up to September 1, 2025) to perform age-stratified disproportionality analyses.</p><p><strong>Main outcomes and measures: </strong>In the clinical cohort, lipid profiles (total cholesterol, LDL-C, triglycerides) were assessed pre- and post-treatment. In VigiBase, adjusted reporting odds ratios (aRORs) for dyslipidemia and time-to-onset (TTO) were estimated across age groups, adjusting for sex, region, and concomitant medications.</p><p><strong>Results: </strong>The cohort included 10 patients (median age, 44.5 days). Post-initiation, 100% developed hypertriglyceridemia and 90% developed hypercholesterolemia. In VigiBase (17,802 sirolimus-related reports), the dyslipidemia reporting signal was highest in infants (28 days-23 months), with an aROR of 147.08 (95% CI 79.67-271.54), substantially exceeding that in adults aged 18-45 years (aROR 2.31; 95% CI 1.75-3.06). Among infant reports with available TTO data, median TTO was 11.0 days (IQR 5.5-15.8), compared with 29.5 days in adults.</p><p><strong>Conclusions and relevance: </strong>Neonates and infants showed a markedly stronger dyslipidemia reporting signal than older age groups, and lipid abnormalities were common in the clinical cohort. These findings support baseline lipid assessment and early monitoring after sirolimus initiation in very young patients, including within the first two weeks of therapy. The pharmacovigilance findings should be interpreted as hypothesis generating.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"417-427"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148101738","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":"Recent Advances in the Pharmacological Management of Pediatric Spondyloarthritis.","authors":"Umit Gul, Elif Kilic Konte, Mehmet Yildiz, Ozgur Kasapcopur","doi":"10.1007/s40272-026-00759-0","DOIUrl":"10.1007/s40272-026-00759-0","url":null,"abstract":"<p><p>Juvenile spondyloarthritis (JSpA) refers to a heterogeneous group of inflammatory diseases affecting peripheral joints and the axial skeleton during childhood and adolescence. Enthesitis-related arthritis (ERA) and juvenile psoriatic arthritis (JPsA), subtypes of juvenile idiopathic arthritis (JIA) under the International League of Associations for Rheumatology (ILAR) classification, are included in this group. They may be associated with human leukocyte antigen (HLA) B27 positivity. Apart from musculoskeletal involvement, pathologies that accompany the disease, such as psoriasis, uveitis, and inflammatory bowel disease, significantly affect patients' quality of life and clinicians' treatment approaches. Guidelines developed for the treatment of JSpA patients primarily address the use of nonsteroidal antiinflammatory drugs (NSAIDs), conventional disease-modifying antirheumatic drugs (csDMARDs), and tumour necrosis factor inhibitors (TNFi). However, not all patients respond adequately to or tolerate the currently available therapies, highlighting the need for novel treatment options. Recent advances in understanding immunopathogenesis have driven the development of new therapeutic targets, including TNF inhibitors, interleukin (IL)-17/IL-23 blockers, and Janus kinase inhibitors. Several of these agents, including secukinumab, have now received approval for the treatment of ERA and JPsA. However, since only a limited number of open-label and randomized controlled trials have specifically evaluated JSpA, current treatment strategies are also informed by evidence from other JIA subtypes and adult studies. Expanding the approved indications of existing biologics-supported by robust trial data-alongside the development of novel therapeutic agents, may broaden treatment options and ultimately improve outcomes for patients with JSpA.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"343-358"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148397423","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":"Epinephrine Underuse for Anaphylaxis in Infants and Toddlers: A Practical Review for Pediatricians.","authors":"Stephanie Leeds, Aikaterini Anagnostou, Michael Pistiner, Jay Lieberman, Nicole B Ramsey, Rachael Griffiths, Julie Wang","doi":"10.1007/s40272-026-00752-7","DOIUrl":"10.1007/s40272-026-00752-7","url":null,"abstract":"<p><p>A recent consensus report defined anaphylaxis as a serious allergic reaction that may involve the skin/mucosa, respiratory, cardiovascular, and/or gastrointestinal systems that can progress rapidly and may cause death. Epinephrine treatment is recommended in suspected anaphylaxis cases; however, numerous barriers exist to consistent, widespread epinephrine use, including difficulty identifying anaphylaxis signs and symptoms; challenges recognizing when to use epinephrine; low propensity of prescribing and filling prescriptions; low epinephrine carrying rates; knowledge gaps in epinephrine administration techniques; fears of contacting emergency medical services; use of antihistamines, inhaled bronchodilators, and other medications as first-line agents over epinephrine; and limited epinephrine device availability in community settings (e.g., schools, daycares, and restaurants). To address these challenges, healthcare providers should provide training and educational resources to caregivers on signs and symptoms of anaphylaxis that warrant epinephrine use, how to use epinephrine devices, and the importance of having epinephrine readily available at all times. System-level strategies, including measures to improve access, legislative efforts to stock epinephrine in childcare centers, and scalable digital education approaches, are also needed. Furthermore, healthcare providers should implement shared decision-making with caregivers to prepare and empower caregivers to make informed decisions when managing anaphylactic events. Understanding barriers to consistent, widespread epinephrine use when infants and toddlers experience anaphylaxis and providing actionable educational tools to caregivers alongside system-level strategies can improve outcomes in this vulnerable population.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"373-385"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379441/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148278404","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":"Real-World Systemic Treatment Patterns and Outcomes of Children With Severe Alopecia Areata: A French Nationwide Study (ESTAPED Cohort).","authors":"Ninon Le Goaziou, Christine Bodemer, Christine Labrèze, Emmanuel Mahé, Annabel Maruani, Helene Aubert, Sébastien Barbarot, Claire Abasq, Nina Sigg, Audrey Lasek, Stéphanie Mallet, Christine Chiaverini, Juliette Miquel, Eve Puzenat, Florence Poizeau, Jasmine Flament, Bertille Bonniaud, Justine Pasteur, Catherine Droitcourt","doi":"10.1007/s40272-026-00757-2","DOIUrl":"10.1007/s40272-026-00757-2","url":null,"abstract":"<p><strong>Background/objective: </strong>Severe forms of alopecia areata (AA) often require off-label systemic treatments (ST) for which data on effectiveness and safety are limited. This study aimed to provide a comprehensive real-world overview of ST patterns in children with severe AA.</p><p><strong>Method: </strong>We conducted a retrospective, longitudinal, multicenter study of children with AA (aged <18 years) receiving at least one ST (2010-2023), identified using a keyword search in Ouest Data Hub Warehouse (a hospital data network in France with a critical mass of 5.1 million patients) or by members of the Société Française de Dermatologie Pédiatrique. The primary outcome was drug survival (DS), defined as time on ST, and the secondary outcomes included reasons for treatment discontinuation and long-term observations.</p><p><strong>Results: </strong>Among the 262 included children (median age at AA onset: 9 years; 58.4% girls, atopic dermatitis in 32.4%), the first-line ST most frequently initiated (median age: 12 years, alopecia totalis in 28.6%) was intravenous corticosteroids (35.9%, median duration: 3 months), followed by methotrexate + intravenous corticosteroids (24.0%, 12.5 months), oral corticosteroids (17.9%, 1 month), methotrexate (10.7%, 9.5 months), methotrexate + oral corticosteroids (8.8%, 9 months), and lastly baricitinib (2.7%, 2.5 months). Specific maintenance treatments were not significantly associated with DS. Psychological impact, atopic dermatitis, later childhood onset and female sex were associated with longer first-line systemic treatment drug survival. Treatment failure was the main reason for discontinuation (51.9%) and no serious adverse events were reported. Five years after starting the first ST (n = 62), 14.5% of patients had achieved remission.</p><p><strong>Conclusions: </strong>In this national, retrospective real-world study, drug survival analyses highlighted two treatment profiles: pulse therapy (corticosteroids) for flare-ups and maintenance treatments (methotrexate, baricitinib) for long-term management. Overall, the effectiveness of therapies appears limited with frequent relapse and a likely need for prolonged treatment. However, data on long-term treatment outcomes of JAKi in children are lacking. This study provides real-life data which could help improve management strategies in children with severe AA.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"429-440"},"PeriodicalIF":3.7,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13379489/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148318652","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":"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":3.7,"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}