Pediatric Drugs最新文献

{"title":"Copper Histidinate: Pediatric First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40272-026-00750-9","DOIUrl":"https://doi.org/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":""},"PeriodicalIF":3.3,"publicationDate":"2026-04-22","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":"Impact of β-Blockers on the Risk of Low-Birth-Weight Infants in Women with Long QT Syndrome or Marfan Syndrome: A Single-Center Retrospective Study from 2008 to 2022 in a Tertiary Care Center.","authors":"Mélissa Grégori, Laurianne Le Gloan, Thomas Goronflot, Jean-Baptiste Gourraud, Emilie Misbert, Norbert Winer, Vincent Dochez","doi":"10.1007/s40272-026-00740-x","DOIUrl":"10.1007/s40272-026-00740-x","url":null,"abstract":"<p><strong>Introduction: </strong>β-Blockers are an essential treatment for cardiovascular disease, the incidence of which is rising among women of childbearing age. Several large cohort and registry studies have reported an association between β-blocker exposure in pregnancy and reduced birth weight, but most have included women with hypertension or structural heart disease. Our study's primary objective was to evaluate the impact of these medications on the birth weight of infants born to mothers with morphologically normal hearts, including women with long QT syndrome (LQTS) or Marfan syndrome (MFS). Secondary objectives were to evaluate the influence of β-blocker type on birth weight, diagnoses that fetuses were small for gestational age (SGA) or growth-restricted (FGR) during pregnancy and at birth, uterine Doppler abnormalities, and neonatal adverse effects (bradycardia and hypoglycemia), which are expected effects of in utero β-blocker exposure.</p><p><strong>Materials and methods: </strong>This retrospective observational single-center study compared pregnancies in patients with LQTS or MFS treated with β-blockers with those of untreated matched control patients. Pregnancies were matched for maternal age, body mass index, parity, gestational age at birth, presence of gestational or pre-existing diabetes, and smoking status.</p><p><strong>Results: </strong>Fifty-seven pregnancies of 40 mothers exposed to β-blockers were matched with 165 control pregnancies. This study's main finding was that the mean birth weight of infants whose mothers used β-blockers during pregnancy was a significant 442 grams lower (unadjusted 2890 g vs 3285 g; p < 0.001) than that of the control group. SGA/FGR during pregnancy and at birth were diagnosed significantly more often in the treated patients, and their incidence of neonatal bradycardia was higher in the exposed group. Among the uterine Doppler examinations available, no clear differences were observed between groups. Data on neonatal hypoglycemia were inconclusive because of differential screening strategies and a high proportion of missing values, particularly in the control group.</p><p><strong>Discussion: </strong>To our knowledge, this is the first study specifically focusing on β-blocker use in pregnant women with LQTS or MFS and structurally normal hearts, using matched controls to minimize confounding by underlying cardiac disease. β-Blocker use-mainly nadolol and bisoprolol-was associated with significantly lower birth weight and higher rates of FGR/SGA and neonatal bradycardia. Our findings support the continuation of β-blocker therapy when clinically indicated, combined with careful fetal growth monitoring and targeted neonatal surveillance.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"197-207"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146132786","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":"Guselkumab: Pediatric First Approval.","authors":"Sheridan M Hoy","doi":"10.1007/s40272-026-00741-w","DOIUrl":"10.1007/s40272-026-00741-w","url":null,"abstract":"<p><p>Guselkumab (TREMFYA^®) is a fully human IgG1λ monoclonal antibody developed by Johnson & Johnson to selectively target the p19 subunit of interleukin (IL)-23, a cytokine that plays a key role in various immune-mediated inflammatory diseases. Guselkumab is approved in multiple countries worldwide, including the USA and those of the EU, for the treatment of adults with moderate-to-severe plaque psoriasis, active psoriatic arthritis, and moderately-to-severely active ulcerative colitis and Crohn's disease. In September 2025, guselkumab received its first pediatric approvals in the USA for the treatment of pediatric patients 6 years of age and older and weighing ≥ 40 kg who either have moderate-to-severe plaque psoriasis and are candidates for systemic therapy or phototherapy, or who have active psoriatic arthritis. Subsequently, guselkumab was approved in December 2025 in the EU for the treatment of moderate-to-severe plaque psoriasis in children and adolescents from the age of 6 years who are candidates for systemic therapy. Johnson & Johnson is currently undertaking phase III development of guselkumab in pediatric patients with Crohn's disease and ulcerative colitis in various countries. This article summarizes the milestones in the development of guselkumab leading to these first pediatric approvals for plaque psoriasis and psoriatic arthritis.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"217-224"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146202535","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":"Established and Emerging Therapies for High-Risk Neuroblastoma.","authors":"Shuo Xu, Jennifer Foster, Andrew Wahba","doi":"10.1007/s40272-025-00731-4","DOIUrl":"10.1007/s40272-025-00731-4","url":null,"abstract":"<p><p>High-risk neuroblastoma (HRNB) accounts for a disproportionately high percentage of cancer-related deaths among pediatric patients, despite intensive multimodal treatment. Current frontline therapy comprising chemotherapy, surgery, autologous stem cell transplantation (ASCT), radiation, immunotherapy, and tumor differentiation agents has improved survival rates; however, overall outcomes have plateaued. Refractory and relapsed cases are associated with even poorer prognoses, underscoring the limitations of existing treatment regimens. Additionally, treatment-related toxicities from intensive therapies pose significant risks to patients' quality of life and long-term health, highlighting the need for novel therapeutic approaches. In response, recent efforts have focused on integrating new modalities into frontline therapy, such as anti-GD2 immunotherapy, iodine-131 (¹³¹I)-metaiodobenzylguanidine (MIBG) therapy, and targeted agents for patients with identifiable molecular mutations, alongside adding eflornithine as maintenance therapy. In parallel, advancements in treating refractory and relapsed neuroblastoma emphasize innovative approaches, including novel targeted therapies, immunotherapy, and radionucleotides. This review discusses both the current standard of care for high-risk neuroblastoma as well as focusing on emerging immunotherapy including cellular therapy in an effort to improve outcomes, both in terms of survival and treatment-related late effects.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"159-175"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147290689","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":"Neurological Adverse Effects of Antipsychotic Medication in Children and Young People.","authors":"Frank M C Besag, Michael J Vasey, Chris Hollis, David Taylor","doi":"10.1007/s40272-025-00730-5","DOIUrl":"10.1007/s40272-025-00730-5","url":null,"abstract":"<p><p>Neurological adverse effects (NAEs) are commonly reported in individuals treated with antipsychotic medications. Children and young people (CYP) may be particularly susceptible to these effects, but few studies have focused on the risk of NAEs in this population. This review provides an overview of the published literature on NAEs in CYP with an emphasis on data from randomised placebo-controlled trials. Most antipsychotics are associated with sedative effects that may impair daily functioning. Akathisia, dystonia and parkinsonism are commonly reported in CYP, although rating scale assessments typically show minimal changes from baseline in short-term randomised studies. Tardive dyskinesia appears to be less common in CYP than in adults, but data are limited. Some antipsychotics, in particular clozapine, are associated with a reduced seizure threshold, but it is unclear whether CYP may be more vulnerable than adults and available studies are subject to various confounding factors. Neuroleptic malignant syndrome, a rare and potentially fatal adverse drug reaction, has been reported in CYP treated with both first-generation and second-generation antipsychotics. Data on risk factors and management strategies for NAEs are largely from studies in adults and may not be relevant to CYP. Future studies should aim to resolve some of the current uncertainties. In particular, within-subject \"self-controlled\" studies using prospectively collected data from large databases would help to clarify the incidence and risk factors, in particular for less common NAEs, while controlling for possible confounders.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"127-157"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12992390/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145912526","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":"Acute Phase Reaction After First Neridronate Infusion in Children with Osteogenesis Imperfecta: An Analysis Based on Questionnaire Data from 65 Patients.","authors":"Felicitas Haselwarter, Stefanie Stasek, Kristina Eva Rehberg, Heike Hoyer-Kuhn, Susanna Reincke, Eva Nüsken, Oliver Semler, Mirko Rehberg","doi":"10.1007/s40272-026-00737-6","DOIUrl":"10.1007/s40272-026-00737-6","url":null,"abstract":"<p><strong>Background: </strong>Bisphosphonates represent the current standard of care for the pharmacological treatment of pediatric patients with osteogenesis imperfecta (OI). However, data on acute phase reactions (APRs) after first-time administration, especially with neridronate, remain limited.</p><p><strong>Objective: </strong> This study evaluates the incidence, characteristics, and severity of APRs following the initial intravenous neridronate infusion in pediatric OI patients.</p><p><strong>Methods: </strong>Sixty-five bisphosphonate-naïve children with genetically or clinically confirmed OI received weight-adjusted intravenous neridronate during a brief hospitalization. Parents documented side effects, including fever onset/duration and skeletal pain, using standardized questionnaires.</p><p><strong>Results: </strong>Neridronate was generally well tolerated. No severe adverse events occurred. Forty percent of patients developed transient fever. Fever typically emerged within 12-24 h and resolved within 1-2 days. New skeletal pain was reported in 40% of patients, with 60% experiencing severe pain. Mean skeletal pain scores decreased below baseline within 4 days post-infusion. Fever duration did not differ significantly between OI types.</p><p><strong>Conclusions: </strong>Fever and skeletal pain are common yet self-limiting side effects after first neridronate infusion in children with OI. Adequate pain medication and caregiver education are important to improve treatment adherence and patient comfort during initiation of bisphosphonate therapy.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"187-196"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12992442/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146197729","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":"Therapeutic Role of Neostigmine and Pyridostigmine in Pediatric Chronic Intestinal Pseudo-Obstruction: A Systematic Review.","authors":"Caterina Cocchi, Vanessa Rossetti, Luisa Zupin, Sara Lega, Grazia Di Leo, Egidio Barbi, Francesca Peri","doi":"10.1007/s40272-025-00736-z","DOIUrl":"10.1007/s40272-025-00736-z","url":null,"abstract":"<p><strong>Background: </strong>Chronic intestinal pseudo-obstruction (CIPO) is a rare and severe disorder presenting with the clinical and radiological features of intestinal obstruction in the absence of a mechanical cause. When it manifests in children, it is referred to as pediatric intestinal pseudo-obstruction (PIPO). Diagnosis remains challenging and is largely based on exclusion. Management is primarily symptomatic, and among the available therapeutic approaches, cholinesterase inhibitors have emerged as potential options for severe gastrointestinal dysmotility, particularly when conventional treatments are insufficient. This review summarizes the current evidence on the use of pyridostigmine and neostigmine in CIPO, with a focus on pediatric patients.</p><p><strong>Methods: </strong>A systematic review was performed using PubMed, Embase, Web of Science, and Scopus, including all studies published up to July 2025 that evaluated the use of pyridostigmine and neostigmine in pediatric patients with CIPO.</p><p><strong>Results: </strong>Our review identified 22 publications reporting a total of 54 patients. Overall, treatment with neostigmine or pyridostigmine was associated with a favorable clinical response in approximately two-thirds of cases, ranging from partial improvement to complete symptoms resolution. Safety outcomes were generally reassuring, with few adverse events and only rare cases requiring treatment discontinuation.</p><p><strong>Discussion: </strong>Pyridostigmine and neostigmine may represent therapeutic options for pediatric CIPO, with neostigmine appearing particularly effective in acute settings and pyridostigmine more suited to chronic management. Although current evidence supports both safety and potential efficacy, robust clinical trials are essential before these agents can be integrated into standard care protocols.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"111-125"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12992420/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146213787","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":"Optimizing Antifungal Therapy for Pediatric Tinea Capitis: A Narrative Review of Species-Specific Efficacy, Dosing Strategies, and Clinical Implications.","authors":"Chester Tyson, Caitlin Haydek, Elizabeth Nieman, Diana McShane, Dean Morrell","doi":"10.1007/s40272-026-00738-5","DOIUrl":"10.1007/s40272-026-00738-5","url":null,"abstract":"<p><p>Pediatric tinea capitis, a common superficial fungal infection of the scalp, is primarily caused by Trichophyton and Microsporum species. While oral antifungal therapy remains the cornerstone of treatment, clinical outcomes vary considerably owing to differences in dosing practices and the species-specific response of the infecting organism. This narrative review synthesizes the existing literature to emphasize contemporary dosing regimens and species-directed efficacy on four widely used agents: griseofulvin, terbinafine, itraconazole, and fluconazole. Across studies published between 1997 and 2025, griseofulvin consistently demonstrated strong performance for both Trichophyton and Microsporum infections when administered at the recommended 20-25 mg/kg/day, confirming its enduring role in pediatric care. Terbinafine achieved reliable results for Trichophyton but was markedly less effective for Microsporum, with wide variability in reported cure rates. Though less extensively studied in children, itraconazole and fluconazole emerged as effective alternatives, while both warrant careful use owing to potential hepatotoxicity and other adverse effects. The evidence underscores the importance of species identification and optimal dosing in guiding antifungal selection. Empirical treatment without microbiologic confirmation risks reduced cure rates and may contribute to emerging antifungal resistance. Future investigations should prioritize long-term follow-up and the potential role of combination therapy to refine and sustain effective management strategies for pediatric tinea capitis.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"177-186"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146086725","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":"Doxecitine and Doxribtimine: First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40272-025-00734-1","DOIUrl":"10.1007/s40272-025-00734-1","url":null,"abstract":"<p><p>Doxecitine and doxribtimine (KYGEVVI^®) are pyrimidine nucleosides developed by UCB for the treatment of thymidine kinase 2 deficiency (TK2d). In November 2025, doxecitine and doxribtimine received its first approval in the USA for the treatment of TK2d in adult and pediatric patients with an age of symptom onset on or before 12 years. This article summarizes the milestones in the development of doxecitine and doxribtimine leading to this first approval for TK2d.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"209-215"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13038758/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146065846","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":"Toward Optimal Treatment Outcomes in New-Onset Polyarticular-Course Juvenile Idiopathic Arthritis.","authors":"Adam Seth Mayer, Yukiko Kimura","doi":"10.1007/s40272-025-00735-0","DOIUrl":"10.1007/s40272-025-00735-0","url":null,"abstract":"<p><p>Polyarticular-course juvenile idiopathic arthritis (polyJIA) is a chronic inflammatory arthritis involving > 4 distinct joints over the course of the disease. PolyJIA tends to be more difficult to treat than other forms of nonsystemic JIA, but advances in immunosuppressive therapies over the past 25 years have dramatically reduced patient morbidity. Despite significant advances in treatment options and outcomes, there is little evidence to guide how and when these treatments should be implemented to be most effective. Optimizing the initial timing and choice of these medications may further enhance outcomes and potentially mitigate the need for serial trials of therapy. There are recent data to suggest that earlier and more aggressive initial immunosuppressive therapy may help patients with polyJIA achieve inactive disease more quickly and may improve long-term outcomes. This approach, however, is counterweighted by the risk of overtreatment and the need for improved risk stratification models that can reliably inform individualized treatment strategies. This article explores the main treatment approaches to new-onset polyJIA and current evidence to support which approach may be optimal.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"103-110"},"PeriodicalIF":3.3,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146011711","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}