Pediatric Drugs最新文献

{"title":"Antipsychotic Medications for Delirium Treatment in the Pediatric Intensive Care Unit: A Systematic Review.","authors":"Francesca Cavagnero, Annalisa Salerno, Chiara Marchegiani Rizzolli, Luca Marchetto, Valentina Stritoni, Alvise Tosoni, Anna Tessari, Angela Amigoni, Marco Daverio","doi":"10.1007/s40272-025-00716-3","DOIUrl":"10.1007/s40272-025-00716-3","url":null,"abstract":"<p><strong>Background and objectives: </strong>Pediatric delirium (PD) is a common but underdiagnosed condition in pediatric intensive care units (PICUs), affecting 17-66% of patients. It is associated with prolonged ventilation and hospitalization, increased healthcare costs, and mortality. While nonpharmacological approaches are considered first-line treatments, pharmacological interventions are used in refractory cases despite limited pediatric-specific evidence. The objective of this systematic review was to evaluate the efficacy and safety of pharmacological treatments for PD in PICUs.</p><p><strong>Methods: </strong>Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines (International Prospective Register of Systematic Reviews [PROSPERO]: CRD42024504618), PubMed, Embase, Scopus, CINAHL, Cochrane, and Web of Science databases were searched for studies published up to February 2024. Eligible studies included children aged 1 month-18 years, diagnosed with PD in the PICU using validated scales or psychiatric evaluation and receiving pharmacologic treatment. Outcomes included delirium improvement or resolution and safety. Risk of bias was assessed using the Risk Of Bias In Non-randomized Studies-of Interventions (ROBINS-I) scale.</p><p><strong>Results: </strong>Of 7,309 records, 10 studies involving 283 patients receiving pharmacological treatment met inclusion criteria. All but one of the studies were retrospective and no randomized controlled trials (RCTs) were identified. Pharmacological treatment was administered to 283 patients, with the most used agents being quetiapine (36%), risperidone (20%), haloperidol (20%), and olanzapine (11%). Seven studies reported variable efficacy, with olanzapine showing significant symptom improvement in one study (olanzapine: N = 31; control: N = 28; F(1,20) = 28.62, r = 0.77, 95% confidence interval [CI] = 0.50-0.90) and the other drugs reporting a trend toward improvement in delirium severity. Adverse events were inconsistently measured and reported throughout studies: 22 cases were reported, with QTc prolongation (11 cases) and dystonia (7 cases) being the most frequent. Dystonia was observed in patients receiving haloperidol, whereas QTc prolongation was reported in those treated with quetiapine or risperidone. Complete resolution of the events was reported in 21/22 cases and occurred after dose adjustment or treatment interruption.</p><p><strong>Conclusions: </strong>Pharmacological interventions for PD in PICU patients showed variable efficacy, and adverse events were reported in a minority of treated patients. The limited sample size, the only modest quality of the studies, and the lack of replication preclude definitive conclusions about the drugs' efficacy. In addition, haloperidol, risperidone, and quetiapine raised some safety concerns. Further research is needed to establish stronger evidence for the pharmacologic treatment of PD in the PICU and to addres","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"707-722"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144993055","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":"Big Data in the Assessment of Medication Safety in Pregnancy: Opportunities and Challenges.","authors":"Per Damkier, Krista F Huybrechts, Hedvig Nordeng","doi":"10.1007/s40272-025-00718-1","DOIUrl":"10.1007/s40272-025-00718-1","url":null,"abstract":"","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"673-677"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144993175","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 of Anti-Interleukin-1 Medications in Children with Rheumatic Diseases: a Systematic Review.","authors":"M Isa, G M Tiller, D F L Liew, W D Renton","doi":"10.1007/s40272-025-00712-7","DOIUrl":"10.1007/s40272-025-00712-7","url":null,"abstract":"<p><strong>Background: </strong>Anti-interleukin-1 (IL-1) biologic disease-modifying anti-rheumatic drugs are the mainstay for several childhood rheumatic and autoinflammatory diseases. Long-term medication safety is a key consideration for chronic disease management.</p><p><strong>Aim: </strong>The objective was to synthesise evidence on the long-term safety of anti-IL-1 medications in children and young people with rheumatic diseases, including autoinflammatory diseases.</p><p><strong>Methods: </strong>The study protocol was registered prospectively (PROSPERO CRD420251000272). Original full text studies of at least ten patients presenting safety data on anti-IL-1 medications in children with rheumatic diseases were eligible for inclusion. Medline, Embase and Web of Science were searched from inception to 27 February 2025. The methodological index for non-randomized studies (MINORS) tool was used to assess risk of bias. All relevant safety outcomes were presented and synthesised. Meta-analysis was not performed owing to study heterogeneity.</p><p><strong>Results: </strong>A total of 1660 unique records were screened, and 57 unique studies (3690 patients) were included. In total, 31 were retrospective cohort studies, and 10 were prospective interventional trials. Most studies were of moderate (n = 31) or high (n = 25) risk of bias. Rates of adverse events varied significantly between studies. Injection site reactions (particularly with anakinra) and minor infections were common. Infections were the most common type of serious adverse event. Drug reaction with eosinophilia and systemic symptoms (n = 3) and interstitial lung disease (including related conditions) (n = 9) were reported in patients with systemic onset juvenile arthritis only. Deaths (n = 16) and malignancies (n = 7) were uncommon, often occurring long after anti-IL-1 medication discontinuation and were often deemed to be unrelated to the anti-IL-1 medication.</p><p><strong>Conclusions: </strong>Our results are consistent with the known safety profile of anti-IL-1 medications and show that they are generally safe for use in the context of childhood rheumatic and autoinflammatory diseases. This review of clinical trial and real-world data will help inform clinical decision-making and family counselling when initiating anti-IL-1 medications in children.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"693-705"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12534318/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964675","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":"Comment on \"Cardioprotective Effect of Nigella sativa in Pediatric Patients with Type 1 Diabetes Mellitus: A Randomized Controlled Study\".","authors":"Syeda Samia Fatima","doi":"10.1007/s40272-025-00713-6","DOIUrl":"10.1007/s40272-025-00713-6","url":null,"abstract":"","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"777-778"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144855967","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 Efficacy and Safety of Secukinumab in Children and Adolescents with Moderate-to-Severe Chronic Plaque Psoriasis: Four-Year Results of a Randomized, Phase III, Open-Label Trial.","authors":"Kulli Kingo, Philemon Papanastasiou, Stefan Beissert, Svetlana Lazareva, Asunción Vicente Villa, Jirina Bartonova, Rosalia Ballona, Amita Bansal, Ruvie Martin, Heng Fan, Charles O'Doherty, Shoba Ravichandran, Nina Magnolo","doi":"10.1007/s40272-025-00715-4","DOIUrl":"10.1007/s40272-025-00715-4","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The efficacy and safety of secukinumab up to week 52 in children and adolescents with moderate-to-severe chronic plaque psoriasis have been demonstrated previously (NCT03668613). Herein, we report the long-term efficacy, safety, and tolerability of secukinumab over a period of up to 208 weeks.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;In this randomized open-label trial, patients (6 to &lt; 18 years) were randomized 1:1 to receive low-dose (LD; N = 42) or high-dose (HD; N = 42) secukinumab stratified by weight (&lt; 25 kg, 25 to &lt; 50 kg, or ≥ 50 kg) and disease severity (moderate or severe). Patients weighing &lt; 25 kg received 75 mg secukinumab (both LD and HD); 25 to &lt; 50 kg received 75 mg (LD) or 150 mg (HD) secukinumab; and ≥ 50 kg received 150 mg (LD) or 300 mg (HD) secukinumab. The study assessed Psoriasis Area and Severity Index (PASI) 75/90/100 response, Investigator's Global Assessment modified 2011 (IGA mod 2011) 0/1 response, Children's Dermatology Life Quality (CDLQI) 0/1 response, and safety. The impact of secukinumab treatment on physical development was also assessed.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Overall, 79.8% (67 of 84) enrolled patients completed 4 years of treatment (secukinumab LD [N = 31] and secukinumab HD [N = 36]). Both treatment groups showed sustained PASI and IGA mod 0/1 responses throughout the treatment period (at week 208, PASI 75/90/100 [secukinumab LD: 96.3%/88.9%/51.9%; secukinumab HD: 87.9%/81.8%/72.7%] and IGA mod 2011 0/1 [secukinumab LD: 85.2%; secukinumab HD: 84.8%]). Mean PASI score remained low in both treatment groups, from week 12 through week 208; at week 208, mean percentage change in PASI scores from baseline was - 95.7% (mean absolute score: 18.46 [baseline]; 0.76 [week 208]) with secukinumab LD and - 94.5% (mean absolute score: 19.25 [baseline]; 1.07 [week 208]) with secukinumab HD. CDLQI 0/1 response remained high in both treatment groups from week 12 through week 208 (secukinumab LD: 75.0%; secukinumab HD: 88.2%). Safety profile of secukinumab with long-term (~313.9 patient-years) treatment was favorable and in line with the known safety profile reported previously for 52-week treatment. No dose-dependent safety observations were noted. There were no deaths recorded throughout the entire duration of the treatment. Nonfatal serious adverse events (SAEs) were reported in four (9.5%) patients in the LD group (coronavirus disease 2019 [COVID-19], Crohn's disease, infectious mononucleosis, intentional self-injury, tibia fracture) and two (4.8%) patients in the HD group (appendicitis, tonsillitis). The incidence of treatment-emergent adverse events was similar in both secukinumab dose groups (LD [78.6%] and HD [83.3%]). No impact on the growth and development, or sexual maturation of pediatric patients, was noted with long-term treatment.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Secukinumab demonstrated sustained long-term efficacy and improved quality of life through week 208 in pediatric pa","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"749-759"},"PeriodicalIF":3.3,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12534278/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964661","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":"History and Current Status of Growth Hormone Treatment in Children.","authors":"Camila C Luz, Paulo F Collett-Solberg","doi":"10.1007/s40272-025-00721-6","DOIUrl":"10.1007/s40272-025-00721-6","url":null,"abstract":"<p><p>Growth hormone (GH) has been used to treat severe GH deficiency for more than 60 years, with other indications dependent upon approval from national medical societies and governmental agencies. Many changes in GH preparations have been made to improve safety, compliance, and efficacy, from the extraction of GH from human pituitary glands to recombinant hormone technology and from the use of vials and syringes to the use of more advanced devices. Adherence to treatment has always been highlighted as a key point in the success of treatment; new depot preparations permitting weekly administration have advantages in this regard. This review summarizes the history of GH use and provides an analysis of the new weekly administration products.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"735-747"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186567","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":"Fremanezumab: Pediatric First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40272-025-00722-5","DOIUrl":"10.1007/s40272-025-00722-5","url":null,"abstract":"<p><p>Fremanezumab (fremanezumab-vfrm; AJOVY^®) is a humanized monoclonal antibody developed by Teva Pharmaceuticals to selectively target calcitonin gene-related peptide (a vasodilatory neuropeptide involved in the pathophysiology of migraine). Fremanezumab was first approved in September 2018 in the USA for the prevention of migraine in adults. In August 2025, it was approved in the USA for the preventive treatment of episodic migraine in pediatric patients aged 6-17 years who weigh 45 kg or more. This article summarizes the milestones in the development of fremanezumab leading to this pediatric first approval for migraine.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"771-775"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186524","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":"Authors' Reply to Fatima Comment on \"Cardioprotective Effect of Nigella sativa in Pediatric Patients with Type 1 Diabetes Mellitus: A Randomized Controlled Study\".","authors":"Doaa El Amrousy, Dalia El-Afify","doi":"10.1007/s40272-025-00714-5","DOIUrl":"10.1007/s40272-025-00714-5","url":null,"abstract":"","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"779-780"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144855966","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":"Vedolizumab Versus Other Biologics and the Risk of Venous Thromboembolism in Patients with Pediatric-Onset Inflammatory Bowel Diseases: A Target Trial Emulation Study.","authors":"Serena Yun-Chen Tsai, Ashwin N Ananthakrishnan, Harland S Winter, Kevin Sheng-Kai Ma","doi":"10.1007/s40272-025-00717-2","DOIUrl":"10.1007/s40272-025-00717-2","url":null,"abstract":"<p><strong>Background: </strong>Pediatric-onset inflammatory bowel diseases (IBD) are associated with an elevated risk of venous thromboembolism (VTE). However, data on the VTE risk in these patients treated with novel biologics, particularly vedolizumab, remain limited.</p><p><strong>Aims: </strong>To evaluate the association between vedolizumab and the risk of VTE compared with other biologics in patients with pediatric-onset Crohn's disease (CD) or ulcerative colitis (UC).</p><p><strong>Methods: </strong>We emulated a target trial using electronic health records from the TriNetX research network. The study included patients with CD or UC diagnosed before the age of 18 years who were treated with vedolizumab, ustekinumab, or anti-tumor necrosis factor (TNF) agents. Comparative groups were generated using 1:1 propensity-score matching based on relevant confounders. The primary outcome was the occurrence of any VTE within 12 months of follow-up. Cox proportional hazards models were used to compare the risk of VTE between vedolizumab-treated patients and those treated with anti-TNF agents and ustekinumab.</p><p><strong>Results: </strong>A total of 1806 matched patient pairs were analyzed across four comparisons: patients treated with vedolizumab versus anti-TNF agents (CD = 407 pairs; UC = 443 pairs), and vedolizumab versus ustekinumab (CD = 563 pairs; UC = 393 pairs), respectively. Patients with CD receiving vedolizumab had a significantly higher risk of VTE compared with those receiving anti-TNF therapy (hazard ratio [HR] 8.63; 95% confidence interval [CI] 1.08-69.10; p = 0.014). A higher VTE risk was also observed in vedolizumab-treated patients with CD compared with those treated with ustekinumab (HR 4.64; 95% CI 1.35-15.97; p = 0.007). In contrast, no significant difference in VTE risk was found between vedolizumab and either comparator group in patients with UC.</p><p><strong>Conclusions: </strong>Treatment with vedolizumab was associated with a higher incidence of VTE than anti-TNF agents or ustekinumab among individuals with CD. Prospective cohort studies are warranted to validate the safety of biologic therapy for pediatric patients with CD.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"761-770"},"PeriodicalIF":3.3,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12534362/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145252245","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":"Navigating the Biosimilars from Bench to Bedside in Juvenile Idiopathic Arthritis.","authors":"Elaine M Yung, Amanda Fridley, Tracy Matheny, Hermine I Brunner","doi":"10.1007/s40272-025-00720-7","DOIUrl":"10.1007/s40272-025-00720-7","url":null,"abstract":"<p><p>Biosimilar use for pediatric rheumatologic conditions, such as juvenile idiopathic arthritis (JIA), is increasing owing to the development and release of multiple biosimilars into the US market. The US biosimilar development process of bio-originator and generic medications differs. Bio-originators typically spend the longest amount of time in research and development and require the largest financial investment, followed by biosimilars and, lastly, generic medications. Data available from European countries, where biosimilars have been made available much earlier than in the USA, support the effectiveness and safety of biosimilars in patients with JIA. However, European rheumatology clinicians surveyed highlight continued concerns regarding biosimilar data and experience. Although there are varying perspectives of major stakeholders on biosimilars in the USA-the patients and caregivers, clinicians, manufacturers, and pharmacy benefit managers (PBMs)-the primary goal of all should be patient benefits. These benefits may include improved medication healthcare access overall and during shortage situations or promoting the innovation of biobetters, but biosimilars may conversely negatively impact provider reimbursement, or manufacturers may drive out competition of competing biosimilar manufacturers. There are risks associated with lack of education for medical staff and patients, such as the nocebo effect, and how to reduce those risks is through assisting patients in understanding their new medication and reasons for the change. Credible biosimilar resources may be used to educate medical staff and patients, including the Food and Drug Administration (FDA), American College of Rheumatology (ACR), and Arthritis Foundation. After a patient has been transitioned to a biosimilar, it is necessary to have appropriate medical follow-up and continuous monitoring for efficacy and safety.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"679-691"},"PeriodicalIF":3.7,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125640","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}