Pediatric Drugs最新文献

{"title":"Current and Emerging Therapies for Prevention and Treatment of Bronchopulmonary Dysplasia in Preterm Infants.","authors":"Margaret A Gilfillan, Adedapo Kiladejo, Vineet Bhandari","doi":"10.1007/s40272-025-00697-3","DOIUrl":"https://doi.org/10.1007/s40272-025-00697-3","url":null,"abstract":"<p><p>Although advances in the care of extremely preterm born infants have yielded improvements in survival and reductions in important morbidities, rates of bronchopulmonary dysplasia (BPD) have remained relatively unchanged. As BPD can have a long-lasting impact on the quality of life for survivors of prematurity and their families, this remains a continuing challenge. Treatments that have consistently shown efficacy in preventing either BPD or the composite outcome of BPD and death prior to 36 weeks post menstrual age (PMA) in large-scale randomized clinical trials (RCTs) include caffeine [adjusted odds ratio aOR for BPD, 0.63; 95% confidence interval (95% CI) 0.52-0.76; p < 0.001)], vitamin A [relative risk (RR) for death or BPD 0.89; 95% CI 0.80-0.99], low-dose hydrocortisone in the first week of life [OR for survival without BPD, 1.45; 95% CI 1.11-1.90; p = 0.007], and post-natal dexamethasone [RR for BPD or mortality; 0.76; 95% CI 0.66-0.87]. Although early caffeine therapy is now a widely used strategy to prevent BPD, the potentially severe side effects of post-natal glucocorticoids and the concerns regarding the cost-benefit of vitamin A have led to inconsistent use of these drugs in clinical practice. Inhaled bronchodilators and diuretics provide differing degrees of symptomatic relief for patients according to their phenotypic pattern of lung injury; however, these medications do not prevent BPD. Currently available pharmaceuticals do not sufficiently address the degree of structural immaturity and immune dysregulation that is present in the growing population of survivors born prior to 25 weeks gestational age. In this article, we provide both an evidence-based summary of pharmacological treatments currently available to prevent and manage BPD and a discussion of emerging therapies that could help preserve normal lung development in infants born preterm.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144079418","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 and Benefits of Pharmacological Treatment for Pediatric Chronic Non-cancer Pain: When Safety Evidence Lags Behind Prescription Pads.","authors":"Dominique Dundaru-Bandi, Kacper Niburski, Rebecca Pitt, Nada Mohamed, Victor Hugo Gonzalez Cardenas, Lisa M Einhorn, Pablo Ingelmo","doi":"10.1007/s40272-025-00698-2","DOIUrl":"https://doi.org/10.1007/s40272-025-00698-2","url":null,"abstract":"<p><p>The evidence to support the efficacy and safety of pharmacological treatments for chronic non-cancer pain in children is limited. In practice, clinicians are often required to establish therapeutic plans using data extrapolated from adult studies, which may not apply to younger patients. Recent systematic reviews and meta-analyses indicate minimal evidence of benefit for these treatments in children; however, the low quality of studies included in these reviews complicates the conclusions that can be derived from them. In this article, we focus on safety, an outcome as critical as efficacy in clinical trial design but often designated as secondary or even exploratory. Specifically, we examine methods for assessing adverse events in clinical research and propose a practical approach for evaluating these events in everyday practice. Additionally, we outline our strategy to conduct a risk-benefit analysis at the individual patient level, highlighting the importance of using a composite risk-benefit metric rather than assessing these outcomes separately. This approach enables real-time monitoring of both drug-related symptom relief and adverse effects, facilitating clinically meaningful risk-benefit discussions with patients and their families. Finally, we advocate for improvements in clinical trial design for pediatric chronic pain treatments, particularly around adverse events. Future trials should incorporate standardized definitions, comprehensive risk-benefit evaluations, and transparent outcome reporting. Implementing these changes may enhance decision-making by balancing the safety and the effectiveness of pharmacological treatments for children and adolescents with chronic pain.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017133","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":"Correction: Optimising Paediatric HIV Treatment: Recent Developments and Future Directions.","authors":"Anne E M Kamphuis, Alasdair Bamford, Alfredo Tagarro, Tim R Cressey, Adrie Bekker, Pauline Amuge, Hilda Angela Mujuru, Francis Ateba Ndongo, Aminata Diack, Alexandra Compagnucci, Marc Lallemant, Angela Colbers, Anna Turkova","doi":"10.1007/s40272-025-00696-4","DOIUrl":"https://doi.org/10.1007/s40272-025-00696-4","url":null,"abstract":"","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143986816","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":"Off-Label Use of Anakinra in Inflammatory Conditions in Neonates and Infants Up to 3 Months of Age: A Case Series and a Review of the Literature.","authors":"Domenico Umberto De Rose, Francesca Campi, Chiara Maddaloni, Sara Ronci, Stefano Caoci, Immacolata Savarese, Iliana Bersani, Maria Paola Ronchetti, Cinzia Auriti, Irma Capolupo, Pietro Merli, Antonella Insalaco, Fabrizio De Benedetti, Andrea Dotta","doi":"10.1007/s40272-024-00679-x","DOIUrl":"10.1007/s40272-024-00679-x","url":null,"abstract":"<p><strong>Background: </strong>Anakinra is an interleukin-1 receptor antagonist (IL-1Ra). Since IL-1 has been shown to play a key role in the etiology of different autoinflammatory diseases, blocking its pathway has become an important therapeutic target, even in neonates.</p><p><strong>Aims: </strong>We aimed to report our experience in using anakinra to treat specific neonatal inflammatory conditions.</p><p><strong>Methods: </strong>We described the clinical management with anakinra of five cases of neonates or infants up to 3 months of age admitted to the neonatal intensive care unit (NICU) of Bambino Gesù Children's Hospital IRCCS in Rome (Italy) from 2020 onwards. Medical history and clinical data concerning NICU hospitalization were collected from the electronic medical records. Furthermore, we performed a literature review of off-label anakinra in the first 3 months of life, up to 5 April 2024. We excluded from this review cases of cryopyrin-associated periodic syndrome, deficiency of the interleukin-1 receptor antagonist, and mevalonate kinase deficiency, for which anakinra is a known treatment.</p><p><strong>Results: </strong>We reported three off-label cardiorespiratory reasons to use IL-1Ra from our series: (i) chronic lung disease with pulmonary hypertension, (ii) interstitial lung disease with pulmonary hypertension to facilitate the weaning from respiratory support, and (iii) post-surgical polyserositis if effusions accumulate despite drainage. In all our patients, the drug was administered at a dosage of 10 mg/kg/day. The route of administration was chosen based on the patient's clinical characteristics, with the subcutaneous and intravenous routes being comparable in efficacy. The duration of therapy was modulated based on the patient's clinical response, with a minimum duration of 4 months. A total of 308 retrieved articles were screened, and then full texts of records deemed eligible for inclusion were assessed. Based on the literature search and our five cases, a total of 17 infants were treated with anakinra outside its approved indications. The major off-label use was for hemophagocytic lymphohistiocytosis/macrophage activation syndrome, followed by multisystem inflammatory syndrome in children and Kawasaki disease, as in two of our cases.</p><p><strong>Conclusions: </strong>According to the results of our case series and review of the literature, the off-label use of anakinra in neonates with inflammatory conditions refractory to first-line therapy could be considered. Prospective, multicenter research is necessary to determine whether anakinra is a safe treatment option for these infants to prevent early inflammatory illnesses and in which situations it could enhance clinical results.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"293-305"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12031743/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142971897","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":"Pharmacological Aspects in the Management of Children and Adolescents with Prader-Willi Syndrome.","authors":"Jennifer Miller, Shivani Berry, Esraa Ismail","doi":"10.1007/s40272-025-00681-x","DOIUrl":"10.1007/s40272-025-00681-x","url":null,"abstract":"<p><p>Prader-Willi syndrome is a rare neurodevelopmental disorder that impacts the musculoskeletal, endocrine, pulmonary, neurologic, ocular, and gastrointestinal systems. In addition, individuals with Prader-Willi syndrome have issues with cognitive development, characteristic behavioral problems, and perhaps most profoundly, appetite control. Currently, the only US Food and Drug Administration-approved therapy for Prader-Willi syndrome is growth hormone, which has been Food and Drug Administration approved for > 20 years for the treatment of growth failure in Prader-Willi syndrome. Growth hormone has shown to improve many aspects of this syndrome, including final height, body composition, developmental milestones, and cognition, but it does not affect hyperphagia, which is the hallmark symptom of this condition. Over the past 15 years, there have been several medication trials for the treatment of hyperphagia in Prader-Willi syndrome, but thus far, all have failed to achieve Food and Drug Administration approval for a variety of reasons. However, hyperphagia is the most life-limiting symptom of Prader-Willi syndrome, thus new pharmacologic therapies are desperately needed. We review ongoing and recently completed clinical trials for hyperphagia. Other issues in Prader-Willi syndrome that significantly impact quality of life include excessive daytime sleepiness and severe behavioral problems. We examine the medication trials to address these issues.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"273-281"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143053171","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":"Tapinarof Cream 1%: Pediatric First Approval.","authors":"Sheridan M Hoy","doi":"10.1007/s40272-025-00689-3","DOIUrl":"10.1007/s40272-025-00689-3","url":null,"abstract":"<p><p>The aryl hydrocarbon receptor (AhR) has an integral role in maintaining skin homeostasis. Tapinarof cream 1% (VTAMA^®) is an AhR agonist developed by Dermavant Sciences, an Organon Company, as a once-daily topical treatment for plaque psoriasis and atopic dermatitis (AD). It was first approved in May 2022 in the USA for the topical treatment of plaque psoriasis in adults. It was then approved in June 2024 in Japan for the topical treatment of plaque psoriasis in adults and AD in adults and pediatric patients 12 years of age and older. In December 2024, it was approved in the USA for the topical treatment of AD in adults and pediatric patients 2 years of age and older. This article summarizes the milestones in the development of tapinarof cream 1% leading to this first pediatric approval.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"383-391"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743748","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":"Macitentan: Pediatric First Approval.","authors":"Susan J Keam","doi":"10.1007/s40272-025-00692-8","DOIUrl":"10.1007/s40272-025-00692-8","url":null,"abstract":"<p><p>Macitentan (Opsumit^®), an endothelin receptor antagonist (ERA) developed by Johnson & Johnson, is well established worldwide as monotherapy or combination therapy for the long-term treatment of pulmonary arterial hypertension (PAH). In September 2024, based on phase 3 clinical data in patients aged < 18 years, macitentan received its first pediatric approval in the EU as monotherapy or in combination for the long-term treatment of PAH in pediatric patients aged 2 years to < 18 years with WHO functional class (FC) II to III. Macitentan has also been approved in the UK for this indication. This article summarizes the milestones in the development of macitentan leading to the first pediatric approval for the long-term treatment of PAH.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"377-382"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143634382","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":"Antibiotics for Paediatric Community-Acquired Pneumonia: What is the Optimal Course Duration?","authors":"Hing Cheong Kok, Anne B Chang, Siew Moy Fong, Gabrielle B McCallum, Stephanie T Yerkovich, Keith Grimwood","doi":"10.1007/s40272-024-00680-4","DOIUrl":"10.1007/s40272-024-00680-4","url":null,"abstract":"<p><p>Despite significant global reductions in cases of pneumonia during the last 3 decades, pneumonia remains the leading cause of post-neonatal mortality in children aged <5 years. Beyond the immediate disease burden it imposes, pneumonia contributes to long-term morbidity, including lung function deficits and bronchiectasis. Viruses are the most common cause of childhood pneumonia, but bacteria also play a crucial role. However, the optimal duration of antibiotic therapy for bacterial pneumonia remains uncertain in both low- and middle-income countries and in high-income countries. Knowing the optimal duration of antibiotic therapy for pneumonia is crucial for effective antimicrobial stewardship. This is especially important as concerns mount over rising antibiotic resistance in respiratory bacterial pathogens, which increases the risk of treatment failure. Numerous studies have focused on the duration of oral antibiotics and short-term outcomes, such as clinical cure and mortality. In contrast, only one study has examined both intravenous and oral antibiotics and their impact on long-term respiratory outcomes following pneumonia hospitalisation. However, study findings may be influenced by their inclusion criteria when children unlikely to have bacterial pneumonia are included. Efforts to differentiate between bacterial and non-bacterial pneumonia continue, but a validated, accurate, and simple point-of-care diagnostic test remains elusive. Without certainty that a child has bacterial pneumonia, determining the optimal duration of antibiotic treatment is challenging. This review examines the evidence for the recommended duration of antibiotics for treating uncomplicated pneumonia in otherwise healthy children and concludes that the question of duration is unresolved.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"261-272"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12031807/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024340","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":"Neuroimaging as a Tool for Advancing Pediatric Psychopharmacology.","authors":"Michael Bartkoski, John Tumberger, Laura Martin, In-Young Choi, Phil Lee, Jeffrey R Strawn, William M Brooks, Stephani L Stancil","doi":"10.1007/s40272-025-00683-9","DOIUrl":"10.1007/s40272-025-00683-9","url":null,"abstract":"<p><p>Neuroimaging, specifically magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), and positron emission tomography (PET), plays an important role in improving the therapeutic landscape of pediatric neuropsychopharmacology by detecting target engagement, pathway modulation, and disease-related changes in the brain. This review provides a comprehensive update on the application of neuroimaging to detect neural effects of psychotropic medication in pediatrics. Additionally, we discuss opportunities and challenges for expanding the use of neuroimaging to advance pediatric neuropsychopharmacology. PubMed and Embase were searched for studies published between 2012 and 2024 reporting neural effects of attention deficit hyperactivity disorder (ADHD) medications (e.g., methylphenidate, amphetamine, atomoxetine, guanfacine), selective serotonin reuptake inhibitors (e.g., fluoxetine, escitalopram, sertraline), serotonin/norepinephrine reuptake inhibitors (e.g., duloxetine, venlafaxine), second-generation antipsychotics (e.g., aripiprazole, olanzapine, risperidone, quetiapine, ziprasidone), and others (e.g., lithium, carbamazepine, lamotrigine, ketamine, naltrexone) used to treat pediatric psychiatric conditions. Of the studies identified (N = 57 in 3314 pediatric participants), most (86%, total participants n = 3045) used MRI to detect functional pathway modulation or anatomical changes. Fewer studies (14%, total participants n = 269) used MRS to understand neurochemical modulation. No studies used PET. Studies that included healthy controls detected normalization of disease-altered pathways following treatment. Studies that focused on affected youth detected neuromodulation following single-dose and ongoing treatment. Neuroimaging is positioned to serve as a biomarker capable of demonstrating acute brain modulation, predicting clinical response, and monitoring disease, yet biomarker validation requires further work. Neuroimaging is also well suited to fill the notable knowledge gap of long-term neuromodulatory effects of psychotropic medications in the context of ongoing brain development in children and adolescents. Future studies can leverage advancements in neuroimaging technology, acquisition, and analysis to fill these gaps and accelerate the discovery of novel therapeutics, leading to more effective prescribing and ensuring faster recovery.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"307-330"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12031650/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143080794","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":"Meningococcal Vaccination in the United States: Past, Present, And Future.","authors":"Sarah Schillie, Lucy A McNamara","doi":"10.1007/s40272-024-00666-2","DOIUrl":"10.1007/s40272-024-00666-2","url":null,"abstract":"<p><p>Meningococcal disease is rare but serious, often striking previously healthy adolescents or young adults, with substantial morbidity and mortality. The incidence of meningococcal disease in the USA declined even prior to the issuance of routine recommendations for vaccination, although an uptick in incidence has occurred since 2022. Routine recommendations for adolescent MenACWY vaccination were issued in 2005, and recommendations for adolescent MenB vaccination based on shared clinical decision-making (SCDM) were issued in 2015. Although meningococcal vaccines are safe and effective, their limited duration of protection coupled with low disease incidence result in a high cost per case averted by vaccination, most notably with MenB vaccines. The low cost-effectiveness raises ethical concerns about resource use and the role of economic analyses in policy decisions. However, the potential for substantial public health impact remains. Outer membrane vesicle (OMV)-containing MenB vaccines provide some protection against gonorrhea infections. The recent development of pentavalent ABCWY vaccines provide the opportunity to reduce the number of injections and simplify implementation, provided MenACWY and MenB vaccine schedules are harmonized. Vaccine attributes, implementation issues, and resource utilization will be important considerations in optimization of the US adolescent meningococcal vaccination strategy.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"331-349"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143468423","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}