Pediatric Drugs最新文献

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Recent Advances in the Management of Seizures in Children. 儿童癫痫发作管理的最新进展。
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-10-04 DOI: 10.1007/s40272-025-00710-9
Frank M C Besag, Michael J Vasey, Richard F M Chin
{"title":"Recent Advances in the Management of Seizures in Children.","authors":"Frank M C Besag, Michael J Vasey, Richard F M Chin","doi":"10.1007/s40272-025-00710-9","DOIUrl":"https://doi.org/10.1007/s40272-025-00710-9","url":null,"abstract":"<p><p>Childhood epilepsies comprise a group of heterogeneous conditions associated with diverse aetiologies, seizure severities/types, comorbidities, degrees of impairment and prognoses. Seizures are refractory to antiseizure medications (ASMs) in around one-third of cases. Alternatives to medication, for example surgical resection, are not always feasible, implying that new treatments are needed. In the past decade, new ASMs have been approved for specific childhood-onset epilepsy syndromes, notably cannabidiol for Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) and tuberous sclerosis complex (TSC); fenfluramine for LGS and DS; everolimus for TSC; and ganaxolone for cyclin-dependent kinase-like deficiency disorder. However, seizure freedom with these medications has rarely been achieved in randomised controlled trials. Alongside ASM development, and surgical strategies such as laser interstitial therapy, neurostimulation modalities have evolved towards responsive systems, such as autostimulation vagus nerve stimulation (VNS) and responsive neurostimulation, and non-invasive devices such as transcutaneous VNS and transcranial direct current stimulation; these have achieved similar decreases in seizure frequency to traditional neurostimulation in some studies. However, data for paediatric epilepsy are limited. Focused ultrasound is being developed not only for seizure focus ablation but also for other approaches to seizure control. In parallel with these developments, accumulating research in the areas of genetic testing, including genetic and related therapies designed to correct or compensate for underlying genetic causes of seizures, suggests that these technologies may have the potential to transform epilepsy treatment in the future. This review summarises major recent developments and current research in the treatment of epilepsy in children.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145225729","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}
引用次数: 0
History and Current Status of Growth Hormone Treatment in Children. 儿童生长激素治疗的历史和现状。
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-29 DOI: 10.1007/s40272-025-00721-6
Camila C Luz, Paulo F Collett-Solberg
{"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":"https://doi.org/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":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-29","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}
引用次数: 0
Fremanezumab: Pediatric First Approval. Fremanezumab:儿科首次批准
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-29 DOI: 10.1007/s40272-025-00722-5
Hannah A Blair
{"title":"Fremanezumab: Pediatric First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40272-025-00722-5","DOIUrl":"https://doi.org/10.1007/s40272-025-00722-5","url":null,"abstract":"<p><p>Fremanezumab (fremanezumab-vfrm; AJOVY<sup>®</sup>) 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":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-29","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}
引用次数: 0
Navigating the Biosimilars from Bench to Bedside in Juvenile Idiopathic Arthritis. 青少年特发性关节炎的生物仿制药从实验到临床应用
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-23 DOI: 10.1007/s40272-025-00720-7
Elaine M Yung, Amanda Fridley, Tracy Matheny, Hermine I Brunner
{"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":"https://doi.org/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":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-23","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}
引用次数: 0
Current Treatment Modalities for Urea Cycle Disorders. 尿素循环障碍的当前治疗方式。
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-22 DOI: 10.1007/s40272-025-00719-0
Nicholas Ah Mew, Uta Lichter-Konecki
{"title":"Current Treatment Modalities for Urea Cycle Disorders.","authors":"Nicholas Ah Mew, Uta Lichter-Konecki","doi":"10.1007/s40272-025-00719-0","DOIUrl":"https://doi.org/10.1007/s40272-025-00719-0","url":null,"abstract":"<p><p>The urea cycle, a metabolic pathway comprising six enzymes and two transporters, is necessary for mammalian nitrogen detoxification. A deficiency of any of these components disrupts this process, leading to the accumulation of nitrogen in the form of ammonia, which is especially toxic to the brain. For decades, treatment of urea cycle disorders has consisted of nitrogen scavengers, dietary protein restriction, arginine or citrulline supplementation, calorie support, and liver transplant. In 2011, carglumic acid became available as a substitute for N-acetylglutamate for N-acetylglutamate synthase deficiency. The past 10 years, however, have seen the development of enzyme therapy for arginase deficiency and gene therapy for ornithine transcarbamylase deficiency. This article reviews the current status and availability of treatment options for urea cycle disorders.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145113495","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}
引用次数: 0
Antipsychotic Medications for Delirium Treatment in the Pediatric Intensive Care Unit: A Systematic Review. 儿童重症监护病房谵妄治疗的抗精神病药物:系统综述。
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-04 DOI: 10.1007/s40272-025-00716-3
Francesca Cavagnero, Annalisa Salerno, Chiara Marchegiani Rizzolli, Luca Marchetto, Valentina Stritoni, Alvise Tosoni, Anna Tessari, Angela Amigoni, Marco Daverio
{"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":"https://doi.org/10.1007/s40272-025-00716-3","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objectives: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;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.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;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":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-04","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}
引用次数: 0
Big Data in the Assessment of Medication Safety in Pregnancy: Opportunities and Challenges. 大数据在妊娠用药安全评估中的机遇与挑战
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-04 DOI: 10.1007/s40272-025-00718-1
Per Damkier, Krista F Huybrechts, Hedvig Nordeng
{"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":"https://doi.org/10.1007/s40272-025-00718-1","url":null,"abstract":"","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-09-04","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}
引用次数: 0
Association of Serum Levels of Ustekinumab, Vedolizumab, and Faecal Calprotectin in Paediatric Patients with Inflammatory Bowel Diseases: A Prospective Observational Study. 儿童炎症性肠病患者血清Ustekinumab、Vedolizumab和粪便钙卫蛋白水平的相关性:一项前瞻性观察研究
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-01 Epub Date: 2025-06-26 DOI: 10.1007/s40272-025-00702-9
J Bronsky, K Zarubova, I Copova, M Durilova, D Kazeka, M Kubat, T Lerchova, K Mitrova, E Vlckova, J Duskova, J Dostalikova, O Hradsky
{"title":"Association of Serum Levels of Ustekinumab, Vedolizumab, and Faecal Calprotectin in Paediatric Patients with Inflammatory Bowel Diseases: A Prospective Observational Study.","authors":"J Bronsky, K Zarubova, I Copova, M Durilova, D Kazeka, M Kubat, T Lerchova, K Mitrova, E Vlckova, J Duskova, J Dostalikova, O Hradsky","doi":"10.1007/s40272-025-00702-9","DOIUrl":"10.1007/s40272-025-00702-9","url":null,"abstract":"<p><strong>Background and objectives: </strong>Ustekinumab (USTE) and vedolizumab (VEDO) are increasingly used in paediatric patients with inflammatory bowel diseases (pIBD). However, data on the usefulness of therapeutic drug monitoring (TDM) in children are scarce. The primary objective of this study was to evaluate the association between disease activity, measured by faecal calprotectin (F-CPT), and serum trough levels (TLs) of USTE and VEDO. Secondary outcomes were to explore factors potentially associated with the outcome and exposure, to determine the optimal USTE or VEDO dose that predicts remission (defined as F-CPT < 250 µg/g), to validate our hypothesis using a proof-of-concept cohort (POCC) and to assess the occurrence of serum antibodies to USTE and VEDO.</p><p><strong>Methods: </strong>This was a prospective single-centre observational study performed at the University Hospital Motol, Prague, Czech Republic. Of the 87 patients (51 Crohn's disease (CD), 30 ulcerative colitis (UC), and 6 IBD unclassified (IBD-U)), drug serum TLs and antibodies were measured in 282 observations (49 treatment courses) of USTE and 359 observations (38 courses) of VEDO. Serum and stool samples were collected before each study drug application during both the induction and maintenance phases of the treatment throughout the entire study period (January 2020 to June 2024). Clinical and laboratory data were obtained from the nationwide prospective registry CREdIT. Patients with perianal disease and those with previous major bowel surgery were not excluded from the study. As a POCC, we analysed a group of pIBD treated at our centre with anti-TNF agents-adalimumab or infliximab.</p><p><strong>Results: </strong>In a linear multiple regression mixed model, an association was observed between logF-CPT levels and USTE treatment duration (β -0.0010, 95% confidence interval (CI) -0.0015 to -0.0006, p < 0.001) but not with USTE TLs (p = 0.12). VEDO TLs and logF-CPT levels were negatively associated both in the linear (β -0.0173, 95% CI -0.0292 to -0.0053, p = 0.005) and categorical models (p = 0.026), even after adjusting for time. A VEDO TL of 15.1 µg/mL showed the best, though still poor, combination of sensitivity (0.82) and specificity (0.32) to predict F-CPT < 250 µg/g (area under the curve (AUC) 0.56, 95% CI 0.49-0.63). Intensification, induction phase, undetectable TLs, and type of IBD (CD, UC, IBD-U) were not associated with logF-CPT. Slightly elevated anti-drug antibodies were detected in 5 USTE and 16 VEDO observations, with no clinical implications.</p><p><strong>Conclusions: </strong>TDM of USTE does not appear to be useful in pIBD. TDM of VEDO may assist in therapeutic strategy decisions, although establishing clinically useful cut-offs remains challenging.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"629-640"},"PeriodicalIF":3.3,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12378899/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144507514","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}
引用次数: 0
Effective Sirolimus Use in Prenatal and Postnatal Management of Symptomatic Extensive Congenital Capillary-Lymphatic-Venous Malformations (CLVMs): A Report of Two Cases. 西罗莫司在产前和产后治疗有症状的广泛先天性毛细血管-淋巴-静脉畸形(clvm)的有效应用:附2例报告
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-01 Epub Date: 2025-07-06 DOI: 10.1007/s40272-025-00708-3
Anna Klosowska, Malgorzata Styczewska, Malgorzata A Krawczyk, Lena Gluszkiewicz, Przemyslaw Adamski, Katarzyna Wartecka-Zielinska, Lukasz Matwiejczyk, Magda Rybak-Krzyszkowska, Daria Dziechcinska-Poletek, Anna Jankowska, Katarzyna Sinacka, Dariusz Wyrzykowski, Anna Taczanowska-Niemczuk, Anna Gabrych, Paulina Kielpinska, Natalia K Mazur-Ejankowska, Iwona Domzalska-Popadiuk, Magdalena Emilia Grzybowska, Dariusz G Wydra, Wojciech Gorecki, Ninela Irga-Jaworska, Ewa Bien
{"title":"Effective Sirolimus Use in Prenatal and Postnatal Management of Symptomatic Extensive Congenital Capillary-Lymphatic-Venous Malformations (CLVMs): A Report of Two Cases.","authors":"Anna Klosowska, Malgorzata Styczewska, Malgorzata A Krawczyk, Lena Gluszkiewicz, Przemyslaw Adamski, Katarzyna Wartecka-Zielinska, Lukasz Matwiejczyk, Magda Rybak-Krzyszkowska, Daria Dziechcinska-Poletek, Anna Jankowska, Katarzyna Sinacka, Dariusz Wyrzykowski, Anna Taczanowska-Niemczuk, Anna Gabrych, Paulina Kielpinska, Natalia K Mazur-Ejankowska, Iwona Domzalska-Popadiuk, Magdalena Emilia Grzybowska, Dariusz G Wydra, Wojciech Gorecki, Ninela Irga-Jaworska, Ewa Bien","doi":"10.1007/s40272-025-00708-3","DOIUrl":"10.1007/s40272-025-00708-3","url":null,"abstract":"<p><strong>Introduction: </strong>Congenital capillary-lymphatic-venous malformations (CLVMs) often result in life-threatening complications, which may begin in utero. Sirolimus, a mammalian target of rapamycin (mTOR) inhibitor, has been successfully used in children and adults with CLVMs due to its antiproliferative and antiangiogenic properties. However, only two cases of prenatal sirolimus treatment of fetal CLVMs have been published to date, with very limited data on optimal therapeutic scheme and drug dosing.</p><p><strong>Case reports: </strong>Here we report two cases of effective prenatal and postnatal sirolimus treatment of extensive, complicated fetal CLVMs. The CLVMs were diagnosed prenatally by ultrasound and confirmed by magnetic resonance. The pregnancies were complicated with intralesional bleeding in both cases and polyhydramnios in one. The pregnant women received oral sirolimus from the 32nd and 33rd weeks of gestation to delivery (for 11 and 31 days, respectively). The dose of oral sirolimus for the pregnant women ranged from 2 to 6 mg/day, with a target trough whole-blood level of 7-12 ng/mL, which resulted in the umbilical cord arterial blood levels of 3.8 and 6.4 ng/mL, respectively. Therapeutic effects of prenatal sirolimus were observed in both fetuses: one experienced reduced intralesional bleeding, while the other had a significant decrease in CLVM size. The sirolimus treatment has been continued postnatally in both children, currently aged 20 and 9 months. The mothers and children experienced no adverse events from the treatment.</p><p><strong>Conclusions: </strong>Administration of sirolimus during pregnancy with maternal blood drug-level monitoring seems to be an efficient and safe treatment option that should be considered in high-risk fetal CLVMs.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"619-627"},"PeriodicalIF":3.3,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12378660/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567730","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}
引用次数: 0
Current and Emerging Therapies for Prevention and Treatment of Bronchopulmonary Dysplasia in Preterm Infants. 预防和治疗早产儿支气管肺发育不良的现有和新兴疗法。
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2025-09-01 Epub Date: 2025-05-15 DOI: 10.1007/s40272-025-00697-3
Margaret A Gilfillan, Adedapo Kiladejo, Vineet Bhandari
{"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":"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":"539-562"},"PeriodicalIF":3.3,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12378787/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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