Pediatric Drugs最新文献

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Drug-Drug Interactions Involving High-Alert Medications that Lead to Interaction-Associated Symptoms in Pediatric Intensive Care Patients: A Retrospective Study. 导致儿科重症监护患者出现相互作用相关症状的高警戒药物间相互作用:一项回顾性研究。
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-09-01 Epub Date: 2024-07-04 DOI: 10.1007/s40272-024-00641-x
Lisa Marie Kiesel, Astrid Bertsche, Wieland Kiess, Manuela Siekmeyer, Thilo Bertsche, Martina Patrizia Neininger
{"title":"Drug-Drug Interactions Involving High-Alert Medications that Lead to Interaction-Associated Symptoms in Pediatric Intensive Care Patients: A Retrospective Study.","authors":"Lisa Marie Kiesel, Astrid Bertsche, Wieland Kiess, Manuela Siekmeyer, Thilo Bertsche, Martina Patrizia Neininger","doi":"10.1007/s40272-024-00641-x","DOIUrl":"10.1007/s40272-024-00641-x","url":null,"abstract":"<p><strong>Background: </strong>Children treated in a pediatric intensive care unit (PICU) often receive several drugs together, among them drugs defined as high-alert medications (HAMs). Those drugs carry a high risk of causing patient harm, for example, due to a higher potential for interactions. HAMs should therefore be administered with caution, especially in a PICU.</p><p><strong>Objectives: </strong>The objective of the current study was to identify drug-drug interactions involving HAMs that increase the risk of interaction-associated symptoms in pediatric intensive care.</p><p><strong>Methods: </strong>In a retrospective study, we analyzed the electronic documentation of patients hospitalized for at least 48 h in a general PICU who received at least two different drugs within a 24-h interval. We assessed potential drug-drug interactions involving HAM on the basis of the two drug information databases UpToDate and drugs.com. Furthermore, we analyzed whether symptoms were observed after the administration of drug pairs that could lead to interaction-associated symptoms. For drug pairs involving HAM administered on at least 2% of patient days, and symptoms observed at least ten times after a respective drug pair, we calculated odds ratios, 95% confidence intervals, and p-values by using a univariate binary logistic regression.</p><p><strong>Results: </strong>Among 315 analyzed patients, 81.3% (256/315) received drugs defined as high-alert medication for pediatric patients. Those high-alert medications were involved in 20,150 potential drug-drug interactions. In 14.0% (2830/20,150) of these, one or more symptoms were observed that could be a possible consequence of the interaction, resulting in 3203 observed symptoms affecting 56.3% (144/256) of patients receiving high-alert medication. The odds ratios for symptoms observed after a drug-drug interaction were increased for eight specific symptoms (each p ≤ 0.05), especially hemodynamic alterations and disturbances of electrolyte and fluid balance. The odds ratio was highest for decreased blood pressure observed after the administration of the drug pair fentanyl and furosemide (OR 5.06; 95% confidence interval 3.5-7.4; p < 0.001). Increased odds ratios for specific symptoms observed after drug-drug interactions resulted from eight combinations composed of eight different drugs: digoxin, fentanyl, midazolam, phenobarbital, potassium salts and vancomycin (high-alert medications), and the diuretics furosemide and hydrochlorothiazide (non-high-alert medications). The resulting drug pairs were: potassium salts-furosemide, fentanyl-furosemide, vancomycin-furosemide, digoxin-furosemide, digoxin-hydrochlorothiazide, fentanyl-phenobarbital, potassium salts-hydrochlorothiazide, and midazolam-hydrochlorothiazide.</p><p><strong>Conclusions: </strong>In a cohort of PICU patients, this study identified eight specific drug pairs involving high-alert medications that may increase the risk of interaction-associa","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"619-629"},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11335839/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141498710","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
Adverse Drug Reactions in Children with Congenital Heart Disease: A Scoping Review. 先天性心脏病患儿的药物不良反应:范围综述。
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-09-01 Epub Date: 2024-07-23 DOI: 10.1007/s40272-024-00644-8
Esmaeel Toni, Haleh Ayatollahi, Reza Abbaszadeh, Alireza Fotuhi Siahpirani
{"title":"Adverse Drug Reactions in Children with Congenital Heart Disease: A Scoping Review.","authors":"Esmaeel Toni, Haleh Ayatollahi, Reza Abbaszadeh, Alireza Fotuhi Siahpirani","doi":"10.1007/s40272-024-00644-8","DOIUrl":"10.1007/s40272-024-00644-8","url":null,"abstract":"<p><strong>Background: </strong>Congenital heart disease (CHD) is one of the leading causes of death. Safe and timely medical interventions, especially in children, can prolong their survival. The drugs prescribed for children with CHD are mainly based on the outcomes of drug therapy in adults with cardiovascular diseases, and their adverse drug reactions (ADRs) might be different. Therefore, the aim of this study was to investigate ADRs in children with CHD.</p><p><strong>Methods: </strong>This was a scoping review conducted in 2023. PubMed, Web of Science, Scopus, the Cochrane Library, Ovid, ProQuest, and Google Scholar databases were searched. All studies that reported ADRs for children with CHD and were published in English by 1 November 2023 were included in this study. Finally, the results were reported using a content analysis method.</p><p><strong>Results: </strong>A total of 87 articles were included in the study. The results showed that symptoms/signs/clinical findings, and cardiovascular disorders were the most common ADRs reported in children with CHD. The results also showed that most of the ADRs were reported for prostaglandin E1, amiodarone, prostaglandin E2, dexmedetomidine, and captopril, respectively.</p><p><strong>Conclusion: </strong>The review underscores the wide array of ADRs in children with CHD, particularly in antiarrhythmics, diuretics, beta-blockers, anticoagulants, and vasodilators, which affected cardiovascular, respiratory, endocrine, metabolic, genitourinary, gastrointestinal, and musculoskeletal systems. Tailored treatment is imperative, considering individual patient characteristics, especially in the vulnerable groups. Further research is essential for optimizing dosing, pharmacogenetics, and alternative therapies to enhance patient outcomes in CHD management.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"519-553"},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141752358","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
Personalized Dosing of Medicines for Children: A Primer on Pediatric Pharmacometrics for Clinicians. 儿童个性化用药:临床医生的儿科药物计量学入门》。
IF 3.3 3区 医学
Pediatric Drugs Pub Date : 2024-07-01 Epub Date: 2024-05-16 DOI: 10.1007/s40272-024-00633-x
Kevin Meesters, Violeta Balbas-Martinez, Karel Allegaert, Kevin J Downes, Robin Michelet
{"title":"Personalized Dosing of Medicines for Children: A Primer on Pediatric Pharmacometrics for Clinicians.","authors":"Kevin Meesters, Violeta Balbas-Martinez, Karel Allegaert, Kevin J Downes, Robin Michelet","doi":"10.1007/s40272-024-00633-x","DOIUrl":"10.1007/s40272-024-00633-x","url":null,"abstract":"<p><p>The widespread use of drugs for unapproved purposes remains common in children, primarily attributable to practical, ethical, and financial constraints associated with pediatric drug research. Pharmacometrics, the scientific discipline that involves the application of mathematical models to understand and quantify drug effects, holds promise in advancing pediatric pharmacotherapy by expediting drug development, extending applications, and personalizing dosing. In this review, we delineate the principles of pharmacometrics, and explore its clinical applications and prospects. The fundamental aspect of any pharmacometric analysis lies in the selection of appropriate methods for quantifying pharmacokinetics and pharmacodynamics. Population pharmacokinetic modeling is a data-driven method ('top-down' approach) to approximate population-level pharmacokinetic parameters, while identifying factors contributing to inter-individual variability. Model-informed precision dosing is increasingly used to leverage population pharmacokinetic models and patient data, to formulate individualized dosing recommendations. Physiologically based pharmacokinetic models integrate physicochemical drug properties with biological parameters ('bottom-up approach'), and is particularly valuable in situations with limited clinical data, such as early drug development, assessing drug-drug interactions, or adapting dosing for patients with specific comorbidities. The effective implementation of these complex models hinges on strong collaboration between clinicians and pharmacometricians, given the pivotal role of data availability. Promising advancements aimed at improving data availability encompass innovative techniques such as opportunistic sampling, minimally invasive sampling approaches, microdialysis, and in vitro investigations. Additionally, ongoing research efforts to enhance measurement instruments for evaluating pharmacodynamics responses, including biomarkers and clinical scoring systems, are expected to significantly bolster our capacity to understand drug effects in children.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"365-379"},"PeriodicalIF":3.3,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12305683/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140958752","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
Pediatric Treatment-Resistant Obsessive Compulsive Disorder: Treatment Options and Challenges. 儿科难治性强迫症:治疗方案与挑战。
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-07-01 Epub Date: 2024-06-14 DOI: 10.1007/s40272-024-00639-5
Sana Younus, Lauren Havel, Jordan T Stiede, Catherine E Rast, Kirti Saxena, Wayne K Goodman, Eric A Storch
{"title":"Pediatric Treatment-Resistant Obsessive Compulsive Disorder: Treatment Options and Challenges.","authors":"Sana Younus, Lauren Havel, Jordan T Stiede, Catherine E Rast, Kirti Saxena, Wayne K Goodman, Eric A Storch","doi":"10.1007/s40272-024-00639-5","DOIUrl":"10.1007/s40272-024-00639-5","url":null,"abstract":"<p><p>Pediatric obsessive-compulsive disorder (OCD) is a chronic, potentially debilitating psychiatric condition. Although effective treatments exist, at least 10% of youth do not achieve remission despite receiving first-line treatments. This article reviews the extant, albeit limited, evidence supporting treatment approaches for youth with treatment-resistant OCD. A literature search for articles addressing pediatric treatment-resistant OCD was conducted through April 11, 2024. These results were augmented by searching for treatment-resistant OCD in adults; treatment strategies discovered for the adult population were then searched in the context of children and adolescents. In general, intensive treatment programs and antipsychotic augmentation of an antidepressant had the most substantial and consistent evidence base for treatment-resistant youth with OCD, although studies were limited and of relatively poor methodological quality (i.e., open trials, naturalistic studies). Several pharmacological approaches (clomipramine, antipsychotics [e.g., aripiprazole, risperidone], riluzole, ketamine, D-cycloserine, memantine, topiramate, N-acetylcysteine, ondansetron), largely based on supporting data among adults, have received varying levels of investigation and support. There is nascent support for how to treat pediatric treatment-resistant OCD. Future treatment studies need to consider how to manage the significant minority of youth who fail to benefit from first-line treatment approaches.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"397-409"},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141321373","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
A Pilot Study of Ketotifen in Patients Aged 8-17 Years with Functional Dyspepsia Associated with Mucosal Eosinophilia. 对 8-17 岁伴有黏膜嗜酸性粒细胞增多症的功能性消化不良患者使用酮替芬的试点研究
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-07-01 Epub Date: 2024-05-21 DOI: 10.1007/s40272-024-00628-8
Chance S Friesen, Valentina Shakhnovich, Paul Toren, Brandon Retke, Jennifer Schurman, Jennifer Colombo, Amanda Deacy, Craig A Friesen, Susan Abdel-Rahman
{"title":"A Pilot Study of Ketotifen in Patients Aged 8-17 Years with Functional Dyspepsia Associated with Mucosal Eosinophilia.","authors":"Chance S Friesen, Valentina Shakhnovich, Paul Toren, Brandon Retke, Jennifer Schurman, Jennifer Colombo, Amanda Deacy, Craig A Friesen, Susan Abdel-Rahman","doi":"10.1007/s40272-024-00628-8","DOIUrl":"10.1007/s40272-024-00628-8","url":null,"abstract":"<p><strong>Background and objective: </strong>Mast cells have been implicated in abdominal pain-associated disorders of gut-brain interaction, such as functional dyspepsia. As such, ketotifen, a second-generation antihistamine and mast cell stabilizer, could represent a viable treatment option in these conditions. The primary aim of the current pilot study was to assess clinical response to ketotifen and assess pharmacokinetics in youth with functional dyspepsia.</p><p><strong>Methods: </strong>We conducted a pilot randomized, double-blind, placebo-controlled, cross-over trial of ketotifen in 11 youth with functional dyspepsia and duodenal mucosal eosinophilia with 4 weeks of active treatment at a dose of 1 mg twice daily. Global clinical response was graded on a 5-point Likert Scale. A single plasma sample was obtained at steady state for pharmacokinetic analysis.</p><p><strong>Results: </strong>Ketotifen was not superior to placebo with regard to global clinical response. Only 18% of patients demonstrated a complete or near-complete clinical response. The estimated half-life was 3.3 h.</p><p><strong>Conclusions: </strong>While ketotifen was not superior to placebo, this study highlights several important challenges for developing drug trials for youth with chronic abdominal pain. Recommendations are made for designing a larger treatment trial for ketotifen in this patient group.</p><p><strong>Clinical trial registration: </strong>This study was registered at ClinicalTrials.gov: NCT02484248.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"451-457"},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141071667","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
Machine Learning: A Potential Therapeutic Tool to Facilitate Neonatal Therapeutic Decision Making. 机器学习:促进新生儿治疗决策的潜在治疗工具。
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-07-01 Epub Date: 2024-06-16 DOI: 10.1007/s40272-024-00638-6
Bo-Hao Tang, Qiu-Yue Li, Hui-Xin Liu, Yi Zheng, Yue-E Wu, John van den Anker, Guo-Xiang Hao, Wei Zhao
{"title":"Machine Learning: A Potential Therapeutic Tool to Facilitate Neonatal Therapeutic Decision Making.","authors":"Bo-Hao Tang, Qiu-Yue Li, Hui-Xin Liu, Yi Zheng, Yue-E Wu, John van den Anker, Guo-Xiang Hao, Wei Zhao","doi":"10.1007/s40272-024-00638-6","DOIUrl":"10.1007/s40272-024-00638-6","url":null,"abstract":"<p><p>Bacterial infection is one of the major causes of neonatal morbidity and mortality worldwide. Finding rapid and reliable methods for early recognition and diagnosis of bacterial infections and early individualization of antibacterial drug administration are essential to eradicate these infections and prevent serious complications. However, this is often difficult to perform due to non-specific clinical presentations, low accuracy of current diagnostic methods, and limited knowledge of neonatal pharmacokinetics. Although neonatal medicine has been relatively late to embrace the benefits of machine learning (ML), there have been some initial applications of ML for the early prediction of neonatal sepsis and individualization of antibiotics. This article provides a brief introduction to ML and discusses the current state of the art in diagnosing and treating neonatal bacterial infections, gaps, potential uses of ML, and future directions to address the limitations of current studies. Neonatal bacterial infections involve a combination of physiologic development, disease expression, and treatment response outcomes. To address this complex relationship, future models could consider appropriate ML algorithms to capture time series features while integrating influences from the host, microbes, and drugs to optimize antimicrobial drug use in neonates. All models require prospective clinical trials to validate their clinical utility before clinical use.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"355-363"},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141331614","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
Inotuzumab Ozogamicin: First Pediatric Approval. 伊诺珠单抗-奥佐加米星:首次获得儿科批准。
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-07-01 Epub Date: 2024-05-23 DOI: 10.1007/s40272-024-00634-w
Sohita Dhillon
{"title":"Inotuzumab Ozogamicin: First Pediatric Approval.","authors":"Sohita Dhillon","doi":"10.1007/s40272-024-00634-w","DOIUrl":"10.1007/s40272-024-00634-w","url":null,"abstract":"<p><p>Inotuzumab ozogamicin (BESPONSA™) is a CD22-targeted monoclonal antibody drug conjugate (ADC) developed by Pfizer for the treatment of CD22-postive B-cell precursor acute lymphoblastic leukaemia (ALL). Inotuzumab ozogamicin comprises a humanized IgG4 anti-CD22 monoclonal antibody covalently linked to the potent DNA-binding cytotoxic agent N-acetyl-gamma-calicheamicin dimethylhydrazide (CalichDMH) via a linker. Inotuzumab ozogamicin binds to CD22-expressing tumour cells, facilitating the delivery of conjugated CalichDMH, which after intracellular activation induces double strand DNA breaks, ultimately leading to cell cycle arrest and apoptotic cell death. Inotuzumab ozogamicin is approved in the USA, Europe and several countries worldwide for the treatment of relapsed or refractory CD22-positive B-cell precursor ALL in adults. On 6 March 2024, inotuzumab ozogamicin received its first pediatric approval in the USA for this indication in patients aged ≥ 1 years. Inotuzumab ozogamicin has since been approved in Japan in March 2024 for the same indication in pediatric patients. This article summarizes the milestones in the development of inotuzumab ozogamicin leading to this first approval for the treatment of relapsed or refractory CD22-positive B-cell precursor ALL in pediatric patients.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"459-467"},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141081840","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
Diagnosis and Management of Pediatric Neuropsychiatric Systemic Lupus Erythematosus: An Update. 小儿神经精神系统性红斑狼疮的诊断和管理:最新进展。
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-07-01 Epub Date: 2024-05-28 DOI: 10.1007/s40272-024-00632-y
Dilara Unal, Veysel Cam, Hulya Ercan Emreol, Seza Özen
{"title":"Diagnosis and Management of Pediatric Neuropsychiatric Systemic Lupus Erythematosus: An Update.","authors":"Dilara Unal, Veysel Cam, Hulya Ercan Emreol, Seza Özen","doi":"10.1007/s40272-024-00632-y","DOIUrl":"10.1007/s40272-024-00632-y","url":null,"abstract":"<p><p>Neuropsychiatric systemic lupus erythematosus (NPSLE) is a potentially serious and life-threatening complication of SLE. The presentation and severity of neuropsychiatric involvement in SLE may show considerable variability. The disease can affect the neural tissue directly or may be associated with vascular involvement, mainly associated with anti-phospholipid (aPL) antibodies. A direct causal link with SLE may sometimes be challenging since there are many confounding factors and the symptoms may be non-specific. Despite its remarkable sensitivity in detecting hemorrhagic and ischemic stroke, transverse myelitis and ischemic infarction, magnetic resonance imaging (MRI) lacks the spatial resolution required to identify microvascular involvement. When standard MRI fails to detect a suspicious lesion, it is advisable to use advanced imaging modalities such as positron emission tomography (PET), single photon emission computed tomography (SPECT) or quantitative MRI, if available. Even with these advanced modalities, the specificity of neuroimaging in NPSLE remains inadequate (60-82% for MRI). Neuropsychiatric syndromes, such as cerebrovascular events, seizures and cognitive impairments appear to be associated with serum aPL antibodies. Some studies have shown that anti-ribosomal P antibodies have a low sensitivity for NPSLE and a limited contribution to the differentiation of different clinical entities. Treatment has two main goals: symptomatic relief and treatment of the disease itself. Commonly used immunosuppressants for NPSLE include cyclophosphamide (CYC), azathioprine (AZA), and mycophenolate mofetil (MMF). According to EULAR's current recommendation, strong immunosuppressants such as CYC and rituximab (RTX) should be preferred. Biologics have also been used in NPSLE. Fingolimod, eculizumab, and JAK inhibitors are potential drugs in the pipeline. Developing targeted therapies will be possible by a better understanding of the pathological mechanisms.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"381-395"},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141158621","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
Real-World Data on the Use of Sirolimus in Asian Children with Vascular Malformations. 亚洲血管畸形儿童使用西罗莫司的真实世界数据。
IF 3.4 3区 医学
Pediatric Drugs Pub Date : 2024-05-01 Epub Date: 2024-01-27 DOI: 10.1007/s40272-023-00605-7
Lu Yu, Zigang Xu, Li Wei, Bin Zhang, Lei Qiu, Lin Ma, Li Li
{"title":"Real-World Data on the Use of Sirolimus in Asian Children with Vascular Malformations.","authors":"Lu Yu, Zigang Xu, Li Wei, Bin Zhang, Lei Qiu, Lin Ma, Li Li","doi":"10.1007/s40272-023-00605-7","DOIUrl":"10.1007/s40272-023-00605-7","url":null,"abstract":"<p><strong>Objectives: </strong>The management of vascular malformations is complex and challenging. This study aimed to explore efficacy, plasma trough concentrations of sirolimus, post-withdrawal conditions, and adverse reactions of sirolimus in treating complex vascular malformations.</p><p><strong>Methods: </strong>In our center, we analyzed vascular malformations treated with sirolimus (and corticosteroid) from August 2017 to June 2021. Meanwhile, we reviewed the medical records, the efficacy, side effects, and laboratory tests. Patients who had stopped taking sirolimus were followed up by telephone.</p><p><strong>Results: </strong>A total of 25 patients with complicated vascular malformations in our center, including 7 females and 18 males aged 4 months to 15 years, were enrolled. In all, 19 patients (76.0%) responded to sirolimus, and the plasma concentration of sirolimus fluctuated between 0.97 and 27.15 ng/ml. In all, 24 patients (96.0%) were in follow-up. A total of 15 patients (62.5%) stopped taking sirolimus during follow-up, and 2 patients (13.3%) discontinued the sirolimus due to side effects. A total of 3 patients (20.0%) restarted sirolimus treatment.</p><p><strong>Conclusion: </strong>Starting dose of 1.5-2 mg/m<sup>2</sup> sirolimus is effective and safe in vascular malformation treatment. The best treatment regimen and discontinuation indications needed more investigation. Most should be done about targeted therapy to improve effectiveness and reduce side effects.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"309-317"},"PeriodicalIF":3.4,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139570677","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
Improving Methylphenidate Titration in Children with Attention-Deficit/Hyperactivity Disorder (ADHD): A Randomized Controlled Trial Using Placebo-Controlled Titration Implemented in Clinical Practice. 改善注意力缺陷/多动障碍(ADHD)儿童的哌醋甲酯滴定:在临床实践中使用安慰剂控制滴定法的随机对照试验》(Randomized Controlled Trial Using Placebo-Controlled Titration Implemented in Clinical Practice)。
IF 3.7 3区 医学
Pediatric Drugs Pub Date : 2024-05-01 Epub Date: 2024-01-27 DOI: 10.1007/s40272-023-00604-8
Karen Vertessen, Marjolein Luman, Pierre Bet, Catharina E Bergwerff, Marco Bottelier, Reino Stoffelsen, James M Swanson, Annemiek Wisse, Jos Twisk, Jaap Oosterlaan
{"title":"Improving Methylphenidate Titration in Children with Attention-Deficit/Hyperactivity Disorder (ADHD): A Randomized Controlled Trial Using Placebo-Controlled Titration Implemented in Clinical Practice.","authors":"Karen Vertessen, Marjolein Luman, Pierre Bet, Catharina E Bergwerff, Marco Bottelier, Reino Stoffelsen, James M Swanson, Annemiek Wisse, Jos Twisk, Jaap Oosterlaan","doi":"10.1007/s40272-023-00604-8","DOIUrl":"10.1007/s40272-023-00604-8","url":null,"abstract":"<p><strong>Background and objectives: </strong>Concerns exist regarding the rising use of methylphenidate. A double-blind, placebo-controlled methylphenidate titration (PCT) for children with attention-deficit/hyperactivity disorder (ADHD) has shown potential to improve titration (i.e., detection of placebo responders and larger ADHD symptom improvement) in experimental settings. This study aims to determine if these advantages can be transferred to clinical settings.</p><p><strong>Method: </strong>Children (aged 5-13 years) with an ADHD diagnosis and an indication to start methylphenidate (MPH) treatment were recruited. Participants were randomized to PCT or care as usual (CAU) in a 1:1 ratio followed by a 7-week randomized controlled trial (T1) and 6-month, naturalistic, open-label follow-up (T2). Parents, teachers, and physicians rated ADHD symptoms, ADHD medication use, MPH dosing, and treatment satisfaction using questionnaires.</p><p><strong>Results: </strong>A total of 100 children were enrolled and randomized to PCT (n = 49) or CAU (n = 51). In the PCT group, we found 8.2% placebo responders, 16.3% non-responders, and 65.3% responders to MPH. With PCT compared with CAU, a significantly larger number of children discontinued MPH (T1: 24.5 vs 5.9%, p = 0.009; T2: 41.7 vs 10.4%, p < 0.001) and refrained from using other pharmacological treatment (T1: 20.4 vs 3.9%, p = 0.013; T2: 20.83 vs 6.25%, p = 0.002). At both timepoints, there were no significant differences between the groups in the average dose of MPH, ADHD symptoms, or treatment satisfaction.</p><p><strong>Conclusions: </strong>PCT can be used to improve detection of children who do not benefit from MPH, and may therefore potentially reduce overtreatment of ADHD with MPH.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"319-330"},"PeriodicalIF":3.7,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139570508","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}
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