Pediatric DrugsPub Date : 2025-05-01Epub Date: 2025-01-18DOI: 10.1007/s40272-024-00665-3
Shan Chong, Lan Sun, Guangyan Mu, Manqi Hua, Qian Xiang, Yimin Cui
{"title":"Adverse Events of Factor Xa Inhibitors in Pediatric Patients: A Meta-analysis and Pharmacovigilance Study.","authors":"Shan Chong, Lan Sun, Guangyan Mu, Manqi Hua, Qian Xiang, Yimin Cui","doi":"10.1007/s40272-024-00665-3","DOIUrl":"10.1007/s40272-024-00665-3","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to provide a comprehensive review of adverse events (AEs) associated with factor Xa (FXa) inhibitors in pediatric patients.</p><p><strong>Methods: </strong>We searched PubMed, Embase, Cochrane Library, ClinicalTrials.gov, and the European Union Clinical Trials Register for English-language records from the establishment of the database up to October 17, 2023. Both randomized controlled trials and single-arm trials were included. AEs were analyzed using a Bayesian hierarchical model. For the pharmacovigilance study, data from the US Food and Drug Administration Adverse Event Reporting System from January 1, 2007, to December 31, 2023, were obtained. The proportional imbalance method and the Medicines and Healthcare products Regulatory Agency method were used to detect AE signals. Further characterization of patients presenting with AEs was performed.</p><p><strong>Results: </strong>Of 451 records identified, 12 eligible studies were included. A total of 50.6% (95% Bayesian credible interval [CrI] 33.1-67.2, τ = 0.796) of patients experienced at least one AE, and 9.9% (95% CrI 3.9-19.5, τ = 0.552) developed at least one serious AE. Major and clinically relevant non-major bleeding occurred in 2.4% (95% CrI 0.8-4.8, τ = 1.61) of patients. The most common bleeding AEs were epistaxis (8.4% [95% CrI 3.9-14.9, τ = 1.96]), subcutaneous hematoma (6.4% [95% CrI 0.5-26.2, τ = 0.54]), and wound hemorrhage (3.7% [95% CrI 0.4-13.3, τ = 0.55]). Non-hemorrhagic AEs were pyrexia (9.2% [95% CrI 4.6-15.3, τ = 1.18]), vomiting (7.8% [95% CrI 4.0-12.3, τ = 0.08]), and abdominal pain (7.4% [95% CrI 1.5-19.4, τ = 0.84]). A total of 39 AE signals were detected in the pharmacovigilance study. The top three highest overall relative odds ratio (ROR) for AEs were observed for haemorrhoidal hemorrhage at 1211.82 (95% CI, 312.69-4696.29), thrombophlebitis at 134.64 (95% CI, 42.18-429.81), and deep vein thrombosis at 68.3 (95% CI, 42.53-109.68). Patients experiencing bleeding AEs had received a mean dosage of rivaroxaban 0.16 mg/kg and apixaban 0.08 mg/kg.</p><p><strong>Conclusions: </strong>Systematically quantified AEs of FXa inhibitors in clinical trials and real-world studies provide an important guide for clinicians. The use of FXa inhibitors in pediatric patients is associated with an acceptable rate of AEs. The most common bleeding AE was epistaxis. Pediatric patients treated with FXa inhibitors were more prone to hemorrhoidal hemorrhage. A safe approach may involve prior use of other anticoagulants followed by careful administration of FXa inhibitors, with a dosing regimen tailored to age and weight. Close monitoring is recommended for peri-procedural anticoagulation and vomiting.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"351-366"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12031795/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009880","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}
Pediatric DrugsPub Date : 2025-05-01Epub Date: 2025-01-28DOI: 10.1007/s40272-025-00682-w
Huan Yang, Ping Li, Hong Shu, Hua Qian, Jing-Ping Chen, Shan Wang, Li-Min Miao, Xiao-Li Li, Yuan Meng, Rong Cao, Xiao-Yan Luo, Lin Ma, Hua Wang
{"title":"Efficacy and Safety of Proactive Therapy with 2% Crisaborole Ointment in Children with Mild-to-Moderate Atopic Dermatitis: A Randomized Controlled Study.","authors":"Huan Yang, Ping Li, Hong Shu, Hua Qian, Jing-Ping Chen, Shan Wang, Li-Min Miao, Xiao-Li Li, Yuan Meng, Rong Cao, Xiao-Yan Luo, Lin Ma, Hua Wang","doi":"10.1007/s40272-025-00682-w","DOIUrl":"10.1007/s40272-025-00682-w","url":null,"abstract":"<p><strong>Background: </strong>The effectiveness of crisaborole for proactive treatment of atopic dermatitis (AD) is not well established.</p><p><strong>Objectives: </strong>This study aims to investigate the efficacy and safety of a proactive treatment strategy with 2% crisaborole ointment for managing mild-to-moderate AD in children.</p><p><strong>Patients and methods: </strong>In this 16-week randomized-controlled trial, children aged 2-17 years with mild-to-moderate AD were enrolled. All participants received treatment with 0.1% mometasone furoate cream for 2 weeks. Those with an IGA score of ≤ 1 were randomly assigned in a 1:1 ratio to either the proactive treatment group, which received crisaborole combined with emollient twice daily, or the reactive treatment group, which received emollients alone, and in the event of disease relapse, both groups received 0.1% mometasone furoate cream as rescue treatment.</p><p><strong>Results: </strong>Of the 153 patients screened, 142 were randomized; 73 to the proactive treatment group and 69 to the reactive treatment group. At the end of the 16-week trial, the proactive treatment group had a relapse rate of 43.84%, significantly lower than the 71.01% relapse rate in the reactive group (P = 0.001). Additionally, the proactive treatment group demonstrated a significant reduction in the need for mometasone furoate prescriptions at weeks 4, 8, 12, and 16 (P < 0.05). Improvements were also observed in IGA, EASI, PP-NRS, and POEM scores at 12 weeks (P < 0.05). No significant differences in adverse events were found between the groups (χ<sup>2</sup> = 2.237, P = 0.135).</p><p><strong>Conclusions: </strong>Proactive treatment with crisaborole ointment for children older than 2 years with mild-to-moderate AD effectively reduces flare-ups and reliance on topical corticosteroids, demonstrating good tolerability and safety.</p><p><strong>Chinese clinical trial registry: </strong>ChiCTR2100054340 (Date of Trial Registration: 14 December 2021).</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"367-376"},"PeriodicalIF":3.4,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143053161","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2025-01-13DOI: 10.1007/s40272-024-00671-5
Malene Galle Madsen, Jin Liang Zhu, Trine Munk-Olsen, Theresa Wimberley, Henrik Larsson, Anna-Sophie Rommel, Xiaoqin Liu, Mette-Marie Zacher Kjeldsen, Sarah Kittel-Schneider, Veerle Bergink, Kathrine Bang Madsen
{"title":"Prevalence and Temporal Trends of Attention Deficit Hyperactivity Disorder Medication Fills During Pregnancy and Breastfeeding in Denmark.","authors":"Malene Galle Madsen, Jin Liang Zhu, Trine Munk-Olsen, Theresa Wimberley, Henrik Larsson, Anna-Sophie Rommel, Xiaoqin Liu, Mette-Marie Zacher Kjeldsen, Sarah Kittel-Schneider, Veerle Bergink, Kathrine Bang Madsen","doi":"10.1007/s40272-024-00671-5","DOIUrl":"10.1007/s40272-024-00671-5","url":null,"abstract":"<p><strong>Background and objectives: </strong>Females of reproductive age are increasingly using attention deficit hyperactivity disorder (ADHD) medication, but its use during pregnancy and breastfeeding is largely unknown. The aim of this study is to examine the prevalence of ADHD medication fills during pregnancy and breastfeeding, including characteristics of these females and cohort differences over time.</p><p><strong>Methods: </strong>We conducted a descriptive study using Danish nationwide registers. Within cohorts of pregnant and breastfeeding females, we calculated the prevalence of ADHD medication (methylphenidate, amphetamine, dexamfetamine, lisdexamfetamine, modafinil, atomoxetine, clonidine and guanfacine) fills and described sociodemographic and clinical characteristics across groups with fills, no fills and previous fills. Cohort differences in ADHD medication fills during pregnancy for 2005-2010, 2011-2016 and 2017-2022 were examined.</p><p><strong>Results: </strong>In this cohort of 1,077,279 pregnancies, ADHD medication fills increased from 0.08 to 7.71 per 1000 individuals between 2005 and 2022. Among 446,485 breastfeeding females, fills increased from 0.55 to 3.67 per 1000 individuals from 2012 to 2022. Compared with the group with no fills, females filling ADHD medication during pregnancy and breastfeeding were younger, had lower levels of education, were more often smoking during pregnancy, utilised more psychiatric healthcare and had concurrent fills of other psychotropic medication. Cohort differences over time revealed that females filling ADHD medication during pregnancy in 2017-2022 were older, had higher levels of education, smoked less during pregnancy, had fewer psychiatric contacts and were less likely to fill other psychotropic medications compared with females in the earlier cohorts.</p><p><strong>Conclusions: </strong>Results showed an increasing prevalence of ADHD medication fills during pregnancy and breastfeeding in Denmark over time, surpassing the increase observed generally in females of reproductive age filling ADHD medication. Results revealed a difference in characteristics of females filling ADHD medication during pregnancy over time, suggesting a shift in pregnancy treatment patterns.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"233-246"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829916/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142978760","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2025-01-08DOI: 10.1007/s40272-024-00678-y
Christian A Maiwald, Karel Allegaert
{"title":"Dexmedetomidine for Less Invasive Surfactant Administration: Another Promising Medicine in an Ever-Growing Toolbox?","authors":"Christian A Maiwald, Karel Allegaert","doi":"10.1007/s40272-024-00678-y","DOIUrl":"10.1007/s40272-024-00678-y","url":null,"abstract":"","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"257-260"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142953196","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2025-01-03DOI: 10.1007/s40272-024-00676-0
Judith A Ten Barge, Gerbrich E van den Bosch, Rebeccah Slater, Nynke J van den Hoogen, Irwin K M Reiss, Sinno H P Simons
{"title":"Visceral Pain in Preterm Infants with Necrotizing Enterocolitis: Underlying Mechanisms and Implications for Treatment.","authors":"Judith A Ten Barge, Gerbrich E van den Bosch, Rebeccah Slater, Nynke J van den Hoogen, Irwin K M Reiss, Sinno H P Simons","doi":"10.1007/s40272-024-00676-0","DOIUrl":"10.1007/s40272-024-00676-0","url":null,"abstract":"<p><p>Necrotizing enterocolitis (NEC) is a relatively rare but very severe gastrointestinal disease primarily affecting very preterm infants. NEC is characterized by excessive inflammation and ischemia in the intestines, and is associated with prolonged, severe visceral pain. Despite its recognition as a highly painful disease, current pain management for NEC is often inadequate, and research on optimal analgesic therapy for these patients is lacking. Insight into the mechanisms underlying intestinal pain in infants with NEC-visceral pain-could help identify the most effective analgesics for these vulnerable patients. Therefore, this comprehensive review aims to provide an overview of visceral nociception, including transduction, transmission, modulation, and experience, and discuss the implications for analgesic therapy in preterm infants with NEC. The transmission of visceral pain differs from that of somatic pain, contributing to the diffuse nature of visceral pain. Studies evaluating the effectiveness of analgesics for treating visceral pain in infants are scarce. However, research in visceral pain models highlights agents that may be particularly effective for treating visceral pain based on their mechanisms of action. Further research is necessary to determine whether agents that have shown promise for treating visceral pain in preclinical studies and adults are effective in infants with NEC as well.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"201-220"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829917/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142922512","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2024-12-20DOI: 10.1007/s40272-024-00673-3
David J Birnkrant, Jane B Black, Daniel W Sheehan, Hollie M Baker, Marielena L DiBartolo, Sherri L Katz
{"title":"A New Perspective on Drugs for Duchenne Muscular Dystrophy: Proposals for Better Respiratory Outcomes and Improved Regulatory Pathways.","authors":"David J Birnkrant, Jane B Black, Daniel W Sheehan, Hollie M Baker, Marielena L DiBartolo, Sherri L Katz","doi":"10.1007/s40272-024-00673-3","DOIUrl":"10.1007/s40272-024-00673-3","url":null,"abstract":"<p><p>New drugs for Duchenne muscular dystrophy (DMD) are emerging rapidly. However, we and others believe these drugs are achieving regulatory approval prematurely. It is the cardiorespiratory complications of DMD that cause the disease's major morbidities and that determine survival. Thus, to be truly effective, a new drug must improve cardiorespiratory function; instead, new drugs are approved for patient use via accelerated regulatory pathways that rely on surrogate outcome measures with unproven clinical benefits (such as tissue levels of non-biologic, truncated dystrophin) and on scales that reflect muscle strength (such as small improvements in timed activities). In DMD, cardiorespiratory complications occur in \"older\" individuals who are in the non-ambulatory stage of the disease. In contrast, accelerated approvals are based on data from young, ambulatory subjects, a group that essentially never experiences cardiorespiratory complications. When drug studies do obtain cardiorespiratory data, their methodologies are suboptimal. We critically review these methodologies in detail, including problems with the use of threshold levels of respiratory function as outcome measures; problems with the use of historical controls, whose results vary widely, and are influenced by uncontrolled variables related to their observational nature; and the limitations of using percent predicted forced vital capacity (FVC %pred), and its single rate of decline across a wide range of age and function, as a preferred respiratory outcome measure. We discuss the advantages of an alternative respiratory outcome, the absolute value of FVC with aging (the \"Rideau plot\"). Unlike FVC %pred, the Rideau plot considers distinct phenotypes rather than aggregating all individuals into a single respiratory trajectory. Key features of the Rideau plot can show the nature and timing of a drug's effect on respiratory function, making it a potentially better outcome measure for assessing the respiratory effects of a drug. With this article, we use our respiratory perspective to critically examine the DMD drug development process and to propose improvements in study methodologies and in the regulatory processes that approve new drugs.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"143-159"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829838/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872754","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2024-12-11DOI: 10.1007/s40272-024-00672-4
A Perri, S Fattore, A Sbordone, G Rotunno, A De Matteis, P Papacci, V D'Andrea, G Vento
{"title":"Intranasal Analgesia in Preterm and Term Neonates.","authors":"A Perri, S Fattore, A Sbordone, G Rotunno, A De Matteis, P Papacci, V D'Andrea, G Vento","doi":"10.1007/s40272-024-00672-4","DOIUrl":"10.1007/s40272-024-00672-4","url":null,"abstract":"<p><p>The prevention, recognition, and treatment of pain is crucial in the management of neonates. Infants do not tolerate pain better than adults; indeed, the immaturity of the endogenous antalgic system means they exhibit an increased stress response. Pain has been associated with worse cognitive and motor scores, reduced growth trend, reduced brain maturation, and altered corticospinal tract structure. The use of the intranasal route for drug delivery is currently expanding because it has many advantages. In certain contexts, it is preferable over the oral route because of the faster entry of drugs into the circulation, the absence of structural changes by the gastrointestinal environment, and the absence of the hepatic first-pass effect. The pharmacokinetics and pharmacodynamics of drugs commonly used for pain management have peculiar characteristics in infants, especially premature infants. In this article, we summarise the evidence regarding pain management in infants using intranasally administered drugs. We then provide a practical guide to the use of intranasal drugs currently being studied in the neonatal population, focusing on appropriate dosages and indications. Intranasal fentanest appears to be an attractive therapeutic alternative for procedural and palliative neonatal pain management when intravenous access is unavailable in preterm infants. Intranasal midazolam is a valid alternative to consider in term or near-term neonates, especially when the aim is to obtain sedation (and not analgesia, i.e. during magnetic resonance imaging), ketamine has favourable cardiovascular effects and should be considered in specific patients and situations. Intranasal dexmedetomidine is well tolerated in premature neonates. Additionally, endonasal dexmedetomidine can be used in combination with other anaesthetic, sedative, hypnotic, and opioid drugs to allow for dose reduction in sedated neonates.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"191-199"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142813457","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2024-11-27DOI: 10.1007/s40272-024-00669-z
Janet Wozniak, Hannah O'Connor, Maria Iorini, Adrian Jacques H Ambrose
{"title":"Pediatric Bipolar Disorder: Challenges in Diagnosis and Treatment.","authors":"Janet Wozniak, Hannah O'Connor, Maria Iorini, Adrian Jacques H Ambrose","doi":"10.1007/s40272-024-00669-z","DOIUrl":"10.1007/s40272-024-00669-z","url":null,"abstract":"<p><p>Despite an opportunity to prevent adult psychopathology associated with bipolar disorder through early diagnosis in children, there is insufficient information and awareness among healthcare providers about the unique features and treatment of mania and its comorbid conditions in children. Converging evidence from disparate sites describe a developmentally distinct presentation of bipolar disorder in youth that is highly morbid, persistent and responds to treatment with the mood stabilizer medications used in the treatment of adult bipolar disorder, such as divalproex sodium and carbamazepine. Some are additionally approved for use in pediatric populations including, for manic or mixed states, risperidone, aripiprazole, and asenapine for those aged 10-17 years and also including lithium and olanzapine for ages 13-17 years. Quetiapine is approved as monotherapy or as adjunct to lithium or divalproex sodium for manic states in those aged 10-17 years. Delayed or missed diagnosis, inappropriate treatment, worsening course, and treatment resistance unfortunately still occur. While an array of mood-stabilizing medications is available for treatment, such as second-generation antipsychotics, lithium, and anticonvulsants, these can be only partially effective and fraught with annoying and serious side effects. This article will review current practice in the diagnosis and treatment of pediatric bipolar disorder and its comorbid conditions, highlighting areas of need for future research.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"125-142"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829910/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142731803","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2024-12-26DOI: 10.1007/s40272-024-00675-1
Rabporn Suntornlohanakul, E Ann Yeh
{"title":"Optimizing Drug Selection in Children with Multiple Sclerosis: What Do We Know and What Remains Unanswered?","authors":"Rabporn Suntornlohanakul, E Ann Yeh","doi":"10.1007/s40272-024-00675-1","DOIUrl":"10.1007/s40272-024-00675-1","url":null,"abstract":"<p><p>Pediatric-onset multiple sclerosis (POMS) refers to multiple sclerosis with onset before 18 years of age. It is characterized by a more inflammatory course, more frequent clinical relapses, and a greater number of magnetic resonance imaging (MRI) lesions compared with adult-onset MS (AOMS), leading to significant impacts on both disability progression and cognitive outcomes in affected individuals. Managing POMS presents distinct challenges due to the unique needs of pediatric patients and the limited number of disease-modifying therapies (DMTs) approved for pediatric use. Notably, only one therapy (fingolimod) is approved by the United States (US) Food and Drug Administration (FDA) and three (fingolimod, teriflunomide, and dimethyl fumarate) by the European Medicines Agency (EMA) for use in youth with MS. However, observational evidence identifies use of almost all agents off-label in this population. This review provides a comprehensive overview of literature supporting the use of DMTs for POMS, including evidence from observational studies. In this paper, we highlight the shift in clinical practice, which has led to increased use of high-efficacy therapies (HETs) at or near disease onset. We review emerging evidence indicating better cognitive and motor outcomes in this population with early initiation of therapy. Finally, in this paper, we provide a suggested treatment algorithm for managing POMS. We underscore the need for personalized approaches in POMS management. We identify special considerations unique to pediatric care, including attention to family dynamics, and strategies to improve medication adherence and a smooth transition to adult care. Further research on DMTs in POMS is essential to optimize outcomes and improve long-term prognosis.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"161-179"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142896379","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}
Pediatric DrugsPub Date : 2025-03-01Epub Date: 2024-11-01DOI: 10.1007/s40272-024-00659-1
Vera Scheckenbach, Frank Fideler
{"title":"Optimizing Pediatric Sedation: Evaluating Remimazolam and Dexmedetomidine for Safety and Efficacy in Clinical Practice.","authors":"Vera Scheckenbach, Frank Fideler","doi":"10.1007/s40272-024-00659-1","DOIUrl":"10.1007/s40272-024-00659-1","url":null,"abstract":"<p><p>Daily, children undergo countless investigations and interventions, which require sedation and immobilization to ensure safety and accuracy. This remains associated with a persistent risk of sedation-induced life-threatening events as children are particularly vulnerable to adverse medical events and complications. Consequently, there is an urgent need to increase the safety of pediatric sedation and anesthesia. An ideal approach involves the use of drugs with fewer intrinsic side effects. In this context, on the basis of their pharmacokinetic properties, remimazolam (RMZ) and dexmedetomidine (DEX) were evaluated for their suitability as ideal sedatives. RMZ and DEX, both of which are currently available in pediatric medicine, have shown great promise in initial publications. To date, only very limited data concerning RMZ in small children are available. RMZ is a novel, ultrashort-acting benzodiazepine that is metabolized by tissue esterase, largely independent of organ function. It has a context-sensitive half-life of approximately 10 min, with minimal accumulation even with prolonged use. Its effects can be completely reversed with flumazenil. DEX, an isomer of medetomidine, is a potent α2-receptor-agonist with multiple indications in anesthesia and intensive care medicine. It has coanalgesic potential, allows for 'arousal sedations' and has a low profile for cardiorespiratory side effects. DEX is metabolized in the liver and is predominantly excreted renally. Both drugs show potential in the prevention and treatment of delirium, with DEX having additional neuroprotective effects. DEX and RMZ possess several properties of an optimal sedative, including clinically insignificant main metabolites and a broad dosage range, indicating their potential to reduce the incidence of sedation-related life-threatening events in children. However, further clinical research is necessary to better evaluate their potential risks.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"181-189"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11829837/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142562425","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}