Pediatric DrugsPub Date : 2024-12-14DOI: 10.1007/s40272-024-00674-2
Lukas Higi, Elisabeth Carydias, Andrea M Burden, Priska Vonbach
{"title":"How Consistent are Anti-Infective Drug Dosing Recommendations Across Three European Paediatric Formularies?","authors":"Lukas Higi, Elisabeth Carydias, Andrea M Burden, Priska Vonbach","doi":"10.1007/s40272-024-00674-2","DOIUrl":"https://doi.org/10.1007/s40272-024-00674-2","url":null,"abstract":"<p><strong>Background: </strong>Drug dosing recommendations in paediatrics are mainly based on the age and bodyweight of the child. Because of the limited amount of label information, several paediatric drug formularies have been developed. This study compares anti-infective drug dosing recommendations across three European formularies.</p><p><strong>Methods: </strong>Recommendations from three paediatric formularies (German Pediatric Formulary [GPF], SwissPedDose [SPD] and the British National Formulary for Children [BNF]) were collected. Using population growth curves, we simulated one child for each month from 1 month up to 18 years of age. The recommendations from each formulary were used to calculate doses for each simulated child. Equivalence and difference in calculated doses were analysed.</p><p><strong>Results: </strong>In total, dosing recommendations for 34 anti-infective substances were collected with 74 corresponding indications, which resulted in 47,154 calculated doses. The mean (± standard deviation) proportion of equivalent doses (difference ≤10%) across all three formularies was 40% (±16), while for pairwise comparisons it was 53% (±19) for GPF versus BNF, 67% (±14) for GPF versus SPD and 52% (±19) for SPD versus BNF. The median [25th quantile, 75th quantile] differences in daily doses across all three formularies were 0%, [0, 26] while for pairwise comparisons it was 4% [0, 32] for GPF versus BNF, 0% [0, 17] for GPF versus SPD and 7% [0, 33] for SPD versus BNF.</p><p><strong>Conclusions: </strong>The majority of recommended anti-infective drug doses were consistent, with the highest equivalence found between GPF and SPD. Maintaining formularies is resource intensive; therefore, a common standard in Europe could prove beneficial when moving towards digitalisation of the healthcare systems.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824419","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 : 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":"https://doi.org/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":""},"PeriodicalIF":3.4,"publicationDate":"2024-12-11","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 : 2024-11-27DOI: 10.1007/s40272-024-00664-4
Totsapol Jirasomprasert, Li-Yuan Tian, Dian-Ping You, Ya-Kun Wang, Lei Dong, Ya-Hui Zhang, Guo-Xiang Hao, John van den Anker, Yue-E Wu, Bo-Hao Tang, Wei Zhao, Yi Zheng
{"title":"Population Pharmacokinetics and Dose Optimization of Piperacillin in Infants and Children with Pneumonia.","authors":"Totsapol Jirasomprasert, Li-Yuan Tian, Dian-Ping You, Ya-Kun Wang, Lei Dong, Ya-Hui Zhang, Guo-Xiang Hao, John van den Anker, Yue-E Wu, Bo-Hao Tang, Wei Zhao, Yi Zheng","doi":"10.1007/s40272-024-00664-4","DOIUrl":"https://doi.org/10.1007/s40272-024-00664-4","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to determine the piperacillin disposition and optimize the dosing regimens for infants and children with pneumonia.</p><p><strong>Methods: </strong>An opportunistic sampling strategy was used in this pharmacokinetic study. High-performance liquid chromatography was used to measure the concentrations of piperacillin in plasma samples. A population pharmacokinetic model was conducted using NONMEM.</p><p><strong>Results: </strong>The pharmacokinetic data of 90 samples from 64 infants and children with pneumonia (age range: 0.09-1.72 years for infants and 2.12-11.10 years for children) were available. A two-compartment model with first-order elimination was the most suitable model to describe the population pharmacokinetics of piperacillin. A covariate analysis indicated that body weight and age were significant factors affecting clearance. Monte Carlo simulations showed that a 50-mg/kg every 8 h or every 12 h dosing regimen results in underdosing. Results both in infants and children showed that an extended infusion (3 h) of various dosing regimens (80, 100, or 130 mg/kg) three times daily or a 300-mg/kg continuous infusion can reach a therapeutic level based on the chosen target for the probability of target attainment threshold of 70%, 80%, and 90% at minimum inhibitory concentration breakpoints of 8 mg/L and 16 mg/L.</p><p><strong>Conclusions: </strong>A population pharmacokinetic model was obtained to evaluate the disposition of piperacillin, and the optimal dosing regimens were provided for use in infants and children with pneumonia.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142731804","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 : 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":"https://doi.org/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":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pediatric DrugsPub Date : 2024-11-25DOI: 10.1007/s40272-024-00660-8
Jose Maria Quintillá, Carmen de la Gala, Ruben Berrueco, Joana Claverol, Bibiana Figueres, Anna Bergós, Lourdes Rodríguez, Anna Mora, Victoria DiBiaso, Cristina Llanos, Begonya Nafria
{"title":"High-Fidelity Clinical Simulation to Improve a Pediatric Clinical Trial Design: Lessons Learned and Conceptualization of the Return on Investment (ROI) and Return on Engagement (ROE) Analysis.","authors":"Jose Maria Quintillá, Carmen de la Gala, Ruben Berrueco, Joana Claverol, Bibiana Figueres, Anna Bergós, Lourdes Rodríguez, Anna Mora, Victoria DiBiaso, Cristina Llanos, Begonya Nafria","doi":"10.1007/s40272-024-00660-8","DOIUrl":"https://doi.org/10.1007/s40272-024-00660-8","url":null,"abstract":"<p><strong>Introduction: </strong>Clinical trials entail complex processes that are more challenging when they are addressed to pediatric patients and include a decentralized design. High-realistic simulation allows for the testing and refining of procedures, organizational systems, and interactions between professionals and patients/families, narrowing the gap between work-as-imagined and work-as-done.</p><p><strong>Objectives: </strong>The aim of this research was to analyze the impact of simulation in three key moments of a clinical trial: the baseline visit, home administration of the investigational drug by parents, and the drawing and processing of pharmacokinetics samples by a home nurse.</p><p><strong>Methods: </strong>Multidisciplinary meetings were held between the team of Barcelona Children's Hospital and the sponsor to define the simulation objectives. Three simulation scenarios were executed in 2 days. Subsequently, a post-process analysis was performed, including the assessment of the patient/family experience.</p><p><strong>Results: </strong>In the baseline visit scenario, minimum duration was calculated, and main aspects related to task dynamics, materials, and training were identified. In the drug administration scenario, the family was able to carry out the infusion within the time established in the protocol, but with some difficulties. In the home nursing scenario, some recommendations for improving safety and patient experience were suggested.</p><p><strong>Conclusions: </strong>High-realistic clinical simulation can contribute to anticipate risks, refine the design of activities, and to identify specific needs prior to the protocol finalization, a timepoint in the process when modifications can still be made that constitutes a significant return on investment and return on the engagement, in the experience of the patients that will participate in the study.</p><p><strong>Clinicaltrial: </strong></p><p><strong>Gov identifier number: </strong>NCT04644575.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142710857","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 : 2024-11-22DOI: 10.1007/s40272-024-00668-0
Hannah A Blair
{"title":"Apremilast: First Pediatric Approval.","authors":"Hannah A Blair","doi":"10.1007/s40272-024-00668-0","DOIUrl":"https://doi.org/10.1007/s40272-024-00668-0","url":null,"abstract":"<p><p>Apremilast (Otezla<sup>®</sup>) is an inhibitor of phosphodiesterase-4 (PDE-4) being developed by Amgen. It is approved in multiple countries worldwide, including the USA and those of the EU, for the treatment of adults with psoriatic arthritis, plaque psoriasis, or oral ulcers associated with Behçet's disease. In April 2024, based on clinical data in patients aged 6-17 years, apremilast received its first pediatric approval in the USA for the treatment of pediatric patients aged ≥ 6 years and weighing ≥ 20 kg with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy. Apremilast was also approved in the EU in October 2024 for the treatment of moderate to severe plaque psoriasis in children and adolescents from the age of 6 years and weighing ≥ 20 kg who are candidates for systemic therapy. This article summarizes the milestones in the development of apremilast leading to the first pediatric approval for plaque psoriasis.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142688044","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":"Dexmedetomidine for Less Invasive Surfactant Administration: A Pilot Study.","authors":"Sagee Nissimov, Amitai Kohn, Rimona Keidar, Ayelet Livne, Mazal Shemer, Ayala Gover, Calanit Hershkovich-Shporen, Matitiahu Berkovitch, Iris Morag","doi":"10.1007/s40272-024-00667-1","DOIUrl":"https://doi.org/10.1007/s40272-024-00667-1","url":null,"abstract":"<p><strong>Introduction: </strong>Less invasive surfactant administration (LISA) involves delivering surfactant to a spontaneously breathing infant by passing a thin catheter through the vocal cords and has become the preferred method for surfactant delivery. However, the role of pre-LISA sedation remains unclear.</p><p><strong>Objective: </strong>The aim of this study was to describe the use of dexmedetomidine for LISA in preterm and early-term infants.</p><p><strong>Methods: </strong>This retrospective study evaluated preterm and early-term infants who received intravenous dexmedetomidine for LISA between December 2022 and March 2024. Primary outcomes included safety parameters such as the absence of bradycardia, hypotension, hypothermia, or respiratory depression, and the success rate of LISA, determined by the lack of endotracheal intubation within 72 h. Intergroup comparison based on a cutoff of 32 weeks post-menstrual age (PMA) was performed.</p><p><strong>Results: </strong>Thirty-seven infants were included. The mean ± SD PMA at birth, birth weight, and age at LISA were 32.2 ± 2.7 weeks, 1879 ± 698 g, and 13.9 ± 12.4 h, respectively. Mean dexmedetomidine dosage was 0.66 ± 0.26 μg/kg. Six patients (16.2%) developed mild hypothermia, and 10 (27%) experienced apnea/bradycardia within 24 h. The success rate of the procedure was 89.2%. Infants born before 32 weeks received lower doses of dexmedetomidine than those born at 32 weeks and above (0.54 ± 0.24 versus 0.76 ± 0.24 μg/kg, p < 0.01). Safety and success rates of LISA were similar across groups.</p><p><strong>Conclusion: </strong>This is the first report on dexmedetomidine as pre-LISA sedation, demonstrating its feasibility with comparable success rates regardless of PMA. These findings may inform future studies on sedation strategies for LISA.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142693105","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 : 2024-11-08DOI: 10.1007/s40272-024-00662-6
Jordan Anderson, Erin B Owen, Shannon Mayes, Janice E Sullivan, Mark J McDonald
{"title":"Pharmacokinetics and Safety of Endotracheal Lidocaine for Endotracheal Intubation in Critically Ill Children.","authors":"Jordan Anderson, Erin B Owen, Shannon Mayes, Janice E Sullivan, Mark J McDonald","doi":"10.1007/s40272-024-00662-6","DOIUrl":"https://doi.org/10.1007/s40272-024-00662-6","url":null,"abstract":"<p><strong>Introduction: </strong>Critically ill pediatric patients are endotracheally intubated in pediatric intensive care units for a variety of illnesses and indications. Management of an endotracheal tube (ETT) requires suctioning to remove patient secretions and prevent occlusion, but this practice can be associated with adverse events, such as hemodynamic deterioration or increases in intracranial pressure. Instillation of lidocaine into the ETT before suctioning may be beneficial in preventing these events. The purpose of this phase I, fixed-dose, open-label pharmacokinetic and safety study, conducted in endotracheally intubated and mechanically ventilated infants and children requiring ETT suctioning, was to determine the serum concentrations and safety of lidocaine after ETT administration.</p><p><strong>Methods: </strong>A total of 21 infants and children meeting inclusion criteria received a single dose of ETT lidocaine per institutional protocol. Blood samples were obtained pre-dose and at 5, 10, 30, and 120 minutes after administration for serum concentration analysis. Lidocaine dosing was driven by weight and age, with infants and children aged > 38 weeks' estimated gestational age and < 3 years receiving 0.5 mg/kg/dose (maximum: 20 mg/dose) and children aged 3-18 years receiving 1 mg/kg/dose (maximum: 100 mg/dose).</p><p><strong>Results: </strong>No subjects aged < 3 years had detectable serum lidocaine concentrations. Four subjects in the group aged ≥ 3 years had detectable serum lidocaine concentrations at 5, 10, and/or 30 minutes, but no serum concentrations were within the toxic range. No adverse effects, such as cough, bronchospasm, seizure activity, or clinically significant depression of cardiac or respiratory function, were noted in any subjects.</p><p><strong>Conclusions: </strong>Pharmacokinetic sampling showed that only 4 of 21 subjects had detectable serum lidocaine concentrations and none were within the toxic range. The safety of ETT administration of lidocaine in endotracheally intubated infants and children requiring ETT suctioning was demonstrated at the doses used in this study. The study was conducted at Norton Children's Hospital. The research team confirmed that this study was not registered with clinicaltrials.gov.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142624640","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":"Therapeutic Drug Monitoring of Antimicrobial Drugs in Children with Cancer: A New Tool for Personalized Medicine.","authors":"Riccardo Masetti, Gianluca Bossù, Edoardo Muratore, Davide Leardini, Milo Gatti, Riccardo Di Sario, Federico Pea, Susanna Esposito","doi":"10.1007/s40272-024-00663-5","DOIUrl":"https://doi.org/10.1007/s40272-024-00663-5","url":null,"abstract":"<p><p>The risk of fungal, bacterial, and viral infections is higher in children with hematological and solid malignancies, particularly during periods of profound neutropenia. Although early administration of antimicrobial agents is common, optimizing pharmacological therapy in pediatric patients with cancer is challenging because of their variable pharmacokinetics compared with adults, including differences in body mass and augmented renal clearance, as well as chemotherapy-induced organ toxicity. Therapeutic drug monitoring, which involves measuring drug concentrations in serum or plasma at specific timepoints and adjusting doses accordingly, can be applied to various medications. While standardized targets for all antimicrobial agents in children are lacking, therapeutic drug monitoring appears to be beneficial in preventing serious toxicity and addressing treatment failure or non-compliance. This narrative review aims to analyze current perspectives on therapeutic drug monitoring for antimicrobial drugs in the special population of children with hematological or oncological diseases, including those undergoing hematopoietic cell transplantation. The review provides evidence on the clinical benefits of this method and explores potential future developments in this area.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":""},"PeriodicalIF":3.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583994","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":" Pediatric Chronic Inflammatory Demyelinating Polyneuropathy: Challenges in Diagnosis and Therapeutic Strategies.","authors":"Issa Alawneh, Asmaa Alenizi, Freddy Paiz, Elisa Nigro, Jiri Vajsar, Hernan Gonorazky","doi":"10.1007/s40272-024-00646-6","DOIUrl":"10.1007/s40272-024-00646-6","url":null,"abstract":"<p><p>Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare autoimmune neurological disorder seen in both pediatric and adult populations. CIDP typically presents with progressive and persistent weakness over at least 4 weeks in addition to sensory symptoms in the extremities. Although CIDP shares common clinical features between children and adults, it sometimes presents as a distinct clinical entity in children that requires close attention and recognition. A major caveat when diagnosing a child with CIDP is the clinical and diagnostic overlap with inherited neuropathies, most commonly Charcot-Marie-Tooth disease (CMT). Demyelinating CMT (dCMT) and CIDP might share similar clinical presentations, and sometimes it might be difficult to differentiate them on the basis of the electrodiagnostic findings or cerebrospinal fluid (CSF) albumino-cytological dissociation. This indeed merits early consideration for genetic testing in patients who do not respond to conventional CIDP therapies. Current treatment options for CIDP include intravenous immunoglobulins (IVIG), corticosteroids (CS), and plasmapheresis (PLEX). The need for novel therapies is essential in instances where patients continue to have symptoms despite the standard therapies or due to adverse effects of long-term use of standard therapies such as CS. This paper reviews the challenges in the diagnosis of CIDP in children and the current as well as novel therapies for CIDP.</p>","PeriodicalId":19955,"journal":{"name":"Pediatric Drugs","volume":" ","pages":"709-717"},"PeriodicalIF":3.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142081145","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}