Characteristics of Reported Pediatric Medication Errors in Northern Ireland and Use in Quality Improvement.
CONCLUSIONS: Incident reports identified neonates, a shortlist of drugs, and specific error types, associated with modifiable behaviors, as priority improvement targets. These findings direct further study and inform intervention development, such as specific training in calculations to prevent dosing errors. Involving experienced practitioners both endorsed the findings and engaged the practice community in their future implementation. The utility of incident reports to direct improvement efforts may offset the limitations in their representativeness. PMID: 32627136 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - July 6, 2020 Category: Pediatrics Authors: Conn RL, Tully MP, Shields MD, Carrington A, Dornan T Tags: Paediatr Drugs Source Type: research

Treat-to-Target in Pediatric Inflammatory Bowel Disease: What Does the Evidence Say?
Abstract The traditional management of inflammatory bowel disease, based on treatment intensification guided by clinical activity alone, has been revised in the last 10 years and a treat-to-target approach has been proposed and is currently under evaluation as a disease-modifying strategy. Treat-to-target focuses on objective and scheduled measures to monitor intestinal damage, with consequent therapeutic adjustments in case of failure to achieve pre-defined targets. Identification of targets has been set out by the Selecting Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) committee in 2015. Mucosa...
Source: Paediatric Drugs - June 23, 2020 Category: Pediatrics Authors: D'Arcangelo G, Aloi M Tags: Paediatr Drugs Source Type: research

Analysis of Reporting Adverse Drug Reactions in Paediatric Patients in a University Hospital in the Netherlands.
CONCLUSIONS: The results of the present study show that a large number of ADRs are not registered in the medical records and are not reported to the national pharmacovigilance system. Furthermore, it is shown that the number of ADRs occurring at our centre is much higher than the number reported to the national pharmacovigilance centre. Only an average of 513 ADRs in paediatric patients are reported per year nationwide, suggesting that there is extensive underreporting. PMID: 32557243 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - June 18, 2020 Category: Pediatrics Authors: Dittrich ATM, Draaisma JMT, van Puijenbroek EP, Loo DMWMT Tags: Paediatr Drugs Source Type: research

Eosinophilic Esophagitis: Update on Diagnosis and Treatment in Pediatric Patients.
Abstract Eosinophilic esophagitis (EoE) is a chronic immune-mediated inflammatory disorder characterized by symptomatic esophageal dysfunction and an eosinophil-predominant inflammation of the esophagus. EoE arises from interaction between genetic and environmental factors. In pediatric patients, clinical manifestations vary depending on age, from a gastroesophageal reflux disease (GERD)-like condition to severe dysphagic symptoms. Upper endoscopy is considered the gold standard for diagnosis and monitoring of EoE; however, significant efforts are underway to identify noninvasive diagnostic tools and biomarkers to...
Source: Paediatric Drugs - June 10, 2020 Category: Pediatrics Authors: Rossetti D, Isoldi S, Oliva S Tags: Paediatr Drugs Source Type: research

Challenges and Opportunities in the Pharmacological Treatment of Acute Venous Thromboembolism in Children.
Abstract Venous thromboembolism (VTE) is an important but historically under-recognized problem in pediatrics, with an incidence concentrated in hospitalized children. A number of specific VTE diseases with discrete triggers have been described, but the most common pediatric trigger is the presence of central venous access devices. VTE diseases, though heterogenous in etiology, are linked by the common therapeutic strategies shared by their management. Historically, the most commonly used drug therapies have been unfractionated heparin, low-molecular-weight heparins, and vitamin K antagonists, based on extrapolati...
Source: Paediatric Drugs - June 10, 2020 Category: Pediatrics Authors: George C, Rahman M, Monagle P Tags: Paediatr Drugs Source Type: research

Target Drug Exposure Attainment in Children: How to Get from Better to Best.
PMID: 32507957 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - June 8, 2020 Category: Pediatrics Authors: Flint RB, Allegaert K Tags: Paediatr Drugs Source Type: research

External Validation of Model-Based Dosing Guidelines for Vancomycin, Gentamicin, and Tobramycin in Critically Ill Neonates and Children: A Pragmatic Two-Center Study.
SN Abstract BACKGROUND: The Dutch Pediatric Formulary (DPF) increasingly bases its guidelines on model-based dosing simulations from pharmacokinetic studies. This resulted in nationwide dose changes for vancomycin, gentamicin, and tobramycin in 2015. OBJECTIVE: We aimed to evaluate target attainment of these altered, model-based doses in critically ill neonates and children. METHODS: This was a retrospective cohort study in neonatal intensive care unit (NICU) and pediatric ICU (PICU) patients receiving vancomycin, gentamicin, or tobramycin between January 2015 and March 2017 in two university hospitals....
Source: Paediatric Drugs - June 8, 2020 Category: Pediatrics Authors: Hartman SJF, Orriëns LB, Zwaag SM, Poel T, de Hoop M, de Wildt SN Tags: Paediatr Drugs Source Type: research

Pediatric Pharmacology of Desmopressin in Children with Enuresis: A Comprehensive Review.
le JV Abstract Desmopressin is a synthetic analogue of the natural antidiuretic hormone arginine vasopressin. Over the years, it has been clinically used to manage nocturnal polyuria in children with enuresis. Various pharmaceutical formulations of desmopressin have been commercialized for this indication-nasal spray, nasal drops, oral tablet and oral lyophilizate. Despite the fact that desmopressin is a frequently prescribed drug in children, its use and posology is based on limited pediatric data. This review provides an overview of the current pediatric pharmacological data related to the different desmopressin...
Source: Paediatric Drugs - June 8, 2020 Category: Pediatrics Authors: Gasthuys E, Dossche L, Michelet R, Nørgaard JP, Devreese M, Croubels S, Vermeulen A, Van Bocxlaer J, Walle JV Tags: Paediatr Drugs Source Type: research

Caffeine for the Treatment of Apnea in the Neonatal Intensive Care Unit: A Systematic Overview of Meta-Analyses.
CONCLUSION: While limited in quantity, there is evidence of non-inferior effectiveness of caffeine against other methylxanthines or doxapram for the management of apnea in neonates. Owing to the limited quality, however, limited evidence exists in support of an optimal administration regimen for caffeine. Further controlled studies are, therefore, needed to confirm the comparative usefulness of caffeine as well as to assess its different potential regimens, including in relation to safety. PMID: 32488731 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - June 3, 2020 Category: Pediatrics Authors: Alhersh E, Abushanab D, Al-Shaibi S, Al-Badriyeh D Tags: Paediatr Drugs Source Type: research

Management of Diabetic Ketoacidosis in Children and Adolescents with Type 1 Diabetes Mellitus.
Abstract Diabetic ketoacidosis (DKA) is the end result of insulin deficiency in type 1 diabetes mellitus (T1D). Loss of insulin production leads to profound catabolism with increased gluconeogenesis, glycogenolysis, lipolysis, and muscle proteolysis causing hyperglycemia and osmotic diuresis. High levels of counter-regulatory hormones lead to enhanced ketogenesis and the release of 'ketone bodies' into the circulation, which dissociate to release hydrogen ions and cause an overwhelming acidosis. Dehydration, hyperglycemia, and ketoacidosis are the hallmarks of this condition. Treatment is effective repletion of in...
Source: Paediatric Drugs - May 25, 2020 Category: Pediatrics Authors: Castellanos L, Tuffaha M, Koren D, Levitsky LL Tags: Paediatr Drugs Source Type: research

Combination of Biological Agents in Moderate to Severe Pediatric Inflammatory Bowel Disease: A Case Series and Review of the Literature.
CONCLUSION: In pediatric IBD, combining biological agents seems to be safe and beneficial in selected patients. The safety should be addressed in long-term follow-up studies. PMID: 32378002 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - May 7, 2020 Category: Pediatrics Authors: Olbjørn C, Rove JB, Jahnsen J Tags: Paediatr Drugs Source Type: research

The Use of TNF α Inhibitors in Treating Pediatric Skin Disorders.
The Use of TNFα Inhibitors in Treating Pediatric Skin Disorders. Paediatr Drugs. 2020 Apr 30;: Authors: Nguyen QD, Starling CT, Hebert AA Abstract Tumor necrosis factor alpha (TNF) inhibitors have had a significant impact in medicine since the approval of the first drug of its class by the US FDA in 1998. New clinical data and indications have emerged for TNF inhibitors in recent years. Currently, four TNF inhibitors have been approved by the US FDA for dermatology, two of which include US FDA-approved pediatric use. In particular, growing evidence supports the use of etanercept and adalimumab a...
Source: Paediatric Drugs - April 30, 2020 Category: Pediatrics Authors: Nguyen QD, Starling CT, Hebert AA Tags: Paediatr Drugs Source Type: research

Why and How Should We Treat Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Syndrome?
Abstract Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome is characterized by recurrent fever flares accompanied by symptoms and signs forming the disease acronym and alternating with asymptomatic periods. Despite the disease having a generally favorable outcome, with spontaneous remission after a few years, it does have a major impact on the quality of life of the child and his or her relatives. Beside symptomatic medications during fever flares, the most used treatment consists of a single dose of corticosteroids at flare onset to interrupt the attack; fever resolves usual...
Source: Paediatric Drugs - April 28, 2020 Category: Pediatrics Authors: Hofer M Tags: Paediatr Drugs Source Type: research

Dealing with COVID-19 in a Pediatric Rheumatology Unit in Italy.
PMID: 32297225 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - April 16, 2020 Category: Pediatrics Authors: Costi S, Caporali R, Cimaz R Tags: Paediatr Drugs Source Type: research

Interleukin-1 Blockade in Systemic Juvenile Idiopathic Arthritis.
This article reviews the efficacy and safety of these drugs for the treatment of sJIA on the basis of published data, and offers the current view on treating sJIA according to its different phenotypes. There are no head to head trials among the different IL-1 inhibitors, and although efficacy has been demonstrated for all of them, it is still unknown which one would be more appropriate for which particular situation. The presence of synovitis in addition to active systemic features might be relevant for these choices. In addition, complications such as macrophage activation syndrome can be important since, on one hand, it ...
Source: Paediatric Drugs - April 15, 2020 Category: Pediatrics Authors: Mejbri M, Theodoropoulou K, Hofer M, Cimaz R Tags: Paediatr Drugs Source Type: research

Pharmacovigilance Reports Received from Children and Young People, and Development of Information to Aid Future Reporting from this Age Group.
This study aimed to determine the contribution of children and young people (CYP) to the Medicines and Healthcare Products Regulatory Agency (MHRA) Yellow Card Scheme (YCS), and to develop age appropriate information with CYP through focus groups to help aid future reporting by this cohort. METHODS: Reports for suspected adverse drug reactions (ADRs) in patients 
Source: Paediatric Drugs - April 7, 2020 Category: Pediatrics Authors: Bhoombla N, Preston J, Ainsworth J, Bird H, Jadeja M, King C, Hawcutt DB Tags: Paediatr Drugs Source Type: research

A Prospective Cohort Study of Factors Associated with Empiric Antibiotic De-escalation in Neonates Suspected with Early Onset Sepsis (EOS).
Abstract BACKGROUND: Prolonged empiric antibiotic use, resulting from diagnostic uncertainties, in suspected early onset sepsis (EOS) cases constitutes a significant problem. Unnecessary antibiotic use increases the risk of antibiotic resistance. Furthermore, prolonged antibiotic use increases the risk of mortality and morbidity in neonates. Proactive measures including empiric antibiotic de-escalation are crucial to overcome these problems. METHODS: This was a prospective cohort study conducted in the neonatal intensive care units of two public hospitals in Malaysia. Neonates with a gestational age greater t...
Source: Paediatric Drugs - March 18, 2020 Category: Pediatrics Authors: Ain Ibrahim N, Makmor Bakry M, Mohd Tahir NA, Mohd Zaini NR, Mohamed Shah N Tags: Paediatr Drugs Source Type: research

The Role of Oral Administration of Immunoglobulin in Managing Diarrheal Illness in Immunocompromised Children.
CONCLUSION: Oral administration of human serum immunoglobulin in immunocompromised children presenting with acute and chronic diarrheal illness appeared helpful in reducing stool output by 50% in the majority of patients. PMID: 32180169 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - March 17, 2020 Category: Pediatrics Authors: Alexander E, Hommeida S, Stephens MC, Manini ML, Absah I Tags: Paediatr Drugs Source Type: research

Does a Dose Calculator as an Add-On to a Web-Based Paediatric Formulary Reduce Calculation Errors in Paediatric Dosing? A Non-Randomized Controlled Study.
CONCLUSIONS: Our study shows that the use of a dose calculator as an add-on to a web-based paediatric formulary can reduce calculation errors. Furthermore, it shows that technologies may introduce new errors through transcription errors and wrongly selecting parameters from drop-down lists. Therefore, dosing calculators should be developed and used with special attention for selection and transcription errors. PMID: 32170636 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - March 14, 2020 Category: Pediatrics Authors: van der Zanden TM, de Hoog M, Windster JD, van Rosmalen J, van der Sijs IH, de Wildt SN Tags: Paediatr Drugs Source Type: research

Correction to: Efficacy of Macrolides on Acute Asthma or Wheezing Exacerbations in Children with Recurrent Wheezing: A Systematic Review and Meta ‑analysis.
Correction to: Efficacy of Macrolides on Acute Asthma or Wheezing Exacerbations in Children with Recurrent Wheezing: A Systematic Review and Meta‑analysis. Paediatr Drugs. 2020 Mar 13;: Authors: Pincheira MA, Bacharier LB, Castro-Rodriguez JA Abstract An Online First version of this article was made available online at https://link.springer.com/article/10.1007/s40272-019-00371-5 on 14 January 2020. An error was subsequently identified in the article, and the following correction should be noted. PMID: 32166730 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - March 13, 2020 Category: Pediatrics Authors: Pincheira MA, Bacharier LB, Castro-Rodriguez JA Tags: Paediatr Drugs Source Type: research

Dupilumab to Treat Type 2 Inflammatory Diseases in Children and Adolescents.
Abstract During the past decade, significant therapeutic progress has been made in the field of allergic diseases, mainly concerning the pathogenic role of type 2 inflammation. Biologics targeting specific key cytokines, such as interleukin (IL)-4, IL-5, and IL-13, as well as IgE, have emerged as promising innovative therapies for allergic disorders. In this context, dupilumab has emerged as one of the most successful therapies targeting the IL-4R axis. Dupilumab is a human IgG4 antibody anti-IL-4 receptor (IL-4R) α-subunit that blocks IL-4R signaling induced by both IL-4 and IL-13, downregulating the molecu...
Source: Paediatric Drugs - March 11, 2020 Category: Pediatrics Authors: Licari A, Castagnoli R, Marseglia A, Olivero F, Votto M, Ciprandi G, Marseglia GL Tags: Paediatr Drugs Source Type: research

Therapeutic Potential of Citrulline as an Arginine Supplement: A Clinical Pharmacology Review.
This article summarizes the preclinical and clinical studies of arginine/citrulline in both adults and children, including currently available pharmacokinetic information. The pharmacology of arginine/citrulline is confounded by several patient-specific factors such as variations in baseline arginine/citrulline due to developmental ages and disease states. Currently available pharmacokinetic studies are insufficient to inform the optimal design of clinical studies, especially in children. Successful bench-to-bedside clinical translation of arginine supplementation awaits information from well-designed pharmacokinetic/pharm...
Source: Paediatric Drugs - March 6, 2020 Category: Pediatrics Authors: Rashid J, Kumar SS, Job KM, Liu X, Fike CD, Sherwin CMT Tags: Paediatr Drugs Source Type: research

Vaccine Hesitancy: A Growing Concern.
Abstract Vaccines are one of the great achievements of medical science. They have eradicated or drastically reduced the incidence of once common diseases. It is estimated that vaccines save between 2 and 6 million lives each year, but 1.5 million more lives could be saved if coverage was increased. Vaccine hesitancy, defined by the World Health Organization as "the reluctance or refusal to vaccinate despite the availability of vaccines", is a barrier to increasing coverage that has received a great deal of attention from the media and public health academics in recent years. It is argued that many parent...
Source: Paediatric Drugs - February 19, 2020 Category: Pediatrics Authors: Kennedy J Tags: Paediatr Drugs Source Type: research

Pharmacokinetics and Clinical Pharmacology of Monoclonal Antibodies in Pediatric Patients.
Abstract Monoclonal antibodies (mAbs) and their derivatives are increasingly used in pediatric pharmacotherapy, and the number of antibody-based drug products with approved pediatric indications is continuously growing. In most instances, pediatric use is being pursued after the efficacy and safety of novel antibody medications have been established in adult indications. The pediatric extrapolation exercise that is frequently used in this context to bridge efficacy and safety from adults to children is oftentimes challenged through uncertainties and knowledge gaps in how to reliably extrapolate pharmacokinetics an...
Source: Paediatric Drugs - February 13, 2020 Category: Pediatrics Authors: Temrikar ZH, Suryawanshi S, Meibohm B Tags: Paediatr Drugs Source Type: research

Pharmacological Management of Pediatric Pustular Psoriasis.
Abstract Therapeutic management of pustular psoriasis remains a challenge despite the rapid advance in psoriasis research and the development of drugs, especially biologics. Treatment guidelines have been established for pustular psoriasis, but no controlled studies are present for juvenile pustular psoriasis (JPP). Search of the literature reveals that current evidence of JPP treatment is limited to case reports and case series. Among the conventional drugs for JPP, oral retinoid is the most commonly used, yet concerns for growth disturbance exist. Cyclosporine and methotrexate have also been administered as firs...
Source: Paediatric Drugs - February 4, 2020 Category: Pediatrics Authors: Huang YW, Tsai TF Tags: Paediatr Drugs Source Type: research

Administration and Dosing of Systemic Antifungal Agents in Pediatric Patients.
Abstract Neonates and immunosuppressed/immunocompromised pediatric patients are at high risk of invasive fungal diseases. Appropriate antifungal selection and optimized dosing are imperative to the successful prevention and treatment of these life-threatening infections. Conventional amphotericin B was the mainstay of antifungal therapy for many decades, but dose-limiting nephrotoxicity and infusion-related adverse events impeded its use. Despite the development of several new antifungal classes and agents in the past 20 years, and their now routine use in at-risk pediatric populations, data to guide the opti...
Source: Paediatric Drugs - January 23, 2020 Category: Pediatrics Authors: Downes KJ, Fisher BT, Zane NR Tags: Paediatr Drugs Source Type: research

The Evolving Diagnostic and Treatment Landscape of NTRK-Fusion-Driven Pediatric Cancers.
Abstract The neurotrophin receptor tyrosine kinase (NTRK1-3) genes have been identified as key fusion partners in a range of pediatric cancers. In childhood cancers, ETV6-NTRK3 fusions are found in the majority of infantile fibrosarcomas and congenital mesoblastic nephromas. NTRK fusions are also found in mammary analog secretory carcinomas (MASC), secretory breast carcinomas, and with modest frequency in high-grade gliomas in very young children. While there are a range of multi-receptor tyrosine kinase inhibitors that show efficacy against TRK kinases, there are now multiple highly selective TRK inhibitors in cl...
Source: Paediatric Drugs - January 22, 2020 Category: Pediatrics Authors: Shulman DS, DuBois SG Tags: Paediatr Drugs Source Type: research

New Developments in the Treatment of X-Linked Hypophosphataemia: Implications for Clinical Management.
r W Abstract X-linked hypophosphataemia (XLH) is due to mutations in phosphate-regulating gene with homologies to endopeptidases on the X chromosome (PHEX) and represents the most common heritable form of rickets. In this condition, the hormone fibroblast growth factor 23 (FGF23) is produced in excessive amounts for still unknown reasons, and causes renal phosphate wasting and suppression of 1,25-dihydroxyvitamin D, leading to low serum phosphate concentrations. Prolonged hypophosphataemia decreases apoptosis of hypertrophic chondrocytes in growth plates (causing rickets) and decreases mineralisation of existing b...
Source: Paediatric Drugs - January 22, 2020 Category: Pediatrics Authors: Saraff V, Nadar R, Högler W Tags: Paediatr Drugs Source Type: research

A Phase IIa Clinical Trial of 2-Iminobiotin for the Treatment of Birth Asphyxia in DR Congo, a Low-Income Country.
Abstract AIM: The main burden of hypoxic-ischemic encephalopathy falls in low-income countries. 2-Iminobiotin, a selective inhibitor of neuronal and inducible nitric oxide synthase, has been shown to be safe and effective in preclinical studies of birth asphyxia. Recently, safety and pharmacokinetics of 2-iminobiotin treatment on top of hypothermia has been described. Since logistics and the standard of medical care are very different in low-resource settings, the aim of this study was to investigate safety and pharmacokinetics of Two-IminoBiotin in the Democratic Republic of Congo (TIBC). METHODS: Near-term ...
Source: Paediatric Drugs - January 21, 2020 Category: Pediatrics Authors: Biselele T, Bambi J, Betukumesu DM, Ndiyo Y, Tabu G, Kapinga J, Bola V, Makaya P, Tjabbes H, Vis P, Peeters-Scholte C Tags: Paediatr Drugs Source Type: research

Drug Treatment of Pulmonary Hypertension in Children.
This article reviews the current drug therapies and their use in the management of PAH in children. PMID: 31960361 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - January 21, 2020 Category: Pediatrics Authors: Avitabile CM, Vorhies EE, Ivy DD Tags: Paediatr Drugs Source Type: research

Drug Treatment of Progressive Myoclonic Epilepsy.
Abstract The progressive myoclonic epilepsies (PMEs) represent a rare but devastating group of syndromes characterized by epileptic myoclonus, typically action-induced seizures, neurological regression, medically refractory epilepsy, and a variety of other signs and symptoms depending on the specific syndrome. Most of the PMEs begin in children who are developing as expected, with the onset of the disorder heralded by myoclonic and other seizure types. The conditions are considerably heterogenous, but medical intractability to epilepsy, particularly myoclonic seizures, is a core feature. With the increasing use of...
Source: Paediatric Drugs - January 14, 2020 Category: Pediatrics Authors: Holmes GL Tags: Paediatr Drugs Source Type: research

Efficacy of Macrolides on Acute Asthma or Wheezing Exacerbations in Children with Recurrent Wheezing: A Systematic Review and Meta-analysis.
CONCLUSIONS: Limited evidence support that a macrolide trial could be considered in children with acute asthma or recurrent wheezing exacerbation. PMID: 31939108 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - January 14, 2020 Category: Pediatrics Authors: Pincheira MA, Bacharier LB, Castro-Rodriguez JA Tags: Paediatr Drugs Source Type: research

A Practical Guide to the Management of Severe Hypertension in Children.
Abstract Severe hypertension in children may result in life-threatening complications. Although there has not been extensive research in this area in children, and recommendations are mostly derived from adult data, in the last few years, there have been more pediatric studies on the safety and effectiveness of antihypertensives. The clinical presentation of a child with severe hypertension varies and may be completely asymptomatic or include signs and symptoms of end-organ damage. Treatment of a child with severe hypertension is emergent and should be done concomitantly with the evaluation. PMID: 31919683 [P...
Source: Paediatric Drugs - January 10, 2020 Category: Pediatrics Authors: Baracco R Tags: Paediatr Drugs Source Type: research

Pediatric Clinical Endpoint and Pharmacodynamic Biomarkers: Limitations and Opportunities.
Abstract Medical research in children typically lags behind that of adult research in both quantity and quality. The conduct of rigorous clinical trials in children can raise ethical concerns because of children's status as a 'vulnerable' population. Moreover, carrying out studies in pediatrics also requires logistical considerations that rarely occur with adult clinical trials. Due to the relatively smaller number of pediatric studies to support evidence-based medicine, the practice of medicine in children is far more reliant upon expert opinion than in adult medicine. Children are at risk of not receiving the sa...
Source: Paediatric Drugs - January 9, 2020 Category: Pediatrics Authors: Dinh JC, Hosey-Cojocari CM, Jones BL Tags: Paediatr Drugs Source Type: research

Update on Drug Management of Refractory Epilepsy in Tuberous Sclerosis Complex.
Abstract Tuberous sclerosis complex (TSC) is a genetic neurocutaneous disorder with epilepsy as a common and early presenting symptom. The neurological phenotype, however, is variable and unpredictable. Early and refractory seizures, infantile spasms in particular, are associated with a poor neurological outcome. Preliminary data suggests early and aggressive seizure control may mitigate the detrimental neurodevelopmental effects of epilepsy. For infantile spasms, vigabatrin is the first line of treatment, and steroids and classic antiepileptic drugs (AEDs) are suitable for second line. Based on retrospective data...
Source: Paediatric Drugs - January 8, 2020 Category: Pediatrics Authors: van der Poest Clement E, Jansen FE, Braun KPJ, Peters JM Tags: Paediatr Drugs Source Type: research

Pharmacologic Management of Chronic Urticaria in Pediatric Patients: The Gap Between Guidelines and Practice.
Abstract Chronic urticaria is an uncommon disorder in children but can present considerable morbidity, as well as frustration for the healthcare provider and parent. The prevalence is 0.1-0.3% but can vary considerably by country. Chronic spontaneous urticaria (no identifiable cause) is responsible for 70-80% of chronic urticaria, about half of this due to a subtype called chronic autoimmune urticaria identified by the presence of autoantibodies to IgE or the IgE receptor. Chronic urticaria that is triggered by external physical stimuli is called chronic inducible urticaria and is present in another 15-20%. Allerg...
Source: Paediatric Drugs - December 20, 2019 Category: Pediatrics Authors: Williams PV Tags: Paediatr Drugs Source Type: research

A Scoping Review of Medications Studied in Pediatric Polypharmacy Research.
CONCLUSION: While characterizing the literature on pediatric polypharmacy in terms of the types of medication studied, we further identified substantive gaps within this literature outside of epilepsy and psychiatric disorders. Medications frequently identified in use of polypharmacy for treatment of epilepsy and psychiatric disorders reveal opportunities for enhanced medication management in pediatric patients. PMID: 31823339 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - December 10, 2019 Category: Pediatrics Authors: Horace AE, Golchin N, Knight EMP, Dawson NV, Ma X, Feinstein JA, Johnson HK, Kleinman L, Bakaki PM Tags: Paediatr Drugs Source Type: research

Molecularly Targeted Agents in the Therapy of Pediatric Brain Tumors.
Abstract Pediatric brain tumors are the leading cause of cancer-related death in children. Recent advances in sequencing techniques, and collaborative efforts to encode the mutational landscape of various tumor subtypes, have resulted in the identification of recurrent mutations that may present as actionable targets in these tumors. A number of molecularly targeted agents are approved or in development for the treatment of various tumor types in adult patients. Similarly, these agents are increasingly being incorporated into pediatric clinical trials, allowing for a targeted approach to treatment. However, due to...
Source: Paediatric Drugs - November 21, 2019 Category: Pediatrics Authors: Bornhorst M, Hwang EI Tags: Paediatr Drugs Source Type: research

CD19 CAR T Cells for the Treatment of Pediatric Pre-B Cell Acute Lymphoblastic Leukemia.
Abstract The development of cluster of differentiation (CD)-19-targeted chimeric antigen receptor (CAR) T cells for the treatment of pre-B-cell acute lymphoblastic leukemia (B-ALL) is an exciting new advancement in the field of pediatric oncology. Tisagenlecleucel and axicabtagene ciloleucel are the first US FDA-approved CD19-targeted CAR T cells. While various different CD19 CAR T cells are in development, tisagenlecleucel is the only CAR T cell approved for pediatric patients. The multicenter phase II trial that led to the approval of tisagenlecleucel demonstrated excellent responses in individuals with highly r...
Source: Paediatric Drugs - November 20, 2019 Category: Pediatrics Authors: Pacenta HL, Laetsch TW, John S Tags: Paediatr Drugs Source Type: research

Macrophage Activation Syndrome and Secondary Hemophagocytic Lymphohistiocytosis in Childhood Inflammatory Disorders: Diagnosis and Management.
Abstract Macrophage activation syndrome (MAS), a form of secondary hemophagocytic lymphohistiocytosis, is a frequently fatal complication of a variety of pediatric inflammatory disorders. MAS has been most commonly associated with systemic juvenile idiopathic arthritis (sJIA), as approximately 10% of children with sJIA develop fulminant MAS, with another 30-40% exhibiting a more subclinical form of the disease. Children with other rheumatologic conditions such as systemic lupus erythematosus and Kawasaki disease are also at risk for MAS. Moreover, MAS also complicates various genetic autoinflammatory disorders suc...
Source: Paediatric Drugs - November 16, 2019 Category: Pediatrics Authors: Henderson LA, Cron RQ Tags: Paediatr Drugs Source Type: research

Treatment Withdrawal Following Remission in Juvenile Idiopathic Arthritis: A Systematic Review of the Literature.
CONCLUSIONS: The published literature on treatment withdrawal in JIA has varied in design and quality, yielding little conclusive evidence thus far on the management of JIA in remission. Given the importance of this question, international collaborative efforts are underway to study clinical and biologic predictors of successful medication withdrawal in JIA. These efforts may ultimately support the development of personalized approaches to withdrawing medication in children with JIA in remission. PMID: 31673960 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - November 1, 2019 Category: Pediatrics Authors: Halyabar O, Mehta J, Ringold S, Rumsey DG, Horton DB Tags: Paediatr Drugs Source Type: research

Update on the Systemic Treatment of Pediatric Localized Scleroderma.
Abstract Juvenile localized scleroderma (jLS) is an orphan disease that can lead to cosmetic disfiguration and orthopedic problems. Two recent publications review the current recommendations regarding diagnosis, assessment, follow up and treatment of pediatric localized scleroderma cases, both of which suggest the Localized Scleroderma Cutaneous Assessment Tool as an important instrument to assess activity and damage. This review focuses on the systemic treatment of jLS. Systemic treatment includes synthetic and biologic disease-modifying antirheumatic drugs. Systemic therapy is indicated if the lesion crosses any...
Source: Paediatric Drugs - October 31, 2019 Category: Pediatrics Authors: Foeldvari I Tags: Paediatr Drugs Source Type: research

Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1.
Abstract Tyrosinemia type 1 (TT1) is a rare metabolic disease caused by a defect in tyrosine catabolism. TT1 is clinically characterized by acute liver failure, development of hepatocellular carcinoma, renal and neurological problems, and consequently an extremely poor outcome. This review showed that the introduction of 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione (NTBC) in 1992 has revolutionized the outcome of TT1 patients, especially when started pre-clinically. If started early, NTBC can prevent liver failure, renal problems, and neurological attacks and decrease the risk for hepatocellular carci...
Source: Paediatric Drugs - October 31, 2019 Category: Pediatrics Authors: van Ginkel WG, Rodenburg IL, Harding CO, Hollak CEM, Heiner-Fokkema MR, van Spronsen FJ Tags: Paediatr Drugs Source Type: research

Pharmacological Prevention and Management of Postoperative Relapse in Pediatric Crohn's Disease.
Abstract Pediatric Crohn's disease (CD) is characterized by an aggressive course that commonly requires more intensive pharmacological and surgical treatments. In spite of the therapeutic advances in monitoring and management, including the widespread use of biologic therapy, the cumulative incidence of surgery in children with CD is still high. However, surgery is usually not curative and disease recurrence after small bowel resection is common. Gastrointestinal endoscopy is currently the gold standard to evaluate disease progression after surgery, but other non-invasive methods have been suggested. Although the ...
Source: Paediatric Drugs - October 18, 2019 Category: Pediatrics Authors: Yerushalmy-Feler A, Assa A Tags: Paediatr Drugs Source Type: research

A Review of Regional Anesthesia in Infants.
Abstract Regional anesthesia provides effective anesthesia and pain relief in infants with age-specific data attesting to safety and efficacy. Regional anesthesia decreases exposure to opioids and general anesthetic agents and associated adverse drug effects, suppresses the stress response, and provides better hemodynamic stability compared to general anesthesia. Regional anesthesia can prevent long-term behavioral responses to pain. As a result, the overall number and variety of nerve blocks being used in infants is increasing. While neuraxial blocks are the most common blocks performed in infants, the introducti...
Source: Paediatric Drugs - October 18, 2019 Category: Pediatrics Authors: Boretsky KR Tags: Paediatr Drugs Source Type: research

Correction to: DTaP ‑IPV‑HepB‑Hib Vaccine (Hexyon®): An Updated Review of its Use in Primary and Booster Vaccination.
Correction to: DTaP‑IPV‑HepB‑Hib Vaccine (Hexyon®): An Updated Review of its Use in Primary and Booster Vaccination. Paediatr Drugs. 2019 Oct 15;: Authors: Syed YY Abstract The article DTaP-IPV-HepB-Hib Vaccine (Hexyon. PMID: 31617083 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - October 15, 2019 Category: Pediatrics Authors: Syed YY Tags: Paediatr Drugs Source Type: research

Applying Pharmacodynamics and Antimicrobial Stewardship to Pediatric Preseptal and Orbital Cellulitis.
Abstract Orbital and preseptal cellulitis are most commonly caused by organisms that originate in the upper respiratory tract or from the skin. There is significant variation in antibiotics used, but ampicillin-sulbactam, ceftriaxone, metronidazole, clindamycin, amoxicillin, amoxicillin-clavulanate, cefuroxime, and vancomycin are often used in the treatment of these infections. The choice of antibiotic, however, is only one consideration. It is also important that antibiotics are dosed to optimize their pharmacodynamic target attainment. Like other serious infections, therapy can be transitioned from initial intra...
Source: Paediatric Drugs - October 14, 2019 Category: Pediatrics Authors: Stimes GT, Girotto JE Tags: Paediatr Drugs Source Type: research

Comment on: "Botulinum Toxin in the Management of Children with Cerebral Palsy".
Comment on: "Botulinum Toxin in the Management of Children with Cerebral Palsy". Paediatr Drugs. 2019 Oct 04;: Authors: Langdon K, Copeland L, Edwards P, Rodwell K, McLennan K, Carroll T, Bandaranayake S, Scheinberg A, Waugh MC, Burnett H, Wimalasundera N, Hennel S, Lim E, O'Flaherty S PMID: 31583614 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - October 4, 2019 Category: Pediatrics Authors: Langdon K, Copeland L, Edwards P, Rodwell K, McLennan K, Carroll T, Bandaranayake S, Scheinberg A, Waugh MC, Burnett H, Wimalasundera N, Hennel S, Lim E, O'Flaherty S Tags: Paediatr Drugs Source Type: research

Authors' Reply to K. Langdon and Colleagues' Comment on: "Botulinum Toxin in the Management of Children with Cerebral Palsy".
Authors' Reply to K. Langdon and Colleagues' Comment on: "Botulinum Toxin in the Management of Children with Cerebral Palsy". Paediatr Drugs. 2019 Oct 04;: Authors: Multani I, Manji J, Hastings-Ison T, Khot A, Graham K PMID: 31583615 [PubMed - as supplied by publisher] (Source: Paediatric Drugs)
Source: Paediatric Drugs - October 4, 2019 Category: Pediatrics Authors: Authors' Reply to K. Langdon and Colleagues' Comment on: "Botulinum Toxin in the Management of Children with Cerebral Palsy". Tags: Paediatr Drugs Source Type: research

Drug-Induced Urolithiasis in Pediatric Patients.
Abstract Drug-induced nephrolithiasis is a rare condition in children. The involved drugs may be divided into two different categories according to the mechanism involved in calculi formation. The first one includes poorly soluble drugs that favor the crystallization and calculi formation. The second category includes drugs that enhance calculi formation through their metabolic effects. The diagnosis of these specific calculi depends on a detailed medical history, associated comorbidities and the patient's history of drug consumption. There are several risk factors associated with drug-induced stones, such as high...
Source: Paediatric Drugs - September 20, 2019 Category: Pediatrics Authors: Sighinolfi MC, Eissa A, Bevilacqua L, Zoeir A, Ciarlariello S, Morini E, Puliatti S, Durante V, Ceccarelli PL, Micali S, Bianchi G, Rocco B Tags: Paediatr Drugs Source Type: research