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Journal Paediatr Drugs

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Management of Diabetic Ketoacidosis in Children and Adolescents with Type 1 Diabetes Mellitus.

Luz Castellanos, Marwa Tuffaha, Dorit Koren, Lynne L Levitsky,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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Combination of Biological Agents in Moderate to Severe Pediatric Inflammatory Bowel Disease: A Case Series and Review of the Literature.

Christine Olbjørn, Jon Bergreen Rove, Jørgen Jahnsen,

BACKGROUND:Treatment with biological agents such as anti-tumor necrosis factors (TNFs) has become standard of care in moderate to severe pediatric inflammatory bowel disease (IBD). However, a significant proportion of patients experience loss of response to anti-TNFs, need treatment escalation, or develop side effects. There is no data in the literature ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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The Use of TNFα Inhibitors in Treating Pediatric Skin Disorders.

Quoc-Bao D Nguyen, Caroline T Starling, Adelaide A Hebert,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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Why and How Should We Treat Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Syndrome?

Michaël Hofer,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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Dealing with COVID-19 in a Pediatric Rheumatology Unit in Italy.

Stefania Costi, Roberto Caporali, Rolando Cimaz,

Paediatr Drugs (Paediatric drugs)
[2020, :]

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Interleukin-1 Blockade in Systemic Juvenile Idiopathic Arthritis.

Manel Mejbri, Katerina Theodoropoulou, Michael Hofer, Rolando Cimaz,

Systemic juvenile idiopathic arthritis (sJIA) is a severe disorder now linked more to autoinflammation than to autoimmunity. Clinical and laboratory evidence support the pathogenetic role of interleukin-1 (IL-1), and blockade of this cytokine has proved to be very effective in the treatment of sJIA. There are now several agents that ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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Pharmacovigilance Reports Received from Children and Young People, and Development of Information to Aid Future Reporting from this Age Group.

Nikita Bhoombla, Jennifer Preston, Jenny Ainsworth, Helena Bird, Mitul Jadeja, Charlotte King, Daniel B Hawcutt,

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. Reports for suspected adverse drug ... Read more >>

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[2020, :]

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Correction to: Efficacy of Macrolides on Acute Asthma or Wheezing Exacerbations in Children with Recurrent Wheezing: A Systematic Review and Meta‑analysis.

Mauricio A Pincheira, Leonard B Bacharier, Jose A Castro-Rodriguez,

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. ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):241]

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Vaccine Hesitancy: A Growing Concern.

Jonathan Kennedy,

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, ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):105-111]

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The Evolving Diagnostic and Treatment Landscape of NTRK-Fusion-Driven Pediatric Cancers.

David S Shulman, Steven G DuBois,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):189-197]

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Drug Treatment of Progressive Myoclonic Epilepsy.

Gregory L Holmes,

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, ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):149-164]

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Administration and Dosing of Systemic Antifungal Agents in Pediatric Patients.

Kevin J Downes, Brian T Fisher, Nicole R Zane,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):165-188]

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New Developments in the Treatment of X-Linked Hypophosphataemia: Implications for Clinical Management.

Vrinda Saraff, Ruchi Nadar, Wolfgang Högler,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):113-121]

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Efficacy of Macrolides on Acute Asthma or Wheezing Exacerbations in Children with Recurrent Wheezing: A Systematic Review and Meta-analysis.

Mauricio A Pincheira, Leonard B Bacharier, Jose A Castro-Rodriguez,

INTRODUCTION:The role of macrolides for treatment of children with acute asthma or wheezing exacerbations is unclear. OBJECTIVE:The aim of this systematic review was to evaluate the effectiveness of macrolides in children with recurrent wheezing presenting with acute asthma or wheezing exacerbation. METHODS:We conducted an electronic search in MEDLINE, EMBASE, CINAHL, ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):217-228]

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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.

Tjitske M van der Zanden, Matthijs de Hoog, Jonathan D Windster, Joost van Rosmalen, I Heleen van der Sijs, Saskia N de Wildt,

OBJECTIVES:The structured digital dosing guidelines of the web-based Dutch Paediatric Formulary provided the opportunity to develop an integrated paediatric dose calculator. In a simulated setting, we tested the ability of this calculator to reduce calculation errors. METHODS:Volunteer healthcare professionals were allocated to one of two groups, manual calculation versus the ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):229-239]

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Pharmacokinetics and Clinical Pharmacology of Monoclonal Antibodies in Pediatric Patients.

Zaid H Temrikar, Satyendra Suryawanshi, Bernd Meibohm,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):199-216]

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Drug Treatment of Pulmonary Hypertension in Children.

Catherine M Avitabile, Erika E Vorhies, David Dunbar Ivy,

Pulmonary arterial hypertension (PAH) is a rare disease in infants and children that is associated with significant morbidity and mortality. The disease is characterized by progressive pulmonary vascular functional and structural changes resulting in increased pulmonary vascular resistance and eventual right heart failure and death. In many pediatric patients, PAH ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(2):123-147]

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A Prospective Cohort Study of Factors Associated with Empiric Antibiotic De-escalation in Neonates Suspected with Early Onset Sepsis (EOS).

Nazedah Ain Ibrahim, Mohd Makmor Bakry, Nurul Ain Mohd Tahir, Nur Rashidah Mohd Zaini, Noraida Mohamed Shah,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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The Role of Oral Administration of Immunoglobulin in Managing Diarrheal Illness in Immunocompromised Children.

Erin Alexander, Salim Hommeida, Michael C Stephens, Mhd Louai Manini, Imad Absah,

INTRODUCTION:Immunocompromised children are susceptible to infectious diarrhea. Oral administration of human serum immunoglobulins to treat immunocompromised patients with viral gastroenteritis caused by viruses like rotavirus and norovirus has been reported. OBJECTIVE:The aim of this study was to assess the efficacy of oral immunoglobulin (OIG) in treating hospitalized immunocompromised children with ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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Therapeutic Potential of Citrulline as an Arginine Supplement: A Clinical Pharmacology Review.

Jahidur Rashid, Shaun S Kumar, Kathleen M Job, Xiaoxi Liu, Candice D Fike, Catherine M T Sherwin,

Supplemental arginine has shown promise as a safe therapeutic option to improve endogenous nitric oxide (NO) regulation in cardiovascular diseases associated with endothelial dysfunction. In clinical studies in adults, L-arginine, an endogenous amino acid, was reported to improve cardiovascular function in hypertension, pulmonary hypertension, preeclampsia, angina, and MELAS (mitochondrial encephalomyopathy, ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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Dupilumab to Treat Type 2 Inflammatory Diseases in Children and Adolescents.

Amelia Licari, Riccardo Castagnoli, Alessia Marseglia, Francesca Olivero, Martina Votto, Giorgio Ciprandi, Gian Luigi Marseglia,

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. ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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A Phase IIa Clinical Trial of 2-Iminobiotin for the Treatment of Birth Asphyxia in DR Congo, a Low-Income Country.

Thérèse Biselele, Jephté Bambi, Dieu M Betukumesu, Yoly Ndiyo, Gabriel Tabu, Josée Kapinga, Valérie Bola, Pascal Makaya, Huibert Tjabbes, Peter Vis, Cacha Peeters-Scholte,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(1):95-104]

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Pharmacological Management of Pediatric Pustular Psoriasis.

Yi-Wei Huang, Tsen-Fang Tsai,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, :]

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A Practical Guide to the Management of Severe Hypertension in Children.

Rossana Baracco,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(1):13-20]

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Pediatric Clinical Endpoint and Pharmacodynamic Biomarkers: Limitations and Opportunities.

Jean C Dinh, Chelsea M Hosey-Cojocari, Bridgette L Jones,

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 ... Read more >>

Paediatr Drugs (Paediatric drugs)
[2020, 22(1):55-71]

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