A Practical Guide to the Management of Severe Hypertension in Children
AbstractSevere 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. (Source: Pediatric Drugs)
Source: Pediatric Drugs - January 9, 2020 Category: Pediatrics Source Type: research

Pediatric Clinical Endpoint and Pharmacodynamic Biomarkers: Limitations and Opportunities
AbstractMedical 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 c hildren is far more reliant upon expert opinion than in adult medicine. Children are at risk of not receiving the same...
Source: Pediatric Drugs - January 8, 2020 Category: Pediatrics Source Type: research

Update on Drug Management of Refractory Epilepsy in Tuberous Sclerosis Complex
AbstractTuberous 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, vigabatr...
Source: Pediatric Drugs - January 7, 2020 Category: Pediatrics Source Type: research

Pharmacologic Management of Chronic Urticaria in Pediatric Patients: The Gap Between Guidelines and Practice
AbstractChronic 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. Chron ic urticaria that is triggered by external physical stimuli is called chronic inducible urticaria and is present in another 15–20%. Allergie...
Source: Pediatric Drugs - December 19, 2019 Category: Pediatrics Source Type: research

A Scoping Review of Medications Studied in Pediatric Polypharmacy Research
ConclusionWhile 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. (Source: Pediatric Drugs)
Source: Pediatric Drugs - December 9, 2019 Category: Pediatrics Source Type: research

Molecularly Targeted Agents in the Therapy of Pediatric Brain Tumors
AbstractPediatric 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 the genet...
Source: Pediatric Drugs - November 20, 2019 Category: Pediatrics Source Type: research

CD19 CAR T Cells for the Treatment of Pediatric Pre-B Cell Acute Lymphoblastic Leukemia
AbstractThe 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 refractory ...
Source: Pediatric Drugs - November 19, 2019 Category: Pediatrics Source Type: research

Macrophage Activation Syndrome and Secondary Hemophagocytic Lymphohistiocytosis in Childhood Inflammatory Disorders: Diagnosis and Management
AbstractMacrophage 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 such as ga...
Source: Pediatric Drugs - November 15, 2019 Category: Pediatrics Source Type: research

Acknowledgement to Referees
(Source: Pediatric Drugs)
Source: Pediatric Drugs - November 1, 2019 Category: Pediatrics Source Type: research

Treatment Withdrawal Following Remission in Juvenile Idiopathic Arthritis: A Systematic Review of the Literature
ConclusionsThe 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. (Source: Pediatric Drugs)
Source: Pediatric Drugs - October 31, 2019 Category: Pediatrics Source Type: research

Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1
AbstractTyrosinemia 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 carcinoma. NTBC...
Source: Pediatric Drugs - October 30, 2019 Category: Pediatrics Source Type: research

Update on the Systemic Treatment of Pediatric Localized Scleroderma
AbstractJuvenile 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 joint, or...
Source: Pediatric Drugs - October 30, 2019 Category: Pediatrics Source Type: research

Pharmacological Prevention and Management of Postoperative Relapse in Pediatric Crohn ’s Disease
AbstractPediatric 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 childr en 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 e ffic...
Source: Pediatric Drugs - October 17, 2019 Category: Pediatrics Source Type: research

A Review of Regional Anesthesia in Infants
AbstractRegional 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 introduction of ultr...
Source: Pediatric Drugs - October 17, 2019 Category: Pediatrics Source Type: research

Correction to: DTaP ‑IPV‑HepB‑Hib Vaccine (Hexyon ® ): An Updated Review of its Use in Primary and Booster Vaccination
The article DTaP-IPV-HepB-Hib Vaccine (Hexyon (Source: Pediatric Drugs)
Source: Pediatric Drugs - October 14, 2019 Category: Pediatrics Source Type: research