recently approved medications for children and adolescents

PURPOSE OF REVIEW: In this review, we discuss new medical and surgical options for the treatment of children and adolescents with obesity. We review the impact of COVID-19 on this vulnerable population. We also discuss the recent availability of screening tests for rare genetic causes of obesity. RECENT FINDINGS: COVID-19 increased the prevalence of obesity among children and adolescents. This population is at increased risk for severe disease. The field of pediatric obesity has benefited from the approval of two new antiobesity medications: liraglutide and setmelanotide...

BACKGROUND: Paediatric atopic dermatitis (AD) can be burdensome, affecting mental health and impairing quality of life for children and caregivers. Comprehensive guidelines exist for managing paediatric AD, but practical guidance on using systemic therapy is limited, particularly for new therapies including biologics and Janus kinase (JAK) inhibitors, recently approved for various ages in this indication. OBJECTIVES: This expert consensus aimed to provide practical recommendations within this advancing field to enhance clinical decision-making on the use of these and other systemics for children and adolescents aged ≥2 years with moderate-to-severe AD...

INTRODUCTION: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal stem cell disease harvesting a somatic mutation in the phosphatidylinositol glycan class A ( PIG-A ) gene. This mutation results in a deficiency in cell membrane complement regulators leading to activation of the terminal complement pathway, clinically presenting as hemolytic anemia and thrombosis, and frequently associated with bone marrow failure. This condition was historically managed with supportive care and bone marrow transplant...

AIMS OF THE STUDY: In Switzerland, COVID-19 vaccines have been approved for children aged 5-11 years only recently, whereas vaccination of adolescents aged 12 years and older was approved in early summer 2021. Although the disease burden in children and adolescents has been reasonably mild, they can transmit COVID-19 to others, thus vaccinating this age group may help to curb the COVID-19 pandemic. The main objective was to investigate the association between five psychological antecedents of vaccination hesitancy in COVID-19 immunised parents and their intention to have their child vaccinated against COVID-19...

Medical therapy for pediatric heart failure is based on a detailed mechanistic understanding of the underlying causes, which are diverse and unlike those encountered in most adult patients. Diuresis and improved perfusion are the immediate goals of care in the child with acute decompensated heart failure. Conversion to maintenance oral therapy for heart failure is based on the results of landmark studies in adults, as well as recent pediatric clinical trials and heart failure guidelines. There will continue to be an important role for newer drugs, some of which are in active trials in adults, and some of which are already approved for use in children...

Many pediatric patients with rare diseases use drugs off-label due to limited data in pediatric patients. Off-label treatment remains an important public health issue for neonates, infants, children, and adolescents, especially for pediatric patients with rare diseases. For patients with rare diseases, the majority of medications have no or limited information in labelling for pediatric use. Children present unique considerations in clinical trials due to ethical and clinical concerns, which have limited and even discouraged testing of drugs in the pediatric population...

BACKGROUND: Bilastine, a non-sedating H1 -antihistamine, is indicated to treat the symptoms of allergic disorders (e.g. rhinoconjunctivitis and urticaria) in adults and adolescents and, more recently, in children. Following its marketing approval, many questions regarding the ideal use of bilastine in various clinical practice situations have been received by the Medical Information Department (MID) of Faes Farma Spain. This article is an update of a previous review, with a focus on recent clinical information on the use of bilastine in paediatric and other populations...

BACKGROUND: Biliary dyskinesia generally refers to a hypofunctioning gallbladder with an ejection fraction (EF) of <35% on hepatobiliary iminodiacetic acid scan with cholecystokinin stimulation (CCK-HIDA testing). In adults, biliary hyperkinesia has a defined association with biliary colic symptoms and can be relieved with surgical intervention. This clinical entity has not been well described in children or adolescents. In fact, only recently have we seen biliary hyperkinesia on HIDA at our centers...

Childhood-onset systemic lupus erythematosus (cSLE) is a prototype of a multisystemic, inflammatory, heterogeneous autoimmune condition. This disease is characterized by simultaneous or sequential organ and system involvement, with unpredictable flare and high levels of morbidity and mortality. Racial/ethnic background, socioeconomic status, cost of medications, difficulty accessing health care, and poor adherence seem to impact lupus outcomes and treatment response. In this article, the management of cSLE patients is updated...

Approximately 470,000 children and adolescents in the United States have epilepsy, 30% of whom experience seizures despite antiseizure drug regimens. School nurses, teachers, caregivers, and parents play integral roles in implementing a care plan that avoids triggers, recognizes signs, and provides supportive care-ideally, guided by a patient-specific seizure action plan, which may include the use of rescue medication. Benzodiazepines are the mainstay of seizure rescue medication; for decades, rectally administered diazepam was the only approved rescue medication for seizure clusters outside the hospital setting...

Hereditary hypophosphatemia with increased FGF23 levels are rare inherited metabolic diseases characterized by low serum phosphate because of impaired renal tubular phosphate reabsorption. The most common form is X-linked hypophosphatemia (XLH), secondary to a mutation in the PHEX gene. In children, XLH is often manifested by rickets, delayed development of gait, lower limb deformities, growth retardation, craniosynostosis, and spontaneous dental abscesses. In adults, patients present diffuse musculoskeletal pain (bone and joints), early osteoarthritis, entesopathies, pseudo-fractures, muscular weakness, and severe dental damage...

A functional hypersalivation reduces patient's quality of life by the need of repeated changes of cloths, skin damage around the mouth and reduced personal contacts. The indication to treat hypersalivation is justified furthermore when respiratory infections by saliva aspiration occur. Transnasal swallowing endoscopy allows to evaluate sufficiently dysphagia with limited risks. With this method therapy options can be judged for effectiveness. There are other additional radiologic assessments to complete diagnostic...

BACKGROUND: Migraine is a primary headache disorder and is the most common disabling primary headache disorder that occurs in children and adolescents. A recent study showed that paranasal air suction can provide relief to migraine headache. However, in order to get the maximum benefit out of it, an easy to use effective air sucker should be available. Aiming to fulfil the above requirement, a randomized, double blind control clinical trial was conducted to investigate the efficacy of a recently developed low-pressure portable air sucker...

The prevalence of type 2 diabetes mellitus (T2DM) in children and adolescents is on the rise, and the increase in prevalence of this disorder parallels the modern epidemic of childhood obesity worldwide. T2DM affects primarily post-pubertal adolescents from ethnic/racial minorities and those from socioeconomically disadvantaged backgrounds. Youth with T2DM often have additional cardiovascular risk factors at diagnosis. T2DM in youth is more progressive in comparison to adult onset T2DM and shows lower rates of response to pharmacotherapy and more rapid development of diabetes-related complications...

Dupilumab has recently been approved by the Food and Drug Administration for use for treatment of moderate to severe atopic dermatitis in children aged 6 to 11 years. It presents a novel treatment option with a favorable safety profile for patients who are currently reliant on immunosuppressants, including cyclosporine A, methotrexate, and mycophenolate mofetil. Particularly during the current COVID-19 pandemic, immunosuppression should be avoided to retain intrinsic antiviral immunity. Transitioning to dupilumab should be executed strategically-tapering immunosuppressants and minimizing risk of flare by overlapping with the biologic...

Atopic dermatitis (AD) is a common skin disease during infancy, which imposes a considerable burden on patients, their families, and the society, requiring effective treatment options that result in rapid and sustained symptom relief. Additionally, early treatment may prevent the development of atopic comorbidities by restoring the skin barrier. Currently, topical standard-of-care for AD in infants includes emollients and topical corticosteroids (TCS) to treat and reduce the risk of flares. However, only few have been approved for infants and long-term maintenance therapy with TCS is not indicated due to potential local and systemic side effects, including skin atrophy...

Objective: Only few psychotropic drugs are approved to treat tic disorders. The aim was to describe use of tic-suppressing medication and other psychotropic drugs in children with tics. Methods: Using nationwide registries, we identified children receiving a tic diagnosis in Denmark during 2006-2017 and extracted data on tic-suppressing medication and other psychotropic drugs. Results: Antipsychotics were used by 12%. Use of tic-suppressing medication increased with age and varied according to sex. Over time less children with tics were medicated (from 44% to 38% in the total use of psychotropic drugs) mainly due to decreased use of antipsychotics (from 18% to 6...

In this clinical guidelines article, we first include a brief review of the epidemiology, pathogenesis, clinical diagnoses, and scoring-scales for pediatric atopic dermatitis (AD). We then offer a set of pharmacologic treatment guidelines for infants and toddlers (<2 years), children (2-12 years), and adolescents (>12 years). We recommend irritant avoidance and liberal emollient usage as the cornerstone of treatment in all age-groups. In infants <2 years, we recommend topical corticosteroids as first-line medication-based therapy...

Autism spectrum disorder (ASD) is a heterogeneous neuropsychiatric condition affecting an estimated one in 36 children. Youth with ASD may have severe behavioral disturbances including irritability, aggression, and hyperactivity. Currently, there are only two medications (risperidone and aripiprazole) approved by the US Food and Drug Administration (FDA) for the treatment of irritability associated with ASD. Pharmacologic treatments are commonly used to target ASD-associated symptoms including irritability, mood lability, anxiety, and hyperactivity...

INTRODUCTION: The incidence of type 2 diabetes mellitus has increased in children and adolescents due largely to the obesity epidemic, particularly in high risk ethnic groups. β-Cell function declines faster and diabetes complications develop earlier in paediatric type 2 diabetes compared with adult-onset type 2 diabetes. There are no consensus guidelines in Australasia for assessment and management of type 2 diabetes in paediatric populations and health professionals have had to refer to adult guidelines...