recently approved medications for children and adolescents

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...

INTRODUCTION: Congenital heart disease (CHD) is the most frequent congenital malformation. With recent advances in medical care, the majority of patients with CHD survive into adulthood. As a result, interest has shifted towards the neurodevelopmental outcome of these patients, and particularly towards the early detection and treatment of developmental problems. A variety of mild to moderate cognitive impairments as well as emotional and behavioural problems has been observed in this population...

Cannabis use in pediatric health care remains limited, however, there is increasing evidence on the pharmacologic benefits of medical marijuana for chronic conditions in childhood. Realizing the need for guidance in practice, the National Council of State Boards of Nursing (NCSBN) published guidelines to aid in decision making in nursing practice. While focusing primarily on adult use of cannabis, the guidelines do address special populations such as children and adolescents. This article reviews the endocannabinoid system, current state of legislation on medical marijuana, policy considerations, recent FDA approval of a cannabis product for pediatric use, NCSBN National Nursing Guidelines for Medical Marijuana, and pediatric implications for nursing practice...

BACKGROUND: Off-label drug use in children is common and potentially harmful. In most previous off-label use research, authors studied hospitalized children, specific drug classes, or non-US settings. We characterized frequencies, trends, and reasons for off-label systemic drug orders for children in ambulatory US settings. METHODS: Using nationally representative surveys of office-based physicians (National Ambulatory Medical Care Surveys, 2006-2015), we studied off-label orders of systemic drugs for children age <18 based on US Food and Drug Administration-approved labeling for age, weight, and indication...

INTRODUCTION: Compromised integrity of the brain due to paediatric acquired brain injury (pABI) has been associated with cognitive impairment, particularly executive dysfunction, in addition to somatic and emotional symptoms and reduced everyday function. Goal Management Training (GMT) is a cognitive rehabilitation intervention for improving executive function (EF) that has received empirical support in studies of adults with ABI. The purpose of the present study is to determine the efficacy of a recently developed paediatric version of GMT (pGMT) for children and adolescents with ABI and reported executive dysfunction...

OBJECTIVE: To evaluate the cost effectiveness of early treatment with direct-acting antiviral therapy in adolescent patients with chronic hepatitis C virus (HCV) infection compared with treatment deferral. STUDY DESIGN: We constructed a Markov model to assess the cost effectiveness of treating a hypothetical cohort of 30 000 adolescent patients with chronic HCV at age 12 years compared with deferring treatment until adulthood from a societal perspective. Model inputs for transition probabilities, HCV treatment and medical care costs, and quality-adjusted life-year (QALY) utilities were derived from the literature and wholesale acquisition estimates...

OBJECTIVES: The use of atypical antipsychotic medications in pediatric patients has become more prevalent in recent years. The purpose of this review is to provide a clinically relevant update of recent selected key publications regarding the use of atypical antipsychotics in this population. METHODS: Studies reviewed included randomized, double-blind, placebo-controlled medication trials conducted within the past 5 years. A PubMed search was conducted for each of the 11 second-generation antipsychotic medications currently approved by the Food and Drug Administration for use in the United States: clozapine, risperidone, olanzapine, quetiapine, aripiprazole, ziprasidone, paliperidone, asenapine, iloperidone, lurasidone, and cariprazine...

Autism spectrum disorder (ASD) is a heterogeneous neuropsychiatric condition that, based on recent CDC estimates affects an estimated 1 in 59 American children. Behavioral treatments remain the mainstay of treatment for the core symptoms of ASD including communication deficits, social interaction deficits and repetitive behavior. However, youth with ASD may also have severe behavioral challenges including irritability, aggression, and hyperactivity. Currently there are only two medications (risperidone and aripiprazole) approved by the FDA for the treatment of irritability associated with ASD in children...

Obesity is an ongoing global health concern and has only recently been recognized as a chronic disease of energy homeostasis and fuel partitioning. Obesity afflicts 17% of U.S. children and adolescents. Severe obesity (≥120% of the 95th percentile of body mass index (BMI) for age, or a BMI ≥ 35 kg/m2 ) is the fastest-growing subgroup and now approaches 6% of all U.S. youth. Health consequences (eg, type 2 diabetes, coronary heart disease) are related in a dose-dependent manner to severity of obesity. Because therapeutic interventions are less effective in severe obesity, prevention is a high priority...

In the vast majority of patients with hereditary angioedema (HAE), angioedema attacks are due to the quantitative or functional deficiency of C1-esterase inhibitor (C1-INH), which leads to increased vascular permeability and unregulated release of bradykinin. Exogenous administration of C1-INH is a rational way to restore the concentration and functional activity of this protein, regulate the release of bradykinin, and attenuate or prevent subcutaneous and submucosal edema associated with HAE. Recent international guidelines for the management of HAE include C1-INH as an option for acute treatment of HAE...

To date, no medication is proven to be effective in treating core symptoms of autism spectrum disorder (ASD). Psychotropic medications are widely used to target emotional and behavioural symptoms in ASD. This article reviewed evidence for pharmacotherapy, novel therapeutic agents, and Complementary and Alternative Medicine (CAM) in children and adolescents with ASD. Currently, only risperidone and aripiprazole have been approved by the US Food and Drug Administration (FDA) for treatment of irritability associated with ASD in children and adolescents...

Restless legs syndrome (RLS) and periodic limb movement disorder (PLMD) are under-recognized sleep disorders in children and adolescents. Several recent epidemiological studies have shown that RLS and PLMD are common in the pediatric population, and if left untreated, may lead to cardiovascular and neurocognitive consequences. Therefore, early diagnosis and intervention may help preventing long-term consequences. The management of RLS and PLMD in children involves both non-pharmacologic and pharmacologic approaches...

Evidence is emerging on the use of long-acting muscarinic antagonists (LAMAs) in the management of asthma. Tiotropium bromide (Spiriva® Respimat® ) is the only LAMA approved in children and adolescents. As the use of tiotropium becomes more common in clinical practice, it is necessary to review the existing data to identify patients who may benefit from the addition of this medication to their daily asthma regimen. This review discusses recent evidence on the safety and efficacy of tiotropium bromide in the management of asthma in children and adolescents...

Multiple sclerosis (MS) is a chronic demyelinating disease of the central nervous system. The disease usually affects young people, although it may also occur in early childhood. As well as the availability of diagnostic methods rises also the possibility of disease-modifying treatment increases. With constant development of diagnostic methods also the possibility of disease-modifying treatment (DMT) increases. Since 2012 in Poland a therapeutic program of the National Health Fund gives the possibility of using first-line therapy treatment in children/adolescents with multiple sclerosis (over 12 years of age), and since June 2015 using the interferon beta in children of 7 years old and body mass below 30 kg has been approved...

Despite the increasing number of medications recently approved to treat obesity among adults, few agents have been formally evaluated in children or adolescents for this indication. Moreover, there is a paucity of guidance in the literature addressing best practices with regard to pediatric obesity pharmacotherapy clinical trial design, and only general recommendations have been offered by regulatory agencies on this topic. The purposes of this article are to (1) offer a background of the current state of the field of pediatric obesity medicine, (2) provide a brief review of the literature summarizing pediatric obesity pharmacotherapy clinical trials, and (3) highlight and discuss some of the unique aspects that should be considered when designing and conducting high-quality clinical trials evaluating the safety and efficacy of obesity medications in children and adolescents...

Attention deficit hyperactivity disorder (ADHD) is a neurodevelopmental disorder - which may persist into adolescence and adulthood. Psychostimulants and atomoxetine (ATX) are frequently prescribed to treat ADHD in Germany. Lisdexamfetamine dimesylate (LDX) is the most recently approved ADHD medication in Germany and other European countries. Data used to support the European registration of LDX is summarised from three phase-3/3b studies in children and adolescents with ADHD. Short-term efficacy (study SPD489 - 325), maintenance of efficacy (study SPD489 - 326) and efficacy in patients who had previously responded inadequately to methylphenidate (MPH) treatment (study SPD489 - 317) were demonstrated...

Obesity is a complex and retractable disease for which effective and durable treatments are elusive. Successful treatment of severe obesity with lifestyle modification therapy alone is highly unlikely, particularly for adolescents. Pharmacotherapy, if appropriately prescribed, can be an effective tool to use in conjunction with lifestyle modification therapy to achieve better weight loss outcomes. Only a few obesity medications have been evaluated in children and adolescents with results suggesting modest efficacy...

Children/adolescents with attention-deficit/hyperactivity disorder (ADHD) may have a poor or inadequate response to psychostimulants or be unable to tolerate their side-effects; furthermore, stimulants may be inappropriate because of co-existing conditions. Only one non-stimulant ADHD pharmacotherapy, the noradrenaline transporter inhibitor atomoxetine, is currently approved for use in Europe. We review recent advances in understanding of the pathophysiology of ADHD with a focus on the roles of catecholamine receptors in context of the α2A-adrenergic receptor agonist guanfacine extended release (GXR), a new non-stimulant treatment option in Europe...

As demonstrated by the 2011 publication of the National Heart, Lung, and Blood Institute Integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents, the information available regarding the treatment of pediatric lipid disorders has greatly expanded. HMG-CoA reductase inhibitor, or statin, therapy is now considered a first-line pharmacologic intervention for pediatric patients with severe dyslipidemias failing treatment with diet and exercise alone. Despite their ability to effectively reduce cholesterol levels, bile acid sequestrants continue to pose challenges for pediatric patients because of their unpalatability and are typically used as adjunctive therapy or for patients not able to tolerate statins...