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"Pediatrics General"
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Assessing methods of measuring medication adherence in chronically ill children–a narrative review
Nonadherence in children who use long-term medication is a serious problem and assessing adherence is an important step to provide solutions to this problem. Medication adherence can be measured by several methods, including (a) self-report questionnaires or structured interviews, (b) therapeutic drug monitoring (TDM), (c) electronic devices, and (d) pick-up/refill rates. The objective of this narrative review is to provide an overview of the literature about methods for the measurement of medication adherence in chronically ill children and adolescents. Therefore, we conducted a literature search by using multiple databases. Four methods of monitoring medication adherence are presented for the most described chronic diseases: asthma, HIV/AIDS, epilepsy, diabetes mellitus and ADHD. First, 10 commonly used self-report questionnaires and structured interviews are described, including the main characteristics, (dis)advantages and their validation studies. Second, the use of TDM in pediatric trials for medication adherence measurement is discussed. New sampling methods (e.g. dried blood spot) and sampling matrices (e.g. hair, saliva and urine) have shown their benefits for TDM in children. Third, electronic devices to measure medication adherence in children are presented, being developed for several drug administration routes. Fourth, the analyses, advantages and disadvantages of pharmacy data are discussed. The usage of this data requires specific calculations and interpretations to assess adherence. As presented in this review, every adherence method has specific (dis)advantages. When deciding which adherence method is applicable, validity and generalizability should be taken into account. Combining multiple methods seems to offer the best solution in the daily clinical practice.
Journal Article
Liraglutide for Children 6 to <12 Years of Age with Obesity — A Randomized Trial
Obesity often develops in childhood. In children (6 to <12 years of age) with obesity, treatment with liraglutide for 56 weeks plus lifestyle interventions induced a greater mean change in BMI than lifestyle interventions alone.
Journal Article
Benefits, Limits, and Risks of GPT-4 as an AI Chatbot for Medicine
Chatbots are computer programs with which one can have a conversation. In this article, the authors describe how the GPT-4 chatbot, which has been given a general education, could affect the practice of medicine.
Journal Article
Current Causes of Death in Children and Adolescents in the United States
Current Causes of Death in U.S. Children and AdolescentsFirearm-related injury is now the leading cause of death among children and teens. We continue to fail to protect our youth from a preventable cause of death.
Journal Article
The Current and Future State of AI Interpretation of Medical Images
Using AI to Interpret Medical ImagesThe authors examine the advantages and limitations of current clinical radiologic AI systems, new clinical workflows, and the potential effect of generative AI and large multimodal foundation models.
Journal Article
Childhood Cardiovascular Risk Factors and Adult Cardiovascular Events
In this study, childhood cardiovascular risk factors including BMI, systolic blood pressure, lipid levels, and smoking were correlated with cardiovascular events in adulthood after a mean follow-up of 35 years. Childhood risk factors and the change in risk score between childhood and adulthood were associated with midlife cardiovascular events.
Journal Article
The Vaccine-Hesitant Moment
The Vaccine-Hesitant MomentThe proliferation of vaccine misinformation and its use for political purposes are placing a large number of people at risk in the Covid-19 pandemic and allowing the pandemic to continue.
Journal Article
Pulmonary Arterial Hypertension
Without effective treatment, pulmonary arterial hypertension results in high morbidity and mortality. This review discusses the pathogenesis of the disorder, the thorough clinical evaluation required to establish the diagnosis, available therapies, and future directions.
Journal Article
GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma
Immunotherapy with chimeric antigen receptor (CAR)-expressing T cells that target the disialoganglioside GD2 expressed on tumor cells may be a therapeutic option for patients with high-risk neuroblastoma.
In an academic, phase 1-2 clinical trial, we enrolled patients (1 to 25 years of age) with relapsed or refractory, high-risk neuroblastoma in order to test autologous, third-generation GD2-CAR T cells expressing the inducible caspase 9 suicide gene (GD2-CART01).
A total of 27 children with heavily pretreated neuroblastoma (12 with refractory disease, 14 with relapsed disease, and 1 with a complete response at the end of first-line therapy) were enrolled and received GD2-CART01. No failure to generate GD2-CART01 was observed. Three dose levels were tested (3-, 6-, and 10×10
CAR-positive T cells per kilogram of body weight) in the phase 1 portion of the trial, and no dose-limiting toxic effects were recorded; the recommended dose for the phase 2 portion of the trial was 10×10
CAR-positive T cells per kilogram. Cytokine release syndrome occurred in 20 of 27 patients (74%) and was mild in 19 of 20 (95%). In 1 patient, the suicide gene was activated, with rapid elimination of GD2-CART01. GD2-targeted CAR T cells expanded in vivo and were detectable in peripheral blood in 26 of 27 patients up to 30 months after infusion (median persistence, 3 months; range, 1 to 30). Seventeen children had a response to the treatment (overall response, 63%); 9 patients had a complete response, and 8 had a partial response. Among the patients who received the recommended dose, the 3-year overall survival and event-free survival were 60% and 36%, respectively.
The use of GD2-CART01 was feasible and safe in treating high-risk neuroblastoma. Treatment-related toxic effects developed, and the activation of the suicide gene controlled side effects. GD2-CART01 may have a sustained antitumor effect. (Funded by the Italian Medicines Agency and others; ClinicalTrials.gov number, NCT03373097.).
Journal Article