Medicine In Pediatric Patients Research Articles

Methods Used for Pediatric Dose Selection in Drug Development Programs Submitted to the US FDA 2012-2020.

Dosing is a critical aspect of drug development in pediatrics that has led to trial failures and the inability to label the drug for pediatric use by the US Food and Drug Administration. Developing a structured approach for pediatric dose selection requires knowledge of the current approaches and their success or failure. This study describes the current experience with pediatric dosing methods from 2012 to 2020 and had 2 primary objectives: (1) to identify how the initial pediatric dose was selected and (2) to identify the pivotal dosing strategy used to identify the initially selected dose for safety and efficacy for pediatric clinical trials. Through September 2020, a total of 275 pediatric drug development programs were characterized for initial and pivotal dosing strategies. The success rate for labeling for pediatric use was 76.4%. The most common initial dosing strategy was previous experience with the product, followed by allometric scaling and exposure matching with adults. The most common pivotal dosing strategy was titration to target response in 33% of programs, with the second and third most common being pharmacokinetic/pharmacodynamic studies (30%) and exposure matching (20%), respectively. Additionally, about one-half of pediatric programs incorporated model-informed drug development. The emergence of titration to target response may signal a shift toward precision medicine in pediatric patients. Future work in pediatric drug dose selection should move toward the development of a structured pediatric dose selection approach.

Use of complementary and alternative medicine in pediatric patients in Saudi Arabia

Aim/Objective/BackgroundPeople all over the world use many forms of complementary and alternative medicine (CAM) and as evidence for efficacy slowly emerges for some therapies, the distinction between CAM and modern medicine is continuously evolving. CAM is commonly practiced in Saudi Arabia. The aim of this study was to assess the use of CAM among pediatric patients. MethodsA cross-sectional prospective study was carried out at the pediatric outpatient clinic at a university hospital in Al-Kharj City, Saudi Arabia. ResultsA total of 131 patient’s parents participated in the study and 90 % reported using CAM. Natural products were used more than body and mind practices (46 % vs. 19 %). Among natural products and body and mind practices, myrrh (53.4 %) and the Holy Quran (44.91 %) were reported to be the ones most used. Family and friends were the most common source of advice to parents regarding the use of CAM, with 77.12 % reporting that they were using CAM to treat diseases and 50 % reporting that using CAM cured their diseases. 77.12 % of parents were strongly satisfied with the use of CAM. ConclusionsHealthcare providers should be adequately trained to help educate parents regarding the efficacy and safety of CAM.

Pharmacogenomic Testing in Child and Adolescent Psychiatry: An Evidence-Based Review

Significant advances have been made in the application of pharmacogenomic testing for the treatment of patients with psychiatric disorders. Over the past decade, a number of studies have evaluated the utility of pharmacogenomic testing in pediatric patients with psychiatric disorders. The evidence base for pharmacogenomic testing in youth with depressive and anxiety disorders as well as attention/deficit hyperactivity disorder (ADHD) is reviewed in this article. General pharmacogenomic principles are summarized and functional polymorphisms in P450 enzymes (and associated metabolizer phenotypes), the serotonin transporter promoter polymorphisms, serotonin 2A receptor genes (e.g., HT2AR) and catecholamine pathway genes (e.g., COMT) are reviewed. These commonly tested pharmacogenomic markers are discussed with regard to studies of drug levels, efficacy and side effects. The translation of pharmacogenomics to individualized/precision medicine in pediatric patients with ADHD, anxiety and depressive disorders has accelerated; however, its application remains challenging given that there are numerous divergent pathways between medication/medication dose and clinical response and side effects. Nonetheless, by leveraging variations in individual genes that may be relevant to medication metabolism or medication target engagement, pharmacogenomic testing may have a role in predicting treatment response, side effects and medication selection in youth with ADHD, depressive and anxiety disorders.

An update on genomic-guided therapies for pediatric solid tumors.

Currently, out of the 82 US FDA-approved targeted therapies for adult cancer treatments, only three are approved for use in children irrespective of their genomic status. Apart from leukemia, only a handful of genomic-based trials involving children with solid tumors are ongoing. Emerging genomic data for pediatric solid tumors may facilitate the development of precision medicine in pediatric patients. Here, we provide an up-to-date review of all reported genomic aberrations in the eight most common pediatric solid tumors with whole-exome sequencing or whole-genome sequencing data (from cBioPortal database, Pediatric Cancer Genome Project, Therapeutically Applicable Research to Generate Effective Treatments) and additional non-whole-exome sequencing studies. Potential druggable events are highlighted and discussed so as to facilitate preclinical and clinical research in this area.

Out-of-hospital emergency medicine in pediatric patients: prevalence and management of pain

Introduction Much less is known about pain prevalence in pediatric patients in an out-of-hospital than emergency department setting. The purpose of this study was to determine pain prevalence in children in a prehospital emergency setting and to identify the factors associated with pain relief. Materials and Methods This prospective cohort study in consecutive patients 15 years or younger was conducted by 5 mobile intensive care units working 24/7 (January-December 2005). The presence of pain, its intensity, and alleviation by the administration of analgesics were recorded. Results A total of 258 of 433 pediatric patients were included, of whom 96 were suffering from acute pain (37%; 95% confidence interval [CI], 31-43) that was intense to severe in 67% of cases. Trauma was the only factor significantly associated with acute pain (odds ratio, 818; 95% CI, 153-4376). Overall, 92% of the children in pain received at least one analgesic drug; 41% received a combination of drugs. Opioid administration was significantly associated with intense to severe pain (odds ratio, 7; 95% CI, 2-25). On arrival at hospital, 67% of the children were still in pain; but 84% had experienced some pain relief regardless of their sex, age, or disorder. Conclusion In a prehospital emergency setting, more than a third of children experience acute pain with a high prevalence of intense to severe pain. Scoring pain in children, and especially in the newborn, is beleaguered by a lack of suitable scales. Despite this, it was possible to treat 90% of children in pain and provide relief in 80% of cases.

Use of complementary medicine in pediatric patients with inflammatory bowel disease: results from a multicenter survey

Use of complementary medicine in pediatric patients with inflammatory bowel disease: results from a multicenter survey

Use of Complementary Medicine in Pediatric Patients With Inflammatory Bowel Disease: Results From a Multicenter Survey

We examined the use of complementary and alternative medicine (CAM) at 3 US pediatric medical centers, comparing a group of children with inflammatory bowel disease (IBD) with children presenting with chronic constipation. Surveys were administered by postal mail and at pediatric IBD centers in San Francisco, Houston, and Atlanta from 2001 to 2003. A comparison group consisting of pediatric patients with chronic constipation also was surveyed. Data were analyzed by t tests and by exact tests of contingency tables. In all, 236 surveys were collected from the IBD group; 126 surveys were collected from the chronic constipation comparison group. CAM therapies were used by 50% in the IBD group and 23% in the chronic constipation group. The overall regional breakdown of CAM use in IBD revealed no differences, although the types of CAM therapy used varied by site. The most commonly used CAM therapies in the IBD group were spiritual interventions (25%) and nutritional supplements (25%). Positive predictors for CAM use in IBD include the patient's self-reported overall health, an increase in the number of side effects associated with allopathic medications, white ethnicity, and parental education beyond high school. This is the first US study to characterize CAM use in pediatric patients with IBD with another chronic gastrointestinal disorder. CAM use was twice as common with the IBD group compared with the chronic constipation group. Regional variations exist with the types of CAM therapy used. Practitioners should know that half of their pediatric patients with IBD may be using CAM in conjunction with or as an alternative to other treatments and that certain predictors can help identify those using CAM therapies.

S1050 The Use of Complementary and Alternative Medicine in Pediatric Patients with Functional and Organic Gastrointestinal Diseases: Results from a Multi-Center Survey

S1050 The Use of Complementary and Alternative Medicine in Pediatric Patients with Functional and Organic Gastrointestinal Diseases: Results from a Multi-Center Survey

The Use of Alternative Medicine in Pediatric Patients with Atopic Dermatitis

Alternative medicine has been defined as forms of therapy or examination that have no scientific basis and for which no effective or diagnostic reliability has been demonstrated by scientific methods. The use of complementary or alternative medicine is increasing and controlled clinical trials on the subject are few. We performed a questionnaire-based study of 80 pediatric patients with atopic dermatitis. This questionnaire assessed the duration of treatment, the reason(s) for trying alternative therapy, the approximate cost and the success of the treatment, the duration of the childhood eczema, and whether the child had ever required hospital admission for eczema. Of the total, 34 (42.5%) patients had used alternative medicine. Herbal remedies and homeopathy were used most often. Most treatments were reported to show no benefit and in three instances deterioration was reported. This study has prompted us to enquire routinely regarding alternative medicine use. Alternative therapies are subject to minimal regulation and have been associated with serious side effects. We would recommend enquiries regarding alternative medicine use in all pediatric dermatology patients.