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Thank you for visiting the Contemporary Pediatrics® website. Take a look at some of our top stories from last week (Monday, November 18, to Friday, November 22, 2024), and click on each link to read and watch anything you may have missed.
The data comes from the phase 3 investigational trials ICONIC-LEAD (NCT06095115) and ICONIC-TOTAL (NCT06095102), that evaluated JNJ-2113 (formerly PN-235), a potential first-in-class targeted oral peptide designed to block the IL-23 receptor in pateints aged 12 years and older with moderate-to-severe plaque psoriasis.
The ICONIC-LEAD trial's inclusion criteria included the diagnosis of plaque psoriasis, with or without psoriatic arthritis, for at least 26 weeks prior to first administration of intervention, a total body surface area greater than or equal to 10% at screening and baseline, total psoriasis area and severity index (PASI) greater than or equal to 12 at screening and baseline, and a total investigator global assessment (IGA) greater than or equal to 3 at screening and baseline, according to study details via ClinicalTrials.gov. Enrollment featured 684 individuals.
Data, published today in The New England Journal of Medicine (NEJM), revealed infigratinib treatment resulted in a statistically significant and sustained increase in annualized height velocity (AHV), positive mean change in baseline in height Z-score, and improved body proportionality, while demonstrating no serious adverse events (SAE) or treatment-emergent adverse events (TEAEs) that led to discontinuation.
According to the NEJM, achondroplasia is a genetic skeletal condition that results in disproportionately short stature and medical complications throughout life. In the phase 2 PROPEL 2 study (NCT04265651), the oral bioavailable FGFR1–3 selective tyrosine kinase inhibitor was evaluated for safety and efficacy in children with achondroplasia aged between 3 and 11 years. There were 72 children enrolled across 5 sequential cohorts.
The investigational oral therapy is a new chemical entity designed to reduce the dimerization of vitamin A without modulating the visual cycle, according to the pharmaceutical company. The disease can result in severe vision impairment in children and young adults, with no treatments currently approved. It is estimated that between 30,000 and 87,000 people in the United States are affected, stated Alkeus.
The investigational therapy has been, and is currently being studied in the Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) clinical program, comprised of 4 independent studies of oral gildeuretinol to treat Stargardt disease: TEASE-1, TEASE-2, TEASE-3 and TEASE-4.
Stephanie Anne Deutsch, MD, MS, MSCR, FAAP, joined us to discuss a new study that highlighted SUID among infants who were prenatally substance exposed.
"At the local level, I am involved in child fatality review processes in the Delaware Valley region, and we were noticing a trend of increasing SUID among infants that had a history of prenatal substance exposure and concerning circumstances amongst those deaths, including report potentially of caregiver impairment," said Deutsch. "That inspired us to look at this topic at the national level and obtain data from the National Center for Fatality Review and Prevention through their case reporting system."
H. Westley Phillips, MD, explains why awareness and availability of epilepsy surgery is important for pediatric providers among drug-resistant epilepsy patients.
"The data has shown that epilepsy surgery is both safe and effective in treating drug-resistant epilepsy, and so the number of patients that come in, undergo a surgical evaluation, and successful surgery—the most common thing I hear is, ‘We wish we would have done this sooner.’”
