Predicting severe outcomes in pediatric trauma patients: Shock index pediatric age-adjusted vs. age-adjusted tachycardia.

INTRODUCTION: When an injured patient arrives in the Emergency Department (ED), timely and appropriate care is crucial. Shock Index Pediatric Age-Adjusted (SIPA) has been shown to accurately identify pediatric patients in need of emergency interventions. However, no study has evaluated SIPA against age-adjusted tachycardia (AT). This study aims to compare SIPA with AT in predicting outcomes such as mortality, severe injury, and the need for emergent intervention in pediatric trauma patients. MATERIAL AND METHODS: This is a retrospective cross-sectional analysis of patient data abstracted from the Trauma Quality Improvement Program Participant Use Files (TQIP PUFs) for years 2013-2020. Patients aged 4-16 with blunt mechanism of injury and injury severity score (ISS) > 15 were included. 36,517 children met this criteria. Sensitivity, specificity, overtriage, and undertriage rates were calculated to compare the effectiveness of AT and elevated SIPA as predictors of severe injuries and need for emergent intervention. Emergent interventions included craniotomy, endotracheal intubation, thoracotomy, laparotomy, or chest tube placement within 24 h of arrival. RESULTS: AT classified 59% of patients as "high risk," while elevated SIPA identified 26%. Compared to AT patients, a greater proportion of patients with elevated SIPA required a blood transfusion within 24 h (22% vs. 12%, respectively; p < 0.001). In-hospital mortality was higher for the elevated SIPA group than AT (10% vs. 5%, respectively; p < 0.001) as well as the need for emergent operative interventions (43% vs. 32% respectively; p < 0.001). Grade 3 or higher liver/spleen lacerations requiring blood transfusion were also more common among elevated SIPA patients than AT patients (8% vs. 4%, respectively; p < 0.001). AT demonstrated greater sensitivity but lower specificity compared to SIPA across all outcomes. AT showed improved overtriage and undertriage rates compared to SIPA, but this is attributed to identifying a large proportion of the sample as "high risk." CONCLUSIONS: AT outperforms SIPA in sensitivity for mortality, injury severity and emergent interventions in pediatric trauma patients while the specificity of SIPA is high across these outcomes.

Casimersen (AMONDYS 45™): An Antisense Oligonucleotide for Duchenne Muscular Dystrophy.

Casimersen (AMONDYS 45TM) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass developed by Sarepta therapeutics. It was approved by the Food and Drug Administration (FDA) in February 2021 to treat Duchenne muscular dystrophy (DMD) in patients whose DMD gene mutation is amenable to exon 45 skipping. Administered intravenously, casimersen binds to the pre-mRNA of the DMD gene to skip a mutated region of an exon, thereby producing an internally truncated yet functional dystrophin protein in DMD patients. This is essential in maintaining the structure of a myocyte membrane. While casimersen is currently continuing in phase III of clinical trials in various countries, it was granted approval by the FDA under the accelerated approval program due to its observed increase in dystrophin production. This article discusses the pathophysiology of DMD, summarizes available treatments thus far, and provides a full drug review of casimersen (AMONDYS 45TM).