Prognostic scoring system for Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis in children

A recent study suggests that a prognostic scoring system identified three high risk groups in children with statistically significant differences in survival curves based on the baseline characteristics that helped in risk prediction. The findings of this study were published in the journal, Pediatric Blood & Cancer.

This multicenter retrospective study included 264 children, aged 0-14 years who were diagnosed with Epstein-Barr virus (EBV)-associated hemophagocytic lymphohistiocytosis (EBV-HLH). The pediatric population was randomly divided into two groups: derivation (n=185) and verification (n=79). In order to establish a prognostic scoring system for death events, risk predictors were investigated using a Cox regression model during the follow-up period. Chronic active EBV infection (CAEBV) history, plasma EBV-DNA copy number, pulmonary infection hemorrhage of digestive tract, and hypoxemia prognostic scoring system (CEPHO-PSS) were developed which demarcated patients into low-risk (0-1 points), middle-risk (2-3 points), and high-risk (4-8 points) groups.

At the end of the study, the three risk groups showed survival curves with statistically significant differences. Receiver operating characteristic (ROC) and calibration curves in the derivation and verification cohorts, respectively, were used for internal and external verification of CEPHO-PSS, confirming good accuracy and applicability.

Thus, it can be concluded that the CEPHO-PSS may identify three risk groups in children with statistically significant differences in survival curves. This may help clinicians to check for risk prediction.

Maralixibat improves health-related quality of life in pediatric patients with Alagille Syndrome

A recent study demonstrated that the ileal bile acid transporter inhibitor maralixibat improves health-related quality of life (HRQoL) in children with Alagille syndrome. This study was published in The Journal of Pediatrics.

The ICONIC trial was a phase 2 study, having a 4-week double-blind, placebo-controlled, randomized drug withdrawal period. The study included 27 children having Alagille syndrome with moderate-to-severe pruritus. From baseline to week 48, the treatment response to maralixibat was noted using Itch-Reported Outcome (Observer) score. The HRQoL was assessed based on the certain scale scores that included Pediatric Quality of Life Inventory Generic Core, Family Impact, and Multidimensional Fatigue scale scores.

At week 48, twenty patients attained an Itch-Reported Outcome (Observer) treatment score response. The mean (SD) change in Multidimensional Fatigue score was higher was higher for responders over non-responders. The Pediatric Quality of Life Inventory Generic Core and Multidimensional Fatigue scores showed smaller and non-statistically significant point estimates.

Based on the results of the study, it can be concluded that maralixibat shows significant improvement in pruritis, thereby enhancing the quality of life in the affected children.

Sustained and moderating effects of a behavioural sleep intervention for autistic children

A recent study suggests that the Sleeping Sound intervention showed sustained improvements in child sleep among autistic children. The study’s findings were published in the Journal of Autism and Developmental Disorders.

This was a randomized controlled trial which included 150 autistic children aged 5-13 years, who had sleep problems. They were randomized either to the Sleeping Sound intervention or Treatment as Usual (TAU).

At the 12-month follow-up, the caregivers of the children reported a greater reduction in sleep problems in the Sleeping Sound group when compared to the TAU group. Those children who were taking sleep medication, children with greater autism severity, and children of parents who were not experiencing psychological distress showed greater long-term benefits with the intervention. Based on the above results, it can be concluded that Sleeping Sound intervention may show sustained improvements in child sleep.

Evaluation of the diagnostic accuracy of point-of-care ultrasound for pediatric distal forearm fractures

According to a recent study, clinician-performed point-of-care ultrasound proved to be more precise in diagnosing clinically nondeformed distal forearm injuries in children and adolescents compared to clinician-interpreted radiographic imaging. The findings of this study were published in the journal, Annals of emergency medicine.

This was an open-label, multicenter, diagnostic randomized controlled trial where 270 participants were enrolled. Eligible patients were randomly assigned to undergo initial imaging either through point-of-care ultrasound conducted by an emergency department (ED) clinician or through radiography (135 randomized to each imaging group). The primary outcome was the treating clinician's diagnostic accuracy in comparison to the reference standard diagnosis. The reference standard diagnosis was determined retrospectively by a panel of experts, which included an emergency physician, pediatric orthopedic surgeon, and pediatric radiologist. This panel thoroughly reviewed all imaging and follow-up data.

At the end of the study, it was observed that in the point-of-care ultrasound group, 132 (97.8%) participants were accurately diagnosed by ED clinicians, while in the radiograph group, 112 (83.0%) participants received correct diagnoses. Point-of-care ultrasound demonstrated superior accuracy in detecting "buckle" fractures (AD=18.5%) and "other" fractures (AD=17.1%).

Thus, it can be concluded that clinician-performed point-of-care ultrasound demonstrated superior accuracy in diagnosing clinically nondeformed distal forearm injuries in children and adolescents compared to clinician-interpreted radiographic imaging in the ED.