Profile of Chronic Non-Epilepsy Drug Use in Children with Epilepsy: Single Centre Experience.

We determined the burden of concomitant chronic non-epilepsy drug use in 100 consecutive outpatient children with a diagnosis of epilepsy (mean (SD) age 7.38 (3.24) y), taking anti-seizure medications (ASM) for at least 6 months. Majority (n = 68) of the children had comorbidities; most commonly global developmental delay (27%). 61 children were receiving chronic non-epilepsy drugs; most common being calcium (38%), multivitamin (18%) and folic acid (16%). Of these, 50 children (82%) were prescribed drugs without any documented indication. Another 24 children (39.4%) were using complementary and alternative medications. The observed chronic non-epilepsy drug use, many of which were not indicated, reiterates the need to limit the burden of medications in children with epilepsy.

Cell-Based Gene Therapy for b-Thalassemia.

The United States Food and Drug Administration (FDA) approved betibeglogene autotemcel (beti-cel), the first cell-based gene therapy for adult and pediatric patients with b-thalassemia in August, 2022. This update details this and other novel therapies that have emerged in the treatment of b-thalassemia, apart from transfusion and iron chelation, with particular focus on newly approved gene therapy.

Factors Affecting Quality of Life in Adolescent Siblings of Children With Autism Spectrum Disorder.

OBJECTIVE: To compare the quality of life (QoL) of adolescent siblings of children with autism spectrum disorder (ASD-Sibs) with siblings of typically developing children (TD-Sibs), and study the factors affecting the QoL. METHODS: Between 1 February, 2021 and 31 September, 2021, 40 children aged 10-18 years, whose sibling was suffering from ASD, were enrolled (Study group). 40 age- and sex-matched siblings of children with no clinically apparent neuro- developmental abnormality or behavioral problem were also enrolled (Control group). Severity of autism was assessed by using the childhood autism rating scale 2 (CARS-2) score. QoL was assessed by a validated version of the World Health Organi-zation Quality of Life questionnaire Brief version (WHO QoL BREF), and compared between cases and controls using Wilcoxon rank sum test. RESULTS: The mean (SD) age of study participants was 13.55 (2.75) years. The mean (SD) CARS-2 score of our sample was 35.78 (5.23). Mild to moderate autism was seen in 23 (57.5%) children, and 13 (32.5%) had severe autism. The median (IQR) QoL in ASD-Sibs was worse than TD-Sibs in physical domain (24 (19,26) vs 32 (29,32); P<0.001), psychological domain (22 (17,23) vs 25 (23,25); P<0.001), social domain (11 (8,12) vs 13 (11,14); P<0.001), and environmental domain (28 (26,31) vs 35 (31,35); P<0.001). Among the ASD-Sibs, severity of the sibling's ASD and the family's socioeconomic status were the only two factors significantly affecting one of the domains of QoL. CONCLUSION: The observed lower QoL score in adolescent siblings of children with ASD, more so in those whose siblings had more severe ASD, suggests the need for targeting the family as a unit while formulating plans for holistic management of children with ASD.