Efficacy and Safety of Remdesivir in Hospitalized Pediatric COVID-19: A Retrospective Case-Controlled Study.

Introduction: While most children experience mild coronavirus disease 2019 (COVID-19) infections, a minority of cases progress to severe or critical illness. This study aimed to assess the efficacy and safety of Remdesivir (RDV) therapy in children with moderate to severe COVID-19, enhancing clinical decision-making and expanding our understanding of antiviral treatments for pediatric patients. Methods: The study included 60 patients, 38 receiving RDV treatment and 22 serving as the control group. Data was collected retrospectively from January 2021 to January 2022 through electronic hospital records. Results: Regarding the main clinical symptoms reported, most patients experienced Upper Respiratory Tract Infections (93.3%), indicating respiratory involvement. Additional symptoms included Central Nervous System (11.7%) and Gastrointestinal (10.0%). Among the 38 cases in the RDV group included in the study, the adverse effects associated with using RDV: Hypoalbuminemia in 19 cases (50.0%) and anemia in 18 cases (47.4%), making them the most common adverse effects. Only one case in the RDV group experienced non-RDV-related death with a different clinical diagnosis. The results showed that RDV treatment was well-tolerated in pediatric patients, with no significant differences in hospital stay and oxygen treatment compared to the control group with P values (0.2, 0.18), respectively. Conclusion: The outcomes indicate that Remdesivir may represent a safe and therapeutic choice for children with coronavirus disease 2019 (COVID-19).

Severe pulmonary hemorrhage in a 3-week-old neonate with COVID-19 infection: A case report.

Our patient is a 3-week-old female neonate, presented with complaints of low-grade fever and a congested nose for one day. Eventually, she developed progressive desaturation, hypotension, and poor perfusion due to severe pulmonary hemorrhage. Then, she developed cardiac arrest and was declared dead.

Neonatal Simulation Program: A 5 Years Educational Journey From Qatar.

We describe the process of implementation, adaptation, expansion and some related clinical intuitional impacts of the neonatal simulation program since its launch in 2016 in a non-simulation neonatal unit. The team has developed 6 types of curricula: 1 full-day course and 5 half-day workshops. A total of 35 free of charge simulation courses/workshops were conducted, 32 in Qatar and 3 abroad with a total of 799 diverse participants. There was a steady increase in the overall success rate of PICC insertion from 81.7% (309/378) to 97.6% (439/450) across 3 years (P < 0.0001). The first attempt PICC insertion success rate has been also increased from 57.7% (218/378) to 66.9% (301/450) across 3 years. The mean duration of PICC insertion has been improved from 39.7 ± 25 to 34.9 ± 12.4 min after implementing the program (P = 0.33). The mean duration of the LISA catheter insertion at the beginning of the workshop was 23.5 ± 15.9 compared to 12.1 ± 8.5 s at the end of the workshop (P = 0.001). When it came to clinical practise in real patients by the same participants, the overall LISA catheter insertion success rate was 100% and the first attempt success rate was 80.4%. The mean duration of LISA catheter insertion in real patients was 26.9 ± 13.9 s compared to the end of the workshop (P = 0.001). The mean duration of the endotracheal intubation at the beginning of the workshop was 12.5 ± 9.2 compared to 4.2 ± 3.8 s at the end of the workshop (P = 0.001). In real patients, the first-attempt intubation success rate has been improved from 37/139 (26.6%) in the first year to 141/187 (75.5%) in the second year after the program implementation (P = 0.001). The mean duration of successful endotracheal intubation attempts has been improved from 39.1 ± 52.4 to 20.1 ± 9.9 s (P = 0.78). As per the participants, the skills learned in the program sessions help in protecting neonates from potential harm and improve the overall neonatal outcome. Implementing a neonatal simulation program is a promising and feasible idea. Our experience can be generalised and replicated in other neonatal care institutions.

Multisystem inflammatory syndrome in children (MIS-C) related to COVID-19 infection in the state of Qatar: Association with Kawasaki-like Illness.

INTRODUCTION: World Health Organization (WHO) is encouraging reporting of children with Multisystem Inflammatory Syndrome (MIS-C) associated with SARS-CoV-2 infection for better understanding and management of the disease. METHODOLOGY: This retrospective study included the first 15 pediatrics patient with a confirmed diagnosis of MIS-C associated with SARS-CoV-2 infection in the state of Qatar. We studied and analyzed their demographic data, clinical manifestations, laboratory tests, treatment, and outcome. RESULTS: A total of 15 children were studied (mean age 3.5 ± 2.7year). Recent severe acute respiratory syndrome coronavirus 2 infection was identified in all of them (100%). The majority of these patients had 4 or more systems involvement. Nine of the 15 presented with Kawasaki disease - picture and all had gastrointestinal symptoms (vomiting and diarrhea). Five required Pediatrics Intensive Care Unit (PICU) admission. Lab investigations revealed high D-Dimer, hyponatremia, and hypoalbuminemia in all. Low hemoglobin (Hb) , thrombocytopenia, and sterile pyuria occurred in 86.6%, 60% and 75% of them, respectively. Treatment with combined anti-inflammatory medications (intravenous immunoglobulin, corticosteroids) was used in along with immunomodulatory agents (Anakinra) in a selected group of refractory patients. No mortality happened. CONCLUSION: Our young children who presented with MIS-C related to SARS-CoV-2 infection had significantly higher Kawasaki-disease picture compared to other reports. One third of them required PICU admission but no mortality occurred.

Gene therapy for spinal muscular atrophy: the Qatari experience.

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by hypotonia, progressive muscle weakness, and wasting. Onasemnogene abeparvovec (Zolgensma®) is a novel gene therapy medicine, FDA-approved in May 2019 for the treatment of SMA. This study aimed to describe Qatari experience with onasemnogene abeparvovec by reviewing the clinical outcomes of 9 SMA children (7 SMA type 1 and 2 with SMA type 2) aged 4‒23 months treated between November 2019 and July 2020. Children <2 years with 5q SMA with a bi-allelic mutation in the SMN1 gene were eligible for gene therapy. Liver function (aspartate aminotransferase [AST], alanine aminotransferase [ALT], and total bilirubin), platelet count, coagulation profile, troponin-I levels, and motor scores (Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders [CHOP INTEND]), were regularly monitored following gene therapy. All patients experienced elevated AST or ALT, two experienced high prothrombin time, and one experienced elevated bilirubin; all of these patients were asymptomatic. Furthermore, one event of vomiting after infusion was reported in one patient. Significant improvements in CHOP INTEND scores were observed following therapy. This study describes the short-term outcomes and safety of onasemnogene abeparvovec, which is well tolerated and shows promise for early efficacy.