pediatric therapeutics

Lipid nanoparticles (LNPs) have established their position as nonviral vectors for gene therapy. Tremendous efforts have been made to modulate the properties of LNPs to unleash their full clinical potential. Among the strategies being pursued, the layer-by-layer (LbL) technique has gained considerable attention in the biomedical field. Illuminated by our previous work, here we investigate if the LbL approach could be used to modify the LNP cores formulated with three different ionizable lipids: DODMA, MC3, and DODAP...

Landiolol is an ultra-short-acting, selective β1-adrenergic receptor blocker that was originally approved in Japan for the treatment of intraoperative tachyarrhythmias. It has gained attention for its use in the management of tachyarrhythmias and perioperative tachycardia, especially atrial fibrillation for both cardiac and non-cardiac surgeries. It can be the ideal agent for heart rate control due to its high β1-selectivity, potent negative chronotropic effect, a limited negative inotropic potential, and an ultrashort elimination half-life (around 4 min); moreover, it may have a potential therapeutic effects for sepsis and pediatric patients...

Since the acceptability of a medicine can significantly impact therapeutic outcomes, this study aimed to determine and compare the preferences of children, parents, and healthcare professionals for the most commonly used pediatric oral medicine formulations (syrup, mini-tablets, oblong tablets, round tablets) addressing all pediatric age groups, 0-<18 years (y). This survey study employed sex-, age-, and participant group-adapted questionnaires for eight cohorts of participants, i.e., children 6-<12 y, adolescents 12-<18 y, parents of children in four age groups (0-<2 y, 2-<6 y, 6-<12 y, and 12-<18 y), nurses, and pediatricians...

Cannabinoid use has surged in the past decade, with a growing interest in expanding cannabidiol (CBD) and delta-9-tetrahydrocannabinol (THC) applications into special populations. Consequently, the increased use of CBD and THC raises the risk of drug-drug interactions (DDIs). Nevertheless, DDIs for cannabinoids, especially in special populations, remain inadequately investigated. While some clinical trials have explored DDIs between therapeutic drugs like antiepileptic drugs and CBD/THC, more potential interactions remain to be examined...

The mosaic form of Edwards syndrome affects 5% of all children with Edwards syndrome. The clinical phenotype is highly variable, ranging from the full spectrum of trisomy 18 to the normal phenotype. The purpose of this publication was to present the therapeutic process in an 18-month-old girl with the mosaic form of Edwards syndrome and hepatoblastoma, against the background of other cases of simultaneous occurrence of this syndrome and hepatoblastoma described so far. It appears that this particular group of patients with hepatoblastoma and Edwards syndrome can have good outcomes, provided they do not have life-threatening cardiac or other severe defects...

Cornelia de Lange syndrome (CdLS) is a complex genetic disorder with distinct facial features, growth limitations, and limb anomalies. Its broad clinical spectrum presents significant challenges in pediatric diagnosis and management. Due to cohesin complex mutations, the disorder's variable presentation requires extensive research to refine care and improve outcomes. This article provides a case series review of pediatric CdLS patients alongside a comprehensive literature review, exploring clinical variability and the relationship between genotypic changes and phenotypic outcomes...

Background : Pediatric odontogenic sinusitis (PODS) is a rare condition with limited research on its clinical features, diagnostic criteria, and treatment options. The current guidelines on pediatric rhinosinusitis do not mention a possible dental origin of the disease. This systematic review aims to summarize and analyze the existing literature on PODS, focusing on epidemiology, etiology, diagnostic tools, complications, treatment options, and outcomes. Methods : A systematic review was conducted following PRISMA reporting guidelines...

(1) Background : Dislocations of the trapeziometacarpal joint (TMC) are uncommon in children and adolescents. Only a few isolated cases are reported in the literature. Therapeutic guidance is minimal and inconclusive. (2) Methods : The authors present four patients treated for this unusual lesion. We evaluated the evolution according to treatment, age, patient activity, and quickDASH. Despite the clear limitation of the small number of patients, it is relevant to try to better understand this lesion and its evolution...

BACKGROUND: Nasal polyps (NPs) represent the end-stage manifestation of chronic rhinosinusitis (CRS), a relatively common pathological condition encountered in all ages of life. METHODOLOGY: The aim of our study was to evaluate the histological features and inflammatory cellular components of NPs in a retrospective cohort (143 cases) of pediatric, adult and elderly populations in order to discuss the possible morphological age-related differences statistically documented...

Viral infections have always been considered a threat to global health, with numerous outbreaks across time. Despite the relative recent experience with coronavirus-associated diseases such as severe acute respiratory syndrome (SARS) and Middle East respiratory syndrome (MERS), severe acute respiratory syndrome-2's (SARS-CoV-2) continuous evolution displays a different behavior. With a tropism for both respiratory and digestive mucosa, coronavirus disease 2019 (COVID-19) and inflammatory bowel disease (IBD) seem to share a particular common background...

Wilms tumor (WT), the most prevalent type of renal cancer in children, exhibits overall survival rates exceeding 90%. However, chemotherapy resistance, which occurs in approximately 10% of WT cases, is a major challenge for the treatment of WT, particularly for advanced-stage patients. In this study, we aimed to discover potential mutation markers and drug targets associated with chemotherapy resistance in advanced-stage WT. We performed exome sequencing to detect somatic mutations and molecular targets in 43 WT samples, comprising 26 advanced-stage WTs, of which 7 cases were chemotherapy-resistant...

Children undergoing antineoplastic treatment often present severe side effects due to the dosage and duration of treatments, with oral mucositis emerging as one of the most prevalent and painful inflammatory conditions. There is a growing body of evidence on therapeutic interventions such as cryotherapy, low-level laser therapy, and natural compounds for this condition. The aim of this systematic review was to identify and compare therapies for the management of cancer treatment-induced oral mucositis in pediatric patients...

BACKGROUND: Osteochondritis dissecans (OCD) is a joint disorder predominantly affecting the knee, elbow, and ankle of children and adolescents. This comprehensive review delves into the epidemiology, etiology, clinical manifestations, diagnostic approaches, and treatment of OCD. RESULTS: The most common cause of OCD is repetitive microtrauma, typically associated with sports activities, alongside other significant factors such as genetic predisposition, ischemia, and obesity...

This case report sheds light on the management of skeletal deformity in a young child with X-linked hypophosphatemia (XLH), emphasizing the significance of a timely orthotic intervention alongside pharmacological treatment, which is a strategy not frequently highlighted in the XLH literature. The patient, a 2-year-and-7-month-old female, presented with classic XLH symptoms, including short stature, pronounced genu varum, and hypophosphatemia, with deformities observed in both the coronal and sagittal planes of the femur and tibia...

BACKGROUND: Cerebral palsy (CP) is one of the primary causes of physical disabilities in children that affects posture and movement. Upper-extremity (UE) function is frequently impaired, which may result in activity and participation limitations in people with CP. The use of kinesiotape (KT) has increased in the treatment of CP for various purposes. The aim of this systematic review was to assess the efficacy of KT for improving UE function in children and adolescents with CP. METHODS: The literature search was carried out in PubMed, Cochrane, PEDro, Web of Science and SCOPUS databases...

The "red code" (RC) represents the highest level of emergency in the emergency department (ED). This study retrospectively analyzed RCs in the Regina Margherita Children's Hospital ED, a regional referral center in north Italy, between 1 July 2020 and 30 June 2023. The aim was to describe RC characteristics and to identify significant correlations between presenting complaints and clinical management. The study includes 934 RCs (0.9% of overall ED admissions); 64% were assigned based on the Pediatric Assessment Triangle alteration...

Childhood glaucoma, a significant cause of global blindness, represents a heterogeneous group of disorders categorized into primary or secondary forms. Primary childhood glaucoma stands as the most prevalent subtype, comprising primary congenital glaucoma (PCG) and juvenile open-angle glaucoma (JOAG). Presently, multiple genes are implicated in inherited forms of primary childhood glaucoma. This comprehensive review delves into genetic investigations into primary childhood glaucoma, with a focus on identifying causative genes, understanding their inheritance patterns, exploring essential biological pathways in disease pathogenesis, and utilizing animal models to study these mechanisms...

We conducted a two-year retrospective evaluation of infants aged under two years with a confirmed, clinical, or suspected diagnosis of scabies in a healthcare center in Alicante (Spain) to determine possible factors associated with diagnostic delay and poor treatment response. We collected epidemiological, clinical, diagnostic, and treatment variables. After describing our findings as mean values and percentages, we compared categorical variables using the Student's t -test and the Mann-Whitney U test, and we compared continuous variables with the Chi2 test and Pearson's correlation coefficient...

OBJECTIVE: This paper reviews the current literature about epidemiology, etiologies, diagnosis, and management of pediatric bilateral vocal fold paralysis (PBVFP). METHODS: According to PRISMA statements, a narrative review of the current literature was conducted through the PubMed, Scopus, and Cochrane Library databases about the epidemiology, etiologies, diagnosis, and management of PBVFP. RESULTS: PBVCP is the second most common congenital laryngeal anomaly in the pediatric population, accounting for 10% to 20% of pediatric laryngeal conditions...

INTRODUCTION: Pediatric cystic kidney disease (CyKD) includes conditions characterized by renal cysts. Despite extensive research in this field, there are no reliable genetics or other biomarkers to estimate the phenotypic consequences. Therefore, CyKD in children heavily relies on clinical and diagnostic testing to predict the long-term outcomes. AIM: A retrospective study aimed to provide a concise overview of this condition and analyze real-life data from a single-center pediatric CyKD cohort followed during a 12-year period...