Safety and outcome of children, adolescents and young adults participating in phase I/II clinical oncology trials: a 9-year center experience.

Introduction: Enrolling children with cancer in early phase trials is crucial to access innovative treatments, contributing to advancing pediatric oncology research and providing tailored therapeutic options. Our objective is to analyze the impact of these trials on patient outcomes and safety, and to examine the evolution and feasibility of trials in pediatric cancer over the past decade. Methods: All patients recruited in pediatric anticancer phase I/II clinical trials from January 2014 to December 2022 were included. Clinical records and trial protocols were analyzed. Results: A total of 215 patients (median age 11.2 years, range 1-29.5) were included in 52 trials (258 inclusions). Patients with extracranial solid tumors (67%), central nervous system (CNS) tumors (24%), and leukemia (9%) were included. The most common investigational drugs were small molecules (28.3%) and antibodies (20.5%). Serious adverse events were experienced by 41% of patients, 4.4% discontinued treatment because of toxicity and two had toxic deaths. Median event-free survival was 3.7 months (95%CI: 2.8-4.5), longer in phase II trials than in phase I (2 vs. 6.3 months; p ≤ 0.001). Median overall survival was 12 months (95%CI: 9-15), higher in target-specific vs. non-target-specific trials (14 vs. 6 months; p ≤ 0.001). Discussion: A significant and increasing number of patients have been included in early clinical trials, suggesting that both oncologists and families consider it valuable to be referred to specialized Units to access new therapies. Moreover, our data suggests that participation in early clinical trials, although not without potential toxicities, might have a positive impact on individual outcomes.

[Controlling carbohydrate content in antiepileptic medications for children on a ketogenic diet]. / Control de carbohidratos en los medicamentos antiepilépticos para niños con dieta cetogénica.

Introduction: Introduction: patients who follow a ketogenic diet for the control of epileptic seizures must carry out a strict control of carbohydrates from the foods they eat and the medicines they are prescribed. In the initiation of a ketogenic diet and when a doctor prescribes a new medication, it is necessary to select the most appropriate pharmaceutical form so that the supply of excipients in the form of carbohydrates from the drugs is minimized. Objectives: the goal of the present paper was to compile a list of carbohydrate and caloric contents in antiepileptic drugs commonly used in pediatric neurology. Methods: in each medication included in the list, the content of excipients considered carbohydrates and derivatives that could influence the patient's ketosis was reviewed. The caloric content from carbohydrates and polyols in each medication was calculated. Results: the table provides the total carbohydrate and caloric content for antiepileptic medications in pediatric patients consuming the ketogenic diet. Conclusions: this table is intended to be a useful tool to help clinicians select a pharmaceutical form that is less likely to affect the ketogenic diet. Additionally, knowing the carbohydrate content of a new medication will allow adjustment of the diet to maintain ketosis.

Introducción: Introducción: los pacientes que siguen una dieta cetogénica para el control de las crisis epilépticas deben llevar a cabo un estricto control de los hidratos de carbono procedentes tanto de los alimentos que consumen como de los medicamentos que tienen pautados. Tanto en la instauración de la dieta cetogénica como cuando el médico prescribe un medicamento nuevo es necesario el ajuste de la medicación a las formas farmacéuticas más adecuadas, de forma que se minimice el aporte de excipientes en forma de hidratos de carbono de los medicamentos. Objetivos: el objetivo que planteamos en el presente trabajo fue elaborar un listado de medicamentos antiepilépticos de utilización habitual en neurología pediátrica que incluyera información sobre su contenido calórico en forma de hidratos de carbono para la administración a pacientes con dieta cetogénica. Métodos: en cada medicamento incluido en el listado se revisó el contenido en excipientes considerados hidratos de carbono y derivados que pudieran influir en la cetosis del paciente. Se calculó el contenido calórico procedente de los hidratos de carbono y polioles de cada medicamento. Resultados: elaboración de una tabla para consulta del contenido calórico de distintos medicamentos antiepilépticos utilizados en neurología pediátrica para pacientes con dieta cetogénica. Conclusiones: la tabla publicada pretende ser una herramienta útil que permita la consulta del contenido calórico de distintos medicamentos antiepilépticos y la selección del medicamento idóneo que menos afecte a la dieta cetogénica. Con el contenido calórico en carbohidratos de las medicaciones pautadas se podrán realizar los ajustes necesarios en la dieta para mantener la cetosis necesaria.

Value of Thyroid Peroxidase Antibodies in Neuroimmune Diseases: Analysis of Interference During Treatment with Intravenous Immunoglobulins.

OBJECTIVE: The absence of specific markers can make the diagnosis of neuroimmune disorders difficult, making other biomarkers such as thyroid peroxidase antibodies (TPO-Abs) more relevant. Laboratory tests are susceptible to interference, especially those tests performed using immunoassay techniques. The effect of treatment with human intravenous immunoglobulin (IVIG) on the results of TPO-Abs assays has not been previously characterized. MATERIALS AND METHODS: We analyzed TPO-Abs levels in 170 children monitored in the neuroimmune disease department of a tertiary hospital. We analyzed the characteristics of patients with increased TPO-Abs values and compared their progress with and without treatment. RESULTS: We found that 97% of patients with elevated TPO-Abs had received IVIG. After withdrawal from IVIG, a mean TPO-Abs decrease of 62.5% at 1 month was observed. The IVIG drug preparation was found to contain 1176 U/mL of TPO-Abs. An interferogram confirmed interference. CONCLUSION: It is advisable to measure levels of TPO-Abs before starting immunotherapy and remain vigilant regarding possible interference in the event of unsubstantiated elevations of this analyte.