Treatment with oral or topical sirolimus in complex vascular anomalies in pediatrics. Experience in a third-level hospital. / Tratamiento con sirolimus oral o tópico en anomalías vasculares complejas en pediatría. Experiencia en un hospital terciario.

OBJECTIVE: The use of sirolimus in vascular anomalies is a special indication not authorized in its data sheet. The objective of this study was to increase the evidence of oral or topical use of sirolimus for this indication in the pediatric population. MATERIALS AND METHODS: An observational, retrospective study of patients under 18 years of age treated with oral or topical sirolimus for vascular anomalies was carried out. Diagnosis and location of lesions, administration route and dosage of sirolimus, blood levels of sirolimus in patients who received oral treatment, treatment duration, response, and toxicity were collected. RESULTS: 18 patients - 7 with oral treatment and 11 with topical treatment - were included. With oral sirolimus, the overall response rate was 85.7%. Sirolimus was discontinued in 2 cases - as a result of full resolution and progression. 57.1% of patients had adverse effects, most of which were mild. Dyslipidemia was the most frequent adverse effect. Blood levels were monitored in all patients for dose adjustment purposes. With topical treatment, the overall response rate was 72.7%. Sirolimus was discontinued in 3 cases -due to progression in 2 cases and to stability in 1. 27.3% of patients had adverse effects, with itching standing out as the most frequent one. CONCLUSIONS: The favorable results of sirolimus treatment in our patients seem to confirm its effectiveness and safety in vascular anomalies, which make it stand as a therapeutic option in pediatric patients. However, further research is required to establish the optimal treatment regimen, treatment duration, and potential long-term adverse effects.

OBJETIVO: El uso de sirolimus en anomalías vasculares es una indicación especial no autorizada en ficha técnica. El objetivo de este estudio es incrementar la evidencia del empleo por vía oral o tópica de sirolimus en esta indicación en población pediátrica. METODO: Estudio observacional retrospectivo de pacientes menores de 18 años tratados con sirolimus oral o tópico para anomalías vasculares recogiendo: diagnóstico y ubicación de lesiones, forma de administración y dosificación de sirolimus, niveles sanguíneos de fármaco en los pacientes con tratamiento oral, duración del tratamiento, respuesta y toxicidad. RESULTADOS: Se incluyeron 18 pacientes (7 con tratamiento oral y 11 tópico). Con sirolimus oral, la tasa de respuesta global fue 85,7%. Se interrumpió sirolimus en 2 casos: por resolución completa y por progresión. El 57,1% experimentó algún efecto adverso, en su mayoría leves; siendo la dislipemia el efecto adverso más frecuente. La monitorización de niveles sanguíneos fue empleada en todos los pacientes para el ajuste de dosis. Con el tratamiento tópico, la tasa de respuesta global fue 72,7%. Se interrumpió sirolimus en 3 casos: progresión en 2 casos y estabilidad en 1. El 27,3% experimentó algún efecto adverso, siendo el prurito el más frecuente. CONCLUSIONES: Los resultados favorables del tratamiento con sirolimus en nuestros pacientes parecen confirmar la efectividad y seguridad del fármaco en anomalías vasculares y lo posicionan como una opción terapéutica en pacientes pediátricos. Aun así, parece necesaria mayor investigación que trate de aclarar, entre otros, el régimen óptimo del tratamiento, la duración del mismo y los potenciales efectos adversos a largo plazo.

[The influence of antiretroviral-resistance tests on treatment effectiveness in patients with HIV and virological failure]. / Influencia de las pruebas de resistencias a antirretrovirales sobre la efectividad del tratamiento en pacientes con infección por VIH y fracaso virológico.

OBJECTIVE: The objective of this study is to determine the influence of antiretroviral-resistance tests on the suppression of HIV (< 400 copies/mL) in patients with virological failure who require an alternative antiretroviral treatment. METHOD: A retrospective observational study on cohorts of adult patients. Two groups were defined: cases in which the prescription of antiretrovirals was based on resistance tests (group A), and controls in which no such test was performed (group B). Each group was divided into two sub-groups according to the number of changes in treatment: first treatment change (A1 and B1); a subsequent change (A2 and B2). The main variable was defined as the proportion of patients with negative viral load (< 400 copies/mL) at the third month of treatment; secondary variables were the proportion of patients with negative viral load at the sixth month and an average variation in the CD4 level at the third and sixth months after this change. RESULTS: A total of 152 patients were included in this study, 59 in group A and 93 in group B (control). No differences were found in the stage of the disease at the time of administering an alternative treatment. 59.3% of the patients in group A and 47.3% of the patients in group B had suppressed the HIV viral load at the third month, although this difference was not statistically significant (p = 0.149). No statistically significant differences were found in the secondary variables. CONCLUSIONS: The use of antiretroviral-resistance tests increased effectiveness in the response to the selected antiretroviral treatment in the study group, although we did not obtain significant differences for the group of patients in which these tests were not performed.