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Treatment with oral or topical sirolimus in complex vascular anomalies in pediatrics. Experience in a third-level
C Fernández Oliveira1, C Martínez Roca1, M Gómez Tellado2
1Pharmacy Department. Complexo Hospitalario Universitario A Coruña (Spain).
Insights
Sirolimus shows effectiveness and safety for pediatric vascular anomalies, with high response rates in both oral and topical treatments. Further research is needed for optimal regimens and long-term effects.
Area of Science:
- Pediatric vascular anomalies
- Pharmacology
- Dermatology
Background:
- Sirolimus is used off-label for vascular anomalies.
- Evidence for its use in children is limited.
Purpose of the Study:
- To evaluate the efficacy and safety of oral and topical sirolimus in pediatric patients with vascular anomalies.
Main Methods:
- Retrospective observational study of 18 pediatric patients.
- Collected data included diagnosis, lesion location, sirolimus route/dosage, blood levels, duration, response, and toxicity.
Main Results:
- Oral sirolimus: 85.7% response rate, 57.1% mild adverse effects (dyslipidemia most common).
- Topical sirolimus: 72.7% response rate, 27.3% adverse effects (itching most common).
Conclusions:
- Sirolimus appears effective and safe for pediatric vascular anomalies.
- Further research needed for optimal dosing, duration, and long-term effects.
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.
