Sophie Le Sage1, Michèle David2, Josée Dubois3, Julie Powell4, Catherine C McCuaig4, Yves Théorêt5,6, Niina Kleiber7,6. 1. Faculty of Medicine, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montréal, QC, Canada. 2. Division of Hematology-Oncology, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montréal, QC, Canada. 3. Department of Radiology, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montréal, QC, Canada. 4. Division of Dermatology, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montréal, QC, Canada. 5. Department of Pharmacology, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montréal, QC, Canada. 6. Clinical Pharmacology Unit, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montréal, QC, Canada. 7. Department of General Pediatrics, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Montréal, QC, Canada.
Abstract
BACKGROUND/ OBJECTIVES: Efficacy of topical sirolimus has recently been described in lymphatic anomalies but not in other types of vascular anomalies. To our knowledge, systemic absorption of topical sirolimus in these lesions has not yet been reported. The objective was to evaluate the efficacy, tolerance, and absorption of topical sirolimus 0.1% with different types of vascular anomalies in children. METHODS: Sirolimus 0.1% was applied on cutaneous vascular anomalies in six children aged 2-17. These anomalies consisted of three extratruncular micro- and macrocystic lymphatic malformations and one each verrucous venous malformation, truncular lymphatic malformation with angiokeratomas, and infantile hemangioma. Sirolimus blood levels were measured after 1 week, 1 month, and 3 months. RESULTS: A rapid decrease in the size of superficial lymphatic malformations in three of six patients and a significant decrease in discharge from oozing lesions were observed. Response occurred in less than 3 months. The truncular lymphatic malformation, verrucous venous malformation, and infantile hemangioma did not respond to topical sirolimus. Sirolimus levels were undetectable. Adverse effects were limited to local irritation. CONCLUSIONS: Topical sirolimus 0.1% is a useful treatment for cutaneous manifestations of extratruncular lymphatic malformations. The only adverse effect is local irritation. No systemic effects are expected, because blood levels are clinically insignificant.
BACKGROUND/ OBJECTIVES: Efficacy of topical sirolimus has recently been described in lymphatic anomalies but not in other types of vascular anomalies. To our knowledge, systemic absorption of topical sirolimus in these lesions has not yet been reported. The objective was to evaluate the efficacy, tolerance, and absorption of topical sirolimus 0.1% with different types of vascular anomalies in children. METHODS:Sirolimus 0.1% was applied on cutaneous vascular anomalies in six children aged 2-17. These anomalies consisted of three extratruncular micro- and macrocystic lymphatic malformations and one each verrucous venous malformation, truncular lymphatic malformation with angiokeratomas, and infantile hemangioma. Sirolimus blood levels were measured after 1 week, 1 month, and 3 months. RESULTS: A rapid decrease in the size of superficial lymphatic malformations in three of six patients and a significant decrease in discharge from oozing lesions were observed. Response occurred in less than 3 months. The truncular lymphatic malformation, verrucous venous malformation, and infantile hemangioma did not respond to topical sirolimus. Sirolimus levels were undetectable. Adverse effects were limited to local irritation. CONCLUSIONS: Topical sirolimus 0.1% is a useful treatment for cutaneous manifestations of extratruncular lymphatic malformations. The only adverse effect is local irritation. No systemic effects are expected, because blood levels are clinically insignificant.
Authors: Laura Macca; Domenica Altavilla; Luca Di Bartolomeo; Natasha Irrera; Francesco Borgia; Federica Li Pomi; Federico Vaccaro; Violetta Squadrito; Francesco Squadrito; Mario Vaccaro Journal: Front Pharmacol Date: 2022-05-26 Impact factor: 5.988