Sirolimus 1% Topico en Malformaciones Vasculares

Published in Dermatology

Journal Scan / Research · February 11, 2020

Treatment of Superficial Vascular Anomalies With Topical Sirolimus

Pediatric Dermatology

TAKE-HOME MESSAGE

• The authors performed a retrospective review of 18 children and young adults with superficial vascular anomalies treated with topical sirolimus 1%. Half of the patients used a compounded ointment or cream, while the other half applied the oral solution topically. Overall, 50% of patients had marked improvement in one or more categories, with blebs and lymphatic exudate the most common symptoms to improve. Minor side effects, including discomfort with application and xerosis, were seen in those patients applying the oral solution topically but not the compounded form.

• Topical sirolimus, an mTOR inhibitor, may be effective in treating superficial vascular anomalies before considering more invasive treatments. Prospective studies are warranted to clarify the degree of improvement and determine the optimal dosage, frequency, and formulation.

– Margaret Hammond, MD

Written by

 

 

Sarah L Chamlin MD

This group treated 18 patients with vascular lesions with topical sirolimus. The included patients mostly had complex combined lesions and capillary lymphatic venous malformations with cutaneous involvement. Treatment indications included bleeding, blebs, drainage, and disfigurement. The mean length of treatment was 10.2 months, and most patients applied sirolimus 1% twice daily. Side effects were minor and tolerable. Notably, the blebs and exudate were the most common findings that improved.

Oral sirolimus has been shown effective to improve the symptoms and size of large and symptomatic vascular malformations, primarily those with lymphatic involvement. Applying topical sirolimus for cutaneous symptomatology makes sense, and the authors demonstrated some improvement in all of their patients, 50% with marked improvement. This case series and others convinces me to prescribe this more frequently. Despite this intention, the challenge remains insurance coverage for the compounded cream or oral solution that is applied topically. The cost of this medication is highly variable. The out-of-pocket cost of a 60-mL bottle of Rapamune is approximately $1800! Working with a regional or national compounding pharmacy that offers lower out-of-pocket pricing may be the best alternative until insurance coverage is possible.

Abstract

BACKGROUND

Systemic sirolimus (rapamycin) has recently been found effective in treating complex vascular anomalies by reducing the size and associated complications. Many vascular anomalies have a cutaneous component, and thus, we sought to determine whether topical administration of sirolimus may be an effective therapy, as data on the use of topical sirolimus are limited.

OBJECTIVE

We reviewed the efficacy and tolerability of topical formulations of sirolimus in the treatment of various simple and combined vascular malformations and tumors.

METHODS

Eighteen patients with any vascular anomaly treated exclusively with topical sirolimus were retrospectively reviewed.

RESULTS

Eleven patients had combined venous lymphatic malformations, three had tufted angiomas, two had a lymphatic malformation, one had a venous malformation, and one had a verrucous venous malformation. All (100%) patients reported some degree of improvement and 50% of patients reported marked improvement in one or more symptoms, most commonly blebs and lymphatic drainage, and bleeding.

LIMITATIONS

The retrospective nature, small number of patients, and differences in topical preparations limit the broad application of the results.

CONCLUSION

Topical sirolimus appears to be a safe and useful non-invasive therapy that is well-tolerated in the treatment of the cutaneous portion of a variety of vascular anomalies.

Pediatric Dermatology

Treatment of Superficial Vascular Anomalies With Topical Sirolimus: A Multicenter Case Series

Pediatr Dermatol 2020 Jan 19;[EPub Ahead of Print], M Dodds, M Tollefson, L Castelo-Soccio, MC Garzon, M Hogeling, K Hook, C Boull, S Maguiness 

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Benjamin Hidalgo-Matlock
Skin Care Physicians of Costa Rica

Clinica Victoria en San Pedro: 4000-1054
Momentum Escazu: 2101-9574

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