Abstract
Introduction Sirolimus has become the third leg in the treatment of
vascular anomalies, initially relegated as rescue therapy for refractory
lesions but lately recommended as first line. Our objective is to
determine when it is best to start sirolimus treatment. Methods A
retrospective review of patients with vascular anomalies treated with
sirolimus during a 9-year period was performed. The variables analyzed
included subtype of vascular anomaly, age at the start of treatment,
sirolimus dosage and levels, response and duration of treatment among
others. Results 129 patients were included and classified according to
ISSVA in: tumor (7), lymphatic malformation (60), venous malformation
(16), combined malformations (26), others (22). Overall response was
positive in 90.8%. Median duration of treatment was 5.5 months (0-110).
Response was not influenced by dosage and levels, neither by subtype of
vascular anomaly, location, extension or symptoms. However the lower the
age of starting sirolimus, the better the response, mainly under 5 years
of age (p=0.004). At 6 months 67% of patients had responded while at 12
months over 84% did. By age, patients under 5 years showed positive
response at a median time of 2 months, compared to 5 and 7 months from
older patients. Time until a positive response was not influenced by
subtype neither by severity. Conclusion Overall response to sirolimus
was good and most patients responded irrespective of their severity in
location, extension or symptoms. Patients under 5 years old respond
better and faster making our goal to attempt for an early treatment.