Patients with Congenital Low-Flow Vascular Malformation Treated with Low Dose Sirolimus.
Veroniek E M HarbersGerard A P J M RongenCarine J M van der VleutenBas H VerhoevenPeter C J de LaatChantal M A M van der HorstWillemijn M KleinLeo J Schultze KoolD Maroeska W M Te LooPublished in: Advances in therapy (2021)
Low dose sirolimus showed a high efficacy in patients with therapy-resistant and low-flow malformation, with a lower incidence of serious adverse events. At the same time a new adverse event, namely menstrual cycle disturbance, was observed in young adolescents, indicating the need for caution when sirolimus is given. This is extremely relevant to patients with low-flow vascular malformation, who are likely to require lifelong treatment for their condition.