The paediatric oncologist and the evolving medical management of complex vascular anomalies: An institutional experience

Abstract

Background: Complex vascular anomalies in children are amenable to medical therapy that can result in complete resolution or improvement in cosmesis and function or serve as a conduit to definitive surgery.

Aim: This study aimed to retrospectively review the management and outcomes of children with complex vascular anomalies.

Setting: The study was conducted at a haematology/oncology unit based out of a paediatric hospital in the Western Cape.

Methods: All patients with biopsy-proven lesions and those diagnosed on magnetic resonance imaging (MRI) from 01 January 2005 to September 2021 were considered eligible for inclusion.

Results: Twenty-five patients presented with a variety of capillary, venous and lymphatic malformations. There were 11 males and 14 females, with a median age of 35 months at presentation (range: 0–156 months). Patients presented with a mass or compartmental enlargement, cutaneous stigmata or bleeding. Hepatic haemangioendotheliomas, kaposiform haemangioendotheliomas and capillary haemangiomas were most common. Kassabach-Merritt syndrome was present in 5/25 (20%) patients. Prednisone, propranolol and vincristine were the most commonly employed first-line medical treatments (15/21; 47.6%). Twelve patients received sirolimus, (11/21; 52%), four as single agent first-line therapy and eight as combination therapy, complicated by transient hyperlipidaemia in only one patient. All but one patient survived: 10 are disease free and 12 are alive with disease. Two patients with Gorham’s disease are maintained on long-term low-dose Sirolimus.

Conclusion: The medical management of complex vascular anomalies yields good results in children. Sirolimus is well tolerated with few manageable side effects with cost being the only prohibitive factor to its broader application.