Role of sirolimus in management of complex vascular anomaliesin children: Complex venous, lymphatic malformations, Vascular tumours- kaposiform haemangioendotheliomas, Generalised lymphatic anomalies and PIKC3A related overgrowth syndromes.

Sydney Children's Hospital, Randwick0 个研究点目标入组 30 人开始时间: 2016年7月15日最近更新: 2020年1月13日

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•Inclusion criteria include children in the age group of zero to eighteen years who have complex vascular anomalies. Examples include children with the following:
•1. Venous Malformations with localised intravascular coagulopathy or severe pain failing to respond to sclerotherapy and /or surgery;
•2. Lymphatic Malformations which compromise the airway or vision or are causing a low albumin level;
•3. Newly diagnosed Kaposiform haemangioendothelioma/tufted angioma: in view of the promising data from the US on the use of sirolimus as initial treatment for KHE (1,2,5), sirolimus will be given to newly diagnosed patients and the results compared to our historical controls, namely children who had received vincristine as initial therapy.
•4. Relapsed Kaposiform haemangioendothelioma: children who have previously been successfully treated with vincristine therapy and then have a recurrence of their tumour will be treated with sirolimus;
•5. Generalised Lymphatic Anomalies with bone erosions, chylous leak or infiltration of lungs and gastrointestinal tract;
•6. localised lymphatic anomalies not amenable to an operation;
•7. Children with various overgrowth syndromes who have benign tumours, asymmetry, pain and coagulopathy that have failed usual therapeutic options and are causing significant symptoms

发起方

Sydney Children's Hospital, Randwick