A novel treatment approach for paediatric Gorham-Stout syndrome with chylothorax.

Aim: To expand the treatment options in paediatric Gorham-Stout syndrome (GSS) when conventional therapy is ineffective.

Method: Two children with biopsy confirmed GSS, a rare disorder with progressive lymphangiomatosis, were treated with a combination of interferon-α-2b, low anticoagulant, low molecular weight heparin, radiotherapy and surgery.

Results: The combined therapy resolved the symptoms in the acute phase, and both patients have since been free of symptoms for >2 years.