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

Nicholas Brodszki1, John-Kalle Länsberg, Michael Dictor

  • 1Department of Clinical Sciences, Section for Paediatrics, The BUT team, Lund University, Sweden.

Acta Paediatrica (Oslo, Norway : 1992)
|May 25, 2011
PubMed

Insights

This study shows that a combination therapy including low molecular weight heparin effectively treated paediatric Gorham-Stout syndrome (GSS). Both patients achieved symptom resolution and remained disease-free for over two years.

Area of Science:

  • Paediatric oncology
  • Vascular anomalies
  • Rare diseases

Background:

  • Gorham-Stout syndrome (GSS) is a rare, progressive lymphangiomatosis with limited treatment options.
  • Conventional therapies for GSS are often ineffective, necessitating novel therapeutic strategies.

Observation:

  • Two paediatric patients with biopsy-confirmed GSS were treated with a multimodal approach.
  • The treatment regimen included interferon-α-2b, low anticoagulant low molecular weight heparin (tafoxiparin), radiotherapy, and surgery.

Findings:

  • The combined therapy led to the resolution of acute symptoms in both patients.
  • Both children remained symptom-free for over two years post-treatment, indicating sustained efficacy.

Implications:

  • The addition of tafoxiparin to existing GSS treatment protocols demonstrates promising results in paediatric cases.
  • This therapeutic approach may offer a viable alternative for managing GSS when standard treatments fail.
Abstract

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