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Octreotide in Hennekam syndrome-associated intestinal lymphangiectasia
Siham Al Sinani1, Yusria Al Rawahi, Hamed Abdoon
1Child Health Department, Sultan Qaboos University Hospital, Muscat 123, Sultanate of Oman. siham_ss@hotmail.com
Insights
Octreotide effectively reduced protein loss in children with Hennekam syndrome (HS) and protein-losing enteropathy (PLE). This treatment minimized hypoalbuminemia, reducing the need for albumin infusions in these patients.
Area of Science:
- Pediatric Gastroenterology
- Rare Genetic Syndromes
- Clinical Pharmacology
Background:
- Protein-losing enteropathy (PLE) in children can stem from primary intestinal lymphangiectasia (PIL).
- Hennekam syndrome (HS) is a rare genetic disorder associated with PIL.
- Standard treatment for PIL involves dietary modifications and albumin infusions, with limited evidence for pharmacologic agents.
Observation:
- This study reports on two pediatric patients diagnosed with Hennekam syndrome (HS) and concurrent protein-losing enteropathy (PLE).
- Octreotide was administered to these patients to manage intestinal protein loss.
- The treatment aimed to mitigate hypoalbuminemia and reduce reliance on supportive albumin infusions.
Findings:
- Both patients exhibited a positive response to octreotide therapy, demonstrating a significant decrease in intestinal protein loss.
- One patient achieved acceptable serum albumin levels, eliminating the need for further albumin infusions.
- The second patient showed a more pronounced response, with serum albumin levels nearing normal and cessation of albumin infusions.
Implications:
- These findings support the use of octreotide as a therapeutic option for managing protein-losing enteropathy in primary intestinal lymphangiectasia.
- The study suggests that octreotide should be considered for patients with PIL secondary to Hennekam syndrome.
- This case report represents the first documented use of octreotide in Hennekam syndrome-associated primary intestinal lymphangiectasia.
Abstract:
A number of disorders have been described to cause protein losing enteropathy (PLE) in children. Primary intestinal lymphangiectasia (PIL) is one mechanism leading to PLE. Few syndromes are associated with PIL; Hennekam syndrome (HS) is one of them. The principal treatment for PIL is a high protein, low fat diet with medium chain triglycerides supplementation. Supportive therapy includes albumin infusion. Few publications have supported the use of octreotide to diminish protein loss and minimize hypoalbuminemia seen in PIL. There are no publications on the treatment of PIL with octreotide in patients with HS. We report two children with HS and PLE in which we used octreotide to decrease intestinal protein loss. In one patient, octreotide increased serum albumin to an acceptable level without further need for albumin infusions. The other patient responded more dramatically with near normal serum albumin levels and cessation of albumin infusions. In achieving a good response to octreotide in both patients, we add to the publications supporting the use of octreotide in PIL and suggest that octreotide should be tried in patients with PIL secondary to HS. To the best of our knowledge, this is the first case report on the use of octreotide in HS-associated PIL.
