Octreotide: A Therapeutic Option for Pediatric Pseudotumor Cerebri Syndrome?

Pseudotumor cerebri syndrome (PTCS) is a condition of unclear etiology, characterized by increased intracranial pressure (ICP) with normal cerebrospinal fluid contents and no associated anomalies evident in the brain parenchyma. It may be associated with concomitant risk factors, including obesity, endocrine disorders, chronic medical illness, and medications. Clinical signs and symptoms of PTCS in children are heterogeneous, but they usually include headache, papilledema at the funduscopic examination, and visual disturbances (e.g., diplopia and/or vision loss). Irreversible blindness, due to persistently raised ICP, has been also reported in up to 10% of cases. Treatment goal of PTCS is the relief of symptoms and the preservation of visual function, by reducing ICP. Diuretics (e.g., acetazolamide, furosemide) could be regarded as the first-line treatments in PTCS; however, up to 30% of patients do not have a clinical response, making important the identification of alternative (conservative) treatment's choices to avoid the PTCS-related complications (e.g., permanent visual loss) and the necessity of invasive surgical (cerebrospinal fluid diverting) procedures. In this context, octreotide (i.e., a synthetic somatostatin analog) has been previously proposed in some studies as a potential (etiologically targeted) treatment able to revert to PTCS-related manifestations. We hereby discuss the possible role of octreotide in PTCS and hypothesize the possible mechanism of action of this drug in reducing ICP.