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Brief Report
September 17, 2020

Association of Adrenal Insufficiency With Pediatric Pseudotumor Cerebri Syndrome

Author Affiliations
  • 1Department of Ophthalmology, Baylor College of Medicine, Houston, Texas
  • 2Abrahamson Pediatric Eye Institute, Division of Pediatric Ophthalmology, Department of Neurology, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
  • 3Department of Ophthalmology, University of Cincinnati, Cincinnati, Ohio
  • 4Section of Pediatric Diabetes and Endocrinology, Department of Pediatrics, Baylor College of Medicine, Texas Children’s Hospital, Houston
JAMA Ophthalmol. 2020;138(11):1187-1191. doi:10.1001/jamaophthalmol.2020.3322
Key Points

Questions  Is there an association between adrenal insufficiency and pediatric pseudotumor cerebri syndrome?

Findings  In this case series, 5 children who were prepubertal, were nonobese, and had pseudotumor cerebri syndrome and optic nerve edema were found to have abnormal cortisol function consistent with adrenal insufficiency. Initiation of physiologic hydrocortisone to treat adrenal insufficiency resulted in the resolution of clinical pseudotumor cerebri syndrome symptoms and optic nerve edema and a shorter duration of conventional medical therapy.

Meaning  Pseudotumor cerebri syndrome is associated with hypocortisolism in the prepubertal pediatric population; identifying risk factors for adrenal insufficiency (exogenous glucocorticoid use), obtaining cortisol testing in the initial workup, and treatment based on causative mechanism may lead to improved outcomes.


Importance  Pediatric pseudotumor cerebri syndrome pathophysiology is complex and not well delineated. Therefore, it is important to identify potential contributors or targets underlying the primary pathogenesis for its development.

Objective  To report cases highlighting the association of pediatric pseudotumor cerebri syndrome with adrenal insufficiency.

Design, Setting, and Participants  This noncontrolled, observational case series included pediatric patients diagnosed with pseudotumor cerebri syndrome and adrenal insufficiency at an urban academic children’s hospital in Houston, Texas, from June 2015 to October 2019.

Main Outcomes and Measures  Monitoring optic nerve edema by clinical examination, fundus photography, and optical coherence topography images of the optic nerve.

Results  Data were collected from 5 pediatric patients (age range, 5-10 years) diagnosed with pseudotumor cerebri syndrome and adrenal insufficiency. One was a girl; all were White and prepubertal. Three patients had unrecognized glucocorticoid exposure. All patients had bilateral optic nerve edema that was initially treated with acetazolamide or topiramate, but cortisol functional testing by either 8 am cortisol or cosyntropin stimulation tests revealed a diagnosis of central adrenal insufficiency. Treatment with physiological doses of hydrocortisone resulted in resolution of optic nerve edema and clinical symptoms of pseudotumor cerebri syndrome, as well as a shorter time receiving medical therapy.

Conclusions and Relevance  In this case series, adrenal insufficiency was associated with both primary and secondary prepubertal pediatric pseudotumor cerebri syndrome. As a potential target specific to causative mechanism, physiologic hydrocortisone therapy resolved the condition. To date, there remains a global unawareness among clinicians about the suppressive outcome that glucocorticoids may have on the developing hypothalamic-pituitary-adrenal axis, resulting in adrenal insufficiency and so-called episodic pseudotumor cerebri syndrome in young children. Ophthalmologists and pediatric subspecialists should implement cortisol testing via either 8 am cortisol or cosyntropin stimulation tests at initial evaluation of all children with pseudotumor cerebri syndrome and risk factors for adrenal insufficiency, no predisposing causes, or nonresponse to conventional treatment. Further management and treatment should be in combination with ophthalmology and endocrine services.

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