Acetazolamide Treatment Duration Shorter in Iatrogenic Pediatric Pseudotumor Cerebri

Acetazolamide treatment duration for pediatric patients with pseudotumor cerebri syndrome (PCS) may be highly variable and may be unrelated to puberty, according to research presented at the North American Neuro-Ophthalmology Society’s 2023 Annual Meeting held in Orlando, FL, from March 11 to March 16, 2023. Pediatric patients with iatrogenic PCS may require acetazolamide treatment for a relatively shorter duration than non-iatrogenic, the report suggests. 

The retrospective, longitudinal study reviewed the acetazolamide treatment duration in 61 patients (18 years and younger at presentation) diagnosed with PCS at a single pediatric neuro-ophthalmology clinic between 2011 and 2022. Researcher Yue Li, MD, analyzed age, gender, weight, puberty status, risk factors, findings on neuroimaging and methods and acetazolamide treatment duration. 

The study sample included 27 patients who were prepubertal (mean age, 6.3±2.8; 24 boys, 3 girls) and 30 pubertal (mean age, 15.5±1.6; 1 boy, 29 girls). The body mass indexes for patients who were prepubertal vs pubertal were 19.4±5.9 and 34.3±8.7 kg/m², respectively. 

The study shows that the interquartile range of months patients required acetazolamide treatment was 4.25-31.75 months in the prepubertal group compared with 3-36 months in the pubertal group. In the pubertal group, 14 patients were non-iatrogenic and received treatment for 10.25-53.75 months and the remaining 16 patients were iatrogenic and received treatment for 2.25-12 months. 

Further surgery (optic nerve sheath fenestration and/or shunt) was performed in 9 prepubertal patients and 2 pubertal patients. The most frequent cause of surgery was intolerance or poor response to a high dose of acetazolamide (8/9), the study notes. A total of 4 patients reached puberty during treatment, and were treated for an average of 31.8 (2-92) months before and 17.5 (6-29) months after puberty. 

“In our limited series, the duration of acetazolamide treatment for PCS was highly variable and unrelated to puberty,” according to the research. “Those who had iatrogenic PCS required treatment for a relatively shorter duration.”

Study limitations include its retrospective nature, single center design, and additional studies are required to determine which patients need a longer course of acetazolamide treatment.