Pseudotumor Cerebri in Acute Promyelocytic Leukemia Patients on Intergroup Protocol 0129: Clinical Description and Recommendations for New Diagnostic Criteria

Background Multiple randomized trials have demonstrated a benefit for all-trans retinoic acid (ATRA) in patients with acute promyelocytic leukemia (APL). Pseudotumor cerebri (PTC) is an infrequently reported adverse effect of ATRA. Methods We examined the incidence, clinical course, and outcomes of patients with APL treated on Intergroup Protocol 0129 (I0129) who developed PTC. This trial evaluated the role of ATRA alone during induction and/or as maintenance therapy. Results Of the patients on trial, 240 received ATRA during induction, maintenance, or both; 8 had a clinical suspicion for PTC. Upon review of individual cases, this was felt to be "probable" in 4 patients, "possible" in 1 and "unlikely" in 3 due to lack of diagnostic criteria or presence of a more likely alternate diagnosis. Conclusions "Probable" PTC occurred in 1.7% of patients who received ATRA during induction and/or maintenance therapy. In agreement with previous reports, the incidence of PTC in APL patients receiving ATRA was higher in the pediatric population. Here, we discuss the method for diagnosing PTC in the setting of ATRA therapy and management strategies.