Catherine C Coombs1, Lisa M DeAngelis2, James H Feusner3, Jacob M Rowe4, Martin S Tallman5. 1. Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY. 2. Department of Neurology, Weill Cornell Medical College, New York, NY; Department of Neurology, Memorial Sloan Kettering Cancer Center, New York, NY. 3. Department of Pediatric Hematology/Oncology, Children's Hospital and Research Center Oakland, Oakland, CA. 4. Department of Hematology, Shaare Zedek Medical Center, Jerusalem, Israel. 5. Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY; Department of Medicine, Weill Cornell Medical College, New York, NY. Electronic address: tallmanm@mskcc.org.
Abstract
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.
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 APLpatients 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.
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