Abstract

Dual differentiation therapy with arsenic trioxide and tretinoin (all-trans-retinoic acid; ATRA) has demonstrated non-inferiority compared to chemotherapy for low and intermediate risk acute promyelocytic leukemia. As clinical experience grows with this regimen, less commonly reported toxicities may begin to surface. At our center, we have treated ten patients thus far with dual differentiation therapy; five (50%) have developed pseudotumor cerebri (PTC). One of these 5 patients was managed with temporary discontinuation of ATRA and initiation of acetazolamide. In the remaining 4 patients, topiramate was substituted after acetazolamide failure, in order to relieve symptoms and permit ATRA dose re-escalation. All patients treated with this approach are in complete remission. Given our experience with dual differentiation therapy, we recommend close monitoring for signs and symptoms of PTC. In patients with inadequate symptom control with ATRA dose reduction and acetazolamide, topiramate should be considered.

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