Abstract Details

Title All-Trans Retinoic Acid-Induced Pseudotumor Cerebri in Patients with Acute Promyelocytic Leukemia: A Review

Topic Neuro-ophthalmology/Neuro-otology

Presentation(s) P1 - Poster Session 1 (9:00 AM-5:00 PM)

Poster/Presentation
Number 439

Objective To examine how dosing of all-trans retinoic acid can be optimized to prevent morbidity from pseudotumor cerebri in patients with acute promyelocytic leukemia.

Background All-trans retinoic acid (ATRA), the most effective treatment for acute promyelocytic leukemia (APL), can induce remission and has improved survival rates up to 80-90%.^1,2 However, ATRA is associated with pseudotumor cerebri (PTC), limiting treatment dose and duration.³

Design/Methods Forty-five patients with APL who were treated with ATRA and developed subsequent PTC were selected via PubMed. Search terms included "all-trans retinoic acid" and one of the following: "papilledema,” "pseudotumor,” or "intracranial hypertension.” Variables recorded included patient age, sex, initial dose, time course of PTC development, cerebrospinal fluid studies, magnetic resonance imaging findings, total length of treatment with ATRA, and any changes in treatment course.

Results Of the 12 patients who continued ATRA despite developing PTC, six were treated with acetazolamide, four received corticosteroids, and four underwent lumbar puncture for symptomatic relief. Another 14 patients experienced an average dose reduction from 45.8 mg/m2/d to 25.5 ± 1.6 mg/m2/d with resolution of PTC and complete remission of APL. Seventeen patients discontinued ATRA entirely upon development or persistence of PTC symptoms.

Conclusions Though ATRA is revolutionary for targeted therapy of APL, patients with intolerable PTC must undergo dose adjustment. In this subset of patients, reducing the dose of ATRA from 45 mg/m²/d to 25 mg/m²/d was effective, as studies show both doses have similar pharmacokinetics and can induce remission of APL with resolution of PTC.^4,5 Additionally, PTC can be effectively managed with serial lumbar punctures, acetazolamide, corticosteroids, or topiramate.^6,7,8 Overall, dose adjustments and symptomatic control of PTC can allow for continued treatment of APL with ATRA.

Authors/Disclosures
Sahar Noorani, MD PRESENTER	Dr. Noorani has nothing to disclose.
	No disclosure on file
Melanie N. Truong-Le, DO (University of Mississippi Medical Center)	Dr. Truong-Le has nothing to disclose.

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