Treatment of central nervous system relapse in PLZF::RARA-positive acute promyelocytic leukemia by venetoclax combined with arubicin, cytarabine and intrathecal therapy: a case report

Abstract

Abstract Objectives Patients suffering from refractory acute promyelocytic leukemia with central nervous system relapse often have a poor prognosis. Among these patients, those with PLZF::RARA rearrangement exhibit poor responses to all-trans retinoic acid and conventional chemotherapy. Venetoclax, a selective inhibitor of B-cell lymphoma-2, can cross the blood–brain barrier and has been widely applied to acute myeloid leukemia therapy recently. Case presentation A case of central nervous system relapse in a patient with acute promyelocytic leukemia harboring PLZF::RARA rearrangement was successfully treated with anthracycline cytotoxic chemotherapy and cytarabine in combination with venetoclax, resulting in complete remission. Liquid chromatography-tandem mass spectrometry revealed that the concentration of venetoclax in the cerebrospinal fluid (CSF) was approximately 1/1,000 of that in plasma. Following the first treatment course, the patient’s bone marrow sample tested negative for PLZF::RARA. After the third treatment course, abnormal promyelocytic leukemia cells in the CSF were not detected using flow cytometry, and the PLZF::RARA test in the CSF remained negative. Conclusions This case report highlights a new approach for the treatment of central nervous system relapse in patients with PLZF::RARA-positive acute promyelocytic leukemia.