Acute promyelocytic leukemia overview

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2]

Overview

Acute promyelocytic leukemia is a subtype of acute myelogenous leukemia (AML), a cancer of the blood and bone marrow.

In APL, there is an abnormal accumulation of immature granulocytes called promyelocytes. The disease is characterized by a chromosomal translocation involving the retinoic acid receptor alpha (RARα or RARA) gene and is unique from other forms of AML in its responsiveness to all trans retinoic acid (ATRA) therapy.

Historical Perspective

The first documentation of the successful treatment of acute promyelocytic leukemia occurred in the late 19th century, at which time physicians and scientists explored the role of arsenic as an anti-leukemic agent. Since that time, multiple advances have been made over the years. Specifically, the use of cytotoxic chemotherapy (anthracycline and cytarabine) has been explored extensively. The use of all-''trans'' retinoic acid in the 20th century has revolutionized the treatment paradigm for acute promyelocytic leukemia. In the early 21st century, a landmark study showed that the combination of arsenic trioxide plus all-''trans'' retinoic acid was superior to conventional chemotherapy for low-risk acute promyelocytic leukemia.

Classification

There are several broad classification schemes for acute promyelocytic leukemia. The most well-accepted classification scheme is risk-based classification, which categories patients into low-risk, intermediate-risk, or high-risk based on the white blood cell count and platelet count. Another classification scheme is based on the origin of the leukemia, which categorized patients as having de novo or therapy-related disease. A final classification scheme is cytogenetic-based, in which case specific chromosomal abnormalities are used to stratify patients.

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