What treatment is used for promyelocytic leukemia?

All-trans-retinoic acid (ATRA) is the primary treatment for promyelocytic leukemia, specifically acute promyelocytic leukemia (APL). APL is characterized by the presence of the promyelocyte and often involves a specific genetic translocation, typically the t(15;17) translocation, which results in the fusion gene PML-RARA. This fusion protein interferes with normal myeloid differentiation.

ATRA functions by promoting differentiation of the abnormal promyelocytes into mature granulocytes, effectively addressing the underlying pathophysiology of APL. By facilitating this differentiation, ATRA considerably improves treatment outcomes and has transformed APL from a highly lethal form of leukemia into a more treatable one.

While chemotherapy is a component of APL treatment regimens, it is not the defining treatment. ATRA is central and often combined with chemotherapy to enhance cure rates. Bone marrow transplant is typically reserved for cases that do not respond to initial therapy or for relapses, while antibiotics are generally used for managing infections that can arise in immunocompromised patients rather than treating the leukemia itself. Thus, ATRA is the key agent that revolutionized the management of promyelocytic leukemia.