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Involvement of the central nervous system with acute myeloid leukemia

Charles A Schiffer, MD
Section Editor
Richard A Larson, MD
Deputy Editor
Alan G Rosmarin, MD


Involvement of the central nervous system (CNS) at the time of acute myeloid leukemia (AML) diagnosis is uncommon, and routine evaluation is not recommended for asymptomatic patients. Clinically overt CNS involvement developing at some point later during the course of treatment is also uncommon, perhaps related to the administration of high dose cytarabine as post-remission therapy.

This topic will discuss the epidemiology, clinical presentation, diagnosis, and treatment of CNS involvement in patients with AML. The clinical features, diagnosis, classification, treatment, and overall prognosis of AML are discussed separately. (See "Clinical manifestations, pathologic features, and diagnosis of acute myeloid leukemia" and "Induction therapy for acute myeloid leukemia in younger adults" and "Treatment of acute myeloid leukemia in older adults" and "Acute myeloid leukemia in children and adolescents".)


Incidence — The exact incidence of CNS involvement in patients with AML is unknown, but it is considerably less common than CNS involvement by acute lymphoblastic leukemia (ALL) in both adults and children [1,2]. As an example, CNS involvement was documented in 0.6 percent of adult patients at the time of initial presentation and in 2.9 percent upon relapse, in a review of 3261 adult patients enrolled in German clinical trials [1]. However, in infants, potential CNS involvement was identified in 29 percent (395 of 1344 children) of those in two Children's Oncology Group trials [2].

The incidence of CNS leukemia appears to have decreased since the incorporation of high dose cytarabine (HiDAC; which can penetrate into the CNS) into initial induction and/or post-remission therapy. Prior to the use of HiDAC, meningeal disease developed in up to 20 percent of children and 16 percent of adults with AML [3].

Risk factors — CNS involvement is more common in patients with AML with the following features [1,2,4,5]:

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Literature review current through: Nov 2017. | This topic last updated: Aug 24, 2017.
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