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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. Likewise, clinically overt CNS involvement developing at some point later during the course of treatment is 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".)


Incidence — The exact incidence of central nervous system (CNS) involvement in patients with acute myeloid leukemia (AML) is unknown, but is thought to be considerably less common than CNS involvement in both adults and children with acute lymphoblastic leukemia (ALL) [1-4]. The incidence of CNS leukemia appears to have decreased since the incorporation of high doses of cytarabine, which can penetrate into the CNS, during both initial induction and post-remission therapy. Prior to the use of cytarabine, meningeal disease developed in up to 20 percent of children and 16 percent of adults with AML. An estimate of the current incidence, based upon patients treated at the author's center or in large clinical trials, is less than 5 percent of patients with AML overall.

Risk factors — CNS involvement may be more common in patients with AML with a prominent monocytic component (eg, acute monocytic leukemia or acute myelomonocytic leukemia), acute promyelocytic leukemia (APL) in systemic relapse, AML with inv(16) or chromosome 11 abnormality, those with hyperleukocytosis or an elevated lactate dehydrogenase, and patients under two years of age [5,6]. The expression of the adhesion molecule CD56 on the surface of leukemia blast cells has also been associated with CNS involvement. In addition, a traumatic lumbar puncture can introduce leukemic cells into the cerebrospinal fluid. It is unclear whether all of these risk factors still apply to patients treated with modern induction regimens.


How acute myeloid leukemia (AML) seeds the central nervous system (CNS) is incompletely understood. Possible mechanisms include [7]:


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Literature review current through: Jul 2017. | This topic last updated: Apr 07, 2017.
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