Treatment of TAM

Source: Childhood Myeloid Proliferations Associated With Down Syndrome Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: September 16, 2024 · Captured 2026-09-09.

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While observation is appropriate for most infants with TAM, therapeutic intervention is warranted in patients with apparent severe hydrops or organ failure. Because TAM eventually spontaneously remits, treatment is short in duration and primarily aimed at the reduction of leukemic burden and resolution of immediate symptoms. Several treatment approaches have been used, including the following:

Exchange transfusion.

Leukapheresis.

Low-dose cytarabine. Of these approaches, only cytarabine has been shown to consistently reduce TAM complications and related mortality.[3,6]; [14][Level of evidence B4] Cytarabine dosing has ranged from 0.4 to 1.5 mg/kg per dose given intravenously (IV) or subcutaneously (SC) once to twice daily for 4 to 12 days.[6] This dosing schedule has produced similar efficacies and less toxicity than higher doses given in continuous 5-day infusions, which led to prolonged severe neutropenia.[3] A prospective trial examined the use of low-dose cytarabine (1.5 mg/kg per day IV or SC for 7 days) to treat symptomatic patients. This trial reported a significant reduction in early death using this regimen, compared with similar patients in the historical control group (12% ± 5% vs. 33% ± 7%, respectively; P = .02).[14][Level of evidence B4]

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