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Blood, 15 June 2001, Vol. 97, No. 12, pp. 3727-3732
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Biology and outcome of childhood acute
megakaryoblastic leukemia: a single institution's
experience
Uma H. Athale,
Bassem I. Razzouk,
Susana C. Raimondi,
Xin Tong,
Frederick G. Behm,
David R. Head,
Deo K. Srivastava,
Jeffrey E. Rubnitz,
Laura Bowman,
Ching-Hon Pui, and
Raul C. Ribeiro
From the Departments of Hematology-Oncology,
Biostatistics and Epidemiology, and Pathology and the International
Outreach Program, St Jude Children's Research Hospital; and
Departments of Pediatrics and Pathology, the University of Tennessee,
Memphis.
To describe the clinical and biologic features of pediatric acute
megakaryoblastic leukemia (AMKL) and to identify prognostic factors,
experience at St Jude Children's Research Hospital was reviewed. Of
281 patients with acute myeloid leukemia treated over a 14-year period,
41 (14.6%) had a diagnosis of AMKL. Six patients had Down syndrome and
AMKL, 6 had secondary AMKL, and 29 had de novo AMKL. The median age of
the 22 boys and 19 girls was 23.9 months (range, 6.7-208.9 months). The
rate of remission induction was 60.5%, with a 48% rate of subsequent
relapse. Patients with Down syndrome had a significantly higher 2-year
event-free survival (EFS) estimate (83%) than did other patients with
de novo AMKL (14%) or with secondary AMKL (20%;
P  .038). Among patients who had de novo AMKL without
Down syndrome, 2-year EFS was significantly higher after allogeneic
bone marrow transplantation (26%) than after chemotherapy alone (0%;
P = .019) and significantly higher when performed during
remission (46%) than when performed during persistent disease (0%;
P = .019). The 5-year survival estimates were
significantly lower for de novo AMKL (10%) than for other forms of de
novo AML (42%; P < .001). Treatment outcome is very
poor for patients with AMKL in the absence of Down syndrome. Remission
induction is the most important prognostic factor. Allogeneic transplantation during remission offers the best chance of cure; in the
absence of remission, transplantation offers no advantage over
chemotherapy alone.
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