Article

Biology and outcome of childhood acute megakaryoblastic leukemia: A single institution's experience

Department of Hematology-Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
Blood (Impact Factor: 10.43). 06/2001; 97(12):3727-32. DOI: 10.1182/blood.V97.12.3727
Source: PubMed

ABSTRACT 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 < or =.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. (Blood. 2001;97:3727-3732)

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Available from: Bassem I Razzouk, Jul 28, 2015
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    • "AMKL is divided into two subgroups: AMKL arising in patients with Down syndrome (DS-AMKL), and leukemia arising in patients without Down syndrome (non-DS- AMKL). Although DS-AMKL patients have an excellent prognosis with an $80% survival, non-DS-AMKL patients do not fare as well, with a reported survival of only 14%–34% despite highintensity chemotherapy (Athale et al., 2001; Barnard et al., 2007; Creutzig et al., 2005). With the exception of the t(1;22) seen in infant non-DS-AMKL, little is known about the molecular lesions that underlie this leukemia subtype (Carroll et al., 1991; Lion et al., 1992; Ma et al., 2001; Mercher et al., 2001). "
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    • "AMKL in DS patients is distinguished by an unusually favorable clinical prognosis, first recognized by POG over a decade ago, and confirmed by the Nordic Society for Pediatric Hematology and Oncology and the Children's Cancer Group (CCG).[7] [27] [28] Current eventfree survival (EFS) for DS AMKL is about 80%, whereas EFS in the rarer subgroup of non- DS AMKL is considerably worse than other AML subtypes at 15-20%.[29] Young age is an important positive prognostic factor in DS AML. "
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    • "In a study conducted by Athale et al. in 2001, two findings were identified as highly diagnostic for AMKL: leukemic cells isolated from the BM had distinct morphologic features, such as surface blebs, cell clumping, and binucleation, and the presence of multifocal punctate cytoplasmic alpha naphthyl acetate esterase cytochemical staining that is inhibited by sodium fluoride. As aforementioned Trisomy 21-AMKL has a much better outcome than AMKL not associated with Trisomy 21 or GATA-1 mutations, and the same study found an estimated 83% two year event free survival rate for Trisomy 21-AMKL, versus 14% and 20% for de novo and secondary AMKL not associated with Trisomy 21 or spontaneous GATA-1 mutations (Athale et al., 2001). "
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