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Improved outcome of pediatric patients with acute megakaryoblastic leukemia in the AML-BFM 04 trial.

Schweitzer J, Zimmermann M, Rasche M, von Neuhoff C, Creutzig U, Dworzak M, Reinhardt D, Klusmann JH - Ann. Hematol. (2015)

Bottom Line: Allogeneic hematopoietic stem cell transplantation in first remission did not provide a significant survival benefit (5-year OS 70 ± 11 % vs. 63 ± 6 %; P Mantel-Byar = 0.85).AMKL patients with gain of chromosome 21 had a superior 5-year OS (80 ± 9 %, P log rank = 0.034), whereas translocation t(1;22)(p13;q13) was associated with an inferior 5-year event-free survival (38 ± 17 %, P log rank = 0.04).Thus, AMKL (excluding DS) remains an AML subgroup with inferior outcome.

View Article: PubMed Central - PubMed

Affiliation: Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.

ABSTRACT
Despite recent advances in the treatment of children with acute megakaryoblastic leukemia (AMKL) using intensified treatment protocols, clear prognostic indicators, and treatment recommendations for this acute myeloid leukemia (AML) subgroup are yet to be defined. Here, we report the outcome of 97 pediatric patients with de novo AMKL (excluding Down syndrome [DS]) enrolled in the prospective multicenter studies AML-BFM 98 and AML-BFM 04 (1998-2014). AMKL occurred in 7.4 % of pediatric AML cases, at younger age (median 1.44 years) and with lower white blood cell count (mean 16.5 × 10(9)/L) as compared to other AML subgroups. With 60 ± 5 %, children with AMKL had a lower 5-year overall survival (5-year OS; vs. 68 ± 1 %, P log rank = 0.038). Yet, we achieved an improved 5-year OS in AML-BFM 04 compared to AML-BFM 98 (70 ± 6 % vs. 45 ± 8 %, P log rank = 0.041). Allogeneic hematopoietic stem cell transplantation in first remission did not provide a significant survival benefit (5-year OS 70 ± 11 % vs. 63 ± 6 %; P Mantel-Byar = 0.85). Cytogenetic data were available for n = 78 patients. AMKL patients with gain of chromosome 21 had a superior 5-year OS (80 ± 9 %, P log rank = 0.034), whereas translocation t(1;22)(p13;q13) was associated with an inferior 5-year event-free survival (38 ± 17 %, P log rank = 0.04). However, multivariate analysis showed that treatment response (bone marrow morphology on day 15 and 28) was the only independent prognostic marker (RR = 4.39; 95 % CI, 1.97-9.78). Interestingly, GATA1-mutations were detected in six patients (11 %) without previously known trisomy 21. Thus, AMKL (excluding DS) remains an AML subgroup with inferior outcome. Nevertheless, with intensive therapy regimens, a steep increase in the survival rates was achieved.

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Overall survival of patients diagnosed with non-DS de novo AMKL (n = 97) or other AML subtypes (n = 1219) in the AML-BFM 98 and AML-BFM 04 studies. Five-year OS is given
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Fig2: Overall survival of patients diagnosed with non-DS de novo AMKL (n = 97) or other AML subtypes (n = 1219) in the AML-BFM 98 and AML-BFM 04 studies. Five-year OS is given

Mentions: With a 5-year OS of 60 ± 5 % (Fig. 2) and 5-year EFS of 47 ± 5 %, children with AMKL revealed a significantly poorer outcome as compared to the total group of children with other AML subtypes (Fig. 2; 5-year EFS 52 ± 1 %, Plog rank = 0.079). The complete remission (CR) rate of non-DS-AMKL patients was comparable to other children with HR-AML (defined by the reported criteria of the AML-BFM study group [14, 23]). Eighty-one patients (83.6 %) achieved CR, 32 % (n = 32) relapsed after first CR, and 4.1 % (n = 4) died in first CR. Thirteen patients (13.4 %) were partial- and non-responders (PR; NR) to induction therapy and 3.1 % (n = 3) suffered from early death (ED). The cumulative incidence of relapse (CIR) was 45 ± 5 % compared to 39 ± 1 % (PGray = 0.07) for patients with other AML subtypes.Fig. 2


Improved outcome of pediatric patients with acute megakaryoblastic leukemia in the AML-BFM 04 trial.

Schweitzer J, Zimmermann M, Rasche M, von Neuhoff C, Creutzig U, Dworzak M, Reinhardt D, Klusmann JH - Ann. Hematol. (2015)

Overall survival of patients diagnosed with non-DS de novo AMKL (n = 97) or other AML subtypes (n = 1219) in the AML-BFM 98 and AML-BFM 04 studies. Five-year OS is given
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC4488462&req=5

Fig2: Overall survival of patients diagnosed with non-DS de novo AMKL (n = 97) or other AML subtypes (n = 1219) in the AML-BFM 98 and AML-BFM 04 studies. Five-year OS is given
Mentions: With a 5-year OS of 60 ± 5 % (Fig. 2) and 5-year EFS of 47 ± 5 %, children with AMKL revealed a significantly poorer outcome as compared to the total group of children with other AML subtypes (Fig. 2; 5-year EFS 52 ± 1 %, Plog rank = 0.079). The complete remission (CR) rate of non-DS-AMKL patients was comparable to other children with HR-AML (defined by the reported criteria of the AML-BFM study group [14, 23]). Eighty-one patients (83.6 %) achieved CR, 32 % (n = 32) relapsed after first CR, and 4.1 % (n = 4) died in first CR. Thirteen patients (13.4 %) were partial- and non-responders (PR; NR) to induction therapy and 3.1 % (n = 3) suffered from early death (ED). The cumulative incidence of relapse (CIR) was 45 ± 5 % compared to 39 ± 1 % (PGray = 0.07) for patients with other AML subtypes.Fig. 2

Bottom Line: Allogeneic hematopoietic stem cell transplantation in first remission did not provide a significant survival benefit (5-year OS 70 ± 11 % vs. 63 ± 6 %; P Mantel-Byar = 0.85).AMKL patients with gain of chromosome 21 had a superior 5-year OS (80 ± 9 %, P log rank = 0.034), whereas translocation t(1;22)(p13;q13) was associated with an inferior 5-year event-free survival (38 ± 17 %, P log rank = 0.04).Thus, AMKL (excluding DS) remains an AML subgroup with inferior outcome.

View Article: PubMed Central - PubMed

Affiliation: Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.

ABSTRACT
Despite recent advances in the treatment of children with acute megakaryoblastic leukemia (AMKL) using intensified treatment protocols, clear prognostic indicators, and treatment recommendations for this acute myeloid leukemia (AML) subgroup are yet to be defined. Here, we report the outcome of 97 pediatric patients with de novo AMKL (excluding Down syndrome [DS]) enrolled in the prospective multicenter studies AML-BFM 98 and AML-BFM 04 (1998-2014). AMKL occurred in 7.4 % of pediatric AML cases, at younger age (median 1.44 years) and with lower white blood cell count (mean 16.5 × 10(9)/L) as compared to other AML subgroups. With 60 ± 5 %, children with AMKL had a lower 5-year overall survival (5-year OS; vs. 68 ± 1 %, P log rank = 0.038). Yet, we achieved an improved 5-year OS in AML-BFM 04 compared to AML-BFM 98 (70 ± 6 % vs. 45 ± 8 %, P log rank = 0.041). Allogeneic hematopoietic stem cell transplantation in first remission did not provide a significant survival benefit (5-year OS 70 ± 11 % vs. 63 ± 6 %; P Mantel-Byar = 0.85). Cytogenetic data were available for n = 78 patients. AMKL patients with gain of chromosome 21 had a superior 5-year OS (80 ± 9 %, P log rank = 0.034), whereas translocation t(1;22)(p13;q13) was associated with an inferior 5-year event-free survival (38 ± 17 %, P log rank = 0.04). However, multivariate analysis showed that treatment response (bone marrow morphology on day 15 and 28) was the only independent prognostic marker (RR = 4.39; 95 % CI, 1.97-9.78). Interestingly, GATA1-mutations were detected in six patients (11 %) without previously known trisomy 21. Thus, AMKL (excluding DS) remains an AML subgroup with inferior outcome. Nevertheless, with intensive therapy regimens, a steep increase in the survival rates was achieved.

Show MeSH
Related in: MedlinePlus