TY - JOUR
T1 - Pediatric acute myeloid leukemia with t(8;16)(p11;p13), a distinct clinical and biological entity: a collaborative study by the International-Berlin-Frankfurt-Munster AML-study group
AU - Coenen, Eva
AU - Zwaan, C.M.
AU - Reinhardt, D
AU - Harrison, CJ
AU - Haas, OA
AU - de Haas, V
AU - Mihal, V
AU - de Moerloose, B
AU - Jeison, M
AU - Rubnitz, JE
AU - Tomizawa, D
AU - Johnston, D
AU - Alonzo, TA
AU - Hasle, H
AU - Auvrignon, A
AU - Dworzak, M
AU - Pession, A
AU - van der Velden, Vincent
AU - Swansbury, J
AU - Wong, KF
AU - Terui, K
AU - Savasan, S
AU - Winstanley, M
AU - Vaitkeviciene, G
AU - Zimmermann, M
AU - Pieters, Rob
AU - Van den Heuvel - Eibrink, Marry
PY - 2013
Y1 - 2013
N2 - In pediatric acute myeloid leukemia (AML), cytogenetic abnormalities are strong indicators of prognosis. Some recurrent cytogenetic abnormalities, such as t(8;16)(p11;p13),are so rare that collaborative studies are required to define their prognostic impact. We collected the clinical characteristics, morphology, and immunophenotypes of 62 pediatric AML patients with t(8; 16)(p11; p13) from 18 countries participating in the International Berlin-Frankfurt-Munster (I-BFM) AML study group. We used the AML-BFM cohort diagnosed from 1995-2005 (n = 543) as a reference cohort. Median age of the pediatric t(8; 16)(p11; p13) AML patients was significantly lower (1.2 years). The majority (97%) had M4-M5 French-American-British type, significantly different from the reference cohort. Erythrophagocytosis (70%), leukemia cutis (58%), and disseminated intravascular coagulation (39%) occurred frequently. Strikingly, spontaneous remissions occurred in 7 neonates with t(8; 16)(p11; p13), of whom 3 remain in continuous remission. The 5-year overall survival of patients diagnosed after 1993 was 59%, similar to the reference cohort (P = .14). Gene expression profiles of t(8; 16) (p11; p13) pediatric AML cases clustered close to, but distinct from, MLL-rearranged AML. Highly expressed genes included HOXA11, HOXA10, RET, PERP, and GGA2. In conclusion, pediatric t(8; 16)(p11; p13) AML is a rare entity defined by a unique gene expression signature and distinct clinical features in whom spontaneous remissions occur in a subset of neonatal cases.
AB - In pediatric acute myeloid leukemia (AML), cytogenetic abnormalities are strong indicators of prognosis. Some recurrent cytogenetic abnormalities, such as t(8;16)(p11;p13),are so rare that collaborative studies are required to define their prognostic impact. We collected the clinical characteristics, morphology, and immunophenotypes of 62 pediatric AML patients with t(8; 16)(p11; p13) from 18 countries participating in the International Berlin-Frankfurt-Munster (I-BFM) AML study group. We used the AML-BFM cohort diagnosed from 1995-2005 (n = 543) as a reference cohort. Median age of the pediatric t(8; 16)(p11; p13) AML patients was significantly lower (1.2 years). The majority (97%) had M4-M5 French-American-British type, significantly different from the reference cohort. Erythrophagocytosis (70%), leukemia cutis (58%), and disseminated intravascular coagulation (39%) occurred frequently. Strikingly, spontaneous remissions occurred in 7 neonates with t(8; 16)(p11; p13), of whom 3 remain in continuous remission. The 5-year overall survival of patients diagnosed after 1993 was 59%, similar to the reference cohort (P = .14). Gene expression profiles of t(8; 16) (p11; p13) pediatric AML cases clustered close to, but distinct from, MLL-rearranged AML. Highly expressed genes included HOXA11, HOXA10, RET, PERP, and GGA2. In conclusion, pediatric t(8; 16)(p11; p13) AML is a rare entity defined by a unique gene expression signature and distinct clinical features in whom spontaneous remissions occur in a subset of neonatal cases.
U2 - 10.1182/blood-2013-02-485524
DO - 10.1182/blood-2013-02-485524
M3 - Article
C2 - 23974201
SN - 0006-4971
VL - 122
SP - 2704
EP - 2713
JO - Blood
JF - Blood
IS - 15
ER -