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Prognostic impact of t(16;21)(p11;q22) and t(16;21)(q24;q22) in pediatric AML: a retrospective study by the I-BFM Study Group
S. Noort, M. Zimmermann, D. Reinhardt, W. Cuccuini, M. Pigazzi, J. Smith, RE. Ries, TA. Alonzo, B. Hirsch, D. Tomizawa, F. Locatelli, TA. Gruber, S. Raimondi, E. Sonneveld, DK. Cheuk, M. Dworzak, J. Stary, J. Abrahamsson, N. Arad-Cohen, M....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, Research Support, N.I.H., Extramural
Grantová podpora
U10 CA180886
NCI NIH HHS - United States
U10 CA180899
NCI NIH HHS - United States
NLK
Free Medical Journals
od 1946 do Před 1 rokem
Freely Accessible Science Journals
od 1946 do Před 1 rokem
Open Access Digital Library
od 1946-01-01
Open Access Digital Library
od 1946-01-01
ROAD: Directory of Open Access Scholarly Resources
- MeSH
- akutní myeloidní leukemie diagnóza genetika MeSH
- dítě MeSH
- kojenec MeSH
- lidé MeSH
- lidské chromozomy, pár 16 genetika MeSH
- lidské chromozomy, pár 21 genetika MeSH
- mladiství MeSH
- nádorové supresorové proteiny genetika MeSH
- předškolní dítě MeSH
- prognóza MeSH
- protein FUS vázající RNA genetika MeSH
- protein PEBP2A2 genetika MeSH
- regulace genové exprese u leukemie MeSH
- represorové proteiny genetika MeSH
- retrospektivní studie MeSH
- transkripční regulátor ERG genetika MeSH
- transkriptom MeSH
- translokace genetická * MeSH
- Check Tag
- dítě MeSH
- kojenec MeSH
- lidé MeSH
- mladiství MeSH
- mužské pohlaví MeSH
- předškolní dítě MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- Research Support, N.I.H., Extramural MeSH
To study the prognostic relevance of rare genetic aberrations in acute myeloid leukemia (AML), such as t(16;21), international collaboration is required. Two different types of t(16;21) translocations can be distinguished: t(16;21)(p11;q22), resulting in the FUS-ERG fusion gene; and t(16;21)(q24;q22), resulting in RUNX1-core binding factor (CBFA2T3). We collected data on clinical and biological characteristics of 54 pediatric AML cases with t(16;21) rearrangements from 14 international collaborative study groups participating in the international Berlin-Frankfurt-Münster (I-BFM) AML study group. The AML-BFM cohort diagnosed between 1997 and 2013 was used as a reference cohort. RUNX1-CBFA2T3 (n = 23) had significantly lower median white blood cell count (12.5 × 109/L, P = .03) compared with the reference cohort. FUS-ERG rearranged AML (n = 31) had no predominant French-American-British (FAB) type, whereas 76% of RUNX1-CBFA2T3 had an M1/M2 FAB type (M1, M2), significantly different from the reference cohort (P = .004). Four-year event-free survival (EFS) of patients with FUS-ERG was 7% (standard error [SE] = 5%), significantly lower compared with the reference cohort (51%, SE = 1%, P < .001). Four-year EFS of RUNX1-CBFA2T3 was 77% (SE = 8%, P = .06), significantly higher compared with the reference cohort. Cumulative incidence of relapse was 74% (SE = 8%) in FUS-ERG, 0% (SE = 0%) in RUNX1-CBFA2T3, compared with 32% (SE = 1%) in the reference cohort (P < .001). Multivariate analysis identified both FUS-ERG and RUNX1-CBFA2T3 as independent risk factors with hazard ratios of 1.9 (P < .0001) and 0.3 (P = .025), respectively. These results describe 2 clinically relevant distinct subtypes of pediatric AML. Similarly to other core-binding factor AMLs, patients with RUNX1-CBFA2T3 rearranged AML may benefit from stratification in the standard risk treatment, whereas patients with FUS-ERG rearranged AML should be considered high-risk.
Children's Oncology Group Monrovia CA
Department of Cytogenetics Saint Louis Hospital Paris France
Department of Pathology St Jude Children's Research Hospital Memphis TN
Department of Pediatric Hematology Oncology Medical School Hannover Hannover Germany
Dutch Childhood Oncology Group The Hague The Netherlands
Fred Hutchinson Cancer Research Center Seattle WA
Pediatrics and Adolescent Medicine Aarhus University Hospital Aarhus Denmark
Women and Children's Health Hematology Oncology Laboratory University of Padova Padova Italy
Citace poskytuje Crossref.org
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