International cooperative study identifies treatment strategy in childhood ambiguous lineage leukemia
Autor: | Simone Stokley, Alexandra Kolenova, Sarah Elitzur, Jan Trka, Barbora Vakrmanova, Neda Marinov, Kirsten Bleckmann, Ales Luks, Karen R. Rabin, Benigna Konatkowska, Hiroto Inaba, Julie Irving, Elaine da Costa, Tamar Feuerstein, Shai Izraeli, Dirk Reinhardt, Ondrej Hrusak, Ester Mejstrikova, Valerie de Haas, Jan Stary, Barbara Buldini, Myriam Campbell, Luciano Dalla-Pozza, Jessa Morales, Olena Kreminska, Marketa Zaliova, Vaclav Capek, John K. Choi, Zuzana Zemanova, Sophia Polychronopoulou, Richard Ratei, Anthony V. Moorman, Kjeld Schmiegelow, Antonis Kattamis, Jorge Rossi, Martin Schrappe, Iveta Janotova, Maria Elena Cabrera, Hanne Vibeke Marquart, Maria S. Felice, Giuseppe Basso, Jitka Stancikova, Peter Svec, Thomas B. Alexander, Anja Möricke, Michael Dworzak, Drorit Luria |
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Rok vydání: | 2017 |
Předmět: |
Male
medicine.medical_specialty Lineage (genetic) Adolescent Biochemistry Immunology Hematology Cell Biology Medizin World health 03 medical and health sciences 0302 clinical medicine Immunophenotyping hemic and lymphatic diseases Internal medicine Biomarkers Tumor Medicine Humans Child Proportional Hazards Models business.industry Proportional hazards model Infant Newborn Myeloid leukemia Disease Management Infant medicine.disease Prognosis Combined Modality Therapy Leukemia Biphenotypic Acute Transplantation Leukemia Treatment Outcome 030220 oncology & carcinogenesis Child Preschool Treatment strategy Female Disease Susceptibility business Biomarkers 030215 immunology |
Zdroj: | Blood. 132(3) |
ISSN: | 1528-0020 |
Popis: | Despite attempts to improve the definitions of ambiguous lineage leukemia (ALAL) during the last 2 decades, general therapy recommendations are missing. Herein, we report a large cohort of children with ALAL and propose a treatment strategy. A retrospective multinational study (International Berlin-Frankfurt-Munster Study of Leukemias of Ambiguous Lineage [iBFM-AMBI2012]) of 233 cases of pediatric ALAL patients is presented. Survival statistics were used to compare the prognosis of subsets and types of treatment. Five-year event-free survival (EFS) of patients with acute lymphoblastic leukemia (ALL)-type primary therapy (80% ± 4%) was superior to that of children who received acute myeloid leukemia (AML)-type or combined-type treatment (36% ± 7.2% and 50% ± 12%, respectively). When ALL- or AML-specific gene fusions were excluded, 5-year EFS of CD19+ leukemia was 83% ± 5.3% on ALL-type primary treatment compared with 0% ± 0% and 28% ± 14% on AML-type and combined-type primary treatment, respectively. Superiority of ALL-type treatment was documented in single-population mixed phenotype ALAL (using World Health Organization and/or European Group for Immunophenotyping of Leukemia definitions) and bilineal ALAL. Treatment with ALL-type protocols is recommended for the majority of pediatric patients with ALAL, including cases with CD19+ ALAL. AML-type treatment is preferred in a minority of ALAL cases with CD19- and no other lymphoid features. No overall benefit of transplantation was documented, and it could be introduced in some patients with a poor response to treatment. As no clear indicator was found for a change in treatment type, this is to be considered only in cases with ≥5% blasts after remission induction. The results provide a basis for a prospective trial. |
Databáze: | OpenAIRE |
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