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- Title
Mixed-phenotype acute leukemia: A cohort and consensus research strategy from the Children's Oncology Group Acute Leukemia of Ambiguous Lineage Task Force.
- Authors
Orgel, Etan; Alexander, Thomas B.; Wood, Brent L.; Kahwash, Samir B.; Devidas, Meenakshi; Dai, Yunfeng; Alonzo, Todd A.; Mullighan, Charles G.; Inaba, Hiroto; Hunger, Stephen P.; Raetz, Elizabeth A.; Gamis, Alan S.; Rabin, Karen R.; Carroll, Andrew J.; Heerema, Nyla A.; Berman, Jason N.; Woods, William G.; Loh, Mignon L.; Zweidler‐McKay, Patrick A.; Horan, John T.
- Abstract
<bold>Background: </bold>Optimal chemotherapy for treating mixed-phenotype acute leukemia (MPAL) and the role of hematopoietic stem cell transplantation (HSCT) remain uncertain. Major limitations in interpreting available data are MPAL's rarity and the use of definitions other than the currently widely accepted criteria: the World Health Organization 2016 (WHO2016) classification.<bold>Methods: </bold>To assess the relative efficacy of chemotherapy types for treating pediatric MPAL, the Children's Oncology Group (COG) Acute Leukemia of Ambiguous Lineage Task Force assembled a retrospective cohort of centrally reviewed WHO2016 MPAL cases selected from banking studies for acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). Patients were not treated in COG trials; treatment and outcome data were captured separately. The findings were then integrated with the available, mixed literature to develop a prospective trial in pediatric MPAL.<bold>Results: </bold>The central review confirmed that 54 of 70 cases fulfilled WHO2016 criteria for MPAL. ALL induction regimens achieved remission in 72% of the cases (28 of 39), whereas AML regimens achieved remission in 69% (9 of 13). The 5-year event-free survival (EFS) and overall survival (OS) rates for the entire cohort were 72% ± 8% and 77% ± 7%, respectively. EFS and OS were 75% ± 13% and 84% ± 11%, respectively, for those receiving ALL chemotherapy alone without HSCT (n = 21).<bold>Conclusions: </bold>The results of the COG MPAL cohort and a literature review suggest that ALL chemotherapy without HSCT may be the preferred initial therapy. A prospective trial within the COG is proposed to investigate this approach; AML chemotherapy and/or HSCT will be reserved for those with treatment failure as assessed by minimal residual disease. Embedded biology studies will provide further insight into MPAL genomics.
- Subjects
WORLD Health Organization; ACUTE leukemia; HEMATOPOIETIC stem cell transplantation; TASK forces; LYMPHOBLASTIC leukemia; ACUTE myeloid leukemia; RESEARCH; CLINICAL trials; RESEARCH methodology; PROGNOSIS; PEDIATRICS; EVALUATION research; MEDICAL cooperation; COMPARATIVE studies; LYMPHOCYTIC leukemia; IMMUNOPHENOTYPING; RESEARCH funding
- Publication
Cancer (0008543X), 2020, Vol 126, Issue 3, p593
- ISSN
0008-543X
- Publication type
journal article
- DOI
10.1002/cncr.32552