Pathophysiological analysis of the pediatric minimally differentiated acute myeloid leukemia and development of a specific therapy

2022 Fiscal Year Final Research Report

• Project/Area Number: 20K16923
• Research Category: Grant-in-Aid for Early-Career Scientists
• Allocation Type: Multi-year Fund
• Review Section: Basic Section 52050:Embryonic medicine and pediatrics-related
• Research Institution: Kyoto University
• Principal Investigator: Saida Satoshi 京都大学, 医学研究科, 特定病院助教 (70638254)
• Project Period (FY): 2020-04-01 – 2023-03-31
• Keywords: 急性骨髄性白血病 / 小児

Outline of Final Research Achievements

Childhood acute leukemia is the most common childhood cancer, and minimally differentiated acute myeloid leukemia (AML-M0) has been reported to have the poor prognosis among childhood AML cases. Elucidating the pathogenesis of these childhood leukemias is an important issue in the treatment of childhood cancer. We collected samples of childhood acute leukemia cohorts and pediatric AML-M0 cases and performed various omics analyses (genomic, transcriptomic, and methylomic analyses). These analyses revealed genetic characteristics and poor prognostic factors specific to pediatric AML-M0. The results of this study provide a basis for better risk stratified treatment of childhood acute myeloid leukemia.

Free Research Field

小児悪性腫瘍・白血病

Academic Significance and Societal Importance of the Research Achievements

希少な小児がんの中において、小児急性骨髄性白血病に占めるAML-M0の割合は非常に低い。我々は、小児AML-M0症例を全国から集積して解析を行った。これは過去の小児AML-M0についてのコホート研究の中では最大のもので、世界的にみても大変貴重なコホートとなる。
今回の解析で、小児急性白血病全体における「小児AML-M0」の遺伝学的特徴（融合遺伝子や遺伝子変異の分布）や遺伝子発現の特徴を明らかにすることができた。さらにそれらの中から予後に関わる因子を抽出することができた。本研究成果は、今後の小児急性骨髄性白血病のリスク層別化治療の開発の一助となることが期待される。