Development of biomarkers to predict immunosuppressive therapy reactivity of aplastic anemia in children

2017 Fiscal Year Final Research Report

• Project/Area Number: 15K09647
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Nagoya University
• Principal Investigator: Muramatsu Hideki 名古屋大学, 医学部附属病院, 助教 (00572570)
• Co-Investigator(Kenkyū-buntansha): 奥野 友介 名古屋大学, 医学部附属病院, 特任講師 (00725533) ; 小島 勢二 名古屋大学, 医学系研究科, 名誉教授 (20313992)
• Co-Investigator(Renkei-kenkyūsha): OKUNO Yusuke 名古屋大学, 医学部附属病院, 特任講師 (00725533)
• Project Period (FY): 2015-04-01 – 2018-03-31
• Keywords: 小児血液学 / 再生不良性貧血

Outline of Final Research Achievements

We analyzed 113 patients with aplastic anemia (AA) in children and identified that absence of PNH-type blood cells and telomere shortening are independent poor prognostic factors for immunosuppressive therapy (IST) reaction (Haematologica 2015). We also identified that patients with high thrombopoietin (TPO) levels showed significantly lower response rate to IST (Pediatr Blood Cancer. 2016). Furthermore, we developed a target sequencing system of 184 genes associated with congenital hematopoietic failure (Genetics in Medicine 2017), and performed on 181 patients with idiopathic AA. In 26 cases, relevant gene mutations were identified, indicating the usefulness of screening for congenital hematopoietic failure genes in pediatric AA patients.

Free Research Field

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