摘要
Primary myelofibrosis (PMF) is a chronic myeloproliferative disorder in human bone marrow. Over 50% of patients with myelofibrosis have mutations in JAK2, MPL, or CALR. However, these mutations are rarely detected in children, suggesting a difference in the pathogenesis of childhood PMF. In this study, we investigated the response to drug treatment of a monozygotic twin pair with typical childhood PMF. The twin exhibited different clinical outcomes despite following
Primary myelofibrosis (PMF) is a chronic myeloproliferative disorder in human bone marrow. Over 50% of patients with myelofibrosis have mutations in JAK2, MPL, or CALR. However, these mutations are rarely detected in children, suggesting a difference in the pathogenesis of childhood PMF. In this study, we investigated the response to drug treatment of a monozygotic twin pair with typical childhood PMF. The twin exhibited different clinical outcomes despite following
基金
supported by the National Natural Science Foundation of China(Grant Nos.31471115,31401160,81300393,and 81300394)
the Strategic Priority Research Program of the Chinese Academy of Sciences,Stem Cell and Regenerative Medicine Research,China(Grant No.XDA01040405)
the National‘‘Twelfth Five-Year”Plan for Science&Technology Support,China(Grant No.2013BAI01B09)
the National Key Scientific Instrument and Equipment Development Projects of China(Grant No.2011YQ03013404)
the National High-tech R&D Program of China(Grant Nos.2015AA020101 and 2015AA020108)
the State Key Laboratory of Experimental Hematology Pilot Project(Grant No.ZK13-05)
the Nature Science Fund of Tianjin Municipal Science and Technology Commission,China(Grant No.12ZCDZSY18100)