Poor outcome with hematopoietic stem cell transplantation for bone marrow failure and MDS with severe MIRAGE syndrome phenotype

Jay Sarthy,Ji Zha,Daria Babushok,Archana Shenoy,Jian-Meng Fan,Gerald Wertheim,Adam Himebauch,Ashley Munchel,Agne Taraseviciute, Samuel Yang,Hirohito Shima,Satoshi Narumi,Soheil Meshinchi,Timothy S Olson

Blood Advances（2018）

引用 31|浏览10

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摘要

Key Points Success of hematopoietic stem cell transplantation for MIRAGE syndrome may be limited by syndrome-specific comorbidities. SAMD9 mutations associated with MIRAGE syndrome are a newly described cause of congenital amegakaryocytic thrombocytopenia.

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