Jameson-Lee Maximilian, Chen Katherine, Ritchie Ellen, Shore Tsiporah, Al-Khattab Omar, Gergis Usama
Weill Cornell Medical College, New York, NY, USA.
New York Presbyterian Hospital, USA.
Hematol Oncol Stem Cell Ther. 2018 Dec;11(4):245-247. doi: 10.1016/j.hemonc.2017.02.001. Epub 2017 Feb 24.
Thrombocytopenia with absent radii (TAR) syndrome is a rare congenital disorder characterized by low platelet counts of various severity, bilateral absent radii but thumbs are usually present. TAR syndrome is not generally associated with bone marrow failure or malignancy. Janus kinase-2, myeloproliferative leukemia protein, and calreticulin are not mutated in TAR patients. Only four cases of leukemia were reported in TAR patients in the literature: three acute myeloid leukemia (AML) and one acute lymphoblastic leukemia. Of the three cases of AML found in TAR patient, only one was reported in an adult. We report a case of myelodysplastic syndrome progressing to AML with calreticulin driver mutation in an adult male with TAR syndrome who was successfully treated with hematopoietic allogeneic stem cell transplantation.
血小板减少伴桡骨缺如(TAR)综合征是一种罕见的先天性疾病,其特征为血小板计数有不同程度的降低、双侧桡骨缺如,但拇指通常存在。TAR综合征一般与骨髓衰竭或恶性肿瘤无关。Janus激酶2、骨髓增殖性白血病蛋白和钙网蛋白在TAR患者中无突变。文献中仅报道了4例TAR患者发生白血病的病例:3例急性髓系白血病(AML)和1例急性淋巴细胞白血病。在TAR患者中发现的3例AML病例中,仅有1例是成人患者。我们报告1例患有TAR综合征的成年男性,其骨髓增生异常综合征进展为伴有钙网蛋白驱动突变的AML,该患者通过异基因造血干细胞移植获得成功治疗。