Department of Hematology and Oncology, Graduate School of Medicine, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo, Japan.
Int J Hematol. 2010 Jul;92(1):194-7. doi: 10.1007/s12185-010-0612-y. Epub 2010 Jun 12.
Familial platelet disorder (FPD) is a rare autosomal dominant disorder which causes moderate thrombocytopenia with or without impaired platelet function. Patients have a propensity to develop acute myeloid leukemia (AML), and various types of second hits have been postulated in the evolution to AML. However, only a few cases of acute lymphoblastic leukemia (ALL) have been reported thus far. Here, we report a family of FPD with a germ-line hemi-allelic mutation R174X in the RUNX1 gene. The proband of the family developed AML and her son had ALL of the T cell lineage. The balanced translocation t(1;7)(p34.1;q22) was detected in the lymphoblasts from the patient with ALL. This translocation was not seen in any other affected members of the family or in the bone marrow sample of this patient in complete remission. Taken together, t(1;7)(p34.1;q22) is thought to be one of the somatic second hits that predisposes FPD to acute leukemia with T cell phenotype.
家族性血小板疾病(FPD)是一种罕见的常染色体显性遗传疾病,导致中度血小板减少症,伴有或不伴有血小板功能受损。患者易发生急性髓系白血病(AML),并假设在向 AML 发展过程中有各种类型的二次打击。然而,迄今为止仅报道了少数急性淋巴细胞白血病(ALL)病例。在这里,我们报告了一个家族性血小板疾病的家族,其 RUNX1 基因存在半等位基因突变 R174X。该家族的先证者发生了 AML,其儿子患有 T 细胞谱系的 ALL。在 ALL 患者的淋巴母细胞中检测到平衡易位 t(1;7)(p34.1;q22)。该易位未在该患者的任何其他受影响的家族成员或骨髓样本中看到。总的来说,t(1;7)(p34.1;q22)被认为是使 FPD 易患具有 T 细胞表型的急性白血病的体细胞二次打击之一。