Ware R E, Parker R I, McKeown L P, Graham M L
Division of Pediatric Hematology/Oncology, Duke University Medical Center, Durham, North Carolina 27710.
Am J Pediatr Hematol Oncol. 1993 Aug;15(3):338-42.
von Willebrand disease (vWD) is a common bleeding disorder characterized by quantitative or qualitative defects in von Willebrand factor (vWF), a protein important for coagulation and platelet adhesion. There are two forms of vWF currently recognized: plasma vWF synthesized by endothelial cells and platelet vWF produced within megakaryocytes. Although both plasma and platelet vWF play important roles in overall hemostasis, the relative contribution of each form is not clear.
We report the results of bone marrow transplantation from a donor with Type I vWD into a hemostatically normal recipient.
Following engraftment, the recipient appears to be a human chimera for vWD, with normal amounts of plasma vWF from endogenous endothelial cells, but low levels of platelet vWF derived from donor megakaryocytes.
Although the vWD chimerism has not been associated with any clinical manifestations, the bleeding time has become prolonged, suggesting that platelet vWF is important for normalization of the bleeding time.
血管性血友病（vWD）是一种常见的出血性疾病，其特征是血管性血友病因子（vWF）存在数量或质量缺陷，vWF是一种对凝血和血小板黏附很重要的蛋白质。目前已确认vWF有两种形式：由内皮细胞合成的血浆vWF和巨核细胞内产生的血小板vWF。虽然血浆vWF和血小板vWF在整体止血过程中都起重要作用，但每种形式的相对贡献尚不清楚。
我们报告了将一名I型vWD供体的骨髓移植到一名止血功能正常的受者体内的结果。
植入后，受者似乎成为了vWD的人类嵌合体，内源性内皮细胞产生的血浆vWF量正常，但供体巨核细胞衍生的血小板vWF水平较低。
虽然vWD嵌合体未出现任何临床表现，但出血时间延长，提示血小板vWF对出血时间的正常化很重要。
