Weiss H J, Meyer D, Rabinowitz R, Pietu G, Girma J P, Vicic W J, Rogers J
N Engl J Med. 1982 Feb 11;306(6):326-33. doi: 10.1056/NEJM198202113060603.
Four members (from four generations) of a family with a mild bleeding disorder and intermittent thrombocytopenia had decreased plasma levels of properties related to factor VIII/von Willebrand factor (FVIII/VWF), an absence of high-molecular-weight forms of FVIII/VWF in the plasma (but normal multimeric structure in the platelets), and increased ristocetin-induced platelet aggregation, as in Type IIB von Willebrand's disease. However, unlike the abnormality in FVIII/VWF in Type IIB disease, the basic defect in this family was in their platelets, which absorbed FVIII/VWF high-molecular-weight multimers at lower concentrations of ristocetin than did normal platelets. In addition, either in platelet-rich plasma or suspended in buffer, their platelets were aggregated by unmodified normal human FVIII/VWF without ristocetin. Since the abnormalities of plasma FVIII/VWF in this family may be secondary to the platelet abnormalities, the term "pseudo-von Willbrand's disease" may be suitably descriptive of their disorder.
一个患有轻度出血性疾病和间歇性血小板减少症的家族中的四名成员(来自四代),其血浆中与因子VIII/血管性血友病因子(FVIII/VWF)相关的特性水平降低,血浆中缺乏高分子量形式的FVIII/VWF(但血小板中的多聚体结构正常),并且瑞斯托霉素诱导的血小板聚集增加,如同IIB型血管性血友病。然而,与IIB型疾病中FVIII/VWF的异常不同,该家族的基本缺陷在于其血小板,与正常血小板相比,它们在较低浓度的瑞斯托霉素下就能吸收FVIII/VWF高分子量多聚体。此外,无论是在富含血小板的血浆中还是悬浮在缓冲液中,他们的血小板在没有瑞斯托霉素的情况下也会被未修饰的正常人FVIII/VWF聚集。由于该家族血浆FVIII/VWF的异常可能继发于血小板异常,“假性血管性血友病”这一术语可能恰当地描述了他们的病症。