Mauz-Körholz C, Budde U, Kruck H, Körholz D, Göbel U
Department of Paediatric Haematology/Oncology, Heinrich-Heine University Medical Centre, Düsseldorf, Germany.
Arch Dis Child. 1998 Mar;78(3):257-60. doi: 10.1136/adc.78.3.257.
Two patients with a long history of unexplained thrombocytopenia, eventually diagnosed with von Willebrand's disease (vWD) type 2B are reported. In one patient with platelet counts of 80 x 10(9)/l 1-desamino-8-D-arginine vasopressin (DDAVP) had a favourable effect during bleeding episodes. The second patient received intermediate purity von Willebrand's factor (vWF)/factor VIII concentrate (Haemate HS), which helped haemostasis during tooth extraction. It increased platelet counts from 15 to 30 x 10(9)/l, whereas platelet transfusions produced no increase, nor prevented severe bleeding during abdominal surgery. Thus the treatment of vWD type 2B might depend on the degree of thrombocytopenia. It is recommended that in patients with mild to moderately decreased platelet counts, DDAVP treatment can be tried, whereas in patients with severely decreased platelet counts intermediate purity vWF/factor VIII concentrate substitution is preferred. In addition, vWD type 2B should be considered in the differential diagnosis of any child with chronic thrombocytopenia as the treatment strategy is different.
本文报告了两名长期原因不明的血小板减少症患者,最终被诊断为2B型血管性血友病(vWD)。其中一名血小板计数为80×10⁹/L的患者,在出血发作期间,1-去氨基-8-D-精氨酸加压素(DDAVP)产生了良好效果。第二名患者接受了中纯度血管性血友病因子(vWF)/凝血因子VIII浓缩物(Haemate HS),这有助于拔牙时的止血。它使血小板计数从15×10⁹/L增加到30×10⁹/L,而血小板输注则未使其增加,也未能防止腹部手术期间的严重出血。因此,2B型vWD的治疗可能取决于血小板减少的程度。建议对于血小板计数轻度至中度降低的患者,可以尝试DDAVP治疗,而对于血小板计数严重降低的患者,首选中纯度vWF/凝血因子VIII浓缩物替代治疗。此外,由于治疗策略不同,在任何慢性血小板减少症儿童的鉴别诊断中都应考虑2B型vWD。