Clin Pediatr Hematol Oncol.  2021 Apr;28(1):54-57. 10.15264/cpho.2021.28.1.54.

Recombinant von Willebrand Factor for Pediatric Patient with von Willebrand Disease: First Report in Korea

• Kim S
• Park YS

• KMID: 2515345
• DOI: http://doi.org/10.15264/cpho.2021.28.1.54

Abstract

von Willebrand disease (VWD) is the most common hereditary bleeding disorder. The treatment of VWD consists mainly of desmopressin and plasma-derived von Willebrand factor (pd-VWF) concentrate. We report on the first patient with VWD to be treated with recombinant VWF (rVWF) concentrate in Korea. Our patient was diagnosed with type 2 VWD at 10 months of age and suffered persistent severe epistaxis despite therapeutic levels of VWF activity and factor VIII (FVIII). At 34 months of age, rVWF was initiated and administered a total of 15 times in the following 8 months. No drug-related adverse events were observed and the patient was neither admitted nor given any transfusions during this period. Unlike pd-VWF/FVIII concentrates, rVWF did not increase FVIII to the excessively high levels that constitute a risk factor for thromboembolism, and was also preferable to pd-VWF/FVIII concentrates in that it contains ultra-large multimers of VWF. This is the first reported case in Korea in which rVWF was used to treat VWD. rVWF may be well tolerated and effective in VWD patients, especially those with refractory bleeding.

Keyword

Recombinant von Willebrand factor concentrate; Plasma derived von Willebrand factor concentrate; von Willebrand disease