Management of severe VWD with cryoprecipitate collected by repeated apheresis of a single dedicated donor.
BACKGROUND
Rare and severe forms of VWD are associated with trace or absent VWF. The feasibility of supporting a child with severe VWD from birth through age 12 with cryoprecipitate derived from DDAVP-stimulated plasma exchange of a single dedicated donor is reported.


METHODS
An infant with excessive hemorrhage at circumcision was found to have Type 3 VWD. His father carried an allele with a mutation at the level of VWF mRNA expression but did not have a history of bleeding. Cryoprecipitate was prepared from serial DDAVP-stimulated plasma exchanges of the father.


RESULTS
Repeated plasma-exchange donations were performed to provide all of the VWF needed for his son. An average of 14 cryoprecipitate units was prepared from each donation, and the units contained markedly elevated levels of FVIII:C. The cryoprecipitate was stored for up to 102 months. Components tested after more than 8 years of storage showed 48 to 130 percent of original FVIII:C activity. Ninety-seven percent of the bleeding episodes, such as epistaxis, tongue-biting accidents, and other minor lacerations, were successfully managed with a single 50- to 100-percent replacement dose of FVIII. The patient experienced normal growth and development and is free of any long-term sequelae attributable to his disease.


CONCLUSIONS
Cryoprecipitate prepared by repeated plasma exchange of a VWD carrier provided excellent hemostatic function, even after storage intervals of more than a year. Plasma exchange of a committed donor was a cost-effective and safe option for long-term management of VWD.
Pomper
Gregory J
GJ
Departments of Transfusion Medicine and Laboratory Medicine, Warren Grant Magnuson Clinical Center, National Institutes of Health, Bethesda, Maryland, USA. gpomper@wfubmc.edu
Rick
Margaret E
ME
Epstein
Jay S
JS
Read
Elizabeth J
EJ
Leitman
Susan F
SF
D001781:Blood Component Removal / Q000009:adverse effects; D001782:Blood Donors; D002944:Circumcision, Male / Q000009:adverse effects; D015925:Cryopreservation; D017707:Erythrocyte Transfusion; D005169:Factor VIII / Q000627:therapeutic use*; D005220:Fathers; D005340:Fibrinogen / Q000627:therapeutic use*; D006470:Hemorrhage / Q000209:etiology / Q000628:therapy; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D010951:Plasma Exchange; D012720:Severity of Illness Index; D013997:Time Factors; D014842:von Willebrand Diseases / Q000150:complications / Q000503:physiopathology* / Q000628:therapy*
C026912:cryoprecipitate coagulum; D005169:Factor VIII; D005340:Fibrinogen