Desmopressin (DDAVP) responsiveness in children with von Willebrand disease.
OBJECTIVE
To determine the pattern and predictors of response to desmopressin (DDAVP) in children with von Willebrand disease (VWD).


METHODS
The authors reviewed the hospital records of all children with type 1 (n = 70) and type 2A (n = 5) VWD who were followed in the institution's Bleeding Disorders Clinic from January 1989 to June 2001 and who had a DDAVP challenge test after diagnosis. The major outcome evaluated was response to DDAVP, defined as an increase of greater than twofold over baseline of von Willebrand factor, ristocetin cofactor (VWF:RCo), and factor VIII coagulant (FVIII:C) and levels above 0.3 IU/mL.


RESULTS
Response to DDAVP was observed in 56 (80%) of the 70 children with type 1 VWD. Age and baseline VWF:RCo and FVIII:C levels were positively associated with DDAVP response. A total of 36 children (28 responders, 8 nonresponders) with type 1 VWD were treated for bleeding episodes or for prophylaxis; of these 75% (6/8) of the nonresponders compared with 7% (2/28) of the responders to a DDAVP challenge test received blood component therapy (P < 0.01). One of the five children with type 2A VWD responded to DDAVP.


CONCLUSIONS
DDAVP challenge tests are recommended in children with newly diagnosed VWD to identify responders in whom DDAVP may be used for the prevention or treatment of bleeding, thus avoiding exposure to blood products. The association of DDAVP response with age merits further investigation.
Revel-Vilk
Shoshana
S
Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Ontario, Canada.
Schmugge
Markus
M
Carcao
Manuel D
MD
Blanchette
Phillip
P
Rand
Margaret L
ML
Blanchette
Victor S
VS
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003894:Deamino Arginine Vasopressin / Q000627:therapeutic use*; D005169:Factor VIII / Q000032:analysis; D005260:Female; D006801:Humans; D008297:Male; D012189:Retrospective Studies; D014842:von Willebrand Diseases / Q000097:blood / Q000188:drug therapy*
D005169:Factor VIII; D003894:Deamino Arginine Vasopressin