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Published on: August 14, 2017
Desmopressin (DDAVP) responsiveness in children with von Willebrand disease
Shoshana Revel-Vilk1, Markus Schmugge, Manuel D Carcao
1Division of Hematology/Oncology, The Hospital for Sick Children, University of Toronto, Ontario, Canada.
Insights
Desmopressin (DDAVP) effectively treats von Willebrand disease (VWD) in most children, particularly those with type 1 VWD. Identifying DDAVP responders early can prevent the need for blood product transfusions.
Area of Science:
- Pediatric Hematology
- Hemostasis and Thrombosis
- Pharmacology
Background:
- Von Willebrand disease (VWD) is a common inherited bleeding disorder.
- Desmopressin (DDAVP) is a potential treatment for VWD, but response varies.
- Predicting DDAVP response in pediatric VWD is crucial for effective management.
Purpose of the Study:
- To determine the response patterns to desmopressin (DDAVP) in children diagnosed with von Willebrand disease (VWD).
- To identify predictors of desmopressin (DDAVP) response in pediatric patients with VWD.
Main Methods:
- Retrospective review of hospital records for children with type 1 (n=70) and type 2A (n=5) VWD.
- Analysis of DDAVP challenge tests conducted between January 1989 and June 2001.
- Response defined as >2-fold increase in VWF:RCo and FVIII:C levels above 0.3 IU/mL.
Main Results:
- 80% (56/70) of children with type 1 VWD responded to DDAVP.
- Age and baseline VWF:RCo and FVIII:C levels were positive predictors of DDAVP response.
- Non-responders (8/36) were significantly more likely to receive blood component therapy (75% vs 7%) compared to responders.
Conclusions:
- DDAVP challenge tests are recommended for newly diagnosed pediatric VWD patients to identify potential responders.
- Identifying DDAVP responders can guide treatment decisions and potentially avoid blood product exposure.
- The association between age and DDAVP response warrants further investigation.
Purpose:
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.
