Response to low-dose desmopressin by a subcutaneous route in children with type 1 von Willebrand disease

Abstract

ObjectiveThe primary objective of this study was to determine responses to low-dose desmopressin (DDAVP) by a subcutaneous route in children with type 1 VWD.MethodsThis study analyzed responses to low doses of DDAVP administered by a subcutaneous route to 14 children between the ages of 3 and 16 with type 1 VWD and a personal and familial history of bleeding. At 0 (baseline) and 1 hour (initial response) after the subcutaneous injection of DDAVP, the vital signs were assessed and blood samples were obtained for VWD panel determinations (VWF:Ag, VWF:RCo, FVIII:C levels, and Col/Epi, Col/ADP). At 4 hours (sustained response), only Col/Epi and Col/ADP were assessed.ResultsThe DDAVP mean (min–max range, μg/kg) based on the patient's weight was 0.15 (0.12–0.18). Laboratory values mean (min–max range in U/dl) baseline for VWF:RCo, VWF:Ag, and FVIII:C were 28 (20–36), 34 (25–42), and 40 (29–48), respectively. After a subcutaneous administration, the laboratory values mean (min–max range in U/dl−1) achieved for 1 hour for VWF:RCo, VWF:Ag, and FVIII:C were 109 (72–144), 132 (88–166), and 151 (96–198), respectively.PFA 100® CT (Col/Epi <134 seconds and Col/ADP <110 seconds) returned to normal values at 1 and 4 hours after a subcutaneous administration.ConclusionSubcutaneous low-dose DDAVP therapy is at least effective as 0.3 µg/kg intravenous therapy for children with type 1 VWD. This study shows that a wider use of DDAVP should be promoted, especially in developing countries.