Evaluation of high concentration intranasal and intravenous desmopressin in pediatric patients with mild hemophilia A or mild-to-moderate type 1 von Willebrand disease.

OBJECTIVES: The objective of the study was to evaluate the safety and efficacy of high-concentration intranasal desmopressin (HCIN-DDAVP) 1.5 mg/mL, in patients weighing < or = 50 kg with mild hemophilia A or mild type 1 von Willebrand disease (VWD). STUDY
DESIGN: This was a single-center, nonrandomized, open-label, single-dose trial of HCIN-DDAVP. Nine boys with hemophilia A, 8 girls with mild VWD, and 8 boys with mild VWD were evaluated. HCIN-DDAVP responses were compared with historic IV-DDAVP responses in 13 of the patients.
RESULTS: HCIN-DDAVP administration resulted in statistically significant mean increases in factor VIII procoagulant activity, ristocetin cofactor, and von Willebrand factor antigen levels in each of the 3 study groups. Mean (+/- 1 SD) increase in factor VIII procoagulant activity was 25.7 +/- 11.9 U/dL in mild hemophilia A. Ristocetin cofactor increased 108.5 +/- 53.8 U/dL in girls and 95.8 +/- 36.0 U/dL in boys with mild VWD. Intravenous desmopressin acetate responses were comparable to HCIN-DDAVP responses in patients who received both preparations. Adverse events were mild and resolved without intervention.
CONCLUSION: We conclude that administration of 150 microg of high concentration intranasal desmopressin is safe and effective in patients weighing < or = 50 kg with mild hemophilia A or mild type 1 VWD.