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 ≤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 μg of high concentration intranasal desmopressin is safe and effective in patients weighing ≤50 kg with mild hemophilia A or mild type 1 VWD. (J Pediatr 2002;140:595-9)