Nasal spray desmopressin (DDAVP) for mild hemophilia A and von Willebrand disease

Objective: To evaluate the effect of a nasal spray preparation of desmopressin (DDAVP) on levels of factor VIII activity, ristocetin cofactor activity, and von Willebrand antigen as well as the length of bleeding time in patients with mild hemophilia A and von Willebrand disease; to determine whether effective hemostatic levels can be attained; and to compare the effect of this spray with that of standard intravenous desmopressin treatment. Design: Before-and-after trial in patients known to respond to intravenous desmopressin. Setting: Regional, comprehensive, hemophilia diagnosis and treatment center. Patients: A total of 22 patients, including 11 patients with von Willebrand disease, 8 patients with mild hemophilia A, and 3 symptomatic hemophilia carriers. Interventions: Patients were infused with desmopressin, 0.3 μg/kg body weight. At least 1 week later, they were taught to self-administer desmopressin nasal spray, 150 μg to each nostril. Measurements: In patients with hemophilia, the level of factor VIII activity was measured; in patients with von Willebrand disease, levels of factor VIII activity, ristocetin cofactor activity, and von Willebrand antigen as well as bleeding time were measured before and after each administration of desmopressin. Main Results: Desmopressin, when administered intravenously or intranasally, elevated levels of factor VIII, ristocetin cofactor, and von Willebrand antigen in both mildly hemophiliac patients and patients with von Willebrand disease when compared with baseline measures (P < 0.05). Factor VIII levels adequate for hemostasis were achieved by 82% of the hemophiliac patients. An abnormal bleeding time was corrected in the majority (62%) of patients with von Willebrand disease. Conclusion: A nasal spray preparation of desmopressin apparently was effective both in treating bleeding episodes and when used prophylactically for minor surgical procedures in several patients.