Desmopressin (1-deamino-8-D-arginine vasopressin), administered intravenously or subcutaneously, has an established role in the treatment of patients with mild to moderate hemophilia A and type I von Willebrand disease [1-3]; it substitutes for plasma-derived coagulation products and avoids the risk for infection with bloodborne viruses. We evaluated the long-term safety and efficacy of home care use of high-concentration intranasal desmopressin in 20 patients with mild hemophilia A or type I von Willebrand disease, in whom coagulation variables were previously shown to respond to this therapy.

Despite promising short-term studies, no data other than one preliminary report [4] have been published on the long-term effectiveness of intranasal desmopressin. Intranasal desmopressin (Centeon, Collegeville, Pennsylvania) contains 1.5 mg of desmopressin per mL; 0.1 mL is administered with each actuation of the pump. The intranasal dosage for adults provides 300 µg from two sprays, one in each nostril (a child's dose is 150 µg) [5]. Thirty-two of 39 persons tested had a favorable response-correction of bleeding time, increase of von Willebrand antigen to at least 50%, increase of von Willebrand factor to at least 50%, and increase of factor VIII:C to at least 30%. These 32 patients were given intranasal desmopressin for home use; 10 patients with type I von Willebrand disease (males and females), 8 male patients with mild hemophilia A, and 2 female symptomatic hemophilia carriers actually used the therapy at home. For as long as 5 years, patients self-administered intranasal desmopressin to treat bleeding episodes and as prophylaxis before surgical and dental procedures. Patients reported excellent (requiring one treatment) or good (requiring two to four treatments) responses in 166 of 184 (90.2%) bleeding episodes. They reported excellent responses in all 27 episodes of trauma, dental work, and surgery. Adverse reactions were mild (similar to those reported in past studies) and did not result in discontinuation of intranasal desmopressin therapy in any case.

We conclude that for many patients with mild hemophilia A and von Willebrand disease, high-concentration intranasal desmopressin offers safe and effective long-term treatment for bleeding episodes and prophylaxis for minor surgical and dental procedures. For patients with adequate hemostatic response, this therapy can be considered first-line treatment for in-home use.