High-dose DDAVP intranasal spray (Stimate) for the prevention and treatment of bleeding in patients with mild haemophilia A, mild or moderate type 1 von Willebrand disease and symptomatic carriers of haemophilia A.
An open-label multicentre trial was conducted to evaluate high-dose DDAVP (desmopressin acetate) intranasal spray (Stimate; 1.5 mg mL(-1)), for the control of bleeding in 333 patients with mild haemophilia A, mild or moderate type 1 von Willebrand disease, or symptomatic carriers of haemophilia A. Overall, 278 patients received 2170 doses of high-dose DDAVP intranasal spray (1.5 mg mL(-1)). Using study-defined guidelines, patients evaluated the efficacy of high-dose DDAVP intranasal spray (1.5 mg mL(-1)) as 'excellent' or 'good' in 743 (95%) of 784 bleeding episodes. It demonstrated 'excellent' results in 384 (93%) of 413 administrations for prophylaxis and in eight of eight uses prior to acute surgical or dental procedures. When used for the treatment of menorrhagia, the efficacy of high-dose DDAVP intranasal spray (1.5 mg mL(-1)) was rated as 'excellent' after 655 (92%) of 721 daily uses. Of 2170 doses of high-dose DDAVP intranasal spray (1.5 mg mL(-1)), 172 (8%) were associated with adverse events. A total of 272 adverse events were reported among 80 patients. Of these, 239 (88%) were mild or moderate in intensity and only one patient was removed from the study due to an adverse event. These results demonstrate the safety and efficacy of high-dose DDAVP intranasal spray (1.5 mg mL(-1)) for control of bleeding episodes in patients with mildly decreased levels of factor VIII, von Willebrand factor, or both.
Leissinger
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C
Tulane University School of Medicine, Department of Hematology/Oncology, New Orleans, LA, USA.
Becton
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D
Cornell
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Cox Gill
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J
D000281:Administration, Intranasal; D000293:Adolescent; D000328:Adult; D016063:Blood Loss, Surgical / Q000517:prevention & control; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003257:Consumer Product Safety; D003894:Deamino Arginine Vasopressin / Q000008:administration & dosage* / Q000633:toxicity; D005260:Female; D005500:Follow-Up Studies; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000188:drug therapy* / Q000209:etiology / Q000517:prevention & control*; D006579:Heterozygote; D006801:Humans; D008297:Male; D008595:Menorrhagia / Q000188:drug therapy / Q000517:prevention & control; D008875:Middle Aged; D013810:Therapeutic Equivalency; D014842:von Willebrand Diseases / Q000150:complications / Q000188:drug therapy*
D003894:Deamino Arginine Vasopressin