Desmopressin for the treatment of haemophilia.
The synthetic vasopressin analogue (1-deamino-8-D-arginine-vasopressin) increases plasma concentration of factor VIII and von Willebrand factor in normal subjects and patients with mild haemophilia A and von Willebrand disease. Since its first clinical use in 1977, desmopressin has become the treatment of choice for patients with haemophilia A and factor VIII coagulant activity (FVIII:C) > 5% and has spared several patients the risk of acquiring blood-borne viral infections due to the use of non-virally inactivated plasma-derived FVIII concentrates. An average two to sixfolds FVIII:C increase is typically observed in most patients and return to baseline occurs usually within 8 hours. Several clinical studies have demonstrated the clinical efficacy and safety of desmopressin and the availability of concentrated formulation for subcutaneous injection and of a nasal spray has paved the way to home-treatment. However, overall it appears that haemophilic children may have a lower rate of biologic response compared to adults and a minority of adult patients are not able to attain clinically useful FVIII:C levels post-desmopressin administration. Thus, in every patient with haemophilia A likely to be treated or candidate to an elective invasive procedure, a test-infusion/injection should be carried out to assess the future usefulness of the compound.
Castaman
G
G
Department of Hematology and Hemophilia, Thrombosis Center, San Bortolo Hospital, Vicenza, Italy. castaman@hemato.ven.it
D000281:Administration, Intranasal; D000328:Adult; D002648:Child; D003894:Deamino Arginine Vasopressin / Q000008:administration & dosage / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy*; D006490:Hemostatics / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D007275:Injections, Intravenous
D006490:Hemostatics; D003894:Deamino Arginine Vasopressin