Desmopressin for the treatment of haemophilia

Abstract

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.

How I treat von Willebrand disease

“…The drug should be used with caution in elderly patients with atherosclerotic disease or in the presence of cardiovascular disorders because a few cases of myocardial infarction and stroke have occurred in hemophiliacs and uremic patients given desmopressin. 47 The present case proved to be responsive to the desmopressin trial infusion. For dental procedures, we usually administer a single desmopressin subcutaneous (or intravenous) injection of the concentrated formulation.…”

Bolnik S Hemofilijo v Sloveniji: Od Leta 1967 Do Danes

“…zdravimo krvavitve v takem primeru s kombinacijo DDAVP, F VIII in antifibrinolitikov. 16,17 Organizacija samozdravljenja oz. zdravljenje na domu.…”

Molecular and clinical predictors of inhibitor risk and its prevention and treatment in mild hemophilia A

“…22,23 Desmopressin (typically at 0.3 mg/kg body weight) is usually administered intravenously, diluted in 50 to 100 mL saline, either infused over 20 to 30 minutes or subcutaneously when concentrated formulation is available, which could be more convenient for home treatment. The drug is also available with an intranasal spray administration; this can, however, result in variable adsorption with less FVIII and von Willebrand factor (VWF) increase.…”

“…Desmopressin induces a 2-to 5-fold increase of plasma FVIII and VWF levels after administration. 22,23 It has been suggested that FVIII levels of at least 30 U/dL are adequate for the treatment of spontaneous or posttraumatic bleeding, whereas FVIII levels higher than 50 U/dL are required to cover major surgery, 22,23 although in the latter case, levels of 80 to 100 U/dL should be achieved. However, no randomized or controlled clinical trial is available, and treatment modality remains almost empirical.…”

“…Although FVIII increase occurs in most cases, only 50% to 60% of patients achieve FVIII levels higher than 50 U/dL. 3,[23][24][25][26] The peak postdesmopressin depends in part on the patient's basal FVIII level 24,25 and age. Young children often have markedly lower responses to desmopressin than adults, but they may become responsive at an older age.…”