Desmopressin in moderate hemophilia A patients: a treatment worth considering

Abstract

Desmopressin increases endogenous factor VIII levels in hemophilia A. Large inter-individual variation in the response to desmopressin is observed. Patients with a lower baseline factor VIII activity tend to show a reduced response, therefore, desmopressin is less frequently used in moderate hemophilia A patients (baseline factor VIII activity 1-5 international units/deciliter), even though factor VIII levels may rise substantially in some of them. We aim to describe the response to desmopressin in moderate hemophilia A patients and to identify predictors. We selected data on 169 patients with moderate hemophilia from the multicenter Response to DDAVP In non-severe hemophilia A patients: in Search for dEterminants (RISE) cohort study. Adequate response to desmopressin was defined as a peak factor VIII level ≥ 30, and excellent response as ≥ 50 international units/deciliter after desmopressin administration. We used univariate and multiple linear regression techniques to analyze predictors of the peak factor VIII level. Response was considered adequate in 68 patients (40%), of whom 25 showed excellent response (15%). Intravenous administration, age, pre-desmopressin factor VIII activity and von Willebrand factor antigen, peak von Willebrand factor activity and desmopressin-induced rise in von Willebrand factor antigen were significant predictors of peak factor VIII level and explained 65% of the inter-individual variation. In 40% of moderate hemophilia A patients, desmopressin response was adequate, thus it is important not to with-hold this group of patients from desmopressin responsiveness. Among the six predictors that we identified for desmopressin-induced factor VIII rise, factor VIII activity and desmopressin-induced rise in von Willebrand factor antigen had the strongest effect.

Pattern of Blood Products Transfusions and Reactions among Multi-Transfused Haemophiliacs in Nigeria : Implications on Haemophilia Care in Low Resource Tropical Settings

“…(5) Desmopressin raises vWF and FVIII levels, while anti-fibrinolytic agents inhibit fibrin clot degradation, and have all been shown to be useful in controlling haemophilic bleeding especially in patients with mild and moderate haemophilia. (31,32) Tranexamic acid in particular has been widely and successfully used alone or in combination with other antihaemophilic agents in the prevention and management of various haemophilic bleedings due to accidental or surgical trauma in haemophiliacs. (32) The only exception to the use of anti-fibrinolytic agents is urinary tract bleeding because of the possible risk of obstructive uropathy that may occur as a result of fibrin clot formation in the urinary tract.…”

Prevalence, pattern and clinical implications of transfusion transmissible viral infections among paediatric haemophiliacs in northern Nigeria

“…Second, the lack of adequate use of pharmacological agents such as desmopressin and antifibrinolytic agents (epsilon amino caproic acid and tranexamic acid) was identified in a previous study as a major limitation in the management of haemophilia in tropical developing countries [8] . Desmopressin raises vWF and FVIII levels, while antifibrinolytic agents inhibit fibrin clot degradation; all have been shown to be useful in controlling haemophilic bleeding, especially in patients with mild and moderate haemophilia [36,37] . Tranexamic acid in particular has been widely and successfully used alone or in combination with other anti-haemophilic agents in the prevention and management of bleeding due to accidental or surgical trauma in haemophilia patients [37] .…”

Inhibitor Risk Stratification and Individualized Treatment in Patients With Nonsevere Hemophilia A: A Single-Institution Practice Audit

“…Given that the RISE study demonstrated a partial/complete DDAVP response in 21% of patients with moderate hemophilia A, DDAVP should not be completely discarded from the therapeutic armamentarium for moderate hemophilia A, especially as an adjunct agent to reduce FVIII exposure. 16…”