TY - JOUR
T1 - Desmopressin in moderate hemophilia A patients: a treatment worth considering
AU - Loomans, Janneke I.
AU - Kruip, Marieke J. H. A.
AU - Carcao, Manuel
AU - Jackson, Shannon
AU - van Velzen, Alice S.
AU - Peters, Marjolein
AU - Santagostino, Elena
AU - Platokouki, Helen
AU - Beckers, Erik
AU - Voorberg, Jan
AU - van der Bom, Johanna G.
AU - Fijnvandraat, Karin
AU - RISE Consortium
PY - 2018/2/28
Y1 - 2018/2/28
N2 - Desrnopressin 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 withhold this group of patients from desmopressin responsiveness. Among the six predictors that we identified for desmopressin-induced factor VIII rise, factor VIII activity and desmo-Nessin-induced rise in von Willebrand factor antigen had the strongest effect.
AB - Desrnopressin 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 withhold this group of patients from desmopressin responsiveness. Among the six predictors that we identified for desmopressin-induced factor VIII rise, factor VIII activity and desmo-Nessin-induced rise in von Willebrand factor antigen had the strongest effect.
KW - VON-WILLEBRANDS DISEASE
KW - FACTOR-VIII
KW - ENDOTHELIAL-CELLS
KW - VONWILLEBRANDS-DISEASE
KW - BLEEDING DISORDERS
KW - INTRANASAL SPRAY
KW - NASAL SPRAY
KW - DDAVP
KW - MILD
KW - TRANSPLANTATION
U2 - 10.3324/haematol.2017.180059
DO - 10.3324/haematol.2017.180059
M3 - Article
C2 - 29305412
SN - 0390-6078
VL - 103
SP - 550
EP - 557
JO - Haematologica-the Hematology Journal
JF - Haematologica-the Hematology Journal
IS - 3
ER -