Background. Because of the very short half-life of factor VII, prophylaxis is considered a difficult endeavor in FVII deficiency. Design and Methods. Clinical efficacy and safety of prophylaxis regimens, and indications for their use,...
moreBackground. Because of the very short half-life of factor VII, prophylaxis is considered a difficult endeavor in FVII deficiency. Design and Methods. Clinical efficacy and safety of prophylaxis regimens, and indications for their use, were evaluated in factor VII-deficient patients in the Seven Treatment Evaluation Registry. Results. Prophylaxis data (38 courses) were analyzed from 34 severe factor VII-deficient patients (<1-45 years, 21 female). Severest phenotypes (central nervous system, gastrointestinal, joint bleeding episodes) were highly prevalent. Twenty-one patients received recombinant activated factor VII (24 courses), four received plasma-derived factor VII, and ten fresh frozen plasma. Prophylaxis schedules clustered into 'frequent' (3 times weekly [n=23]) and 'infrequent' (≤2 times weekly [n=15]) courses. Excluding courses for menorrhagia, 'frequent' and 'infrequent' courses produced 18/23 (78%) and 5/12 (41%) 'excellent' outc...
