12th Congress of the European Hematology ... - Haematologica

trials. The aim of this retrospective study is to verify the efficacy and
safety of highly purified VWF/FVIII concentrates in treatment and prevention
of bleeding in inherited Von Willebrand disease (VWD). Methods.
103 patients were treated with study concentrates (Fanhdi and Alphanate)
from 2001 to 2006 at 15 Hemophilia Centers according to Italian guidelines
for VWD management. 47 males and 56 females, median age 50
years (range 6-83 years), had types 3 (10), 2A (14), 2B (20), 2M (7) and
DDAVP-unresponsive 1 VWD (52) with a median bleeding severity score
(BSS) of 8 (range 0-27). Results. Study drugs were given to treat 113 bleeding
episodes in 51 cases and to prevent excess bleeding during 111 surgical
procedures in 72 cases with a good/excellent clinical response in 97%
(bleedings) and 99% (surgeries). To prevent the recurrence of GI bleeding
(8) CNS hemorrhage (1) hemarthroses (1), menorrhagia/urogenital
bleeding (6), secondary prophylaxis was also carried out in 16 cases with
types 3 (3), 2A (2), 2B (2), 1 VWD (9) all characterized by BSS >6. A regimen
of 40 U VWF:RCo/kg every other day or twice a week, given over
a median period of 505 days (range 24-1484) totally prevented bleeding
in 13 cases, strongly reducing the incidence in the other ones. No adverse
reactions occurred in 4.909 infusions for a total of 10.505.000 IU FVIII.
Conclusions. These results confirm the efficacy and safety of study concentrates
not only in the management of bleedings and surgeries but also
in secondary prophylaxis in severe inherited VWD.
0093
PHARMACOKINETICS OF THE REFORMULATED B-DOMAIN DELETED RECOMBINANT
FACTOR VIII CONCENTRATE USING CHROMOGENIC AND ONE-STAGE ASSAYS WITH
POOLED NORMAL PLASMA AND REFACTO LABORATORY STANDARD
C. Santoro, 1 M.G. Mazzucconi, 1 P. Pignoloni, 1 A. Rago, 1 F. Biondo, 1
A. Iorio, 2 F. Ferrante, 2 F. Torelli, 1 G. Agnelli, 2 R. Foà1 1 Ematologia, ROME; 2 Internal and Vascular Medicine, PERUGIA, Italy
Background. The use of RLS in one stage clotting assay has been proposed
to reduce the underestimation of FVIII plasma concentration
measurements after infusion of BDD-rFVIII in hemophilia A patients.
Both BBD-rFVIII and RLS have been recently reformulated. Aims. Aim of
this study was to evaluate the performance of reformulated RLS in the
measurement of FVIII plasma concentration after infusion of the reformulated
BDD-rFVIII. Methods. In 13 hemophilia A patients, FVIII