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A Retrospective Study to Evaluate the Efficacy and Safety of a Plasma-Derived, von Willebrand Factor-Containing FVIII Concentrate, in Patients with Von Willebrand Disease (VWD) in Spain: Interim Analysis Results

V. Jiménez-Yuste1, R. Mir2

1Hospital Universitario La Paz, Autónoma University, Madrid, Spain, 2Grifols, Scientific and Medical Affairs, Barcelona, Spain

Abstract Number: PB1551

Meeting: ISTH 2020 Congress

Theme: Platelet Disorders and von Willebrand Disease » VWF and von Willebrand Factor Disorders - Clinical Conditions

Background: Positive results on VWD therapy with Fanhdi® (Grifols) a plasma-derived, von Willebrand factor-containing FVIII concentrate (pdFVIII/VWF) have been reported in clinical trials. However, studies on use of Fanhdi® in standard clinical practice are scarce.

Aims: The main objective of this observational study was to retrospectively evaluate in an interim analysis the clinical efficacy and safety of Fanhdi® in standard clinical practice in patients with VWD in Spain.

Methods: Patients diagnosed with congenital VWD and treated with Fanhdi® for bleeding episodes or during surgical prophylaxis, when desmopressin is ineffective or contraindicated, were included in the study, which was granted the approval from the hospital Ethics Committee. Medical records of patients followed up from January 2011 to December 2017 were retrospectively collected. Efficacy of Fanhdi® achieving haemostasis was evaluated in bleeding episodes (mean dose ± SD: 45.33±8.24 IU/Kg) and in the prevention of surgical bleeding (mean dose ± SD: 36.66±11.28 IU/Kg) according to the following criteria: Excellent, Good, Poor, and No Response. For safety endpoints, related adverse events were assessed.

Results: Thirty-six patients (male [n=14]; female, [n=22]) with VWD type 1 (n=10), type 2 (n=17), and type 3 (n=9) were analysed. Median (P25;P75) age was 34.5 (19.5;50.0) years old (Table 1). A total of 61 bleeding episodes and 28 surgical procedures were reported. Overall, replacement therapy with Fanhdi® showed an Excellent to Good clinical efficacy in 96.7% of bleeding episodes and in 100% for the prevention of excessive bleeding during surgical procedures (Table 2). No related adverse events occurred during 318 infusions.

Conclusions: Awaiting the final study results, this interim analysis suggests that Fanhdi® can be efficacious and safe in the management of bleeding episodes and in the prevention of excessive bleeding during surgery in patients with VWD.

    TYPE OF VWD DIAGNOSED
  All types Type 1 Type 2A Type 2B Type 2M Type 2N Type 3
Parameter n=36 (100.0%)1 n =10, (27.7%) n =11, (30.5%) n=4, (11.1%) n=0, (0%) n =1 (2.7%) n=9 (25.0%)
Male/female, n (%) 14 (38.9)/ 22 (61.1) 5 (50.0)/ 5 (50.0) 4 (36.4)/ 7 (63.6) 2 (50.0)/ 2 (50.0) — 0 (0.0)/ 1(100.0) 3 (33.3)/ 6 (66.7)
Age, years, Median (P25;P75) 34.5 (19.5;50.0) 35.5 (20.0;69.0) 35.0 (22.0;62.0) 33.5 (21.0;45.5) — 36.0 (36.0;36.0) 34.0 (15.0;39.0)
1One of the patients with VWF Type 2 was not subclassified due to lack of information in the eCRD at the time of the interim analysis. This information will be included in the final report of the study.

[Table 1.]

Clinical situations n (%) episodes reported Efficacy of treatment with Fanhdi®
Bleeding episodes 61 (61.6%) Excellent 41 (67.2%)
  Good 18 (29.5%)
  Poor 2 (3.3%)
  No response 0 (0.0%)
Prevention of surgical bleeding 28 (28.3%) Excellent 19 (67.9%)
  Good 9 (32.1%)
  Poor 0 (0.0%)
  No response 0 (0.0%)

[Table 2.]

To cite this abstract in AMA style:

Jiménez-Yuste V, Mir R. A Retrospective Study to Evaluate the Efficacy and Safety of a Plasma-Derived, von Willebrand Factor-Containing FVIII Concentrate, in Patients with Von Willebrand Disease (VWD) in Spain: Interim Analysis Results [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/a-retrospective-study-to-evaluate-the-efficacy-and-safety-of-a-plasma-derived-von-willebrand-factor-containing-fviii-concentrate-in-patients-with-von-willebrand-disease-vwd-in-spain-interim-analy/. Accessed August 15, 2022.

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