Abstract Number: PB0722
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Rare Bleeding Disorders
Background: Congenital fibrinogen deficiency (CFD) is a rare disease associated with severe and/or frequent bleeding episodes (BEs). Human fibrinogen concentrate (HFC) has been demonstrated to restore hemostasis in the event of such bleeding.
Aims: To collect real-world evidence from clinical practice concerning the safety and efficacy of HFC for treatment of acute BEs in CFD.
Methods: FORMA-07 is a prospective, non-interventional, multicenter observational study in patients of any age with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia expected to require on-demand BE treatment with HFC. The study will examine this purified, plasma-derived, double virus inactivated/eliminated, lyophilized HFC (Fibryga®, Octapharma). This Octapharma-sponsored study will seek approval from independent ethics committees prior to study start. Freely given, written informed consent will be obtained from all patients at screening. Patients with other bleeding disorders, including acquired fibrinogen deficiency and dysfibrinogenemia; history of anti-fibrinogen inhibitors; or participating in concurrent clinical studies will be excluded. Study outline is shown in Figure 1.
FORMA-07 will investigate the use of fibrinogen concentrate in treatment of CFD, focusing on the safety profile, including serious adverse drug reactions (ADRs), and ADRs of special interest (i.e., incidence of thromboembolic ADRs and anaphylaxis). Hemostatic efficacy, assessed by the investigator using a 4-point objective scale within the first 2–24 hours following treatment, will also be assessed.
The study aims to enroll a minimum of 25 patients treated with HFC over 7 years to describe 105 BEs (at least 10 major BEs in 10 patients).
Results: FORMA-07 has commenced and is currently enrolling. Completion is expected by Q4 2027. Results will be monitored on an ongoing basis.
Conclusions: Real-world evidence from routine clinical use will further assess the safety profile of HFC, supporting clinical study data on the use of this HFC for bleeding treatment in patients with congenital fibrinogen deficiency.
To cite this abstract in AMA style:
Schwartz B, Solomon C, Knaub S, Peyvandi F. Safety and Efficacy of Fibrinogen Concentrate in Congenital Fibrinogen Deficiency: A Post-marketing Observational Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/safety-and-efficacy-of-fibrinogen-concentrate-in-congenital-fibrinogen-deficiency-a-post-marketing-observational-study/. Accessed March 22, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/safety-and-efficacy-of-fibrinogen-concentrate-in-congenital-fibrinogen-deficiency-a-post-marketing-observational-study/