To report an adverse event or product quality complaint, call 1-877-TAKEDA-7 (1-877-825-3327)
To report an adverse event or product quality complaint, call 1-877-TAKEDA-7 (1-877-825-3327)
Not an actual patient.
As with Hemophilia A, the gene for encoding FIX is on the X chromosome (recessive), which means Hemophilia B occurs primarily in males.1 Females are typically carriers of the gene and can pass it on to their children, but can occasionally be symptomatic carriers.
Globally, it is estimated that more than 1.1 million males have hemophilia, including 418,000 with severe hemophilia.1 Hemophilia B accounts for an estimated 15-20% of cases worldwide.
Within the U.S., it is estimated that 1 in 19,283 males are born with Hemophilia B.2 Data from the U.S. Centers for Disease Control and Prevention (CDC) estimated that the total number of patients with Hemophilia B in the U.S., between 2012 and 2022, was 7,253.3
According to the hemophilia treatment centers population profile (HTC PP), about 28.1% of male patients with Hemophilia B have mild disease, 40.9% have moderate disease, and 30.0% have severe disease.4
Hemophilia B is caused by a variety of inherited or spontaneous mutations in the gene encoding FIX.1
FIX plays a key role in the contact activation pathway, or intrinsic pathway, for blood coagulation following an injury.6 Following damage to endothelial surface, FIX is cleaved by Factor VIIa into its active form, FIXa. FIXa, in turn, cleaves Factor X to create Factor Xa, which is instrumental in the generation of thrombin, necessary for the formation of fibrin clots.
The level of deficiency in functional FIX, as detected in factor assays, is associated with the severity of bleeding symptoms in Hemophilia B.1
The diagnosis of hemophilia is based on 3 principles:1
A surveillance project evaluating 864 male infants with hemophilia seen at U.S. hemophilia treatment centers (aged 0 to 2 years) found that 73% of them were diagnosed by 1 month of age.5 The diagnosis in these cases was prompted by a known carrier mother (47.2% of cases), a family history (23.2% of cases), and a bleeding event or unusual bruising (28.8% of cases).
In approximately one-third of severe cases there is no known family history, suggesting a spontaneous mutation.1,5
Joints (most commonly the elbow, knee, and ankle) are the site of approximately 92% of bleeds in severe hemophilia.1,7 Other common sites of bleeding are the muscles, the brain, and mucosal tissues (mouth, epistaxis, gastrointestinal, and genitourinary tract).1 Recurrent bleeding into the same joint may eventually cause hypertrophic synovitis, progressive cartilage degradation, hemophilic arthropathy, and significant impairment of joint function.7,8
Treatment of Hemophilia B involves replacement therapy in which the deficient endogenous FIX is replaced via intravenous infusion.9,10 The standard of care in Hemophilia B has shifted towards prophylactic treatment, with the goal of preventing all bleeds, rather than on-demand or episodic treatment in response to a bleeding episode. World Federation of Hemophilia (WFH) guidelines recommend individualized prophylaxis based on bleeding phenotype, joint status, individual pharmacokinetics, and patient self-assessment and preference.1
The most severe treatment-related complication in hemophilia is the development of inhibitors, which are alloantibodies to administered factor replacement therapy.11 The lifetime incidence of inhibitors is lower in Hemophilia B than in Hemophilia A (3-5% of severe Hemophilia B patients vs 25-30% of severe Hemophilia A patients). In contrast with FVIII inhibitors, FIX inhibitors are associated with severe anaphylactic reactions to the infusion of FIX-containing products in about half of patients with inhibitors.
This is not intended to be a comprehensive resource of all congresses and congress materials across therapeutic and disease areas. Congress materials may include information about investigational use(s) of compounds/products that are not approved for use by the U.S. Food and Drug Administration (FDA) and/or are inconsistent with the Prescribing Information. Takeda does not recommend the use of any Takeda product beyond the approved labeling. Any decisions regarding the usage of a Takeda product beyond the approved labeling are left to the discretion of the healthcare professional. Takeda makes no representations about whether investigational compounds or unapproved uses will be approved by the FDA.
The premier global congress from the world's largest professional society serving both clinicians and scientists working in malignant and classical hematology.
The THSNA Summit is a collaboration of the 13 leading non-profit organizations in the fields of Thrombosis and Hemostasis. The Summit provides a focused forum for over 1,000 attendees with an interest in bleeding and clotting disorders to network, learn, and share across disciplines and disease states. The educational programming is organized in a series of plenary presentations, educational track session, oral abstract presentations and digital poster sessions.
Conference delivers vital education and networking opportunities to subspecialist investigators and practitioners from every sector of the field as well as hospitalists, physician assistants, advanced nurse practitioners, other advanced practice providers, and allied healthcare professionals.
Brings together leading experts in blood disorders research and top clinicians to share their perspectives through ground-breaking presentations and robust Q&A sessions. Together, we’ll explore how ATHN’s unique data infrastructure supports research projects that leverage data to answer scientifically and clinically meaningful questions.
The BIC International Conference is traditionally addressed to a relatively narrow selection of medical specialties since it is meant to feature only the most novel advances of basic science and clinical research in haemophilia, rare inherited coagulation disorders, von Willebrand factor and disease, gene therapy and thrombotic microangiopathies.
Global congress featuring the world’s leading experts on thrombosis, hemostasis and vascular biology presenting the most recent advances, the latest science, and the newest clinical applications designed to improve patient care.
Annual event with more than 2,500 members and non-members of the AMCP to engage on the latest innovations and most intentional networking in managed care pharmacy.
Congress focusing on the practical management of patients living with or at risk for cancer and best practices for cancer care delivery.
Scientific meeting open to all medical, research, and allied health professionals interested in disorders of hemostasis and thrombosis.
The premier global congress from the world's largest professional society serving both clinicians and scientists working in malignant and classical hematology.
This resource provides information on Takeda medications available in the Hemophilia B category and is not intended to represent a complete list of therapeutic options.
(anti-inhibitor coagulant complex)
[Coagulation Factor IX (Recombinant)]
Watch videos focused on Hemophilia.
Learn more about GOAL-Hēm, a hemophilia-specific patient-centered outcome measure and clinical engagement tool to aid in individualized goal setting.
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