ASH Clinical News February 2016 | Page 38

CLINICAL NEWS Written in Blood Study Provides New Molecular Insight into the Increased Risk of VTE in African Americans African Americans are at a dramatically higher risk for developing venous thromboembolism (VTE), with a 30- to 60-percent higher risk than other ethnicities. The reasons behind this increased incidence are largely unknown, but the authors of a study published in Blood have found that certain genetic variants may be responsible for African Americans’ susceptibility to VTE. Previous studies of white and European patients with VTE have identified specific genetic risk variants, prompting Wenndy Hernandez, PhD, from the Department of Medicine in the Section of Genetic Medicine at the University of Chicago, and colleagues to study whether genetic factors could also contribute to VTE risk in African Americans. “Clinicians know that VTE is a multifactorial disease with genetics explain- FEIBA [Anti-Inhibitor Coagulant Complex] For Intravenous Use, Lyophilized Powder for Solution Brief Summary of Prescribing Information. Please see package insert for full Prescribing Information. WARNING: THROMBOEMBOLIC EVENTS • Thromboembolic events have been reported during post-marketing surveillance following infusion of FEIBA, particularly following the administration of high doses and/or in patients with thrombotic risk factors. • Monitor patients receiving FEIBA for signs and symptoms of thromboembolic events. INDICATIONS AND USAGE FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for: • Control and prevention of bleeding episodes • Perioperative management • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes. FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX. CONTRAINDICATIONS • Known anaphylactic or severe hypersensitivity reactions to FEIBA or any if its components, including factors of the kinin generating system. • Disseminated intravascular coagulation (DIC). • Acute thrombosis or embolism (including myocardial infarction). WARNINGS AND PRECAUTIONS Thromboembolic Events Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur with FEIBA, particularly following the administration of high doses (above 200 units per kg per day) and/or in patients with thrombotic risk factors [see ADVERSE REACTIONS]. Patients with DIC, advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with recombinant factor VIIa have an increased risk of developing thrombotic events due to circulating tissue factor or predisposing coagulopathy. Potential benefit of treatment with FEIBA shou ld be weighed against the potential risk of these thromboembolic events. Monitor patients receiving more than 100 units per kg of body weight of FEIBA for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue the infusion and initiate appropriate diagnostic and therapeutic measures. Hypersensitivity Reactions Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur following the infusion of FEIBA. The symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. These reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of FEIBA and provide appropriate supportive care. Transmission of Infectious Agents Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents, e.g., viruses, and the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. The risk has been minimized by screening plasma donors for prior exposure to certain viruses, by testing for the presence of certain current virus infections and by inactivating and removing certain viruses during the manufacturing process [see DESCRIPTION in full Prescribing Information]. Despite these measures, the product may still potentially transmit human pathogenic agents. There is also the possibility that unknown infectious agents may still be present. All infections thought by a physician to have been possibly transmitted by this product should be reported by the physician or other healthcare providers to Baxter Healthcare Corporation, at 1-800-423-2862 (in the U.S.) and /or to FDA Med Watch (1-800-FDA-1088 or www.fda.gov/medwatch). ADVERSE REACTIONS The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting. Baxalta and Feiba are trademarks of Baxalta Incorporated January 2016 USBS/145/15-0065 The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis. Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. The safety assessment of FEIBA is based on the review of the data from two prospective clinical trials in which FEIBA was used for the treatment of acute bleeding episodes and a prospective trial that compared the use of FEIBA prophylactically versus on-demand treatment. The adverse reactions reported from two prospective clinical trials in which FEIBA was used for the treatment of acute bleeding episodes were chills, chest pain, chest discomfort, dizziness, dysgeusia, dyspnea, hypoesthesia, increase of inhibitor titer (anamnestic response), nausea, pyrexia, and somnolence. Specifically, the first trial was a multicenter randomized, double-blind trial in 15 hemophilia A subjects with inhibitors to factors VIII. The second trial was a multicenter FEIBA study conducted in 44 hemophilia A subjects with inhibitors, 3 hemophilia B subjects with inhibitors and 2 acquired factor VIII inhibitor subjects. Of the 489 infusions used to treat acute bleeds during the second trial, 18 (3.7%) caused minor transient reactions of chills, fever, nausea, dizziness and dysgeusia. Out of 49 subjects, 10 (20%) had a rise in their inhibitor titers after treatment with FEIBA. Five of these subjects (50%) had increases that were, tenfold or more, and 3 (30%) of these subjects received factor VIII or IX concentrates within 2 weeks prior to treatment with FEIBA. These anamnestic rises were not associated with decreased efficacy of FEIBA. Table 2 lists the adverse reactions in >5% of subject reported in the randomized, prospective prophylaxis trial comparing FEIBA prophylaxis with on-demand treatment in 36 hemophilia A and B subjects with inhibitors to factors VIII or IX. The trial population included 33 (92%) subjects with hemophilia A and 3 (8.3%) subjects with hemophilia B. Four (11%) subjects were ≥7 to <12 years of age, 5 (14%) were ≥12 to <16 years of age, and 27 (75%) were ≥16 years of age. A total of 29 (80.6%) subjects were Caucasian, 3 (8.3%) Asian, 2 (5.6%) Black/African American, and 2 (5.6%) other. The subjects received a total of 4,513 infusions (3,131 for prophylaxis and 1,382 for on-demand). Adverse reactions were defined as adverse events that occurred (a) within 24 hours after being infused or (b) adverse events assessed related or possibly related or (c) adverse events for which the investigator’s or sponsor’s opinion of causality was missing or indeterminate. Table 2 Prophylaxis Study Adverse Reactions (ARs) in >5% of Subjects MedDRA System Organ Class Blood And Lymphatic System Disorders Gastrointestinal Disorders Investigations Musculoskeletal And Connective Tissue Disorders Number of ARs Preferred Term Number of Subjects Percent of Subjects (N=36) Anemia 2 2 5.6 Diarrhea Nausea Vomiting Hepatitis B Surface Antibody Positive 2 2 2 2 2 2 5.6 5.6 5.6 4 4 11.1 5 3 8.3 Hemarthrosis Post-Marketing Experience Because post-marketing reporting of adverse reactions is voluntarily and from a population of uncertain size, it is not always possible to reliably estimate the frequency of these reactions or establish a causal relationship to product exposure. BLOOD AND LYMPHATIC SYSTEM DISORDERS: disseminated intravascular coagulation CARDIAC D ISORDERS: tachycardia, flushing RESPIRATORY, THORACIC, AND MEDIASTINAL DISORDERS: bronchospasm, wheezing GASTROINTESTINAL DISORDERS: abdominal discomfort SKIN AND SUBCUTANEOUS TISSUE DISORDERS: pruritus GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS: malaise, feeling hot, injection site pain DRUG INTERACTIONS Concomitant Medications Consider the possibility of thrombotic events when systemic antifibrinolytics such as tranexamic acid and aminocaproic acid are used during treatment with FEIBA. No adequate and wellcontrolled studies of the combined or sequential use of FEIBA and recombinant factor VIIa or antifibrinolytics have been conducted. Use of antifibrinolytics within approximately 6 to 12 hours after the administration of FEIBA is not recommended. ing only one piece of the risk,” Minoli A. Perera, PharmD, PhD, corresponding author of the study, told ASH Clinical News. “Our study shows that the genetic tests used clinically for VTE risk assessment are not informative for African Americans because, though they have a higher prevalence of the disease, they do not carry these genetic mutations as a population.” Dr. Hernandez and investigators conducted a genome-wide association study that included a two-stage analysis of African-American patients with VTE: a discovery cohort followed by an examination of the most significant single nucleotide polymorphisms (SNPs) in an independent replication cohort. Patients included in both parts of the study were unrelated, selfdescribed as African-American, and were 18 years old or older. The following data related to potential risk factors for VTE were collected: • Age • Height • Weight • Ethnicity • Sex Patients had a documented history of VTE (specifically proximal deep-vein thrombosis or pulmonary embolism) and did not have strong known risk factors such as prolonged hospitalization, surgery, active cancer or history of malignancy, pregnancy or puerperium, oral contraceptive use, menopausal replacement therapy, or protein C/S deficiency. The discovery cohort included 146 patients with VTE and 433 controls, while the replication cohort had 94 patients with VTE and 65 controls. In the discovery cohort, seven SNPs were identified that more than doubled the risk of VTE. The following SNPs reached genome-wide significance: • rs73692310 on chromosome 7 (odds ratio [OR] = 3.04; 95% CI 2.0-4.7; p=0.1.73x109) • rs58952918 on chromosome 18 (OR=2.48; 95% CI 1.7-3.7; p=1.07x10-8) • rs28496996 on chromosome 18 (OR=2.44; 95% CI 1.6-3.6; p=1.0x10-8) The following SNPs also had a strong suggestive association with VTE risk: • rs2144940 on chromosome 20 (OR=2.18; 95% CI 1.6-2.9; p=3.52x10-7) • rs2567617 on chromosome 20 (OR=2.17; 95% CI 1.6-2.9; p=4.01x10-7) February 2016