Hematology:
Key Publications
Have a Question? Use Our New Chat Feature! Chat with our Medical Information Specialists by clicking on the Chat with us now available in the lower right corner of this screen.
Gene Therapy Research in Hemophilia Publications
Gene therapy with etranacogene dezaparvovec for hemophilia B
Pipe SW, Leebeek FWG, Recht M, et al. N Engl J Med. 2023Request | Ask a Question | View Article
Gene Therapy Research in Hemophilia Posters
Etranacogene dezaparvovec-drlb hemophilia B gene therapy Phase 2b trial final results: Stable and durable FIX level expression over 5 years
Please see full prescribing information for HEMGENIX
IX-TEND: A Phase IV Observational, Long-term Follow-up Study on the Safety and Efficacy of Etranacogene Dezaparvovec in Patients with Hemophilia B
Please see full prescribing information for HEMGENIX
First Real-world Experience Administering Etranacogene Dezaparvovec Gene Therapy for People with Hemophilia B
Please see full prescribing information for HEMGENIX
Sustained efficacy and safety 3 years following infusion with etranacogene dezaparvovec in adults with severe or moderately severe hemophilia B in the Phase 3 HOPE-B clinical trial
Please see full prescribing information for HEMGENIX
HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec
Please see full prescribing information for HEMGENIX
Adult Patients With Hemophilia B and With a History of Chronic HCV/HBV Infection Receiving Liver Directed Gene Therapy Demonstrated Long-Term Bleeding Protection And Sustained FIX Activity: Efficacy and Safety Results 3 Years After Administration of a Single Dose of Etranacogene Dezaparvovec
Please see full prescribing information for HEMGENIX
Stable and durable factor IX levels over 4 years after etranacogene dezaparvovec gene therapy administration in a Phase 2b trial in patients with haemophilia B
Please see full prescribing information for HEMGENIX
Management of Infusion Reactions: Lessons from the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene Dezaparvovec in Adults with Hemophilia B
Please see full prescribing information for HEMGENIX
Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals With and Without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) With Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
Please see full prescribing information for HEMGENIX
Improvements in health-related quality of life in adults with severe or moderately severe hemophilia B after receiving etranacogene dezaparvovec gene therapy
Please see full prescribing information for HEMGENIX
Analysis of elevated alanine transaminase in HOPE B, a Phase 3 recombinant adeno associated viral 5 gene therapy trial in people with haemophilia B
Please see full prescribing information for HEMGENIX
Hemophilia B Publications
Tap Image to view Graphical Abstract
Request | Ask a Question | View Article
Hemophilia B Posters
Efficacy and Safety of Recombinant Factor IX Fusion Protein (rIX-FP) in Previously Untreated Patients with Hemophilia B
Please see full prescribing information for IDELVION
Safety and efficacy of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in pediatric patients with hemophilia B: A review of results from phase 3 trial studies
Please see full prescribing information for IDELVION
Hemophilia A Publications
Acquired Bleeding Publications
KCENTRA was approved by the United States (US) food and Drug Administration (FDA) on April 29, 2013. KCENTRA, under the trade name Beriplex or Confidex, has been approved in other countries since 1996. The specific drug product name that was used in the cited publications is referenced below:
Acquired Bleeding Posters
The Relationship of 4 Factor PCC or Plasma to INR and Factor Levels: An Exploratory Analysis of 2 Clinical Trials
Please see full prescribing information for KCENTRA
Management of oral anticoagulant reversal prior to emergency surgery: are we following guidelines in the United States?
Please see full prescribing information for KCENTRA
Von Willebrand Disease Publications
Pharmacokinetics, efficacy and safety of Humate-P® in von Willebrand disease
Dobrkovska, A et al, Haemophilia 1998Request | Ask a Question | View Article
Von Willebrand Disease Posters
Intermittent prophylaxis for heavy menstrual bleeding in von Willebrand disease
Please see full prescribing information for HUMATE-P
Fibrinogen Deficiency Publications
Request | Ask a Question | View Article
Pharmacokinetic properties of a pasteurized fibrinogen concentrate
W. Kreuz, E. Meili, K. Peter-Salonen., et al. Transfusion and Apheresis Science 2005Request | Ask a Question | View Article
Factor XIII Deficiency Publications
Important Safety Information for KCENTRA
WARNING: ARTERIAL AND VENOUS THROMBOEMBOLIC COMPLICATIONS
Patients being treated with Vitamin K antagonist therapy have underlying disease states that predispose them to thromboembolic events. Potential benefits of reversing VKA should be weighed against the risk of thromboembolic events, especially in patients with history of such events. Resumption of anticoagulation therapy should be carefully considered once the risk of thromboembolic events outweighs the risk of acute bleeding. Both fatal and nonfatal arterial and venous thromboembolic complications have been reported in clinical trials and postmarketing surveillance. Monitor patients receiving KCENTRA, and inform them of signs and symptoms of thromboembolic events. KCENTRA was not studied in subjects who had a thromboembolic event, myocardial infarction, disseminated intravascular coagulation, cerebral vascular accident, transient ischemic attack, unstable angina pectoris, or severe peripheral vascular disease within the prior 3 months. KCENTRA might not be suitable for patients with thromboembolic events in the prior 3 months.
KCENTRA is contraindicated in patients with known anaphylactic or severe systemic reactions to KCENTRA or any of its components (including heparin, Factors II, VII, IX, X, Proteins C and S, Antithrombin III and human albumin). KCENTRA is also contraindicated in patients with disseminated intravascular coagulation. Because KCENTRA contains heparin, it is contraindicated in patients with heparin-induced thrombocytopenia (HIT).
Hypersensitivity reactions to KCENTRA may occur. If patient experiences severe allergic or anaphylactic type reactions, discontinue administration and institute appropriate treatment.
In clinical trials, the most frequent (≥2.8%) adverse reactions observed in subjects receiving KCENTRA were headache, nausea/vomiting, hypotension, and anemia. The most serious adverse reactions were thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.
KCENTRA is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent and its variant (vCJD), cannot be completely eliminated.
Indications for KCENTRA
KCENTRA®, Prothrombin Complex Concentrate (Human), is a blood coagulation factor replacement product indicated for the urgent reversal of acquired coagulation factor deficiency induced by Vitamin K antagonist (VKA—eg, warfarin) therapy in adult patients with acute major bleeding or the need for urgent surgery or other invasive procedure. KCENTRA is for intravenous use only.
Please see full prescribing information for KCENTRA.
To report SUSPECTED ADVERSE REACTIONS, contact the CSL Behring Pharmacovigilance Department at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Important Safety Information for Humate-P
Antihemophilic Factor/von Willebrand Factor Complex (Human), HUMATE-P® is indicated for treatment and prevention of bleeding in adult patients with hemophilia A (classical hemophilia). HUMATE-P is also indicated in adult and pediatric patients with von Willebrand disease (VWD) for (1) treatment of spontaneous and trauma-induced bleeding episodes, and (2) prevention of excessive bleeding during and after surgery. This applies to patients with severe VWD, and patients with mild and moderate VWD for whom use of desmopressin is known or suspected to be inadequate. HUMATE-P is not indicated for the prophylaxis of spontaneous bleeding episodes.
HUMATE-P is contraindicated in individuals with a history of anaphylactic or severe systemic response to antihemophilic factor or von Willebrand factor preparations. Monitor for intravascular hemolysis and decreasing hematocrit values in patients with A, B, and AB blood groups who are receiving large or frequent doses. Also monitor VWF:RCo and FVIII levels in VWD patients, especially those undergoing surgery.
Thromboembolic events have been reported in VWD patients receiving coagulation factor replacement. Caution should be exercised and antithrombotic measures considered, particularly in patients with risk factors for thrombosis.
HUMATE-P is derived from human blood. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent and its variant (vCJD), cannot be completely eliminated.
In patients receiving HUMATE-P in clinical studies for treatment of VWD, the most commonly reported adverse reactions (reported by >5% of subjects) were allergic/anaphylactic reactions, including urticaria, chest tightness, rash, pruritus, and edema. For patients undergoing surgery, the most common adverse reactions are postoperative wound or injection-site bleeding, and epistaxis.
Please see full prescribing information.
To report SUSPECTED ADVERSE REACTIONS, contact the CSL Behring Pharmacovigilance Department at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
The information provided here is primarily intended for use by physicians and other healthcare professionals in the United States. The CSL Behring product listed may not have been approved in other countries and may not be available everywhere.
Important Safety Information for HEMGENIX
Warning and PrecautionsInfusion Reactions
Infusion reactions, including hypersensitivity reactions and anaphylaxis, may occur. Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or interrupt administration. Re-start administration at a slower infusion once resolved.
Hepatotoxicity/Hepatocellular Carcinoma
Post-dose, monitor for elevated transaminase levels. Consider corticosteroid treatment should elevations occur. The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. For patients with preexisting risk factors for hepatocellular carcinogenicity, perform regular (eg, annual) abdominal ultrasound and alpha-fetoprotein testing following administration.
Immune-mediated neutralization of the AAV5 vector capsid
Preexisting neutralizing anti-AAV antibodies may impede transgene expression at desired levels.
Monitoring Laboratory Tests
In addition to monitoring liver function, monitor for Factor IX activity and Factor IX inhibitors after administration.
Adverse ReactionsThe most common adverse reactions (incidence ≥5%) were elevated ALT, headache, blood creatine kinase elevations, flu-like symptoms, infusion-related reactions, fatigue, nausea, malaise, and elevated AST.
Indication for HEMGENIX
HEMGENIX®, etranacogene dezaparvovec-drlb, is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who:
- Currently use Factor IX prophylaxis therapy, or
- Have current or historical life-threatening hemorrhage, or
- Have repeated, serious spontaneous bleeding episodes.
HEMGENIX is for single use intravenous infusion only.
Contraindications: None.Please see full prescribing information for HEMGENIX.
To report SUSPECTED ADVERSE REACTIONS, contact the CSL Behring Pharmacovigilance Department at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.