Hematology
35+ Years Treating Hemophilia
CSL Behring has over 35 years of experience in the field of hematology. From disorders that span the coagulation cascade to the management of urgent reversal of acquired bleeding, CSL Behring continues to invest in this area and remains committed to continuing our innovations in this field.
Key Publications
Gene Therapy Research in Hemophilia B
Gene therapy with etranacogene dezaparvovec for hemophilia B
Pipe SW, Leebeek FWG, Recht M, et al. N Engl J Med. 2023
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Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy
von Drygalski A, Gomez E, Giermasz A, et al. Blood Adv 2022
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Gene Therapy Research in Hemophilia B Posters
Adults With Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection After 24 Months’ Follow-up
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
Multiple-year durability data from a Phase 2b trial of gene therapy with etranacogene dezaparvovec in people with hemophilia B
Please see full prescribing information for HEMGENIX
Hemophilia B
Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children. Results of a phase 3 trial
G. Kenet, Chambost H.. J. Thrombosis and Hemostasis, 2016
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Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial
E. Santagostino, U. Martinowitz, et al. Blood, April 2016
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Efficacy and safety of long-acting recombinant fusion protein linking factor IX with albumin in haemophilia B patients undergoing surgery
E. Negrier et al. Haemophilia 2016
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Hemophilia A
Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A
Mahlangu, J. Blood, 2016
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Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial
Stasyshn, O., J. Thrombosis and Hemostasis, 2017
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Acquired Bleeding
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:
Go et al. 2022
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Hood et al. 2022
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Milling TJ Jr, et al., Ann Emerg Med 2016
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Goldstein JN, et al., Lancet 2015
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Refaai, MA, et al., Transfusion 2015
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Sarode R, et al., Circulation 2013
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Pabinger I, et al., Ann Hematol 2010
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Pabinger I, et al., Thromb Haemost 2008
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Ostermann H, et al., Thromb Haemost 2007
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Lorenz R, et al., Eur J Gastroenterol Hepatol 2003
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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
Pharmacokinetics, efficacy and safety of Humate-P® in von Willebrand disease
Dobrkovska, A et al, Haemophilia 1998
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von Willebrand factor/factor VIII concentrate (Humate-P) for management of elective surgery in adults and children with von Willebrand disease
Gill JC, et all. Haemophilia 2011
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von Willebrand factor/factor VIII concentrate (Haemate P) dosing based on pharmacokinetics: a prospective multicenter trial in elective surgery
Lethagen, S. et al J Thromb Haemost. 2007
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Fibrinogen Deficiency
Efficacy and tolerability of a pasteurized human fibrinogen concentrate in patients with congenital fibrinogen deficiency
Kreuz W, Meili E, Peter-Salonen K, et al. Transfusion and Apheresis Science 2005
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Pharmacokinetic properties of a pasteurized fibrinogen concentrate.
W. Kreuz, E. Meili, K. Peter-Salonen., et al. Transfusion and Apheresis Science 2005
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Factor XIII Deficiency
Pharmacokinetics and safety of plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency
Nugent, D.j et al , Haemophilia 2015
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Efficacy and safety of prophylactic treatment with plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency
Ashley, C. Et al, Haemophilia 2015
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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.