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This European website, initiated and developed by CSL Behring, contains product specific information and promotional content on gene therapy in haemophilia B and is intended for an international audience of European healthcare professionals only. For more detailed information on the use of the product in your country, please visit www.cslbehring.com.
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Enter SitePatient experience
HEMGENIX® treatment is the first of its kind in haemophilia B.1,2 Innovative treatments like this require guidance and support from the very beginning of a patient’s journey. Understanding the benefits, risks, adverse events, impact on quality of life, eligibility and managing expectations is vitally important to achieving treatment success. A well supported journey will allow a patient to make informed decisions before, during, and after treatment.3
The Gene Therapy Patient Journey1,4
This timeline provides a guide to help you prepare your patients for their gene therapy journey, so they know what to expect from their healthcare team from beginning to end.
Current Schematic Patient Journey for Haemophilia
0-18 years of age
Diagnosis
- Start of close relationship between people with haemophilia and their health care professionals from the haemophilia treatment centre
Prophylactic treatment
- Started at less than 2 years of age
- Parents learn how to administer infusions to their children with haemophilia, then people with haemophilia learn to self-infuse
- Coagulation factor levels and bleeding events are monitored
Aware of Gene Therapy (GT)
- Transition to adult care
- Most people with haemophilia and/or their families have been aware of gene therapy since childhood
- Information received through health care professionals, patient associative groups and online sources
Long-term follow-up
- Bi-yearly or yearly follow up, with monitoring of bleeding rates and treatment adherence
- Individualised treatment adaptation
Can take years
Consider Gene Therapy
- Health care professionals identify those people with haemophilia who could benefit from gene therapy based on their medical circumstances
- Discussions are mostly driven by health care professionals, but can also be driven by people with haemophilia
Patient Journey for Haemophilia including Gene Therapy
18 years of age
Can last 1 to 6 months
Eligibility Assessment
- Health care professionals ensure patients meet gene therapy eligibility criteria, and consent for testing eligibility
- NAb AAV Test, and liver health assessment (blood test, ultrasound/FibroScan)
Shared-Decision Making Process
- Patient is informed of expected efficacy/safety outcomes and known unknowns about the selected gene therapy
- Implications are discussed about the 12-week intensive monitoring period and the need for long-term follow up
Final Go and Informed Decision
- Medical Team Assessment (checklist) is performed and the infusion is scheduled
- Person with haemophilia makes an informed decision to proceed, with confirmation of commitment for monitoring and long-term follow-up
Pre-Infusion
- Health care professionals inform the person with haemophilia about the procedure and post-infusion monitoring
gene therapy administration
- The gene therapy infusion is prepared
- Gene therapy is administered to the person with haemophilia as a one-time only intravenous infusion delivered in an outpatient setting
- Each dosing kit is personalized to the person with haemophilia
Gene Therapy Infusion Day
Short-Term Monitoring
- After administration of gene therapy, health care professionals will have regular follow-ups with the person with haemophilia to track how well gene therapy is working
- They will also support the person with haemophilia in their transition to infusion-free life
- The person with haemophilia will record use of coagulation factor products/bleeding episodes
- Weekly routine blood tests are performed for the first 3 months to confirm continuously elevated FIX level and to check for liver enzyme elevations
1-12 weeks post infusion
Long-Term Follow-Up
- The person with haemophilia is to record use of on-demand coagulation factor product/bleeding episodes
- Regular long-term monitoring of liver health, endogenous coagulation factor levels
- Patient experience sharing
12+ weeks post infusion
Service Materials
Resources
- Download Patient Brochure: A Brief Introduction To Gene Therapy
- Download Healthcare Professional Brochure: Gene Therapy In Haemophilia
- Download HEMGENIX® Mechanism Of Action Infographic
- View HEMGENIX® Mechanism Of Action Video
- View Mechanism Of Action Video Adapted For Patients: Gene Therapy In Haemophilia
- View Mechanism Of Action Video: Gene Therapy In Haemophilia
RISK MANAGEMENT PLAN AND ADDITIONAL RISK MINIMISATION MEASURES MATERIALS
DOSING AND ADMINISTRATION FOR HEMGENIX®1
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CLINICAL EVIDENCE
Find out about the efficacy, safety, and tolerability data behind HEMGENIX®
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About HEMGENIX®
Discover more about HEMGENIX® design, development and mechanism of action
References
- EU SmPC HEMGENIX® (European Medicine Agency, 2023).
- Pipe SW, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023;388:706-718
- Wang M, et al. The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making. Patient Preference and Adherence. 2022;16:1440-1447.
- Le Quellec S et al. The paradigm shift of gene therapy for haemophilia: impact on the patient journey. Poster presentation at ESGCT 2022, Edinburgh.