PATIENT JOURNEY

The image is used for illustrative purposes only and does not represent actual patients.

WHAT MIGHT YOUR PATIENT’S HEMGENIX® JOURNEY LOOK LIKE?

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 start to finish.

HCP with clipboard

PATIENT IDENTIFICATION, COUNSELLING, AND SHARED DECISION-MAKING

Is HEMGENIX® suitable for your patients?

Consider discussing HEMGENIX® as a treatment option with your adult patients who may not be meeting their treatment goals.

Clinical1,2

  • Not achieving a trough FIX level to match their clinical needs
  • Have frequent or uncontrolled bleeding episodes
  • Suspected asymptomatic internal bleeding

Practical and logistical2

  • Have issues with the demands of their current treatment regimen
  • Limitation on where and how long they can travel due to the threat of running out of treatment and/or having to carry the vials with them
  • Have poor venous access, are needle phobic, and/or struggle with venous access skills

Mindset and lifestyle2

  • Dissatisfied with their current treatment regimen and looking for a different option that minimises restrictions on physical activity and travel
  • Have a higher expectation of what is possible living with haemophilia B and want to reduce the impact on their mental health and quality of life
  • Would like to be part of establishing a change within the management of haemophilia B
HCP talking with patient

ASSESS ELIGIBILITY

There are several clinical factors to consider, such as:1,2

  • FIX inhibitor tests*
  • Liver health screenings including ALT, AST, ALP, total bilirubin, hepatic ultrasound, and elastography
  • Assessment of comorbidities that might impact safety or effectiveness of gene therapy
  • Prior to treatment with HEMGENIX®, patients should be assessed for the titre of pre-existing neutralising anti-AAV5 antibodies†
Patient infusing

PRIOR TO INFUSION

Before prescribing HEMGENIX®, the specific eligibility tests must be completed.1

Following completion, eligible patients will be taken through the infusion day protocol and may be referred to one of the 3 hub sites in the UK, depending on their situation.4

The product is prepared and ready for administration.

Infusion equipment

INFUSION DAY

When planning for infusion day, there are important considerations to keep in mind:1,2

  • Confirm eligibility and that all required screenings are completed prior to scheduling a patient’s infusion
  • Consult with your patient to schedule their infusion day (which may require discussion and referral to the administration centre). Remind the patient that they need to maintain a consistent weight from prescription to administration
  • Review infusion day expectations with your patient
  • Once your patient’s infusion day is set, schedule and confirm your patient’s post-treatment monitoring appointments, and reinforce the importance of post-monitoring progress of HEMGENIX®
  • HEMGENIX® is a one-time dose that should take 1–2 hours‡ 
Patient looking at graph on computer monitor

POST INFUSION

Post-infusion, patients will be monitored for liver health and to assess FIX levels.1

Weekly monitoring is recommended for the first 3 months post-treatment to assess:1

  • Liver enzymes: Check for potential elevations that may require intervention. Continue to monitor transaminases in all patients who develop liver enzyme elevations until liver enzymes return to baseline
  • FIX activity: Continue to monitor FIX activity. Use of exogenous FIX concentrates before and after administration may impede assessment of endogenous, HEMGENIX®-derived FIX activity. It may take several weeks after treatment with HEMGENIX® for endogenous FIX levels to rise. Until then, infusions of FIX replacement product may be needed

After 3 months, the follow-up monitoring requirements for patients will decrease over time.1

Longer-term monitoring is recommended to assess the safety and efficacy of HEMGENIX® gene therapy. It will depend on your patient’s FIX activity levels, their stability, and any evidence of bleeds.1

If your patient has FIX activity levels >5 IU/dL, consider monitoring:1

  • Every 6 months during the second year post-administration
  • Every 12 months after the second year post-administration

After administration of HEMGENIX®, your patients are expected to enrol in the follow-up study, in which their progress will be monitored for 15 years.1

Patient talking to doctor

NAVIGATING LIFE AFTER TREATMENT WITH HEMGENIX®

Since HEMGENIX® is a one-time treatment for haemophilia B, it is believed to remove the need for lifelong FIX replacement therapy.1

As a result, patients who receive HEMGENIX® may likely experience a sudden change in their lifestyle that could make them feel that they have lost of part of their identity, and they may struggle with the adjustment to a life without regular FIX replacement.5

It is important that you check in with your patients both prior to treatment and during their follow-up appointments after receiving HEMGENIX®.

*In the case of a positive test result for human FIX inhibitors, a re-test within 2 weeks should be performed. If both the initial test and re-test results are positive, the patient should not receive HEMGENIX®.1

†Pre-existing neutralising anti-AAV5 antibodies above a titre of 1:678 may impede transgene expression at desired therapeutic levels and thus reduce the efficacy of HEMGENIX® therapy. There is a lack of data in patients with neutralising anti-AAV5 antibodies above 1:678. In 1 patient with a pre-existing neutralising anti-AAV5 antibody titre of 1:3212 in the clinical study, no FIX expression was observed and restarting of exogenous FIX prophylaxis was needed.1,3

‡The diluted product should be administered at a constant infusion rate of 500 mL/hour (8 mL/min). In the event of an infusion reaction during administration, the infusion rate should be slowed or stopped to ensure patient tolerability. If the infusion is stopped, it may be restarted at a slower rate when the infusion reaction is resolved (see section 4.4 of the SmPC).1

AAV5, adeno-associated viral vector serotype 5; ALP, alkaline phosphatase; ALT, alanine aminotransferase; AST, aspartate aminotransferase; FIX, factor IX.

References

1. HEMGENIX® (etranacogene dezaparvovec). Summary of product characteristics. 2. CSL Behring. Gene Therapy Academy. From Clinical Trial to Clinical Practice and Beyond. Amsterdam, 25–26 January 2024. 3. Pipe S et al. Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B. Blood. 2023;142(Suppl 1):1055. 4. Pipe SW et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023;388(8):706–718. 5. CSL Behring. Gene Therapy in Haemophilia B: An introduction. 2024.