Andrea shares her experience treating people with HEMGENIX gene therapy
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Andrea, APNP, works at a center in Wisconsin, and has administered HEMGENIX gene therapy to some of the first people in the U.S. following FDA approval. Andrea also currently sits on the Region V-West Regional Council and Regional Gene Therapy Working Group.
We recently caught up with Andrea to talk about her experience treating people with HEMGENIX, and highlights from our discussion follow.
Please note that Andrea is sharing her patients’ experiences and others’ experiences may vary.
Hi Andrea, let’s start with the news everyone wants to hear. What results have you seen in the people you treated with HEMGENIX?
The people I’ve treated have been early responders—their factor IX levels started coming up after the first week, and they have been able to stop prophylaxis after week two.
Patients almost a year out are still experiencing fantastic results. They’ve remained off prophylaxis. They’ve had some injuries that in the past would have caused a bleed, but none required any factor IX infusions.
How did your team identify the patients you would end up treating with HEMGENIX?
We looked primarily at people with hemophilia B on prophylaxis therapy that were still having bleeds, but we wanted all who were potentially eligible to be made aware that it’s a new option for them.
Those we treated were born with hemophilia B, were very afraid of needles, still experienced bleeds, and even as young men they had identified target joints. Also, they were very motivated and excited to know that there was a treatment option that could potentially improve their quality of life.
We went over the clinical data and the risk of adverse events that could occur. We sent them home with written information and said we would be following up with them.
Did any patients have any issues or concerns?
They were looking at this therapy as something that could potentially really change things for them. And what if it didn’t work? I told them that while we don’t know how every person will react, the data that we do have is reassuring. And in the clinical trial, we know reasons why it did not work for specific people. (Of the 54 people in the trial, 2 experienced lack of efficacy. One person had a high neutralizing antibody titer of 1:3212, and 1 person received about 10% of the planned dose.)
What is your eligibility process leading up to Infusion Day?
It is about a 4-month process from the time our patients decide they want to move forward with HEMGENIX until Infusion Day.
For the patients we’ve treated at the center, we completed an initial consent form and brought them in right away to do their AAV5 neutralizing antibody testing. Even though this wouldn’t have excluded them, we wanted to make sure they didn’t have abnormally high levels. We set up their labs and their liver diagnostics in the interim and chose to do a psychosocial assessment.
While that is going on, we work with both CSL Behring and the HEMGENIX ConnectSM program on the financial side for coverage. We make sure there is great communication between the clinical team and the financial team. Our patients have found the HEMGENIX Connect program helpful with the benefits investigation and providing education on CSL Behring’s travel assistance program.
That brings us to your Infusion Days! What have those been like?
Infusion Days have been very exciting!
Our policy for anyone undergoing gene therapy is to visit us the day before for one final check-in. They stay in town overnight, and then arrive the next morning, ready for their infusions. I think most people will say there may be a few nerves when you’re doing a new therapy, but I don’t think we could have asked for better days for their infusions. We have not had any infusion reactions, and when the patients have finished there have been lots of hands in the air and cheers.
We have our patients stay in town overnight for a follow-up visit the next day just to make sure things are going well. We then provide them with calendars with their weekly lab monitoring for the next 3 months and choose to send them home with steroids in the event they would need them. They have had some liver enzyme elevations and took steroids to bring those levels back down.
That follow-up visit is really important to go through what’s next for them.
Where do post-monitoring visits take place?
If a person lived quite a distance away, we did all of their baseline labs at the hospital we knew they would be using so that we could keep labs very consistent. We also did monthly video visits with them so we could track their progress.
If another person chose to come to our center for all of their labs, we did that as well. And if there were a few times where they were traveling for work, we did have to send orders to a different location. We were able to set it all up pretty easily.
What did the people you treated tell you was the biggest difference they felt since Infusion Day?
They always say they can’t believe they’ve gone this long without a factor IX infusion. Patients have had some injuries in that time frame—either getting a finger caught in the car door or slicing a thumb with a knife—and they expressed a little bit of shock over the fact that those injuries didn’t bleed like they did prior to HEMGENIX.
If someone with hemophilia B is considering HEMGENIX, what advice would you give them?
I would advise talking with every person with hemophilia B about their quality of life. What their goals are for themselves and their bleeding disorder, and making sure they know there is this option out there that could help them meet those goals.
There are great resources from CSL Behring for you, and now that other healthcare providers like our center have gone through the process, I’m sure they’re willing to answer questions too.
And that joy you see in a person when you tell them that they’re making their own factor IX level is well worth it!
IMPORTANT SAFETY INFORMATION
What is HEMGENIX?
HEMGENIX®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who:
- Currently use Factor IX prophylaxis therapy, or
- Have current or historical life-threatening bleeding, or
- Have repeated, serious spontaneous bleeding episodes.
HEMGENIX is administered as a single intravenous infusion and can be administered only once.
What medical testing can I expect to be given before and after administration of HEMGENIX?
To determine your eligibility to receive HEMGENIX, you will be tested for Factor IX inhibitors. If this test result is positive, a retest will be performed 2 weeks later. If both tests are positive for Factor IX inhibitors, your doctor will not administer HEMGENIX to you. If, after administration of HEMGENIX, increased Factor IX activity is not achieved, or bleeding is not controlled, a post-dose test for Factor IX inhibitors will be performed.
HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.
What were the most common side effects of HEMGENIX in clinical trials?
In clinical trials for HEMGENIX, the most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell. These are not the only side effects possible. Tell your healthcare provider about any side effect you may experience.
What should I watch for during infusion with HEMGENIX?
Your doctor will monitor you for infusion-related reactions during administration of HEMGENIX, as well as for at least 3 hours after the infusion is complete. Symptoms may include chest tightness, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure. If an infusion-related reaction occurs, the doctor may slow or stop the HEMGENIX infusion, resuming at a lower infusion rate once symptoms resolve.
What should I avoid after receiving HEMGENIX?
Small amounts of HEMGENIX may be present in your blood, semen, and other excreted/secreted materials, and it is not known how long this continues. You should not donate blood, organs, tissues, or cells for transplantation after receiving HEMGENIX.
Please see full prescribing information for HEMGENIX.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.
HEMGENIX is manufactured by uniQure Inc. and distributed by CSL Behring LLC.
HEMGENIX® is a registered trademark of CSL Behring LLC.
HEMGENIX ConnectSM is a service mark of CSL Behring LLC.
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