A man with hemophilia B shares his gene therapy story, part 2

Second in a series. Read part one.

In part one of my interview with Chip Hailey, a 75-year-old from Tennessee, we explored what led him to pursue gene therapy for hemophilia B, his lifelong journey with the condition, his hopes for a new chapter, and the road to participating in a clinical trial. In this second installment, Chip opens up about the decision-making process: the hopes and fears he carried into gene therapy, the crucial support he found in his family, and the moments of anticipation before receiving his Hemgenix (etranacogene dezaparvovec) infusion.

Excerpts from our email exchange follow, with light editing for style.

JL: What were your biggest hopes and concerns going into gene therapy?

CH: I was really focused on the best that could happen. My biggest hope was that it would work well, that my body would start to produce its own factor IX, and I would not need to worry about my prophylaxis treatment again. My biggest concern was that it wouldn’t work — but what would be the difference? I’d just continue my prophylaxis treatment like nothing happened. Thinking like that, it was a no-brainer for me.

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Was the decision to pursue gene therapy an easy one? Who helped you make it?

I’d tried to participate in several clinical trials, so I knew the risks better than most, but honestly, none of it worried me. I was excited about the opportunity, and nothing was going to scare me away.

My wife was by my side the entire time. We’ve been married for over 50 years, and we always make decisions with each other in mind. For me, this clinical trial was such a life-changing event, and she supported me in pursuing the opportunity.

Did your hemophilia treatment center or physician help prepare you for the emotional or physical aspects?

Yes, I was very informed going into the process. I [knew] the potential benefits and risks of joining the gene therapy clinical trial, but I was just ready for it. I was 65 years old and I’d seen it all from a lifetime of treating my hemophilia. I had already done so much to educate and prepare myself for this moment, I was excited to get started.

Interestingly, a couple of weeks before the trial I was a little anxious — not that it wouldn’t work, but that something would stop me from getting the treatment. I had been disappointed before with trials, so I prepared myself, just in case this one didn’t happen, either. But after my last visit with the team, I thought, OK, this is going to happen, and it did.

Can you walk us through what the day of your infusion was like?

On the day of my Hemgenix infusion, I arrived at around 8 a.m. My wife was with me, and we were both excited and a bit anxious. The clinical trial team was great. They were very supportive and made sure everything was ready for the infusion. I had some blood work done and answered a bunch of questions. They brought in the Hemgenix, and I was asked to lie down.

The infusion itself went smoothly, and I didn’t experience any physical reactions. In fact, I was so relaxed that I even took a nap while receiving it!

After the infusion, I stayed at the clinic for a while to ensure there were no immediate side effects. I didn’t have any allergic reactions or other issues that day. The team monitored me closely, and everything went fine. Nothing unexpected. I remember feeling very happy and relieved once it was over and I was heading home.

What side effects, if any, did you experience, and how were they managed?

I was fine. I didn’t experience any side effects and didn’t have any physical or allergic reactions during or after. Overall, the infusion day was smooth and uneventful.

How long did it take before you noticed any changes?

I was really excited by the results. I had regular blood tests following the infusion to monitor my progress. The first blood test was a week after the infusion, and my factor levels were already up. I was so excited each week for my blood tests to see where my factor levels were. It was so amazing to see it rise. I’m now doing blood work only twice a year and everything is still good. And I haven’t used any factor since the clinical trial, which is a significant change for me. I used to infuse once a week, but now I don’t have to do that anymore.

Thank you, Chip! Stay tuned for part three, where Chip shares how Hemgenix has transformed his daily routine and his outlook on living with hemophilia B.

Note: Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Hemophilia News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to hemophilia.