Emicizumab's Impact on Quality of Life in Hemophilia


Before sharing their closing take-home messages, Doris V. Quon, MD, and patient Eric Mamos reflect on the improvements in Eric’s quality of life since being started on prophylactic emicizumab.


Doris V. Quon, MD: Just to review, I know that you had no bleeds on the clinical trial. You were part of this group that we talked about, patients with 0 bleeds. It was nearly 60% of patients in both the once-weekly and the once-every-other-week arms who had 0 bleeds. That must resonate with you.

Eric Mamos: Oh, absolutely. It’s funny how we define a bleed. I’m sure there were bleeds, they just never turned into hemophiliac bleeds, right? They just ended up fixing themselves like in any other normal person. Nothing ever turned into something I would have used an infusion for.

Doris V. Quon, MD: Yes, so you really had no treated bleeds.

Eric Mamos: Nothing, no.

Doris V. Quon, MD: Do you think that affected your quality of life? How did it affect your life?

Eric Mamos: It’s an interesting question. It affected my life in that for the first time, I didn’t have to think as much before I did something. A good example of that is last Thursday. I coach my boys in basketball, and the other coach was not able to be there. It was me and an odd number of kids, and it’s hard to play basketball with an odd number of kids. So I stepped in, and I said to our tallest kid, “Look, kid, I’m still taller than you. I’m playing center because it’s zone defense, and I know I’m not moving much if I’m playing center.” So I played center. I still had to get up and down the court at some sort of relatively decent speed, but if you asked me to do this a year and a half or 2 years ago, it would have been impossible. They would have just played with odd numbers. But this time, I thought, “I’m going to give this a shot, let’s jump into this and see what happens.” I was able to do that.

Doris V. Quon, MD: Do you wish that you had known about this or had this drug when you were growing up? Do you think it would have made a difference?

Eric Mamos: Oh yes, probably 3 surgeries less of a difference, maybe more. I don’t think I would have had the target joints if that was the case.

Doris V. Quon, MD: Are there still things that you struggle with?

Eric Mamos: Hemophilia-related, yes. The fused ankles are still a pain. Going up and down stairs is not the easiest thing I look forward to because I can’t bend my ankles. To try going up the stairs walking on your heels takes some acclimation. At the same time, there’s the ITP [idiopathic thrombocytopenic purpura], and the weight gain never really helps. I’m still dealing with those ramifications combined with the hemophilia. I think it’s all related and makes those things difficult. But in the scheme of things, if that’s my biggest problem, that’s not a problem.

Doris V. Quon, MD: Do you have any final words of wisdom or advice for patients who are newly diagnosed? Actually, in this case, it would be moms who have children who are newly diagnosed or babies who are newly diagnosed.

Eric Mamos: I don’t know if this particular medication is available for infants.

Doris V. Quon, MD: It was actually approved. The FDA label was for adults as well as children and newborns.

Eric Mamos: Then my advice is to get them on this medication because if they’re on this medication, I don’t see why they won’t have at least 80% to 90% of a normal life from day 1. That’s huge. That’s a game-changer. Things like camping or hiking, that’s the fun. I was not doing that, no way. Skiing? No, not happening. But if an infant or a child is brought on to this kind of medication, or maybe something better in the future, then hemophilia really becomes more of a nonissue.

Doris V. Quon, MD: For patients, there are a number of things to consider before starting a new medication. What’s important is for the physician and the patient—or mother, father, parents—to have a long, detailed conversation about what’s important. Patient preference is a key factor in making a decision as well as patient goals or family goals. In adult patients who have comorbidities, such as yourself—target joints, things like that—those have to be taken into consideration.

Eric Mamos: But if you can avoid target joints, just avoid them. Take the medication. The only other parting advice I would have is that somebody recently told me a lot of hemophiliacs like to be defined by their hemophilia. I would say that if you are newly diagnosed or the mothers of newly diagnosed patients, I don’t know if I like that. I don’t think we should be defined by the diseases that we have. We are made from the sum of challenges and the sum of good things in our lives. Let that define you. Don’t let hemophilia be a definition of your life.

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