Moving the Field of Hemophilia Management Forward


Novel therapeutic approaches to managing hemophilia are reviewed alongside a conversation about appropriate follow-up with patients to ensure positive outcomes.


Peter L. Salgo, MD: I just want to take a little detour here because I talked to these guys before we went on the air. You were telling me about viral therapy, embedding a new genome into the liver and patients suddenly making factor VIII.

Robert F. Sidonio Jr, MD: Gene therapy has been something where I feel like we keep saying in 5 years, it’s going to happen. They say that every couple of years. But I think in 5 years, it’s going to be a reality. Certainly, at your center, I know they have these studies available and we do, too. They take a specific virus that’s usually nonpathogenic and they strip out all the bad things.

Peter L. Salgo, MD: We hope.

Robert F. Sidonio Jr, MD: Then they insert the factor VIII or factor IX gene. A lot of them have developed such that they don’t insert into the genome, so it goes into the liver cell. You inject it just like a regular injection, an IV [intravenous] injection. It’s been tagged to find the liver. It goes into the liver and forms what’s called an episome, circular DNA outside the nucleus. It doesn’t integrate, or its integration rate is extremely low, and you hope that it will continue to produce factor as long as you have your liver. You have to wait for the liver to be fully grown, so we’re not typically going to be doing this until patients are older.

You have that time period, the first 18 years of their life, where you’ve got to give them something. But there are trials where patients have 50% factor VIII and patients have factor IX levels in the 30% or 40% range. It’s becoming a reality. Gene therapy will be available for multiple diseases in the future.

Tim Boonstra, RPh: But you were talking about cost.

Peter L. Salgo, MD: Yes.

Tim Boonstra, RPh: Nobody knows exactly what gene therapy is going to cost. Is it going to be a million dollars per patient?

Peter L. Salgo, MD: But it’s just once.

Robert F. Sidonio Jr, MD: It’s a one-time therapy, yes.

Tim Boonstra, RPh: Exactly. You do the math.

Robert F. Sidonio Jr, MD: But we don’t know. Some patients may get 100% factor and some patients may get 2% factor: do you base it on that? As we know, people who have insurance when they’re 18 do not have the same insurance when they’re 35. Sometimes people don’t want to pay for future benefit, and that’s why it makes it complicated in the United States.

Peter L. Salgo, MD: Let me ask a really naïve question. If you can’t use this gene therapy until they’re 18 but they’ve had a lifetime of nonexposure to factor VIII and suddenly they get factor VIII, why won’t they develop blockers to that factor VIII now that they’re making their own?

Robert F. Sidonio Jr, MD: Typically, they’re done in previously treated patients who we call PTPs. We know that in patients who receive factor and are able to get past those first 20 to 50 exposures, the chance of them developing a neutralizing antibody afterwards is very small. There’s a little bit of a peak as they get older, since any time you see older patients their immune system changes. We’re talking 70- or 80-year-olds who may develop that. But we’ve done these big studies, where 400 or 500 patients have received and switched products and they don’t develop inhibitors. We haven’t seen that with gene therapy, and that’s another reason why it probably isn’t going to be feasible in young children.

Peter L. Salgo, MD: Now, let’s talk about the nuts and bolts from a pharmacy perspective. I know you guys have your boots on the ground. You’re getting the granular structure of all of this. How frequently do you review the outcomes with the patients in your practice? Do you do this on the phone? Do you do this in person? Do you text them? Lead me through some of this.

Lacey Chapman, RPh: We usually call them every month.

Peter L. Salgo, MD: Old-fashioned, you call them.

Robert F. Sidonio Jr, MD: You don’t text them.

Peter L. Salgo, MD: Audio, audio.

Lacey Chapman, RPh: We actually call them. Some people have patient representatives in the field who contact them and then the representative will email us.

Peter L. Salgo, MD: My daughter would call you a Neanderthal. “Dad, nobody talks to anybody anymore.”

Lacey Chapman, RPh: But we do have some younger patients who won’t answer their phone, and they want us to text them.

Peter L. Salgo, MD: I knew it.

Lacey Chapman, RPh: An app might be a good thing for them.

Robert F. Sidonio Jr, MD: It totally works, yes.

Tim Boonstra, RPh: We do have a pharmacy cellphone and we do text people.

Peter L. Salgo, MD: You do?

Robert F. Sidonio Jr, MD: Honestly, we have to.

Peter L. Salgo, MD: How often do you follow up? What do you do with the data? When do you intervene? Do you go directly? Do you call the doctor? Help me out here.

Tim Boonstra, RPh: As Lacey said, I think we probably all call once a month or have patients call us once a month.

Lacey Chapman, RPh: Yes.

Tim Boonstra, RPh: As I said earlier, we just go through a list of questions and determine what’s going on with them in that particular month. At least at our center, we can certainly communicate that back to the care team—the doctor, the nurses—and say, “John Doe has had 2 bleeds in the last month and his pharmacokinetics says that he shouldn’t be bleeding.” They’ll call and go through all of that.

Peter L. Salgo, MD: Do you have a part of your algorithm for, “Oops, we didn’t get a call?”

Lacey Chapman, RPh: We have a calendar and we know when patients are due, so we will call them.

Robert F. Sidonio Jr, MD: The old-fashioned strategies still work, right?

Peter L. Salgo, MD: That could be very, very good or very, very bad.

Lacey Chapman, RPh: But we know if they’re being [adherent] or not.

Tim Boonstra, RPh: That’s right.

Lacey Chapman, RPh: If they’re not refilling in a month, we call them and ask them why. “How many doses do you have on hand? 10? Why do you have 10 doses on hand?”

Robert F. Sidonio Jr, MD: That means you missed 10 doses, yes.

Lacey Chapman, RPh: Or it could be a patient who had a soccer season and soccer is over, so now he’s not infusing as much.

Tim Boonstra, RPh: What our business person at the pharmacy actually puts together is what we call a possession time.

Peter L. Salgo, MD: What is a possession time?

Tim Boonstra, RPh: It’s a rate of how often we filled it and how much the patient has used. We can tell if they have a possession time of 1 [of] everything [that] we’ve sent them, they’ve used. But if they have a possession time of half, or 0.5, they’ve only used half of what they were supposed to. That communicates back to our care team. When that patient comes in for an appointment, they know what kind of conversation to have with them.

Robert F. Sidonio Jr, MD: We rely on that because we can’t see patients that often. We have a very busy clinic, probably the second or third largest in the country. We’re seeing hundreds of bleeding disorder patients. We don’t typically see them more often than every 3 months, so we heavily rely on our pharmacists to call us and say, “Johnny, he’s got a lot of doses at home, I think something is going on.” They’ll alert the social worker. Maybe something’s going on at home. Maybe the parents are getting divorced. We have to know everything about these patients. We know everything about them.

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