Optimizing Care for Patients With Hemophilia


A panel of experts provides considerations for the optimal management of hemophilia with regard to treatment planning, challenges in traditional pharmacologic approaches, and access to effective treatment.


Peter L. Salgo, MD: Let’s talk about initial treatment plans for hemophilia and how effective these plans are. Not just for acute bleeding but the global planning for someone.

Tim Boonstra, RPh: If somebody has been prescribed standard half-life factor VIII or factor IX, or even an extended half-life product, they’re extremely effective. To enhance that effectiveness, because our pharmacy is located right at a hemophilia treatment center, I’m fortunate to be able to participate in pharmacokinetics. We do some blood assay tests and determine what the best dose for a patient might be, and then determine what their ideal dosing interval would be. That really makes the treatment plan very effective.

Peter L. Salgo, MD: How do you monitor that, though? You start with plan A, but no plan survives, and you’re back with the enemy.

Tim Boonstra, RPh: Yes.

Peter L. Salgo, MD: Let’s get back to what I was asking you before. In other words, you’ve got more to do than just dispensing, right? You were telling me that you follow up. But what are the questions you ask? How do you do it? What do you communicate?

Lacey Chapman, RPh: At our monthly refill assessment, like Tim, we ask how many doses they have on hand, have they had any bleeds that month, are they having any pain, and what their current dose is—because it does change a little bit every month depending on assay availability. Sometimes you might give 4200 units; sometimes it might be 3800, depending on that plus-or-minus-10% range. We’ll know if they’re having a lot of bleeds. With some patients, we’ll use a bleed log for them because they’re having sporadic bleeding. We ask about their exercise level, how they’re doing, and how they’re feeling—those kinds of things.

Robert F. Sidonio Jr, MD: With children, their exercise might change by the sport, right? If there are times when they’re not playing baseball, they might not need as much factor. But if they’re participating in sports for a few months, they might bleed more and you need to change that. We rely on these updates. Nowadays, we have these electronic logs of bleeds, thankfully, and hopefully people are utilizing those more.

Tim Boonstra, RPh: As Lacey was describing with the treatment logs, there’s a website that people can go to called MicroHealth. People can track their bleeds and scan their box of factor, and they’ll immediately input how many units and what exactly was used.

Robert F. Sidonio Jr, MD: These paper calendars, some parents like them, but we’ve got to move into the electronic age, so you can scan the product and keep track of it.

Peter L. Salgo, MD: That’s crazy.

Tim Boonstra, RPh: But it’s important. Even insurance companies are now requiring them to show what their treatment logs are.

Peter L. Salgo, MD: Just upload it, right?

Robert F. Sidonio Jr, MD: Yes, they just have that log; it stays there. Then they have the ability to communicate with us, so we can see if they’re missing doses. All this is helpful for better care, and adherence is obviously important with any factor treatment.

Peter L. Salgo, MD: All I can think of is, “There’s an app for that.”

Robert F. Sidonio Jr, MD: There is. There’s always an app.

Peter L. Salgo, MD: There’s an app for everything.

Robert F. Sidonio Jr, MD: There is.

Peter L. Salgo, MD: What are the challenges you encounter with the traditionally available options here?

Robert F. Sidonio Jr, MD: The biggest one is the route of administration. They’re all intravenous (IV) therapies, right? The last time I checked, most children do not like to be stuck with needles, right? That’s the biggest challenge with that, the route of administration. The half-life of these products is relatively short. For hemophilia B, they need at least twice-a-week prophylaxis to prevent bleeds, and for hemophilia A, they need at least 3-times-a-week prophylaxis, maybe as often as every other day.

Peter L. Salgo, MD: IV.

Robert F. Sidonio Jr, MD: IV therapy, yes. They’ve got to start the IV, mix their product, and do all the stuff. This is the one patient group that is able to perform much medical care. They’re doing things that a nurse typically would do, and we have to teach them how to do this, which is really important.

Peter L. Salgo, MD: We should introduce them to some of the diabetics who use IV. “I have to give myself a shot.” Look at these kids.

Robert F. Sidonio Jr, MD: Yes. They’re doing it themselves. It’s not unusual to see a 6- or 7-year-old get up in the morning—mom watches him—and he cleans it, he’s got a little mat, he learned the process, and he does everything from mixing the vial to infusing. It’s pretty amazing.

Peter L. Salgo, MD: It is amazing, but it’s also sad.

Robert F. Sidonio Jr, MD: It is hard to see them have to grow up so early. But the great thing I like about pediatrics is you see patients as babies and you follow them until they’re young men, when you see them participate in patient advocacy groups. We see them go back to the camps. It’s awesome to see these 16-year-olds come to camp and help out these 5- and 6-year-olds who are out there.

Tim Boonstra, RPh: It’s an extended family, almost.

Robert F. Sidonio Jr, MD: Yes.

Peter L. Salgo, MD: Speaking of family, the pharmacists are in the middle of all this, right?

Robert F. Sidonio Jr, MD: Yes.

Peter L. Salgo, MD: You’re keeping track of everything. With all due respect, you’re not a hematologist.

Robert F. Sidonio Jr, MD: No, no.

Peter L. Salgo, MD: You’ve got vaccinations to worry about. You’ve got other illnesses. How do you integrate all this into a pharmacologic practice?

Lacey Chapman, RPh: We keep a chart on every patient and for the hemophiliac patients.

Peter L. Salgo, MD: I’ll bet you do.

Lacey Chapman, RPh: Insurance requires it as well. As Tim was saying, some insurances want to actually see the physical calendar of bleed doses, when they’ve infused, and all their medication history. They want it all right there, so it’s easy to look at and you can track them chronologically, what has happened from this month to this month. You can see how many bleeds they’re having.

Peter L. Salgo, MD: What are the things you watch out for in terms of other illnesses? In other words, it’s easy to focus on this. This is a life-threatening problem. Kids get stuff. Kids get colds, kids get viruses, kids need vaccines, which means injections and potential bleeding. How do you integrate all of that?

Tim Boonstra, RPh: We’re in Minnesota, and our center is an adult center, so we don’t see all those things.

Peter L. Salgo, MD: In other words, he gives them to you.

Robert F. Sidonio Jr, MD: Yes, we just give them to you.

Peter L. Salgo, MD: The sniffly kids.

Tim Boonstra, RPh: But we do vaccines, and one of my jobs, because I’m right on-site, is that I do a look ahead in their electronic medical record and just see what vaccines they are due for. I can administer them while they’re in clinic, while they’re there for their appointments.

Robert F. Sidonio Jr, MD: They have to deal with it, as you said. They don’t always have just one medical problem. We have kids with asthma. There are obviously a lot of patients who are still dealing with HIV and hepatitis C and all those medication interactions. Even in doing something like starting an SSRI [selective serotonin reuptake inhibitor], there are some potential risks of bleeding. And so, they keep up with that. I just got an e-mail yesterday about someone who is starting a new drug and wanted to clear it with us. That’s awesome that they alert us to those kinds of things. For us, we have to make sure we’re managing all that stuff, and so we do provide that medical home and make sure we’re managing every part of the patient, not just the hemophilia aspect.

Peter L. Salgo, MD: I realized this only recently. For 10 or 15 years, we’ve had a PharmD go on rounds with us in the intensive care unit. What a dramatic improvement in care. I realized I didn’t know everything. It’s sad, but true.

Robert F. Sidonio Jr, MD: Only 10 years ago you realized that?

Peter L. Salgo, MD: Only 10 years. My kids could have informed me of this long ago. But it’s dramatic. There’s another whole specialty PharmD who provides dramatic input here. It’s good.

Tim Boonstra, RPh: One of the cautions that I know our hematologists say is that our center is not their primary care.

Robert F. Sidonio Jr, MD: Yes, exactly.

Tim Boonstra, RPh: They like to say, “Well, Dr. So-and-so, you’re the person I go to for everything.” Primary care is a specialty, and they are a specialty for a reason. And so we make sure that they’re connected with primary care just as they’re connected with a dentist.

Robert F. Sidonio Jr, MD: I take it as a compliment because they trust us to do everything. But I say, “If you have allergy problems or reflux, you should probably go see your primary doctor for that as well. I’m happy to help, but remember, you do have a primary doctor.” But it feels like a compliment because, as I remind our nurses, they trust us enough to call us for everything, and that’s just a test of the level of care we’re providing.

Peter L. Salgo, MD: But I’ll bet you get calls from the primary care physician too.

Robert F. Sidonio Jr, MD: Yes.

Peter L. Salgo, MD: “I’ve got one of your patients with hemophilia, and that person has asthma. Is it safe to use this?”

Robert F. Sidonio Jr, MD: And the other thing is that some of the pharmacists will also send people out to the centers for education. In pediatrics, you might be the only hemophiliac in that school. Kids don’t want to always say, “I have hemophilia” to every kid they see. It’s nice for us to go out there and educate the nurses and educate the teachers so that they know when to call us, so they’re not calling us for everything. They’re not obviously waiting to call us for everything.

Peter L. Salgo, MD: It just occurred to me, as you were talking about schools, that there was a big dust-up about schools not wanting to carry EpiPen [epinephrine auto-injector].

Robert F. Sidonio Jr, MD: Yes.

Peter L. Salgo, MD: Do schools have to carry, or should they carry, emergency medications if there’s a hemophiliac in the school?

Robert F. Sidonio Jr, MD: We work with the schools. Sometimes, oftentimes, they can give the therapy before school. It’s a little easier, and less paperwork. It’s hard for some of the kids to carry it, so they’ll have to keep it with the nurse. Those nurses keep in contact with other specialty pharmacy nurses who are in contact with the pharmacist and us, so yes, that communication is very important.

Peter L. Salgo, MD: I have this vision: Johnny or Jane falls in gym class. The nurse better have something there, right?

Robert F. Sidonio Jr, MD: Yes, of course. That’s the most important thing: early recognition and treatment, not calling us, and asking the nurses for advice.

Peter L. Salgo, MD: Is there pushback from the schools? Do you get that?

Tim Boonstra, RPh: Well, with our adult center, we don’t work with the schools.

Peter L. Salgo, MD: Do you see it?

Lacey Chapman, RPh: I don’t. I’ve had patients ask us to have a nurse go out to the school to do the training. We have nurses who go out to do initial training with children and help them with their infusions.

Robert F. Sidonio Jr, MD: Yes.

Tim Boonstra, RPh: But if there is a school nurse, I think they would welcome it. They want to be prepared.

Robert F. Sidonio Jr, MD: Yes. They want to be prepared. They want to make sure they don’t cause harm. But they also don’t want to be alarmists at any time and keep the child from doing the things they would love to do on the jungle gym and such.

Related Videos
Related Content
© 2024 MJH Life Sciences

All rights reserved.