The Role of Inhibitors in Treatment Strategies for Hemophilia A


Lisa Schrade, PharmD, shares insight on the impact of inhibitors in the hemophilia A treatment landscape.

Lisa Schrade, PharmD: Inhibitors impacting treatment can be very scary and even more exceptionally demanding on the families. If you have a patient that’s already infusing every day, or every other day, now you’re talking about a regimen that requires them to infuse every 2 hours. A family member has to try to access a vein for these little guys. Or you have to get them a surgical procedure to have a port placed because the infusions are so frequent. It affects the patients, the families. It’s so demanding, and it puts so much pressure on everybody to really step up to the plate. And you’re already mentally, emotionally exhausted, and to add that it’s a lot.

And then, too, from a payer perspective, it gets financially burdensome, not only to the families but also to the insurers. It’s a high out-of-pocket expense for the patient. We try to avoid that at all costs.

Some of the current challenges for patients with factor replacement therapy is access to care. Not all patients are fully insured. Some of them don’t meet the criteria to be supported by state-funded plans, so their availability to be able to get the medication that they need when they need it can be very restrictive, which then starts to cause a whole cascade of other issues. If they’re stressed out about trying to afford their medication or stressed out about losing their job because if they do have a bleed that requires them to take time off from work, they worry about jeopardizing their livelihood with their employer. It adds another level of stress. Possibly it can cause depression. Sometimes they’ll go without treatment to try to prevent that from happening. And then something more substantial will happen, like a joint bleed, an intracranial bleed, or GI [gastrointestinal] bleeds. Things that are preventable, but having that limitation with how they can get their medication when they need it because of finances is one of the biggest challenges.

The human burden with hemophilia A is how demanding it is, not only to the patient, but to the families, making sure that everyone is aware of what the patient’s needs are. And unfortunately we see it happen all too often with younger families. If we have a child that’s born with hemophilia, parents might not have known that they were carriers, or maybe it’s a patient that is an anomaly and it’s the first mutation in the family to experience hemophilia. There’s that feeling of guilt from the family members, that they had passed this trait down to their child. And then it causes a lot of turmoil within the family. There’s sometimes blame on yourself, [or] fingers being pointed at other family members, that it’s their fault that this happened.

But, too, the demand of the therapy itself is a burden. Having to take time off from work to take your child to the doctor because there’s a bleed, or going to school to pick them up. Missing days off from work yourself, if you’re having a bleed or some joint disruption from something as simple as just bending over. Things that we take for granted, simple tasks that become very restrictive with our patients, and affect their quality of life. So we do whatever we can do to try to make that easier for them. They’re already dealing with so much; the least we can do is make sure that they have what they need when they need it.

Transcript edited for clarity.

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