Challenges Caused by Factor Replacement Therapy

Atta Chowdhry, RPh, discusses challenges with factor replacement therapy in patients with hemophilia A.

Atta Chowdhry, RPh: For monitoring of the treatment complications, specifically the development of inhibitors, that patient will have a higher frequency of bleeds. So, if the patient is taking their prophylactic treatment on a regular basis and still having a lot of bleeds, then definitely they need to get tested for inhibitors and to rule out if there are any inhibitors. And most patients usually need to be tested at least once a year to check for the inhibitors.

Usually, in factor VIII patients, there is a prevalence of 30% of patients who end up developing inhibitors. And in factor IX patients, it's a very small percentage, it's anywhere from one-and-a-half to 3% of the patients develop inhibitors, and usually, it is accompanied by anaphylaxis.

The factor VIII replacement therapy has challenges like any other disease state, but it is potentially a fatal disease if it's not being treated properly. And so, the challenge for the patient is always an insurance issue. They are constantly explaining themselves to insurance companies for getting the reimbursement for the product. The second thing is that these patients physicians also have to access their veins and there is a lot of issues in having vein access where sometimes it works, sometimes it doesn't so that is another issue, vein access.

The last thing that I’d like to add is that with these patients that have factor replacement therapy, the first issue is the compliance that a lot of these young kids, specifically anybody who is between the ages of 17 to 25, they have a bigger issue in compliance. The kids go to college, they stop infusing themselves regularly, and then they have a lot of joint bleeds, which can end up causing some severe joint problems or other kinds of bleeds. So, the compliance is the first issue with the patients. The second thing is the cost of the therapy. It's a really high-dollar therapy and a lot of these patients have to go through a lot of different hoops in order to get these therapies approved; constantly explaining themselves to their insurance companies and going over these therapies with their physicians. And then also finding a good physician where they can go to the treatment center, having a physician who understands the therapy because it's such a rare disease that a lot of the physicians and a lot of the nonregular physicians don't have a full grasp of the disease state.

The relationship between factor IX levels and bleeding or other complications is that factor IX patients are very tricky. If their factor IX level is low, they end up having bleeds, bruises all over their bodies, having issues, but if their factor IX level is high, which they end up infusing more than they need, then they end up with thrombotic events. So, they end up with pulmonary hypertension in the long run or some other disease states. So, the management of factor IX patients with the right amount of factor as an individual basis, is very critical.

Transcript edited for clarity.

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