Jonathan Ogurchak, PharmD, CSP: How do you approach this exactly? Is there a difference in how you plan to approach these types of tactics for primary to secondary to tertiary prophylaxis?
Robert Sidonio Jr., MD, M.Sc.: We will often talk about continuous prophylaxis, this idea of giving it regularly. The definition is a bit vague, but generally you factor in at least once a week, for a period of 3 to 6 months compared to intermittent or short-term prophylaxis, during which they may have had bleeding. You are trying to break this cycle. Often times, we do this for our non-severe patients. The guidelines were published and said that even in moderate patients, if they have a severe bleeding phenotype, they should also be offered prophylaxis. Can they be offered this secondary because you’re trying to see if this moderate patient will develop bleeding symptoms that you would typically see in a severe patient?
However, if you look at the trends, we are obviously more aggressive than just 10 years ago. Just look at our internal American Thrombosis & Hemostasis Network datasets [ATHN] and the Centers for Disease Control and Prevention [CDC]. At least half of our moderate patients, those with rates of 1% to 5%, are on prophylaxis for at least a period of time before the age of 18. This number was significantly lower years ago. We will continue to insist that we do more primary prophylaxis. However, usually in a young child, you can’t start maybe 3 times a week, every other day, or every 48 hours or every 72 hours. It’s really difficult from the start. We usually start once a week and try to get the family and the child to come to terms with this emotionally. Then we increase the dose in young children to twice a week for factor VIII or factor IX, which is usually effective early on, apart from the devastating bleeding. Finally, we try to get them to a more typical strategy, which is usually about 3 times per week for standard half-life products for factor VIII, and generally 2-3 times per week for factor IX. Obviously, some extended products allow administration once a week.
Jonathan Ogurchak, PharmD, CSP: Awesome. Once prophylaxis is started, can it be stopped? What is this approach from there?
Robert Sidonio Jr., MD, M.Sc.: Alexis, I’ll let you take the lead, then I intervene.
Alexis Kuhn, PharmD, BCOP: Thank you. With prophylaxis, as we look to the future and play the long-term game, in an ideal state, we would continue and reap the benefits of long-term prophylaxis. However, as we have mentioned, prophylaxis carries significant logistical, emotional and physiological burdens. Yes, in very individualized cases absolutely, we would consider discontinuing prophylaxis if the burdens outweigh the benefits that are realized in the individual.
Robert Sidonio Jr., MD, M.Sc.: It’s always a compromise with patients. There are situations where it’s just not a good time, or the child is really struggling with it, or a center line is the obstacle. Young children often need a central line, even a temporary one, and we all know every child is different. For some children, you could do this. Some children learn to glue from the age of 5. With some children, it takes up to 12 or 13 years. We need to be very supportive and tell people that our goal is to try to get prophylaxis. We try to prevent life-threatening bleeding events. We are trying to make life more normal. It will never be normal, but we try to involve them in life and physical activity – some sports, obviously non-contact. It’s always a negotiation, especially in early childhood. With adults, we have to say, “We are thinking about your long term benefits. We want you to be functional. We don’t want you to lose your ability to infuse because you’ve had so much elbow bleeding.
Jonathan Ogurchak, PharmD, CSP: It makes perfect sense. Now, let’s talk a bit about this patient preference to approach. How does this patient preference play a role in deciding whether or not to opt for therapy over prophylactic therapy?
Anastasia Abramson, PharmD, MBA: Patient preference is certainly important in the selection of treatment and should be taken into account in decision making, so that we can help make the optimal treatment choice not only for favorable results, but also for sustainability in terms. of observance. It is a little different for the younger populations in the care of hemophilia treatment centers, but in the older populations that have not been established on prophylaxis, we certainly see some patient preferences and introduce the prophylaxis in the treatment plan. Of course, we want to keep the patient’s personal goals in mind when selecting this approach. With respect to some patient preferences that we see at the pharmacy level, besides cost, one of the concerns is the development of inhibitors against factor VIII substitutes. We meet adult patients who have been treating their bleeds on demand their entire lives, and when the conversation about prophylactic therapy gets started, they’re not entirely comfortable taking the risk rather than the benefits. Also, there is the downside of the frequency and intimidating intravenous administration with the products, but it is something that we can provide education on and help make the patient more comfortable.
Transcription edited for clarity.