Ease of use, fear of the unknown, and healthcare providers’ advice are all major factors that play a role in whether people with hemophilia want to switch to new treatment options, a small study suggests.
The study, “Patient Perspectives on Novel Treatments in Haemophilia: A Qualitative Study,” was published in the journal The Patient.
The current standard-of-care for people with hemophilia is replacement of the missing coagulation factors, given via injection. But there are numerous imperfections with these therapies, from the need for frequent injections to the possibility of developing resistance.
In recent years, new hemophilia treatments, including products with longer half-lives and gene therapies, are being developed or entering the market. Although these therapies may be improvements over standard care in some regards, they have drawbacks of their own — and since they are newer, some of these issues may not even be known yet.
Still, there exists the possibility that people with hemophilia may want to switch treatment regimens as new options become available. In this study, researchers set out to better understand the factors that influence such a choice.
To do this, they conducted interviews with 12 people with moderate or severe hemophilia, as well as two parents of children with hemophilia. All participants were Dutch, and they represented a range of backgrounds, including different ages, hemophilia types, infection statuses (such as HIV and hepatitis C), and even fear of needles.
The participants generally reported satisfaction with their current treatment methods (all but one were treated at home; one went to the hospital for bleeds), though they acknowledged some challenges, such as the “hassle” of self-injecting.
Cost was a particular concern; six participants reported avoiding injections when possible to save money — even against the advice of their healthcare providers.
When asked how they felt about switching to a new therapy, eight participants said they were open to the idea, but did not feel they urgently needed something different, while three participants — all of whom were younger and had few bleeding problems — said they didn’t feel the need to switch. The two parents said they were adopting a “wait-and-see” approach.
The researchers summarized factors that participants said would make them more or less likely to switch therapies. Factors in favor of switching included the possibility of the new medication being more effective and being easier or more convenient to use.
“If I had to switch to a different medication, I would switch to one with a longer half-life. That would be a bit better for me so I have to inject less often,” one participant said.
However, participants expressed a general fear of the unknown and a reluctance to take therapies that have relatively less research to back them up, saying they “didn’t want to be a guinea pig or research subject.”
There is also the possibility that a new therapy — even one that works well in general — might not work for a given individual, posing a risk for switching from a treatment that is known to work.
“I know two guys that participated in a gene therapy trial. One was out of luck, he didn’t achieve higher factor levels. The other one did. Yes, fantastic if it works,” said one participant. “On the other hand, if you have good treatment, why would you change it?”
More broadly, participants expressed confidence that their healthcare providers would inform them if new therapies that would benefit them became available.
“The doctors are specialists,” one participant said, “and at some point they’ll say: ‘hey, we have this new treatment for you’, so I’ll say: ‘sure, bring it on!'”
Overall, the study highlights the factors that are important to people with hemophilia who are considering their treatment options.
“It is important for hemophilia treatment teams to be aware of these factors in providing information to facilitate shared decision making,” the researchers concluded.