Page 24 - HFA Dateline 2022 Special Edition

The Future of Self-infusing? mild hemophilia phenotype, whether The Future Role of HTCs
One question that comes up when it’s the result of their mutation or the Currently, there are 145 HTCs in
considering advances in treatments for medications or treatments they take, the United States and its territories.
hemophilia is the future of self-infusions. it might be easier to find a medical These critical treatment centers will
provider who can aid in infusion continue to have an important place
Parents of children with hemophilia instead of learning themselves. in the future, too, if they can continue
often learn how to give their to receive enough support from the
children infusions until the children Plus, some products may not be patient community, Pugliese said.
are old enough to learn how to do available to some patients. And even
it themselves. Many parents and if they are, some people may not HTCs will continue to be a resource for
children learned how to do regular choose to take advantage of them. For people who need factor replacement,
infusions through their hemophilia example, some people may choose especially those who can’t give
treatment centers (HTCs), home gene therapy, while others might not. themselves infusions or choose not to.
care or specialty pharmacies, or Wheeler said she plans to show her But they can be a resource for people
hemophilia summer camps. patients the factor activity graphs and who opt to use newer products and
emerging therapies, as well. Bleeding
issues will still need to be monitored
“For some people, yes, they’re going to ensure that a patient’s bleeding

to have to learn and maintain that skill disorder is well-managed, regardless
of treatment.
set of self-infusion” “They have the best expertise
to make sure that gene therapy
—Allison Wheeler, MD, MSCI
procedures are going to be
successful,” Pugliese said. “I think
discuss their expectations with them. it’s the most logical, safe and cost-
But if enough products come along That can help patients decide if they’re effective way to do it: to work with
that don’t require regular self-infusions, satisfied with the therapies that they’re the established experts in the field.”
it’s possible that many people will already using or if they want to try
lose the ability to give themselves something new. That decision may What’s Next?
infusions—or they may never learn how also affect their decision about self- Now, we wait and see how well the
to do it in the first place. If their need infusing and if that’s a skill that they emerging therapies, including gene
for infused factor is just an infrequent want to master. therapy, deliver on their promises. For
occurrence, they might just visit their some, infusing factor may become an
local emergency department or HTC or Sidonio acknowledged that with infrequent event, while others may
access home health services. the acceleration of new therapies in not have the choice to give up regular
recent years, self-infusing is already
“For some people, yes, they’re going becoming a lost art for some people infusions. But all patients should
to have to learn and maintain that with hemophilia. But he does have continue to have a relationship with
skill set of self-infusion,” said Allison some reservations about people not their HTC, Pugliese said.
Wheeler, MD, MSCI, a hematologist- learning how to give themselves And, at least for the foreseeable
oncologist and research director of infusions and hopes that more people future, factor replacement therapy
the Vanderbilt Hemophilia Treatment don’t completely discard that skill will continue to remain an important
Center in Nashville. set. For example, imagine a trip therapy for people with hemophilia.
But others won’t need to, she said. If a or a vacation when a person with “If you’re on a non-factor therapy,
person only needs occasional infusions, hemophilia develops a bleed and you will still need some therapy to
they may not feel they need to learn needs factor but doesn’t know how to elevate your factor levels to protect
how to give themselves infusion of infuse themselves and doesn’t have you from bleeding if you’re having
factor. And that might be the right easy access to a hospital or HTC. “It’s surgery or in response to a bleeding
decision for them. If a person has a worth retaining that life skill,” Sidonio event,” Sidonio said.
said. “It’s a key to independence.”

24 DATELINE FEDERATION < www.hemophiliafed.org
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DATELINE FEDERATION < www.hemophiliafed.org