Most people with hemophilia inherit the condition at birth — they probably find out they have it early in childhood and spend the rest of their lives learning how to manage it. For the small number of folks with acquired hemophilia, the diagnosis comes late in life and without warning.
“People with acquired hemophilia have not had bleeding issues in the past. The majority may have had previous surgeries or tooth extractions with no bleeding issues whatsoever,” says “Bobby” Duc Tran, M.D., a hematologist and chief medical officer at Hemophilia of Georgia.
Often, they are hospitalized and experience prolonged bleeding after surgery or have routine blood work done and get abnormal results back.
“Usually, they are diagnosed by an outside provider — like a primary care doctor, a surgeon, or an emergency room doctor — and then they’re referred to the hemophilia treatment center for follow-up care,” Tran says. “Because they’ve never had bleeding issues before, they’ve never had a reason to be referred to hematology.”
What Is Acquired Hemophilia and What Makes it Different
Acquired hemophilia occurs when the body’s immune system begins to develop antibodies (known as inhibitors) that attack factor VIII, a protein that is essential for normal blood clotting. This leads to uncontrolled bleeding.
Tran says that sometimes acquired hemophilia is idiopathic, meaning there is no known cause, but other times there are underlying conditions such as autoimmune conditions or certain cancers. Around 1 or 2 people in 1 million are diagnosed each year — that’s around 440 to 510 people in the U.S. — and the average age of onset is 65 to 75.
Because it is so rare, acquired hemophilia is not something that medical providers necessarily know to look for.
“The general practitioners, the dental providers, the emergency room doctors — if they are not thinking about acquired hemophilia as a potential condition, then it may just be a missed diagnosis as opposed to a misdiagnosis,” Tran says.
Also, because of the rarity and the lateness of diagnosis, people are left without the support that people with other types of bleeding disorders have built up over time.
“Where there is a challenge for acquired hemophilia is that they’re not familiar with what resources are out there,” Tran explains. Individuals and families dealing with this new diagnosis and trying to understand the treatment options may feel overwhelmed and unsure about where to find support services in the bleeding disorders community.
Why Specialized Care at Hemophilia Treatment Centers Matters
People with inherited hemophilia largely know the value of the comprehensive care model at HTCs. The good news is that a lot of the treatment options for inherited hemophilia are applicable to the acquired type.
“The doctors, nurses, and support staff who are at hemophilia treatment centers are well versed at how those medications should be used and the potential complications and side effects,” Tran says.
“For acquired hemophilia, having the HTC as the quarterback, so to speak, is very important, just because the local hematologist may never have prescribed these medications,” he says. “The HTC is leading and managing the care, but then the local hematologist may help deliver the treatments that are recommended by the HTC.”
Acquired Hemophilia Treatment: Controlling Bleeding and Restoring Balance
The approach to treating acquired hemophilia is two-pronged, Tran says.
“The first prong is looking at bleed control.” Because acquired and inherited hemophilia with inhibitors both have this goal, many treatment options are similar — namely, bypassing treatments such as recombinant factor VIIa or activated prothrombin complex concentrates (aPCC).
“The second prong is to try to get rid of the inhibitor to allow the patient’s body to start and keep making factor VIII and not have it be cleared out of the body by the inhibitor,” Tran says.
When there is a condition underlying the acquired hemophilia, treatment is as much about optimizing the care for the underlying condition that caused the development of the inhibitor in the first place.
Looking Ahead: Recovery, Support, and Hope
Although people with inherited hemophilia must manage it their whole lives, those with acquired hemophilia could see the condition resolve over time.
If the inhibitor can be eradicated, then the patient will start using their own factor VIII again without it being cleared out of the body by the inhibitor.
In that case, he explains, the condition would be monitored frequently, and if there is no detectable inhibitor and factor levels are normal after some time, the frequency of regular hematology monitoring can decrease significantly and may require no additional treatments.
This is hopeful news, though Tran cautions that for some people, the inhibitor never goes away, and the condition remains.
Research is underway to develop new and improved treatment options for acquired hemophilia, Tran explains. “A lot of these clinical trials are started and carried out in hemophilia treatment centers” because of their expertise with the condition.