Using Drug for Prevention Might Help in Hard-to-Treat
WEDNESDAY, Nov. 2 (HealthDay News) -- A compound currently used
to stop bleeding episodes in a subset of hard-to-treat hemophilia
patients also seems to be effective in preventing bleeds before
Although this study, published in the Nov. 3 edition of the
New England Journal of Medicine, was a small one and didn't last long, the results do raise the possibility that, over time, this type of use will also prevent the much-feared joint damage that is a hallmark of the condition.
"This study shows that it is at least possible to keep these people relatively healthy, without bleeds, by using chronic prophylactic [preventive] agents," said Dr. Thomas Harrington, director of the Adult Hemophilia and Adult Sickle Cell programs at the University of Miami Miller School of Medicine. He was not involved with the study.
The research was funded by Baxter BioScience, which makes FEIBA
(Factor VIII Inhibitor Bypassing Activity), the agent tested in
Most people with hemophilia have a genetic defect in a clotting
factor known as factor VIII. This results in excessive bleeding and
bruising either as the result of traumas -- even small ones -- or
sometimes occurring spontaneously.
About half of the bleeds occur in the joints and can lead to
painful and debilitating joint disease.
Clotting factor concentrates that contain factor VIII are
extremely effective both to stop bleeds when they happen (called
"on-demand" treatment) and to prevent bleeds when given three or so
times a week.
The problem is that about one-third of hemophilia patients
develop antibodies to factor VIII, meaning the compound just stops
So-called "bypassing agents" have been developed for patients
who have factor VIII inhibitors, but these aren't nearly as
effective and have only been used on an on-demand basis.
The authors designed this study to see if bypassing agents might
prevent bleeds in this subset of patients in the same way that
factor VIII concentrates successfully prevent bleeds in people
Thirty-four patients over the age of 2 were randomly assigned to
receive either six months of preventive thrice-weekly FEIBA (also
known as AICC or anti-inhibitor coagulant complex) or to receive
After a three-month "washout" period, the groups were
Twenty-six participants completed the study. The bleeding rates
were reduced by 62 percent in the prophylaxis group compared to the
Even patients with the most severe disease saw benefits, with
the average number of bleeding episodes decreasing from 3.8 to 1.1
in the seven patients who had the most frequent bleeding before the
study. Two of these individuals had no bleeding at all during the
There was one allergic reaction to FEIBA, while several
participants had complications from the hemophilia: Two adults died
from bleeding episodes and two experienced brain bleeds.
Cost remains a significant issue.
Using the bypassing agent prophylactically would cost more than
double what it would for on-demand (nearly $500,000 versus nearly
$210,000 per patient in this study), the authors said, although
these calculations didn't subtract for hospitalizations avoided and
prevention of long-term complications.
"The cost is so extreme but the benefits are pretty extreme, too," said Harrington. "Three times a week is very, very costly [both for factor VIII concentrates and bypassing agents] but it keeps the joints healthy and provides good quality of life so these people can live normal lives."
FEIBA is licensed in the United States for on-demand treatment
of hemophilia only. According to study author Dr. Cindy Leissinger,
Baxter BioScience has started a study that "I feel quite sure is
being done with a labeling indication in mind."
In the meantime, FEIBA could be used preventively on an
off-label basis, something insurance companies might not cover.
And even so, the authors noted, more, longer and bigger studies
are needed to assess potentially beneficial effects on long-term
health, particularly joint health.
National Hemophilia Foundation has more on this
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