Results
As of June 2022, 743 females under 18 years of age who were identified
as having hemophilia A or B or were carriers of hemophilia A or B were
enrolled in the ATHNdataset. Demographic information regarding these
subjects is found in table 1. P values for all comparisons were
>0.05. Three-hundred-forty-three were children under 11
years of age and 400 were adolescents between 11 and 17.99 years of age.
Roughly three-fourths were hemophilia A carriers or had hemophilia A,
and one-fourth were hemophilia B carriers or had hemophilia B. Of the
626 with known factor activity levels, 305 (49%) had activity levels of
40 IU/dL or less, thereby meeting criteria for having hemophilia. Of
these, 18 had activity levels in the severe range, and 9 were moderate.
Adolescents with hemophilia B were more likely to report factor activity
levels less than 40 IU/dL than adolescents with hemophilia A (p=0.02).
There was no statistical difference in children reporting factor
activity levels < 40 IU/dL between hemophilia A and B (p=0.3).
The mean factor activity (standard deviation) for children was 45 (28)
and for adolescents was 47 (28) (p=0.5). Figure 1 shows the distribution
of factor activity levels for the entire pediatric population of
subjects enrolled in the ATHNdataset.
Two-hundred-twenty-three subjects reported an ISTH-BAT BLS and
demographics regarding this population is found in table 2. Ninety-eight
percent of subjects reporting a BLS participated in the MLOFgenotype initiative. The mean BLS for adolescents (1.35) compared to
children (0.86), was statistically different (p=0.03), but not a
clinically relevant difference. Only 30 (13.5%) subjects reported an
abnormal bleeding score for age. Seventeen percent of adolescents
reported an abnormal BLS for age compared to 7.7% of children
(p=0.045). There was no statistically significant difference in those
reporting an abnormal BLS between hemophilia A and B for children or
adolescents (p=0.3 and p=0.8). Of the 223 subjects who submitted an
ISTH-BAT BLS, 195 submitted both a BLS and factor activity level.
Twenty-seven (13.8%) reported an abnormal BLS for age. Figure 2 shows
the proportion with an abnormal bleeding score by factor activity level
for children and adolescents. Fourteen (23%, 3 children) subjects with
factor activity levels under 40 IU/dL reported an abnormal BLS for age
while 13 (9.7%, 4 children) of those with factor activity levels over
40 IU/dL did. Because the sample size of subjects reporting an abnormal
BLS was so small, additional evaluations to determine factors
contributing to an abnormal BLS were not undertaken.