PB1244 - Early joint damage detection in pediatric patients with moderate and severe hemophilia: usefulness for treatment individualization

Background: Early detection of hemophilic arthropathy is crucial to assure joint health in patients with hemophilia. The standard of care for periodic joint evaluation is the clinical examination using specific scales such as Hemophilia Joint Health Score (HJHS). Ultrasound evaluation has been also recently included in the follow-up using the HEAD-US (Hemophilia Early Arthropathy Detection with Ultrasound) protocol. Joint health evaluation could be useful to individualize treatment.

Aims: Description of joint damage detection in pediatric patients and outcomes after treatment modification.

Methods: Retrospective periodic evaluation (every 6 months) of joint health using HJHS and HEAD-US in pediatric patients (3-18 years-old) diagnosed with moderate or severe hemophilia from January 2019 and December 2022. Treatment approach due to new/persistent synovitis during joint evaluation were assessed.

Results: A total of 37 patients were included (severe n=36; moderate n=1). All of them were receiving prophylactic treatment (table 1). First joint evaluation showed that higher HJHS and HEAD-US scores were related to age, inhibitor persistence and longer inhibitor duration. HEAD-US score was 0 in 20 patients (54%) and 1-2 in 9 patients (14.2%). Synovitis was seen in 28 of a total of 222 joints (12.6%); elbows were the most frequent damaged joints.
During the follow-up, a total of 257 HJHS/HEAD-US evaluations in 36 patients were collected. A statistically significant correlation between HJHS and HEAD-US was found, but coefficient was moderate (p < 0.0001; r=0.458). Of note, HJHS score < 4 with absence of cartilage/bone joint damage was described in all patients but five. Treatment was modified 38 times in 24 patients; 15 times due to joint health findings (figure 1).

Conclusion(s): Joint health periodic evaluation is useful to detect an early joint damage. However, the role of transient joint synovitis in tailoring treatment modifications is still unknown. Long-term follow-up will help us to draw any further conclusions in this regard.