OC 12.1 - First evidence of improvement of long-term joint outcomes through ultrasound for hemophilia; from 3-years prospective J-DaUPHIN study

Background: Early-stage joint damage in hemophilia can be evaluated using point-of-care ultrasound (POCUS). Yet, the relationship between the POCUS and long-term outcomes has not been investigated.

Aims: Provide final results for 3 years of J-DaUPHIN (Joint Damage Ultrasonography Patients Hemophilia IN Japan) study.

Methods: Prospective, observational, non-interventional, ethics-approved study conducted at Ogikubo hospital. Inclusion criteria was for participants (≥ 6 years) who are congenital severe hemophilia without inhibitors, received prophylaxis. The POCUS (elbows, knees, and ankles of staged I to III according Arnold classification), adherence to prophylaxis, patient-reported joint bleedings and HJHS are planned to perform and collect every half-year for 3 years. Clinical visits for acute joint bleedings were excluded. Wilcoxon signed-rank test was used for significant differences.

Results: In total 174 patients with median age 34 years old and 682 joints [238 elbows, 293 knees and 151 ankles] were enrolled and followed for 3 years. Total 3772 joints were scanned and evaluated. Figures showed the long-term outcome of synovitis and joint bleeding. Synovitis scores on elbows and knees, spontaneous patient-reported joint bleeding and HJHS were significantly improved at 3 years (visit 7) compared to visit 1 (fig1, 2). Synovitis was detected in 472/3776(12.5%) joints among whole study periods. Surprisingly, 421/472(89.2%) of the synovitis had no record of bleeding in the infusion records for the six months prior to the ultrasound. This was not characterized by age group. For those 85 patients with synovitis, prophylaxis regimen was modified [66 once, 19 twice or more]. Increasing the dose of the extended half-life product was the most common method as step1, with 22 attempts, but also included many ways to modification including switching to non-factor products.

Conclusion(s): This is the first evidence that the ultrasound for hemophilia improves their long-term joint outcomes. Joint ultrasound detects asymptomatic synovitis and improves joint prognosis.