Abstract Number: OC 07.3
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Early-stage joint damage in hemophilia can be evaluated using point-of-care ultrasound (POCUS). However, the relationship between POCUS and long-term follow-up outcomes has not been investigated.
Aims: To provide the 2-year preplanned interim results of the Joint Damage Ultrasonography Patients Hemophilia in Japan (J-DaUPHIN) study.
Methods: This is an ongoing, prospective, observational, non-interventional, ethics-approved study conducted at Ogikubo Hospital. Patients (≥ 6 years) with congenital severe hemophilia without inhibitors, receiving prophylaxis, were included. Data regarding POCUS (elbows, knees, and ankles at stages I–III according to the Arnold-Hilgartner classification), adherence to prophylaxis, joint bleeding, and HJHS is collected every 6 months (for a planned period of 3 years). Spontaneous annual joint bleeding rate (AjBR) is evaluated by home infusion records every 6 months. Significance was assessed using the Wilcoxon signed-rank test.
Results: Results are as of March 31, 2020. In total, 174 patients with a median age of 34 years and 689 joints (240 elbows, 296 knees, and 153 ankles) were enrolled and followed for 2 years. Within this 2-year period, a total of 1,252 joints were scanned by ultrasound and evaluated. Synovitis was detected in 396 joints (129 elbows, 210 knees, and 57 ankles). Patient records confirmed bleeding in 42 (10.6%) of these. Symptomatic bleeding was not observed in the remainder. The hemostatic treatment regimen was modified for 49 patients (58 joint synovitis). Their adherence to prophylaxes improved from 94.4% to 100%, and synovitis was resolved in 27 joints (26 patients). Figures 1 and 2 show the interim outcomes of synovitis, adherence, and HJHS. There was a tendency for improvement, but this was not statistically significant. Median spontaneous AjBR was consistently 0.
Figure 1. Long-term outcome of synovitis through ultrasoundFigure 1
Figure 2. Long-term outcome of adherence to prophylaxis and HJHS through ultrasoundFigure 2
Conclusions: The results after 2 years of this ongoing, prospective study regarding long-term outcomes for hemophilia though ultrasound showed the possibility of improved outcomes.
To cite this abstract in AMA style:
Nagao A, Takedani H, Orita S, Fukutake K, Suzuki T. Long-term Joint Outcomes in Hemophilia through Ultrasound: 2-year Interim Results from the Prospective J-DaUPHIN Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/long-term-joint-outcomes-in-hemophilia-through-ultrasound-2-year-interim-results-from-the-prospective-j-dauphin-study/. Accessed September 24, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/long-term-joint-outcomes-in-hemophilia-through-ultrasound-2-year-interim-results-from-the-prospective-j-dauphin-study/