Abstract Number: PB0546
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Progressive arthropathy is the main cause of morbidity in hemophilia. Prophylaxis significantly reduces acute bleeds but subclinical disease may still occur. The ultrasound detection of early joint damage may contribute to optimize prophylaxis.
Aims: To assess the joint status in a series of hemophilia patients under prophylactic treatment by means of the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) system.
Methods:
Starting from January 2016, we prospectively performed the ultrasound examination of the elbows, the knees and the ankles in a series of 39 consecutive hemophilia patients under prophylactic treatment (Table). Ultrasound assessments were performed during the annual follow-up visit. When synovial hypertrophy was detected, an additional ultrasound study was planned 6 months later than the baseline examination.
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Table: Patients’ characteristics. |
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diagnosis (n°of patients) |
severe HA (27) |
moderate HA (4) |
severe HB (8) |
Total (39) |
|
median age (range) |
14 yrs (3-37) |
9 yrs (6-13) |
12 yrs (4-33) |
14 yrs (3-37) |
|
prophylaxis with SHL products |
8 |
3 |
1 |
12 |
|
prophylaxis with EHL products |
13 |
1 |
7 |
21 |
|
prophylaxis with Emicizumab (n° of patients with/without inhibitors) |
6 (2/4) |
n.a. |
n.a. |
6 |
|
HA = hemophilia A; HB = hemophilia B; SHL = standard half-life; EHL = extended half-life; n.a.= not applicable |
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Patients’ characteristics.
Results: A total of 105 ultrasound examinations (630 joints) using the HEAD-US protocol were performed. At the first examination, 11 patients under primary prophylaxis (5 severe HA; 2 moderate HA, 4 HB) had HEAD-US score=0, whereas 5 patients with severe arthropathy (4 HA; 1 HB) presented worse HEAD-US scores related to osteochondral damage. The median HEAD-US score at baseline was 3. A second follow-up examination was performed in 35 patients (29 HA, 6 HB) with a median follow-up of 28 (range 6-60) months. Out of the 7 patients with HEAD-US score=0 evaluable for follow-up, 5 (2 severe HA, 1 moderate HA and 2 HB treated with rVIIIFc, moroctocog alfa and rIX-FP respectively) didn’t change joint status and HEAD-US score during follow-up (median 44 months, range 17-60), whereas 2 patients with severe HA treated with SHL products worsen their synovial HEAD-US subscore. The follow-up results of patients with HEAD-US>0 is illustrated in the Figure.
Three-years follow-up of patients with arthropathy
Conclusions: Ultrasound performed with the HEAD-US protocol may help clinicians to monitor synovial hypertrophy and osteochondral damage during prophylaxis. Although preliminary, our data suggest progressive reduction of active joint disease during optimized prophylaxis.
To cite this abstract in AMA style:
Ricca I, Pollio B, Albiani R, Martinoli C. Ultrasound Assessment of the Joint Status in Hemophilia Patients under Prophylaxis Using the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Score [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/ultrasound-assessment-of-the-joint-status-in-hemophilia-patients-under-prophylaxis-using-the-hemophilia-early-arthropathy-detection-with-ultrasound-head-us-score/. Accessed October 2, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/ultrasound-assessment-of-the-joint-status-in-hemophilia-patients-under-prophylaxis-using-the-hemophilia-early-arthropathy-detection-with-ultrasound-head-us-score/