OR02 - MRI-Based Assessment of Joint Outcomes in Children with Severe Hemophilia A on Emicizumab Prophylaxis: A Prospective Observational Study
OR02
MRI-Based Assessment of Joint Outcomes in Children with Severe Hemophilia A on Emicizumab Prophylaxis: A Prospective Observational Study
L. M. Sherief1,*, O. S. El agamy2, A. sobhy2, A. Darwish3, A. Nazim4, B. K. Soliman5
1Pediatic, Zagazig University, Zagazig, 2Pediatic, Kafrelsheikh University, kafrelsheikh, 3Pediatic, Mansoura University, 4Pediatic, Mansura Health Insurance Hospital, Mansoura, 5Radiodiagnosis, Zagazig University, Zagazig, Egypt
Introduction: Children with severe hemophilia A experience recurrent joint bleeds leading to long-term arthropathies and impaired quality of life. Emicizumab is the first bispecific monoclonal antibody that was approved for routine prophylaxis in children with hemophilia A. While most studies focused on clinical outcomes, this study evaluated the real-world effectiveness of Emicizumab in improving structural outcomes using MRI-based evaluation and clinical scoring
Methods: This prospective, observational, multi-center study included male children (<18 years) diagnosed with severe hemophilia A , who received Emicizumab prophylaxis. Each patient underwent clinical and MRI evaluation of the index joints at baseline and after 12 months. Clinical assessments included changes in annual bleeding rate (ABR), Hemophilia Joint Health Score (HJHS), and MRI scores using the International Prophylaxis Study Group (IPSG) version 1.0 scale.
Results: This study included 39 children with severe hemophilia A with a median age of 8 years (range: 4-17 years). A total of 54 joints were assessed, including 33 knees, 12 ankles, and 9 elbows. After one year of Emicizumab prophylaxis, patients showed marked clinical and radiological improvements. The mean ABR significantly decreased from 50 to 0.46 (p<0.001), and the mean HJHS improved from 13 to 8 (p<0.001). Baseline MRI evaluation revealed joint effusions in 100% of joints, with 50.0%, 22.2%, and 27.8% having small, moderate, and large effusions, respectively. After one year, moderate joint effusions were significantly reduced to 11.1%, and none had large effusions. Synovial hypertrophy decreased from 83.3% to 61.1%, and haemosiderin deposition decreased from 66.7% to 16.7% (p<0.001). Osteochondral changes, improved in 22.2% of joints, while only 5.6% showed progression. Almost 88.9% of joints demonstrated improvement on MRI, with 11.1% remaining stable after one year of prophylaxis.
Discussion/Conclusion: Emicizumab prophylaxis resulted in both clinical and radiological improvements in children with severe hemophilia A. These findings support the role of Emicizumab in controlling joint disease progression and improving long-term musculoskeletal outcomes in children with severe hemophilia A.
Disclosure of Interest: None declared