Impact of the Individualized 4-Year Physiotherapy on the Musculoskeletal System and Quality of Life of Patients With Severe Hemophilia A With Inhibitors.

INTRODUCTION

The development of factor VIII inhibitors is a significant complication of hemophilia A, increasing the risk of bleeding and resulting in high morbidity. Recurrent joint bleeds lead to severe arthropathy and a reduced quality of life compared to patients without inhibitors. Although recent therapies have improved outcomes, many patients with longstanding inhibitors already experience irreversible joint damage. Research on rehabilitation in hemophilia, particularly for patients with inhibitors, remains limited.

AIM: This 4-year study evaluated the impact of individualized physiotherapy on musculoskeletal health and quality of life (QoL) in severe hemophilia A patients with inhibitors.

METHODS

Twenty-five adults (aged 19-69) with severe hemophilia A and inhibitors received personalized home physiotherapy supervised by a specialist, alongside biannual 5-day rehabilitation camps. Prophylactic activated prothrombin complex concentrate (aPCC) was administered. Joint status was assessed biennially via Hemophilia Joint Health Score (HJHS) and MRI of target joints; QoL was measured using EQ-5D questionnaires.

RESULTS

No bleeding episodes occurred during prophylaxis. Thirteen patients completed follow-up. MRI scores stabilized (mean change: +1.3 points; 95% CI: 0.3-2.3), while HJHS improved significantly from 41.1 (95% CI: 32.5-49.7) to 24.9 (95% CI: 16.9-32.7). EQ-5D scores rose from 54.0 (95% CI: 48.2-59.8) to 86.7 (95% CI: 81.9-91.5), reflecting enhanced mobility, reduced pain and greater independence.

CONCLUSION

Individualized rehabilitation with aPCC prophylaxis significantly improved joint function (notably pain and mobility) and QoL, despite limited MRI changes due to pre-existing arthropathy. These results advocate for long-term, tailored physiotherapy in inhibitor patients.