CO002 | RETROSPECTIVE LONGITUDINAL ASSESSMENT OF JOINT HEALTH AND QUALITY OF LIFE IN ADOLESCENT AND ADULT PATIENTS AFFECTED BY MODERATE OR SEVERE HAEMOPHILIA A WITH OR WITHOUT INHIBITOR SWITCHING FROM INTRAVENOUS FVIII TREATMENT TO SUBCUTANEOUS PROPHYLAXIS WITH EMICIZUMAB
S. Ceglie1, R. Gualtierotti1|2, S. Arcudi1, A. Ciavarella1, S.M. Siboni1, F. Peyvandi1|2 | 1Centro Emofilia e Trombosi Angelo Bianchi Bonomi, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy; 2Dipartimento di Fisiopatologia Medico-Chirurgica e dei Trapianti, Università degli Studi di Milano, Milano, Italy
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Background and Aims. Joint damage and impaired quality of life (QoL) remain major long-term complications in patients with hemophilia A (HA). Real-world longitudinal data after switching to emicizumab prophylaxis are limited. The aim of our study is to evaluate longitudinal changes in joint health and QoL in moderate/severe HA patients switching to emicizumab.
Methods. Among 101 patients switching from FVIII to emicizumab at our center, 41 underwent systematic joint health assessment (between 2020 and 2024), at baseline, 1 year after the switch, and after 2 years (n=21). Joint health was assessed using the Hemophilia Joint Health Score (HJHS) and the Hemophilic Early Arthropathy Detection with Ultrasound (HEAD-US) score, the QoL with the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) questionnaire, and the EuroQol Visual Analogue Scale (EQ-VAS). The Anualized Bleeding Ratio (ABR) was recorded. Changes between −1 and +1 points in HEAD-US were considered stable; ≥2-point variation indicated improvement or worsening. Continuous variables are reported as median (IQR/range). Fisher’s exact test compared worsening in target vs. non-target joints. QoL changes were analyzed with Wilcoxon and Friedman tests (p<0.05).
Results. The HEAD-US score remained mostly stable from baseline (median 12, IQR 3-19) to 1 (14, IQR 4-20) and 2 years (11, IQR 2-18). For HJHS, 32/41 patients (78.0%) were stable or improved at 1 year, and 15/21 (73.7%) showed stability or improvement at 2 years. EQ-VAS remained stable. EQ-5D-5L showed significant improvements in pain/discomfort (p=0.024) and mobility (p=0.030) (Figure). To provide a joint-level perspective, 224 joints were evaluated at 1 year and 106 at 2 years, with stability in HEAD-US score predominating (>95% unchanged at 1 year and similarly high rates at 2 years). At 1 year, worsening occurred only in 6/103 target joints compared with 1/121 non-target joints (OR 7.42, p=0.050); at 2 years, worsening was observed in 3/39 target joints versus 1/67 non-target joints (OR 5.5, p=0.14). From baseline to 1 year, patients with an ABR of 0 increased (16/41 vs 22/41; risk difference RD 15%), whereas the proportion of patients with ABR ≥ 3 decreased (10/41 vs 5/41; RD 12%).
Conclusions. In our cohort of HA patients switching to emicizumab, joint health was preserved. Most joints remained stable, with limited worsening mainly involving target joints. Improvements in pain/discomfort and mobility further support the clinical benefit of emicizumab beyond structural outcomes.
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