OC 71.5 - Joint health and quality of life scores from baseline to one year after switching to emicizumab

Wednesday, June 26, 2024

11:15 – 11:30 ICT

Room: Ballroom B2

Presenting Author(s)

Sara Arcudi, MD, PhD

Clinician
Foundation I.R.C.C.S. Ca’ Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy

Co-Author(s)

Roberta Gualtierotti, PhD

Associate Professor
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy
Milan, Lombardia, Italy
AG

Andrea Giachi

Physician
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
Milan, Lombardia, Italy
VB

Valentina Begnozzi

Physioterapist
Foundation I.R.C.C.S. Ca’ Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi and Thrombosis center, Milan, Italy.
Milan, Lombardia, Italy
EB

Elena Boccalandro

Physioterapist
Foundation I.R.C.C.S. Ca’ Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi and Thrombosis center, Milan, Italy.
Milan, Lombardia, Italy
Alessandro Ciavarella

Clinician
Fondazione IRCCS Ca’Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
SS

Simona Maria Siboni

Physician
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
Milan, Lombardia, Italy
Flora Peyvandi, MD, PhD

Full Professor
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Pathophysiology and Transplantation
Milan, Lombardia, Italy

Background: The efficacy and safety of emicizumab in severe emofilia A (HA) were established by several clinical studies and real-world reports, although less is known concerning the effect on joint health and quality of life.

Aims: To evaluate joint health and quality of life before switching and one year after emicizumab prophylaxis.

Methods: Consecutive HA patients starting emicizumab were enrolled in a prospective cohort study. Joint health was assessed with the Hemophilia Joint Health Score (HJHS version 2.1) and the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score. The synovitis total score was assessed with the HEAD-US. Quality of life was assessed only in adults with the 5-level EQ-5D version tool (EQ-5D-5L). All scores were collected by the same operators before switching to emicizumab and 12 months after. Paired-samples T-test was performed.

Results: Twenty-five patients on emicizumab prophylaxis were enrolled, 5 children (median age 4, IQR 3-8) and 20 adults (median age 43, IQR 33-50). Three adult patients had inhibitor and 7 adult patients had one or more prosthetic joint. Fourteen adult patients had synovitis according to the HEAD-US score before emicizumab switch and in 10 out of 14 patients the total synovitis score diminished after 1 year (median synovitis score before 1, IQR 0-1; median synovitis score after 0, IQR 0-1), p-value < 0.01 (Figure 1). The osteochondral and subchondral bone damage score did not change before and after one year of prophylaxis with emicizumab. The median HJHS score was 15 before switching (IQR 4-23) and 11 after 1 year (IQR 2-20), p-value 0.07. Regarding the EQ-5D-5L tool, 4 out of 5 domains improved after switching prophylaxis (Figure 2).

Conclusion(s): In conclusion, the total synovitis score diminished in 10 out of 14 patients after 12 months of emicizumab prophylaxis. Based on the quality-of-life assessment tool, 4 out of 5 domains improved after switching prophylaxis.