PB0238 - Clinical and patient-centered outcomes among Italian people with severe haemophilia A receiving prophylaxis: real-world findings from ‘Cost of Haemophilia: a Socio-economic Survey’ (CHESS) data base

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

Giancarlo Castaman

Medical Director Hematologist
Department of Oncology Careggi University Hospital, Florence, Italy
Florence, Toscana, Italy

Presenting Author(s)

rS

rita carlotta Carlotta Santoro

MD
UO Emostasi e Trombosi, Azienda Ospedaliero-Universitaria Dulbecco, Catanzaro, Italy
Catanzaro, Calabria, Italy

Co-Author(s)

MD

Matteo Nicola Dario Di Minno

MD
Dipartimento di Medicina Clinica e Chirugia, Università di Napoli Federico II, Napoli, Italy
Napoli, Campania, Italy
EZ

Ezio Zanon

Physician
Haemophilia Centre, General Medicine, Padua University Hospital, 35121 Padua, Italy
Padova, Veneto, Italy
LS

Luigi Sannino

Medical Manager
Roche Italy
Monza, Lombardia, Italy
RT

Rosaria Tempre

Evidence Generation Manager
Roche Italy
MONZA, Lombardia, Italy
SB

Sara Bendinelli

Blood Rare Disease Medical Lead
Roche Italy
Monza, Lombardia, Italy
TB

Tom Burke

Principal Health Economist
HCD Economics
Knutsford, England, United Kingdom
TB

Tom Blenkiron

Health Economist
HCD Economics
Knutsford, England, United Kingdom
EF

Enrico Ferri Grazzi

Senior Health Economist
HCD Economics
Knutsford, England, United Kingdom

Background: Limited evidence is available describing how burden of illness measures change in the context of an evolving treatment paradigm for people with severe haemophilia A (PwSHA).

Aims: This analysis aimed to evaluate clinical and patient-reported outcomes (PROs) in a sample of Italian PwSHA, alongside current/historical clinical characteristics and treatment approaches.

Methods: Descriptive analysis examined data on non-inhibitor PwSHA receiving emicizumab or FVIII prophylaxis from the CHESS data platform (2019-2022) (a repeated European cross-sectional retrospective, burden-of-illness study conducted in adult PwH) within two non-mutually-exclusive groups: one comprising individuals with a physician questionnaire (Full-cohort) and another with complete patient forms (PRO-cohort).

Results: Of 238 PwSHA enrolled in Italy with ≥6months on current treatment, 121 (51%) were on emicizumab and 117 (49%) on FVIII prophylaxis, with a mean age of 35.4 and 38.3. Of these, 125 (52%) completed a patient-form [78 (62%) on emicizumab and 47 (38%) on FVIII] (Table 1). Mean-all-bleed annualized bleeding rate (ABR) was 2.0 and 3.8, and number of problem joints 0.5 and 1.2 for the emicizumab and FVIII cohorts, respectively. Moderate/severe chronic pain was reported in 12.4% and 38.4% of the emicizumab and FVIII cohorts, respectively.
Among emicizumab patients with pre-switch bleeding data (n=108), a reduction in ABR was observed vs. previous treatment regimen (1.8 vs 2.8), with 78.7% switching from FVIII prophylaxis.
Comparing the emicizumab and FVIII cohorts, PRO analysis showed higher EQ-5D scores (0.76 vs. 0.59) and lower caregiver requirement (25.6% vs 44.7%), work productivity loss (25.7% vs. 34.2%) and activity impairment (23.3% vs. 30.0%) and anxiety/depression, measured with GAD-7 (4.4 vs. 9.0) and PHQ-8 (6.0 vs. 7.8) in emicizumab cohort (Table 2).

Conclusion(s): These findings suggest that PwSHA in this cohort experiencing sub-optimal bleed control switching to emicizumab, generally experience reductions in ABR. The emicizumab cohort reported favorable joint and pain outcomes and EQ5D, mental health and productivity scores.