Abstract Number: PB0456
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Basic
Background: Haemophilia A (HA; factor VIII [FVIII] deficiency), characterised by prolonged trauma-related and/or spontaneous intra-articular bleeding events, is associated with adverse impacts on physical functioning and quality of life (QoL). The breadth of clinical and patient relevant outcomes/experiences of people with HA (PWHA) in Spain – and variation between those with mild, moderate and severe HA – is not well understood.
Aims: To examine demographic, clinical and patient-centric outcomes across condition severity among adult PWHA in Spain, as captured in the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey – II’ (CHESS II) study.
Methods: Data for non-inhibitor PWHA living in Spain were extracted from CHESS II and stratified based on clinician-reported baseline endogenous FVIII level. Participant demographics, annualized bleeding rate (ABR), incidence of target joints (ISTH definition) and joints exhibiting symptoms of chronic damage (‘problem’ joints), treatment regimen and QoL (EQ-5D-5L index score) were assessed. Outcomes by HA severity were compared using descriptive statistics.
Results: One hundred and eighty-seven PWHA were included in the analysis (mild n=33, moderate n=66, severe n=88). The proportion of participants in full-time employment declined with increasing HA severity; reporting of part-time employment increased with increasing severity (Table 1). Participants with mild/moderate HA received on-demand FVIII; prophylaxis regimens were most common in participants with severe HA. Mean ABR, incidence of target joints and EQ-5D-5L index score increased with severity. Incidence of problem joints was highest in participants with moderate HA (Table 2).
Severity of HA | Mild (n = 33) |
Moderate (n = 66) |
Severe (n = 88) |
TOTAL (n = 187) |
Age, mean ± SD | 43.58 ± 17.03 | 40.77 ± 16.08 | 39.44 ± 14.28 | 40.64 ± 15.42 |
Body Mass Index (BMI), mean ± SD | 25.08 ± 2.45 | 24.63 ± 2.65 | 24.76 ± 2.75 | 24.77 ± 2.65 |
Employment status, n (%) | ||||
Employed full time | 20 (61%) | 25 (38%) | 31 (35%) | 76 (41%) |
Employment part-time | 2 (6%) | 8 (12%) | 22 (25%) | 32 (17%) |
Self-employed | 3 (9%) | 1 (2%) | 4 (5%) | 8 (4%) |
Unemployed | 1 (3%) | 4 (6%) | 3 (3%) | 8 (4%) |
Student | 4 (12%) | 7 (11%) | 9 (10%) | 20 (11%) |
Other | 3 (9%) | 21 (32%) | 19 (22%) | 43 (23%) |
Severity of HA | Mild (n = 33) |
Moderate (n = 66) |
Severe (n = 88) |
TOTAL (n = 187) |
Receiving factor replacement therapy, n (%) | 8 (24%) | 25 (38%) | 88 (100%) | 121 (65%) |
Primary on-demand | 8 (100%) | 20 (80%) | 37 (42%) | 65 (54%) |
Primary prophylaxis | 0 (0%) | 0 (0%) | 10 (11%) | 10 (8%) |
Secondary on-demand | 0 (0%) | 5 (20%) | 6 (7%) | 11 (9%) |
Secondary prophylaxis | 0 (0%) | 0 (0%) | 35 (40%) | 35 (29%) |
Annualised bleed rate, mean ± SD | 1.09 ± 0.96 | 2.77 ± 3.57 | 4.58 ± 10.65 | 3.34 ± 7.73 |
Target joints, mean ± SD | 0.06 ± 0.24 | 0.53 ± 0.83 | 1.00 ± 1.30 | 0.67 ± 1.08 |
Problem joints, mean ± SD | 0.33 ± 0.48 | 0.86 ± 1.18 | 0.78 ± 1.12 | 0.73 ± 1.07 |
EQ-5D-5L, n; mean ± SD | 15; 0.87 ± 0.19 | 28; 0.81 ± 0.15 | 55; 0.77 ± 0.19 | 98; 0.80 ± 0.18 |
Conclusions: The CHESS II study enrolled a large cohort of PWHA in Spain and provides an important resource for exploring differences in clinical outcomes and QoL across the spectrum of HA severity. This analysis observed increasing clinical burden and lower QoL associated with greater condition severity. Collection of further country-specific data will help to validate these findings and provide further insight into the differences observed in the present study.
To cite this abstract in AMA style:
Shaikh A, Burke T, Hawes C, O'Hara J, Camp C. Real-world Clinical and Patient-centric Outcomes in People with Haemophilia A in Spain: Findings from the CHESS II Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/real-world-clinical-and-patient-centric-outcomes-in-people-with-haemophilia-a-in-spain-findings-from-the-chess-ii-study/. Accessed March 22, 2024.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/real-world-clinical-and-patient-centric-outcomes-in-people-with-haemophilia-a-in-spain-findings-from-the-chess-ii-study/