Abstract Number: PB0479
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 the United Kingdom (UK) – 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 the UK, as captured in the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey – II’ (CHESS II) study.
Methods: Data for non-inhibitor PWHA living in the UK 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. Study methodology and interpretation of results were informed by representatives from the UK Haemophilia Society.
Results: Sixty-nine PWHA were included in the analysis (mild n=11, moderate n=22, severe n=36). The proportion of participants in full-time employment decreased with increasing HA severity (Table 1). The majority of participants received on-demand treatment regimens. Mean ABR, incidence of problem joints and EQ-5D-5L index score were all suggestive of greater impairment with increasing HA severity. No similar pattern was observed in target joint outcomes (Table 2).
Severity of HA | Mild (n = 11) |
Moderate (n = 22) |
Severe (n = 36) |
TOTAL (n = 69) |
Age, mean ± SD | 43.82 ± 18.31 | 29.68 ± 10.41 | 27.78 ± 8.38 | 30.94 ± 12.31 |
Body Mass Index (BMI), mean ± SD | 24.50 ± 3.36 | 23.93 ± 5.38 | 24.54 ± 4.42 | 24.34 ± 4.55 |
Employment status, n (%) | ||||
Employed full time | 4 (36%) | 4 (18%) | 3 (8%) | 11 (16%) |
Employment part-time | 1 (9%) | 5 (23%) | 9 (25%) | 15 (22%) |
Self-employed | 2 (18%) | 5 (23%) | 14 (39%) | 21 (30%) |
Unemployed | 0 (0%) | 2 (9%) | 1 (3%) | 3 (4%) |
Student | 1 (9%) | 2 (9%) | 4 (11%) | 7 (10%) |
Other | 3 (27%) | 4 (18%) | 5 (14%) | 12 (17%) |
Severity of HA | Mild (n = 11) |
Moderate (n = 22) |
Severe (n = 36) |
TOTAL (n = 69) |
Receiving factor replacement therapy, n (%) | 4 (36%) | 14 (64%) | 36 (100%) | 54 (78%) |
Primary on-demand | 4 (100%) | 8 (57%) | 6 (17%) | 18 (33%) |
Primary prophylaxis | 0 (0%) | 0 (0%) | 8 (22%) | 8 (15%) |
Secondary on-demand | 0 (0%) | 6 (43%) | 13 (36%) | 19 (35%) |
Secondary prophylaxis | 0 (0%) | 0 (0%) | 9 (25%) | 9 (17%) |
Annualised bleed rate, mean ± SD | 1.18 ± 0.87 | 3.00 ± 1.54 | 4.28 ± 1.77 | 3.38 ± 1.93 |
Target joints, mean ± SD | 0.00 ± 0.00 | 0.18 ± 0.50 | 0.11 ± 0.40 | 0.12 ± 0.40 |
Problem joints, mean ± SD | 0.00 ± 0.00 | 0.18 ± 0.39 | 0.36 ± 0.49 | 0.25 ± 0.43 |
EQ-5D-5L, n; mean ± SD | 3; 0.88 ± 0.10 | 8; 0.81 ± 0.18 | 4; 0.61 ± 0.06 | 15; 0.77 ± 0.17 |
Conclusions: Data from the CHESS II study provides important insights into the clinical experience and QoL of people with mild, moderate and severe HA in the UK. A pattern of increased burden in clinical outcomes and QoL was observed with declining baseline FVIII levels. This study highlights the importance of real-world data in understanding the burden across the spectrum of HA in the UK setting.
To cite this abstract in AMA style:
Shaikh A, Burke T, Hawes C, McKeown W, Morgan D, O'Hara J, Camp C. Real-world Clinical and Patient-centric Outcomes in People with Haemophilia A in the United Kingdom: 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-the-united-kingdom-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-the-united-kingdom-findings-from-the-chess-ii-study/