Abstract Number: PB0452
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 France – 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 France, as captured in the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey – II’ (CHESS II) study.
Methods: Data for non-inhibitor PWHA living in France were extracted from CHESS II and stratified based on clinician-reported baseline endogenous FVIII level. Participant demographics, clinical outcomes (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 a representative from l’Association Française des Hémophiles (AFH) patients’ organisation.
Results: Sixty PWHA were included in the analysis (mild n=10, moderate n=19, severe n=31) with a mean age of 26 years. Forty-five percent of participants were full-time students; for the remainder of the cohort, full-time employment decreased with increasing HA severity (Table 1). Prophylaxis regimens were most common for participants with severe HA. Low incidence was observed for all clinical outcomes, and only target joint incidence increased with increasing severity of HA. EQ-5D-5L index scores were similar across subgroups (Table 2).
| Severity of HA | Mild (n = 10) |
Moderate (n = 19) |
Severe (n = 31) |
TOTAL (n = 60) |
| Age, mean ± SD | 25.10 ± 4.43 | 23.68 ± 4.89 | 28.48 ± 12.99 | 26.40 ± 10.05 |
| Body Mass Index (BMI), mean ± SD | 23.35 ± 2.65 | 25.03 ± 1.96 | 23.83 ± 2.18 | 24.13 ± 2.25 |
| Employment status, n (%) | ||||
| Employed full time | 6 (60%) | 6 (32%) | 6 (19%) | 18 (30%) |
| Employment part-time | 0 (0%) | 1 (5%) | 2 (6%) | 3 (5%) |
| Self-employed | 0 (0%) | 0 (0%) | 2 (6%) | 2 (3%) |
| Unemployed | 1 (10%) | 0 (0%) | 1 (3%) | 2 (3%) |
| Student | 3 (30%) | 8 (42%) | 16 (52%) | 27 (45%) |
| Other | 0 (0%) | 4 (21%) | 4 (13%) | 8 (14%) |
Table 1: Participant demographics by HA severity
| Severity of HA | Mild (n = 10) |
Moderate (n = 19) |
Severe (n = 31) |
TOTAL (n = 60) |
| Receiving factor replacement therapy, n (%) | 3 (30%) | 5 (26%) | 31 (100%) | 39 (65%) |
| Primary on-demand | 3 (100%) | 3 (60%) | 8 (26%) | 14 (36%) |
| Primary prophylaxis | 0 (0%) | 0 (0%) | 2 (6%) | 2 (5%) |
| Secondary on-demand | 0 (0%) | 2 (20%) | 7 (23%) | 9 (23%) |
| Secondary prophylaxis | 0 (0%) | 0 (0%) | 14 (45%) | 14 (36%) |
| Annualised bleed rate, mean ± SD | 1.40 ± 0.97 | 1.06 ± 0.94 | 2.94 ± 2.35 | 2.10 ± 2.01 |
| Target joints, mean ± SD | 0.10 ± 0.32 | 0.11 ± 0.46 | 0.68 ± 1.05 | 0.40 ± 0.85 |
| Problem joints, mean ± SD | 0.10 ± 0.32 | 0.00 ± 0.00 | 0.19 ± 0.48 | 0.12 ± 0.37 |
| EQ-5D-5L, n; mean ± SD | 3; 0.98 ± 0.02 | 8; 0.96 ± 0.04 | 22; 0.96 ± 0.06 | 33; 0.96 ± 0.05 |
Table 2: Treatment strategy, clinical and patient-centric outcomes by HA severity
Conclusions: Real-world data from CHESS II provides preliminary insights into the clinical outcomes and QoL of PWHA in France. Participants enrolled comprised a young cohort of PWHA with low reported incidence of clinical outcomes and high QoL. Further data is needed to contextualize the burdens experienced by PWHA in France.
To cite this abstract in AMA style:
Shaikh A, Burke T, Hawes C, Duport G, O'Hara J, Camp C. Real-world Clinical and Patient-centric Outcomes in People with Haemophilia A in France: 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-france-findings-from-the-chess-ii-study/. Accessed November 28, 2022.« 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-france-findings-from-the-chess-ii-study/