Abstract Number: PB0487
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 Germany – 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 Germany, as captured in the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey – II’ (CHESS II) study.
Methods: Data for non-inhibitor PWHA living in Germany 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 German patients’ organisation IGH.
Results: Forty-seven PWHA were included in the analysis (mild n=10, moderate n=16, severe n=21). The proportion of participants in full-time employment decreased with increasing HA severity (Table 1). Two-thirds of participants with severe HA received prophylaxis FVIII regimens. Mean ABR and incidence of target joints and problem joints were suggestive of greater impairment with increasing severity of HA. EQ-5D-5L index scores showed a potential downward trend (Table 2).
| Severity of HA | Mild (n = 10) |
Moderate (n = 16) |
Severe (n = 21) |
TOTAL (n = 47) |
| Age, mean ± SD | 40.70 ± 11.14 | 44.75 ± 15.72 | 39.95 ± 11.07 | 41.74 ± 12.76 |
| Body Mass Index (BMI), mean ± SD | 24.69 ± 1.84 | 24.73 ± 2.62 | 25.45 ± 3.13 | 25.04 ± 2.70 |
| Employment status, n (%) | ||||
| Employed full time | 8 (80%) | 12 (75%) | 14 (67%) | 34 (72%) |
| Employment part-time | 0 (0%) | 1 (6%) | 2 (10%) | 3 (6%) |
| Self-employed | 1 (10%) | 1 (6%) | 4 (19%) | 6 (13%) |
| Unemployed | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) |
| Student | 1 (10%) | 1 (6%) | 0 (0%) | 2 (4%) |
| Other | 0 (0%) | 1 (6%) | 1 (5%) | 2 (4%) |
Table 1: Cohort demographics by HA severity
| Severity of HA | Mild (n = 10) |
Moderate (n = 16) |
Severe (n = 21) |
TOTAL (n = 47) |
| Receiving factor replacement therapy, n (%) | 3 (30%) | 4 (25%) | 21 (100%) | 28 (78%) |
| Primary on-demand | 3 (100%) | 4 (100%) | 7 (33%) | 14 (50%) |
| Primary prophylaxis | 0 (0%) | 0 (0%) | 8 (38%) | 8 (29%) |
| Secondary on-demand | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) |
| Secondary prophylaxis | 0 (0%) | 0 (0%) | 6 (29%) | 6 (21%) |
| Annualised bleed rate, mean ± SD | 0.70 ± 0.48 | 1.25 ± 1.57 | 1.86 ± 1.53 | 1.40 ± 1.44 |
| Target joints, mean ± SD | 0.00 ± 0.00 | 0.19 ± 0.40 | 0.43 ± 0.81 | 0.26 ± 0.61 |
| Problem joints, mean ± SD | 0.10 ± 0.32 | 0.19 ± 0.40 | 0.57 ± 1.25 | 0.34 ± 0.89 |
| EQ-5D-5L, n; mean ± SD | 2; 0.97 ± 0.04 | 4; 0.90 ± 0.08 | 0; – | 6; 0.92 ± 0.08 |
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 people with mild, moderate and severe HA in Germany. A low incidence of clinical outcomes was reported, though a pattern of increased clinical burden with declining baseline FVIII levels was observed. Additional QoL data could provide insight into how clinical outcomes impact upon daily functioning and lived experiences of PWHA in Germany.
To cite this abstract in AMA style:
Shaikh A, Burke T, Hawes C, Becker T, Brandt S, O'Hara J, Camp C. Real-world Clinical and Patient-centric Outcomes in People with Haemophilia A in Germany: 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-germany-findings-from-the-chess-ii-study/. Accessed November 29, 2023.« 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-germany-findings-from-the-chess-ii-study/