Abstract Number: PB0454
Meeting: ISTH 2021 Congress
Background: Haemophilia is a genetic bleeding disorder characterised by prolonged trauma-related or spontaneous bleeding events and associated chronic complications such as synovitis, pain, and, eventually, destruction of the joint. Life-long adherence to prophylactic treatment with factor replacement is required for protection from bleeds and improved haemophilia-related clinical outcomes.
Aims: To investigate the relationship between real world adherence to prophylaxis and long-term clinical outcomes and hospitalisations in adults with severe haemophilia across Europe.
Methods: Physician reported data were extracted from the CHESS II dataset, a prevalence-based cross-sectional, burden-of-illness study in adults with mild, moderate, and severe haemophilia A and B throughout Europe (n=1337). The cohort analysed consisted of people with severe haemophilia receiving prophylaxis, without a current inhibitor diagnosis (n=467). ‘Full adherence’ (FA) was defined as missing fewer than 15% of annual factor infusions (n=385) and ‘moderate or low adherence’ (MLA) was defined as missing ≥15% of annual factor infusions (n=82). Clinical outcomes evaluated were level of chronic pain (none, low, moderate, or high), joint health (a composite measure of no joint issues: absence of both problem joints [PJ] and target joints [TJ]) and annual hospitalisations related to haemophilia.
Results: Full adherence to a prescribed prophylactic regimen was associated with superior clinical outcomes in adults with haemophilia. Those with FA had lower levels of chronic pain, better joint health (higher proportion with 0 TJ/PJ) and fewer annual hospitalisations related to haemophilia, compared to those with MLA (Table 1).
Table 1. Adherence to prophylactic treatment and real-world outcomes in haemophilia
|Joint health, n (%)|
|0 TJ/PJ||203 (52.7)||24 (29.3)|
|Zero haemophilia-related hospitalisations, n (%)||272 (70.6)||32 (39.0)|
|Haemophilia-related hospitalisations, mean (SD, median)||0.51 (0.98, 0)||1.30 (1.49, 1)|
|Chronic pain, n (%)|
|No pain||89 (23.1)||10 (12.2)|
|Mild pain||183 (47.5)||26 (31.7)|
|Moderate pain||100 (26.0)||36 (43.9)|
|Severe pain||13 (3.4)||10 (12.2)|
Conclusions: This study suggests that adherence to current haemophilia prophylactic regimens remains a challenge and this contributes to significant morbidity as evidenced by real-world data. Gene therapy approaches, which eliminate the need for adherence to factor supplementation regimens, may have the potential to provide more consistent levels of factor and therefore improved patient outcomes.
To cite this abstract in AMA style:Burke T, Asghar S, O'Hara J, Yee D, Kar S. Impact of Adherence to Prophylactic Treatment on Real-world Outcomes in Adults with Severe Haemophilia: A CHESS II Analysis in Europe [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/impact-of-adherence-to-prophylactic-treatment-on-real-world-outcomes-in-adults-with-severe-haemophilia-a-chess-ii-analysis-in-europe/. Accessed September 24, 2021.
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