O’Mahony B, Pastarnak A, Noone A, Skinner M. Patient Reported Outcome Data on Switch from Standard to Extended Half Life in Irish Adults with Haemophilia. Abstract OC 42.4. Res Pract Thromb Haemost. 2019;3(S1):1-228. https://doi.org/10.1002/rth2.12227
Background
From 2017 to the end of 2018, all people with Haemophilia B and the majority with Haemophilia A (PwH) in Ireland were switched to extended half-life FIX (rFIXFc) or FVIII (rFVIIIFc) concentrates.
Objectives
The Irish Haemophilia Society surveyed measurable impact on their health-related quality of life.
Methods
Utilising the Patient Reported Outcomes, Burdens and Experiences (PROBE) survey, the Society surveyed members of the organisation with Haemophilia A/B pre-change and a minimum of 10 months post product change. There were 12 paired responses from people with Haemophilia B and 5 with haemophilia A.
Results
Three patients switched from episodic treatment to regular prophylaxis (2-HB, 1-HA).
Post-switch, there were reductions in PWH reporting the use of mobility aids (67% to 53%), acute pain (78% to 47%), frequency of use of pain medications, activities of daily living (ADL) being affected, reduced range of motion (83% v 65%) and chronic pain in a target joint (patient defined) (67% to 13%).
There was a significant reduction in reported bleeds. Pre-switch 29% reported having greater than 8 bleeds/year and 41% having 1 or less bleed/year. Post-switch there we no responses greater than 8 bleeds/year and 59% having 1 or less bleed/year.
Overall, VAS (68 v 73), EQ-5D (0.71 v 0.77) and PROBE (0.72 v 0.78) score improved post-switch, which equates to ~5-6% increase in utility in a year. All dimensions of the EQ-5D showed improvement with the most significant changes seen in mobility, self-care and pain/discomfort. (Table 1).
Conclusions
From this initial data set, there is a demonstrated benefit to PWH switching to EHL products in reduced bleeding rates as well as in the physical and pain limitations that haemophilia imposes on daily life. Data collection and analysis are ongoing.
View Presentation: Data on switch from standard to extended half life in Irish adults with haemophilia
