O’Donovan M, Johnston K, Singleton E, Benson J, Ryan K, O’Connell N. Impact of Switching to Prophylaxis with Recombinant Factor IX Fc Fusion Protein Concentrate on Patient Reported Outcomes in People with Haemophilia B Using the PROBE Questionnaire [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/impact-of-switching-to-prophylaxis-with-recombinant-factor-ix-fc- fusion-protein-concentrate-on-patient-reported-outcomes-in-people-with-haemophilia-b-using-the- probe-questionnaire/. Accessed July 22, 2020.
Background
In 2017 all patients with severe Haemophilia B in Ireland switched to prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) concentrate from treatment with standard half-life (SHL) recombinant FIX. The Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire was developed to assess patient reported outcomes (PRO). Ithas four major sections; demographics, general health problems, haemophilia related health problems and health related quality of life.
Aims
To evaluate impact of switching to prophylaxis with rFIXFc from treatment with SHL FIX on PRO at two years after switchover.
Methods
Following ethical approval and informed consent, patients completed PROBE questionnaires pre switchover and at 24 months. Descriptive statistics were used for analysis.
Results
23 paired responses from 23 patients, median age 49 (31-72) years, with severe Haemophilia B were analysed. Acute pain, chronic pain and use of pain medication were reported at high rates at both 0 months (48%, 83% & 83%) and 24 months (64%, 74% & 95%). There was a reduction in chronic pain (83% to 74%) after 24 months of treatment with rFIXFc with a corresponding reduction in difficulties with activities of daily living (ADL). At 0 months, 61% reported :53difficulties with ADL. In comparison, at 24 months 68% reported :53 ADL difficulties, with greatest improvement seen with doing heavy domestic tasks (17%), bending down (13%), walking (13%) and going upstairs (13%). There was a reduction in reported number of bleeds; 50% :53 bleeds/annum in year prior to switchover, compared to 87% :53 bleeds/annum at 24months, with 35% reporting no bleeds.
Conclusions
From this initial data analysis there is a PRO benefit following a switch to rFIXFc prophylaxis with improvement in ADL, reduced chronic pain and bleeding rates. To further understand the patient experience post rFIXFc switch we plan to continue data analysis and perform qualitative interviews.
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