Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life. Issue 7 (11th October 2021)

• Record Type: Journal Article
• Authors: O'Donovan, Mairead; Quinn, Eimear; Johnston, Kate; Singleton, Evelyn; Benson, Julie; O'Mahony, Brian; Noone, Declan; Duggan, Cleona; Gilmore, Ruth; Ryan, Kevin; O'Donnell, James S.; O'Connell, Niamh M.
• Other Names: Mahlangu Johnny handlingEditor.
• Is Part Of: Research and practice in thrombosis and haemostasis. Volume 5:Issue 7(2021)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2021-10-11
• Subjects: extended half‐life concentrate -- hemophilia B -- health‐related quality of life -- pain -- patient‐reported outcomes; Thrombosis -- Periodicals; Hemostasis -- Periodicals; 616.135005
• DOI: 10.1002/rth2.12602
• Languages: English
• ISSNs: 2475-0379

Abstract

Abstract: Introduction: In 2017, all people with severe hemophilia B in Ireland switched to recombinant factor IX Fc fusion protein concentrate (rFIXFc) prophylaxis. Patient‐reported outcomes (PROs) and health‐related quality of life (HRQoL) are important to evaluate with new treatments. Aims: To assess HRQoL in people with severe hemophilia B and their experience after switching to rFIXFc prophylaxis. Methods: Participants completed a Patient Reported Outcomes Burden and Experience (PROBE) questionnaire on initiation and following two years of rFIXFc prophylaxis. The PROBE questionnaire has four domains: demographics, general health, haemophilia‐specific, and European Quality of Life 5‐Dimensions (EQ‐5D‐5L) questionnaire. Results: Twenty‐three participants completed the questionnaire at both time points. The number of activities where chronic pain occurred and interfered with the activity was reduced by 25% and 33%, respectively ( P < .001), following two years of rFIXFc prophylaxis. There was a 9% decrease in chronic pain during the second year of rFIXFc prophylaxis compared to baseline, but the rate remained high, at 74%. A 25% reduction in the number of affected activities of daily living (ADLs) was reported following 2 years of rFIXFc prophylaxis ( P = .007). The most common health problems were arthritis, hypertension, anxiety/depression, and gingivitis. The median EQ‐5D‐5L score was similar following two years of rFIXFc prophylaxis, 0.76 (range, −0.01 to 0.95), compared to 0.77 (range, 0.36‐1) at baseline. Conclusion: This study of real‐world patient experience using PROs demonstrates a reduction in chronic pain and improvement in ADLs in participants after switching to rFIXFc prophylaxis. It provides important insights into patient‐identified health care needs and living with severe hemophilia B.