The impact of factor infusion frequency on health-related quality of life in people with haemophilia

Abstract

BackgroundSome studies suggest that people with haemophilia (PwH) who use prophylaxis value low frequency of clotting factor administration more than a lower risk of bleeding. However, more frequent infusions offer the potential of reducing joint disease and pain, which in turn may improve functioning and quality of life.AimsTo explore the impact on health-related quality of life (HRQoL) aspects of haemophilia associated with adherence and annual infusion rate in the context of factors influencing treatment that are important to patients, including prophylaxis, chronic pain, concomitant conditions and hospital admission.Materials and methodsHRQoL was assessed in participants with severe haemophilia in the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS) study who were using prophylaxis. Patients using on-demand treatment were excluded. This multivariate analysis examined the interaction between factors potentially influencing treatment and HRQoL, and minor and major bleeds.ResultsFrom the total CHESS population (n=1,285), 338 (26%) participants provided responses for major and minor bleeds and target joints, and 145 (11%) provided EQ-5D-3L responses. Major and minor bleeds were associated with pain. Patients with severe chronic pain reported a substantial negative impact on HRQoL; but this was significantly improved by increases in the annual infusion rate. This was not apparent in participants with mild or moderate pain.ConclusionIncreasing the frequency of prophylaxis infusions is associated with improved quality of life in PwH who have severe chronic pain. However, increasing the number of infusions per week in those with mild or moderate chronic pain with the intention of improving prophylactic effect may not have the same effect.

Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A

“…23 However, patients without inhibitors are expected to have a higher quality of life; therefore our utilities may be underestimated. 5 Also, our approach to use FVIII to simulate bleeding rates in the gene therapy arm was done to incorporate treatment durability as well overcome the limitation that valrox and prophylactic FVIII benefits are FVIII-level driven, and emicizumab is not. 72 The last limitation is that we did not take into account the costs and burden of administration of FVIII (intravenously) and emicizumab (subcutaneously) by the patients at home 3 times a week (around 1500 times and 250 times over 10 years, respectively), which may mean we underestimated societal costs and burden.…”

“…Longer-acting FVIII substitutes and nonfactor replacement therapies allow less frequent (intravenous) administrations increasing patient mobility and quality-of-life. 5 The latest innovation in HA-treatment is the emergence of gene therapies. 6 The promise of one-time treatments inducing prolonged or sustained near-normal FVIII is considered a potential transformative innovation and creates high expectations among patients and physicians.…”

Recombinant factor IX‐Fc fusion protein in severe hemophilia B: Patient‐reported outcomes and health‐related quality of life

“…The presence of the disability paradox among people with hemophilia has been recently described, with people with hemophilia reporting higher health states than the general population when assessed with the EQ‐5D. 36 Other important factors such as pain, joint disease, and ability to perform ADLs also impact on HRQoL, 37 , 38 and the PROs in this study illustrate the high disease burden experienced by Irish adults with severe hemophilia B. The majority of this study population did not receive childhood prophylaxis and problems secondary to chronic muscle and joint deterioration may not demonstrate improvement, particularly with a short observation period.…”

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

“…Although increasing dosing frequency can achieve elevated factor levels, this approach comes with challenges, including the potential need for central venous access devices (CVAD) in young children, and the possibility of reduced adherence in older children and adults 1,8 . There is a balance between the benefits of increasing dosing frequency and the negative impact this has on burden of treatment and HRQoL 58 . In the CHESS study, improvements in HRQoL were observed in participants with severe chronic pain who increased their dosing frequency; however, participants with mild or moderate pain did not experience improvements in HRQoL, potentially due to the negative impact of additional infusions on their HRQoL 58 …”

“…1,8 There is a balance between the benefits of increasing dosing frequency and the negative impact this has on burden of treatment and HRQoL. 58 In the CHESS study, improvements in HRQoL were observed in participants with severe chronic pain who increased their dosing frequency; however, participants with mild or moderate pain did not experience improvements in HRQoL, potentially due to the negative impact of additional infusions on their HRQoL. 58…”