Health‐related quality of life in paediatric haemophilia B patients treated with rIX‐FP

Abstract

IntroductionFrequent infusions and bleeds can impact on the health‐related quality of life (HRQoL) of paediatric haemophilia B patients. rIX‐FP (IDELVION®) is a fusion protein linking recombinant factor IX with recombinant albumin, and is associated with low bleeding rates with a weekly regimen, which could improve HRQoL.AimsTo measure the effect of rIX‐FP prophylaxis on the HRQoL of paediatric patients and treatment satisfaction in their caregivers using the Haemo‐QoL and Hemo‐SATP questionnaires, respectively.MethodsAt baseline and end‐of‐study (EOS), patients 4‐11 years old participating in the PROLONG‐9FP program answered the Haemo‐QoL questionnaire and gave information on their socio‐demographic data and physical activity. Caregivers completed the Hemo‐SatP. Minimal important differences (MID) (|Cohen's d| > 0.5) between baseline and EOS and the number of responders (patients with meaningful subject‐level improvements over time) at EOS were calculated.ResultsTwenty patients (age group I: 4‐7 years old [n = 12]; age group II: 8‐12 years old [n = 8]) completed the Haemo‐QoL questionnaire at baseline. MIDs were found in age group I representing improvement for “physical health” (d = −0.547) domain; 60% of patients were responders for “physical health.” In age group II, MIDs were seen in most domains; 71.4% patients were responders in “total score.” In caregivers, improvements were seen for most domains of the Hemo‐SatP with a small effect size. Fewer patients missed school when treated with rIX‐FP and 94.1% patients maintained their physical activity level.ConclusionProphylaxis with rIX‐FP led to substantial improvements in HRQoL in paediatric patients and treatment satisfaction in caregivers.

Real-World Utilisation and Bleed Rates in Patients with Haemophilia B Who Switched to Recombinant Factor IX Fusion Protein (rIX-FP): A Retrospective International Analysis

“…In the UK, 90.9% of patients who switched to prophylaxis with rIX-FP received once-weekly prophylaxis, which was in line with published guidance on prophylaxis with EHL coagulation factor concentrates from the UK Haemophilia Centres Doctors' Organisation [19]. Reduced dosing frequency may improve treatment adherence and has the potential to improve treatment experience by reducing treatment burden [20]. The higher trough levels achieved with rIX-FP also have the potential to improve patient management and allow the patient to live a more active lifestyle [21].…”

“…The higher trough levels achieved with rIX-FP also have the potential to improve patient management and allow the patient to live a more active lifestyle [21]. While treatment efficacy should always remain the most important outcome when selecting a treatment regimen, the enhanced features of rIX-FP are allowing patients with haemophilia the opportunity to live a healthier and less burdensome life [20].…”

Assessing bleeding rates, related clinical impact and factor utilization in German hemophilia B patients treated with extended half-life rIX-FP compared to prior drug therapy

“…Clinical trials have already demonstrated an improvement in the QoL of patients treated with EHL FIX products. 15,16 Additionally, improving QoL was reported as the most frequent reason to switch to an EHL product according to real-world data collected in Canada. 17…”

Real‐life experience in switching to new extended half‐life products at European haemophilia centres

“…12 Clinical trial outcomes have shown for both EHL recombinant FVIII and FIX drugs that prophylaxis regimens are associated with very low annualized bleeding rates (ABRs) compared to on-demand treatment. 17,18 EHL products have been licensed at different times in the USA and Europe. 17,18 EHL products have been licensed at different times in the USA and Europe.…”

“…11,[13][14][15][16] These results make such therapies significantly less distressing to the patient (particularly in the case of FIX) and are likely to improve their quality of life. 17,18 EHL products have been licensed at different times in the USA and Europe. In the USA, EHL products were available much earlier than in Europe due to the differences between US Food and Drug Administration (FDA) and European Medicines Agency (EMA) regulations for authorizing new products.…”