Real-world outcomes associated with standard half-life and extended half-life factor replacement products for treatment of haemophilia A and B

Abstract

Standard-of-care treatment for haemophilia A or B is to maintain adequate coagulation factor levels through clotting factor administration. The current study aimed to evaluate annualised bleeding rates (ABR) and treatment adherence for haemophilia A or B patients receiving standard half-life (SHL) vs. extended half-life (EHL) factor replacement products. We analysed data from the Adelphi Disease-Specific Programmes, a health record–based survey of United States and European haematologists. Analysis included 651 males with moderate-to-severe haemophilia A or B (the United States, n = 132; Europe, n = 519). The haemophilia A analysis included 501 patients (SHL, n = 435; EHL, n = 66). In the combined United States/European population, mean (SD) ABR was 1.7 (1.69) for the SHL group and 1.8 (2.00) for the EHL group. A total of 72% of patients receiving SHL factor VIII and 75% of patients receiving EHL factor VIII in the combined population were fully adherent (no doses missed of the last 10 doses), as reported by physicians. The haemophilia B analysis included 150 patients (SHL, n = 114; EHL, n = 36). The mean (SD) ABR in the combined population was 2.1 (2.16) for patients receiving SHL factor IX (FIX) and 1.4 (1.48) for patients receiving EHL FIX. The percentage of fully adherent patients (physician-reported) was similar in both treatment groups (SHL FIX, 68%; EHL FIX, 73%). In this preliminary real-world survey in a relatively small sample of patients, measures of ABR and adherence between SHL and EHL products were evaluated. Additional real-world research on prescribing patterns, SHL vs. EHL effectiveness, and adherence is warranted.

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

“…Similar to our findings, Tortella and colleagues reported 30% lower mean monthly dispensed IUs and 54% higher mean monthly costs for EHL versus SHL regimens among 296 commercially insured US patients with moderate or severe hemophilia B [ 26 ]. While this study was not meant to compare EHL versus SHL regimens, it is important to note that assessment of direct costs only did not capture the potential clinical benefits and reduced treatment burden associated with an EHL regimen [ 37 , 38 ]. In addition to FIX replacement therapy, the costs of medical encounters such as hospitalizations (and intensive care unit admissions) and physician office visits represent additional financial burden to the healthcare system.…”

Cost of patients with hemophilia A treated with standard half-life or extended half-life FVIII in Spain

“…Preliminary results of a study evaluating real-world outcomes with SHL and EHL in the US and several European countries (Spain included) found numerical differences in the mean total dose per week [34]. This study included 501 HA patients but only 66 patients were under EHL rFVIII treatment, and interestingly there were more moderate patients (59%) than severe patients.…”

“…This study included 501 HA patients but only 66 patients were under EHL rFVIII treatment, and interestingly there were more moderate patients (59%) than severe patients. Regarding mean weekly dose, it was reported 106.2 and 101.29 IU/kg for SHL vs. EHL group, respectively, in the US, and in Europe, 102.8 and 71.5 (IU/kg), respectively [34]. This could suggest that the total per patient dose of FVIII product may differ between Spain and the United States.…”

“…Pivotal clinical trials of EHL for hemophilia A usually report results on the efficacy and safety of severe patients under prophylaxis treatment. Nonetheless, when reviewing the limited publications on the real-life experience with EHL rFVIII we observe an evolving trend of use of FVIII clotting factors also in non-severe population, especially in moderate patients [34,35].…”

Converting factor and nonfactor usage into a single metric to facilitate benchmarking the resources consumed for haemophilia care across jurisdictions and over time

“…Table 5 provides mean values of the annual factor utilization (IU/kg/patient) of real‐world non‐switching studies for SHL and EHL products in the United States and Europe 31,32 . For FVIII products, the weighted–average is 5419 IU/kg (SHL) and 5038 IU/kg (EHL), resulting in a SHL/EHL conversion ratio of 1.08.…”