Unmet Medical Need in Severe Hem B Care, European Study Shows

Patricia Inacio PhD avatar

by Patricia Inacio PhD |

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Adults with severe hemophilia B have a persistent number of bleeds per year, as well as joint problems and mild to moderate chronic pain, revealing an unmet medical need in these patients, according to real-world data from the European CHESS II study.

The study, “Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe,” was published in the Orphanet Journal of Rare Diseases.

Since hemophilia B is four to five times less common than hemophilia A, real-world studies focused on assessing the current burden of severe hemophilia B are still limited.

To better understand the clinical and economic burden of severe hemophilia B in Europe, a team of researchers at uniQure, along with colleagues at HCD Economics and the University of Chester, analyzed data from the CHESS II study.

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CHESS II investigated the burden of illness in adults with severe hemophilia A or B across the U.K. and seven other European countries: Denmark, France, Germany, Italy, the Netherlands, Romania, and Spain.

Specifically, they analyzed retrospective data, collected from 2019 to 2020, of severe hemophilia B patients from France, Germany, Italy, Spain, and the U.K.

Patients had not developed FIX inhibitors — neutralizing antibodies that target therapies meant to replace FIX, the clotting factor missing in hemophilia B patients — at the time of data collection and were being treated with either standard (SHL) or extended half-life (EHL) replacement therapies on a preventive basis.

The analysis included both patient- and physician-reported clinical, humanistic, and economic outcomes. Participants completed questionnaires on bleeding, joint status, quality of life, and hemophilia-related direct and indirect costs.

In total, they reviewed data from 75 hemophilia B patients, including 42 (56%) who were treated with SHL and 33 (44%) with EHL. Patient-reported outcomes were available for 40 (53%) patients.

Most patients (70%) had two or more bleeding events per year, with a mean annualized bleed rate (ABR) of 2.4. ABR was similar for patients receiving EHL or SHL preventive therapy (2.5 vs. 2.4).

The proportion of patients with a history of joint surgery rates was also similar for those receiving EHL or SHL replacement therapies (33% vs. 24%, respectively).

However, a greater proportion of patients receiving EHL reported having at least one target joint (30% vs. 17%). Target joints were defined as joints where three or more bleeds occurred over a period of six months.

Over three-quarters of the patients reported mild or moderate chronic pain (76%). Also, most patients (63%) reported that hemophilia had an impact on their daily lives, and 20% stated they had to adapt their treatment regimen before physical activity.

The mean annual medical costs due to hemophilia per patient amounted to €235,723 (about $262,430). Most of these costs were driven by FIX treatment costs, which totaled €232,328 (about $258,650). Costs were higher for EHL (€290,620, or about $323,547) than for SHL (€186,528 or about $207,661).

FIX treatment costs varied substantially between countries, with the highest costs seen in Germany (€393,263, or about $437,820) and the lowest in the U.K. (about €182,219, or about $202,864). The mean annual indirect costs (€8,973 or $9,995) were driven by early retirement or the need to stop working due to hemophilia.

Patients had a mean score of 0.67 on the European Quality of Life 5-Dimensions (EQ-5D), a standardized measure of overall health and quality of life. Its scores range from zero (worse possible state) to one (best possible health state).

Overall, these real-world data “demonstrate the substantial clinical and humanistic burden of severe haemophilia B, together with a consistent economic burden,” despite preventive therapy, the researchers wrote.

Moreover, according to researchers, these findings highlight the “persistent unmet needs for these patients, their caregivers, and society.”