Hemophilia B Patients in US Face Great Financial, Personal Burden

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Severe hemophilia B patients receiving prophylactic, or preventive treatment with factor IX face a significant financial and personal burden, according to data from two U.S. population-based medical databases.

These findings were reported in a study, “Clinical, humanistic, and economic burden of severe hemophilia B in the United States: results from the CHESS US and CHESS US+ population surveys,” published in the Orphanet Journal of Rare Diseases.

Hemophilia B is an inherited blood disorder caused by the lack of functional factor IX (FIX), an essential blood clotting protein. Preventive treatment consisting of the administration of the missing FIX — a form of treatment known as replacement therapy — is the standard care for these patients, and has significantly improved their life expectancy.

The disease is estimated to affect around 6,000 people, particularly men, in the U.S. However, accurate estimates of the real burden of hemophilia B — key for health management and policy decisions — are lacking in the U.S.

Two databases — the Cost of Severe Hemophilia across the US: a Socioeconomic Survey (CHESS US) and the CHESS US+ — were designed to provide real-world insights into the burden of severe hemophilia B in the U.S. CHESS US includes data from medical records and notes provided by clinicians and healthcare providers, while CHESS US+ contains additional information from questionnaires completed by adult patients with hemophilia.

In this study, researchers used both databases to assess the clinical and economic impact of hemophilia B to U.S. patients who were on FIX prophylaxis between 2017 and 2019.

In total, they reviewed data from 44 patients who were part of CHESS US and for whom data on FIX prophylaxis were available.

Out of the 44 patients, 20 received FIX therapy with standard half-life (SHL) and 24 received extended half-life (EHL) therapies. Therapies with EHL — meaning those whose levels take longer in the body to drop by half — were designed to allow for less-frequent infusions while maintaining bleed protection, and improve patients’ compliance, compared to standard treatments.

The researchers also analyzed data from 57 patients with severe hemophilia B who were part of CHESS US+. More than half (59%) had a record of FIX prophylaxis and were included in the analysis (22 received SHL, 35 received EHL).

Patients in CHESS US+ were in general older than those in CHESS US (mean age of 35.6 vs. 27.6 years) and heavier (mean weight of 85.6 vs. 75.7 kg).

Approximately half of the patients in both databases had full-time jobs (52% in the CHESS US and 49% in the CHESS US+). Nearly all patients in CHESS US (82%) and all in CHESS US+ were covered by insurance.

The most common additional conditions reported included anxiety, depression, osteoarthritis, and type 2 diabetes.

Data from CHESS US showed that patients bled, on average, 1.73 times a year. Also, 9.1% of the patients reported at least one bleed-related hospitalization in the previous year.

Approximately one-fifth of the patients (18%) reported having at least one target joint, meaning a joint that had a tendency to bleed, and 11% of patients reported at least one joint problem.

The majority of patients (84%) in CHESS US+ reported chronic pain in the past year. Over half of them (56%) also stated the disease had a direct impact on their daily lives, and most (91%) said they adapted their FIX treatment schedule to accommodate the impact on their physical activity.

Calculations based on data from CHESS US showed that the mean annual direct costs of FIX replacement therapy amounted to $614,886. Also, the mean annual costs were higher for EHL FIX therapy ($788,491) than for SHL ($397,491). The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931.

“We observed a substantial clinical and humanistic burden of hemophilia B on patients receiving FIX prophylaxis in the US, and substantial FIX treatment-driven costs to the society,” the researchers wrote.

“Despite recent therapeutic advances in the treatment of severe hemophilia B, tangible unmet needs remain to better serve patients with this lifelong condition in a sustainable manner,” they added.