Limited Access to Hemophilia Treatment and Expert Care in Developing Countries Reasons Joint Damage Seen, Study Reports

Inadequate access to treatment centers, lack of expert care, and high bleeding rates lead to similar joint complications in hemophilia patients with and without inhibitors, and joint problems in 70% of the people taking part in a recent study in five developing countries.

The research, “HAEMOcare: The First International Epidemiological Study Measuring Burden of Hemophilia in Developing Countries,” appeared in the journal TH Open.

Optimizing care can be beneficial for hemophilia patients, especially those in developing countries where resources are limited and access to treatment may be nonexistent. However, unlike in developed countries, large-scale studies aiming to improve disease management and outcomes are still scarce.

To address this gap, the HAEMOcare study (NCT01503567) was conducted in five countries — India, Algeria, Morocco, Oman, Egypt and South Africa — to specifically assess the link between hemophilia-related joint damage and inhibitors against clotting factors, treatment history, disease management, quality of life, and economic aspects.

A total of 282 males with severe hemophilia A (250) or B (32) and a mean age 20.8 were enrolled in 2012. All were receiving on-demand treatment for bleeding.

The pediatric group – ages 6 to 18 – included 128 patients, 24 (18.8%) of whom had inhibitors. Of the 154 adults, 26 (16.9%) were with inhibitors. More patients with inhibitors had a family history of inhibitors than those without. The disease was diagnosed at a mean age of 34 months, and inhibitors found at a mean age of 14.6 years.

The mean annualized bleeding rate was 25.8 and was similar comparing the groups with and without inhibitors. This rate was higher than that reported in studies in developed countries, the researchers noted.

Hemophilia-related orthopedic disabilities had a significant impact on the Hemophilia Joint Health Score (HJHS) — an assessment of hemophilia-related joint clinical and functional health — in 70% of patients. No differences in HJHS and in the Pettersson score of the severity of arthropathy (joint disease) were seen between patients with or without inhibitors. Adults had higher (worse) HJHS and Pettersson scores than children.

As for disease management, in the 12 months prior to assessment, 206 patients without inhibitors (88%) received on-demand treatment with factor VIII or IX, the two clotting proteins that are missing in patients with hemophilia A and B, respectively.

Among the 50 patients with inhibitors, the more commonly used treatment was activated recombinant factor VII. It was used by 21 patients, including 12 of the 26 adults in this group.

Twenty-six patients had used prophylaxis treatment before enrollment, most (20) being adults without inhibitors. More patients with inhibitors received hemostatic treatment within the first two hours of bleeding compared to those without, but no significant differences were seen in hemophilia-related orthopedic surgeries, use of orthopedic aids, or regular participation in physical therapy sessions or sports.

A greater proportion of those with inhibitors (50%) received home treatment compared to those without (35%). The mean distance to a hemophilia treatment center (HTC) was 79.4 kilometers or almost 50 miles. About 133 patients, or 47.2%, had regular access to an orthopedic expert, which was associated with less severe joint complications.

A majority of participants reported complications related to quality of life, as assessed with the EuroQol five-dimension questionnaire (EQ-5D-3L). Most often, they were related to pain/discomfort (65.2%) and mobility (56.4%), and were similar between adults and children, and between patients with or without inhibitors.

Total hemophilia-related costs — including direct (treatment and transportation costs) and indirect expenses (lost working days/family productivity), as well as ability to cover expenses — were significantly higher in patients with inhibitors than those without. Most patients without reimbursement were from India (64 people) and Morocco (41). In contrast, all patients in Algeria were reimbursed in full.

Overall, “inadequate access to HTCs and expert care, along with high bleeding rates, led to equivalent hemophilia-related orthopedic morbidity between hemophilia patients without and with inhibitors,” the researchers wrote.