South American men with severe hemophilia face ‘large burden’
Direct and indirect costs, most related to treatment, also affect quality of life
Men in South America with severe hemophilia A or B are hit with both direct and indirect costs related to the genetic disease, which also affect quality of life, a new study reports.
As much as 99% of the direct costs of hemophilia were related to factor replacement therapy — one of the standard treatments for hemophilia, typically used as a prophylaxis. Regardless of whether preventive or on-demand treatments are used, such therapies are typically required over a patient’s lifetime, adding to the disease’s direct costs.
When also including indirect costs, such as lost wages, the average total annual spending per person — the economic burden of the disease — was estimated to be more than $100,000. However, that figure varied across surveyed countries and disease types, as did quality of life outcomes.
“The results of the … study highlight the large burden faced by patients with severe hemophilia A and B in Latin America,” the researchers wrote, noting that the burden “was shown to be high in South America across Argentina, Brazil, Chile, and Colombia with factor replacement therapy as the key driver of cost.”
The team added, however, that “despite the dominating treatment costs, the indirect impact of hemophilia on the patients and those who care for them was still substantial and should not be dismissed.”
The study, “Impact of Severe Hemophilia on Costs and Quality of Life in South American Men: Findings From the CHESS LATAM Study,” was published in the journal Value in Health Regional Issues.
In hemophilia, problems with blood clotting lead to the disease’s characteristic symptoms of easy and prolonged bleeding. Genetic mutations — affecting different genes for these two disease types — typically cause these issues by disrupting the production or function of specific clotting proteins.
Different types of hemophilia are related to deficiencies in different clotting proteins. In hemophilia A, there is a deficiency of factor VIII (FVIII), while in hemophilia B, there is a lack of factor IX (FIX). Both of these conditions are more common in men.
Study focused on 4 countries: Argentina, Brazil, Chile, and Colombia
Treatment for hemophilia A or B may involve on-demand therapies to control active bleeding and prophylactic treatments to prevent future bleeds. Factor replacement therapies, which provide the body with a version of the missing clotting factor, are common preventive treatments.
The disease may lead to long-term reductions in health-related quality of life and work productivity, particularly in severe cases. It’s typically also coupled with increased health resource use and costs. Nonmedical expenses, such as the cost of traveling to a treatment center, may also accrue.
“Although the comprehensive burden of hemophilia has been studied in Europe and North America, the impact of severe hemophilia … in Latin and South America has not been well characterized,” the researchers wrote.
To learn more, an international research team assessed the costs incurred by men with severe hemophilia A and B receiving care in South America.
A total of 830 men from four countries, namely, Argentina, Brazil, Chile, and Colombia, participated in the study. These men had a mean age of 33.6, and most (81%) had hemophilia A.
Data on medical costs came from the men’s physicians. A subset of participants also provided information on nonmedical and indirect costs, as well as on quality of life data. The researchers translated costs to U.S. dollars.
Annual cost of severe hemophilia? $104K per person.
On average, direct medical costs unrelated to factor replacement therapy were similar across disease types: $1,559 for hemophilia A and $1,563 for hemophilia B. However, the cost of factor replacement therapies and Hemlibra (emicizumab-kxwh), a nonfactor prophylactic therapy, was higher among patients with hemophilia A than in those with hemophilia B, the data showed.
Direct nonmedical costs and indirect costs were also higher for the hemophilia A patients, the researchers noted.
“It is important to consider indirect costs to truly appreciate the burden faced by not only patients but caregivers and the healthcare systems themselves in these countries,” the team wrote.
The total annual cost of treating severe hemophilia across the four countries was estimated to amount to slightly less than $104,000 per patient.
It is important to consider indirect costs to truly appreciate the burden faced by not only patients but caregivers and the healthcare systems themselves in these countries.
There were also differences in quality of life measures across disease types. On a scale of 0 (worse health) to 1 (perfect health), mean health-related quality of life scores were higher for men with hemophilia A than for those with hemophilia B (0.59 vs. 0.46).
“Despite showing a smaller socioeconomic burden of disease, men with severe [hemophilia B] showed worse [health-related quality of life] outcomes, suggesting that there is an unmet need associated with this population in the [four] South American countries studied,” the researchers wrote.
The team noted the findings of this study, dubbed “Cost of Hemophilia in Latin America: A Socioeconomic Survey,” or CHESS LATAM, generally match those of similar studies carried out in other locations.
“The findings of the CHESS LATAM study are consistent with those found in previous CHESS studies that prophylaxis treatment makes up the vast majority of the economic burden in severe hemophilia, although other medical costs and patient costs are still of importance,” the researchers wrote.
As a key limitation of the study, the team noted the unequal participant response rate in surveys across different countries.
As such, “subsequent work extending and further exploring these findings will be essential for providing the fullest and most accurate picture of the impact of hemophilia on clinical outcomes, costs, and [health-related quality of life ],” the scientists wrote, adding that the results “may be useful to inform hemophilia management strategies for both clinicians and policy makers.”
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