Severe hemophilia still tough to manage, study shows

Men with severe hemophilia were more likely than men with mild hemophilia to report missing more than 10 days of work or school during the previous year. In Era A, the proportions were 6.9% and 2.6%, respectively. In Era D, the proportions were 5.6% and 3%, respectively.

“Clear disparities remain in terms of frequent bleeding and disability between men with severe hemophilia and mild hemophilia across every decade of adult life,” Dr Monahan said.

“We thought the difference in functional outcomes would have narrowed over the years. That is, men with severe hemophilia should look more like those with

mild disorder, given improved therapeutics and access to care, but this wasn’t the case.”

“What needs examination is why, despite widespread availability of preventive and on-demand therapies for home use, we still see disparities. It speaks to the need for continued disease surveillance to monitor and inform hemophilia interventions and outcomes.”

Mortality

There were 551 deaths during the study period. The Era A and B cohorts accounted for 82% of the deaths in the severe hemophilia population and 96% of the deaths in the mild hemophilia population.

The researchers noted that liver failure has surpassed bleeding issues and HIV as the leading cause of death among US men with hemophilia.

Although there were no liver-related deaths in the 2 youngest cohorts, liver failure was the most commonly reported cause of death across all the cohorts, for both severe hemophilia (33% of deaths) and mild hemophilia (26% of deaths).

The researchers said this finding underscores the need to swiftly evaluate and treat HCV infections.

“Liver disease worsens bleeding, so eradicating hepatitis C infections needs to be a priority, especially as we now have remarkably effective therapies,” Dr Monahan said.

Across all the birth cohorts, hemophilia-related deaths accounted for 14.6% of deaths in patients with severe hemophilia and 10.7% of deaths in those with mild hemophilia.