Preventive therapy, also known as prophylaxis, is proving to be a game-changer for patients aged 50 and older with severe hemophilia types A and B. Recent findings from a post hoc analysis of four Phase 3 clinical trials have revealed that this therapy not only significantly reduces bleeding episodes, including those affecting target joints, but also enhances quality of life for elderly patients suffering from this condition.

The analysis was detailed in a letter titled “Long-term clinical outcomes of prophylaxis with an rFVIIIFc or rFIXFc in adults aged ≥50 years with hemophilia A or B,” published in Blood Advances. Hemophilia, a genetic disorder, results when specific clotting factors—namely factor VIII (FVIII) in type A and factor IX (FIX) in type B—are deficient. Prophylactic treatment, considered the gold standard, involves regularly administering the missing clotting factors to prevent bleeding episodes before they occur.

Traditionally, such treatments are most effective when started early in life, which means older patients who did not receive prophylaxis earlier could face severe complications, including hemophilic arthropathy—a painful and debilitating condition characterized by chronic joint damage due to repeated bleeds.

Significant Reductions in Bleeding Episodes

The recent study focused on patients aged 50 and older who participated in pivotal clinical trials: the A-LONG (for hemophilia A) and B-LONG (for hemophilia B) studies, along with their respective extension trials. Notably, these trials evaluated extended half-life products, such as efmoroctocog alfa (marketed as Elocta) and Alprolix (eftrenonacog alfa).

The results were striking: patients with hemophilia A showed a median age of 57 at the start of A-LONG, with 81% experiencing target joints—joints that had experienced three or more spontaneous bleeds within a short time frame. Meanwhile, in B-LONG, the median age was 56 with over half of the participants also showing signs of target joints.

Before entering these studies, the majority of patients had been relying on on-demand treatment rather than prophylaxis, but the shift to preventive therapy led to notable reductions in annualized bleeding rates (ABR). Those who had previously experienced more frequent bleeds saw the most significant improvements upon starting prophylaxis.

Improvement in Joint Health and Overall Quality of Life

Not only did the bleeding episodes decline, but joint health improved remarkably as well. All 42 previously troubled target joints in 12 participants from the A-LONG/ASPIRE studies and all 19 in B-LONG/B-YOND were healed following the introduction of prophylactic therapy.

The analysis also revealed enhanced quality of life for these patients, as measured by the Hemophilia Quality of Life Questionnaire for Adults. Improvements were particularly noted in areas such as physical health and recreational activities. The researchers concluded that prophylactic treatment may enable older patients with hemophilia to engage in more physical activities, enhancing their overall well-being.

These findings underscore the potential long-term benefits of prophylaxis, proving that therapies like Eloctate and Alprolix can deliver significant advantages, regardless of a patient’s age or severity of hemophilia. This breakthrough highlights the importance of reevaluating treatment approaches for older hemophilia patients, suggesting that it’s never too late to initiate a proactive strategy against this challenging condition.