Does Prophylaxis Help Prevent Joint Damage?

By Dr. David Clark

2/19/24  With the increasing use of prophylaxis, the incidence and severity of joint damage has decreased.  A group of researchers from The Netherlands has looked at this in more detail.  They studied X-rays of 1064 joints in 363 patients with severe or moderate hemophilia A or B at their center.  Seventy-seven percent of patients with severe hemophilia developed arthropathy (joint damage).  However, when the patients were divided by birth cohort (born before 1970, born between 1970 and 1980, born 1981 to 1990, or born after 1990) they could see the decreasing incidence of joint damage. 

Factor concentrates were first developed in the 1960s and by the 1970s patients in Europe were using prophylactic treatment.  (Prophylaxis started later in the U.S.)  In addition, the median age for starting prophylaxis decreased from 18 years to 2.1 years over time.  (Note that over time, as the younger cohorts continue to age, their joint damage may increase.)In severe patients, ankles were the most common affected joint, followed by elbows and knees.  In the “before 1970” cohort, the incidence of joint damage in at least one ankle was 97.8%.  This was reduced to 23.5% in the “after 1990” cohort.  Knees were affected the least but still saw a decrease from an incidence of 86.7% in the “before 1970” cohort to 3.9% in the cohort born after 1990. 

Moderate patients also saw a decrease in joint damage but to a lesser extent, probably since fewer moderate patients are on prophylaxis.  Only 26.1% of moderate patients were on prophylaxis compared to 94.3% of the severes.  Interestingly, the most affected joint for moderates was the knee, which was the least affected for severes.  The study also had a small number of inhibitor patients in the severe group.  Since the development of inhibitors tends to happen in the first 50 exposure days of treatment, the age for first inhibitor development has fallen from a median of 17.4 years for the oldest cohort to 1.5 years in the youngest.  Overall, though, inhibitor development did not appear to be associated with any increase in joint damage. 

This study emphasizes the importance of prophylactic treatment.  Prophylaxis is regular, continuing treatment with clotting factor, as opposed to on-demand treatment in which you infuse only when a joint bleed has already started.  [van Heel DAM et al., Res Pract Thromb Haemost, 8(2), 102335, 2024]