ISTH Academy

Arthropathy following repeated bleeding is common in persons with hemophilia. Since the introduction of prophylaxis, treatment has intensified and joint health has improved. However, data on the long-term development of arthropathy are still scant.

To evaluate long-term arthropathy development since the introduction of prophylaxis according to birth cohort, hemophilia severity, and inhibitor status.

This single-center historic cohort study included persons with severe and moderate hemophilia A and hemophilia B born between 1935 and 2005. Arthropathy on X-rays was evaluated using the Pettersson score. Patient and joint characteristics were studied per birth cohort (<1970, 1970-1980, 1981-1990, and >1990) and compared according to hemophilia severity. The distribution of affected joints and cumulative incidence of arthropathy were analyzed. The association of Pettersson score with birth cohort and inhibitor characteristics was explored using multivariable regression analyses adjusted for age at evaluation.

In total, 1064 X-rays of 363 patients were analyzed. Of persons with severe hemophilia (n = 317, 87.3%), 244 (77.0%) developed arthropathy. Prophylaxis was started at younger ages over time, from a median of 18 to 2.1 years, and concomitantly, arthropathy decreased in consecutive birth cohorts. Ankles were most commonly affected in 188 of 258 (72.9%) patients. Persons with moderate hemophilia (n = 46, 12.7%) had a lower risk of arthropathy (27/46 [58.7%]), but a reduction over time was less pronounced. In the multivariable analyses, birth cohort and age at evaluation were predictors for the development of arthropathy, while inhibitor status showed no association.

The development and severity of arthropathy have decreased over the past decades. However, patients have remained at risk for developing arthropathy, especially in their ankles.

• Joint damage due to bleeds is common in persons with hemophilia, impairing quality of life. • Long-term joint damage development and its predictors were studied in 1064 X-rays of 363 patients. • Birth cohort and age at X-ray evaluation were independent determinants of joint damage. • Ankles remained most susceptible to joint damage in the youngest birth cohorts.