22 June 2020

A new study supports the use of prophylactic therapy for children with severe haemophilia A to reduce the risk of joint damage into young adulthood, researchers say.

Treatment with intravenous factor VIII (FVIII) in early childhood was investigated by Dr Beth Warren of the University of Colorado, USA, and her team.

An earlier trial, the Joint Outcome Study (JOS), found that preventative intravenous FVIII for boys with severe haemophilia A led to better joint health at the age of six. The current study continued to followed 33 of the JOS participants until the age of 18.

The Joint Outcome Continuation Study (JOS-C) involved 15 participants who had early prophylaxis, at an average age of 1.3 years, and 18 participants who were not given FVIII until an average age of 7.5 years.

MRI scans showed that of those on early prophylactic FVIII, only 35% had damage to the cartilage of a joint and/or the bone underneath, compared with 77% of those on later treatment. Early treatment was also linked to a lower bleeding rate.

However, very few participants reached adolescence with no joint damage at all, so there remains a need for improved prophylactic therapies.

Details of the study were published on 3rd June in Blood Advances.

Dr Warren said: “Prophylaxis has been the standard of care for preventing joint damage, and this study establishes the importance of starting as early as possible. However, we still have room for improvement in treatment options for severe haemophilia A, including possibilities like subcutaneous methods and gene therapy.”

She added: “We need to keep track of patients on those new products as rigorously as we have for this group, to ensure we are not trading convenience for worse outcomes, for joint damage.”

Source: Warren BB, Thornhill D, Stein J, Fadell M, Ingram JD, Funk S, Norton KL, Lane HD, Bennett CM, Dunn A, Recht M, Shapiro A, Manco-Johnson MJ (2020) “Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study”, Blood Advances, doi: 10.1182/bloodadvances.2019001311


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