New treatment can help patients with common bleeding disorder, study shows

A new medicine can help people living with the bleeding disorder von Willebrand disease by slowing its clearance from the bloodstream, new research from the Royal College of Surgeons has found. 

A study published recently by the RCSI University of Medicine and Health Sciences has identified how the drug Rondaptivon pegol (also known as BT200) can help people living with the bleeding disorder von Willebrand disease.

The discovery opens the way for more targeted treatments of the condition, according to the study authors.

Von Willebrand disease affects as many as one in 100 individuals. It is caused by a reduced quantity or function of von Willebrand factor in the blood.

The researchers investigated how BT200 slows that clearance and keeps levels of von Willebrand factor high. In a series of lab experiments, they showed that BT200 makes it more difficult for immune system cells called macrophages to bind to von Willebrand factor and remove it from the bloodstream.  

They identified a section of von Willebrand factor where macrophages typically bind to remove it from the blood, and showed how BT200 partially interferes with this binding.

This means the macrophages cannot remove the factor as quickly, and the factor stays in the bloodstream for longer.

The full paper was published in the journal Blood.