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Yorkshire patient is the first person globally to join study into new treatment for rare bleeding disorder

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A patient in Yorkshire and Humber is the first to be recruited globally to a study to understand the real-world performance of a new treatment for people who have a rare autoimmune bleeding condition known as ITP at Leeds Teaching Hospitals NHS Trust.

The rare disorder – immune thrombocytopenia (ITP) - leads to bruising or bleeding because of low levels of platelets in the blood and is usually caused by something going wrong with the immune system.

Supported by the NIHR Clinical Research Network Yorkshire and Humber, the new study welcomed its first participant globally at Leeds Teaching Hospitals NHS Trust.

The study is designed to describe the real-world clinical outcomes of patients treated with a new treatment for ITP, called Avatrombopag. This includes looking at the percentage of patients who respond to the treatment, the time it takes to respond to the treatment and how long it lasts.

Dr Quentin Hill, consultant haematologist and the national chief investigator leading the study at Leeds Teaching Hospitals NHS Trust, said: “We are delighted to have recruited the first patient globally to this study, which aims to understand the real-world performance of Avatrombopag, a new treatment for immune thrombocytopenia (ITP).

“Collecting real world evidence is especially important in rare diseases, where the number of patients treated in phase III trials may be small.”

Phase III trials compare a new treatment to the standard treatment for a condition or to a placebo drug.  

The study is open across a number of sites across Europe and will run until January 2025. Patients taking part in the study give their consent to have their data collected and reviewed and there are no additional visits to hospital required. 

Did you know that anyone – with or without a health condition - can take part in research? To find out more about getting involved in health and care research and find studies taking place near you, visit the Be Part of Research website: www.bepartofresearch.nihr.ac.uk

For more information about ITP visit: Immune thrombocytopenia (ITP) | disease | symptoms | Sobi