Rare disease study aiming to find better treatment pathway for people with Immune Thrombocytopenia (ITP)

A team at University Hospitals Bristol NHS Foundation Trust is striving to find better treatment for the rare disease Immune Thrombocytopenia (ITP) in the first UK independently funded multicentre Haematology trial, which is a significant breakthrough for this condition.

Treatment of this non-cancerous disease has not been a research priority, leading to ‘unsatisfactory’ treatment pathways for patients.The FLIGHT study aims to test standard steroid treatment of newly diagnosed ITP against a treatment of combined steroids and Mycophenolate (MMF), an immunosuppressant drug.

Immune Thrombocytopenia (ITP) is a disease which causes bruising, bleeding, and fatigue due to a low platelet count in the blood. In standard treatment for ITP, patients are given high-dose steroids to boost their platelet count. Platelets are blood cells that are essential for normal blood clotting. However, this treatment can cause negative side-effects including difficulty sleeping, weight gain, moods swings, high blood pressure and diabetes. Furthemore, once steroid treatment has stopped, patients frequently become ill again, with only 20% of patients remaining well long-term.

Current ITP treatment pathways can be extremely disruptive, particularly for young patients. Following treatment with steroids on a standard treatment pathway, MMF is often given to patients as a next stage treatment, which tends to be effective, although patients have to continue steroid treatment whilst waiting for MMF to take effect. This often takes a few months and during this time patients are still required to come into hospital for frequent blood tests and observation.

The study opened to recruitment in October 2017, and aimed to recruit 120 patients across 20 hospitals in the UK. The Research Ethics Committee (REC) approved 48 sites for the FLIGHT trial, and the study opened in 43 of those sites. The FLIGHT trial was open to male and female patients over the age of 16, with a platelet count of less than 30x109/L and a clinical need for first line treatment. The study also includes a translational laboratory sub study, providing patients the opportunity to give a blood sample for research into predictive biomarkers of treatment response and illness outcomes.

As this is a rare disease area, many trusts only see a few newly diagnosed ITP patients per year. The FLIGHT team expected that sites would recruit between 1 and 4 patients; however 9 sites have recruited 5 or more patients to the trial, and the study achieved its target recruitment by 7 February 2019. Whilst the trial has been active, the FLIGHT team have published one paper, with another paper to follow. Currently, follow up for the FLIGHT study is ongoing, and will close at the end of the year. The study is due to close on time in April 2020.

Charlotte Bradbury, Chief Investigator of the FLIGHT Trial, says:

“Standard initial treatment for ITP is unsatisfactory for patients but has been unchallenged for decades due to the lack of research in this illness to test alternative approaches. The NIHR funded Flight trial represents a big step forward for patients with this rare condition. It has been remarkable to see the enthusiasm from doctors and patients for this trial as reflected by the number of hospitals keen to take part and the successful patient recruitment. The trial demonstrates it is possible to do research on rare autoimmune conditions and provides hope for others suffering from similar illnesses.”