Manchester Royal Infirmary team recruit first patient in the UK to new study testing treatment for condition that can occur after stem cell or bone marrow transplantation

A research team at Manchester Royal Infirmary has recruited the first patient in the UK to a new study testing medication for the treatment of Chronic Graft versus Host Disease, which can occur after a stem cell or bone marrow transplant.

Graft versus Host Disease (GVHD) happens when particular types of white blood cell (T cells) in the donated bone marrow or stem cells attack the transplant patient’s own body cells. This happens because the donated cells (the graft) see the body cells (the host) as foreign.^[1]

Chronic GVHD usually starts more than 100 days after a transplant, and can last for several months or even years.  Like acute GVHD, it could affect the skin, gut, liver or mouth, but it can also target other body parts including eyes, lungs, vagina and joints.

The Adult Haematology Oncology research team at Manchester Royal Infirmary has recruited the first patient in the UK to a new study to test experimental medication for the condition, Entospletinib (ENTO). The research is sponsored by Gilead Sciences, Inc., with around 100 participants aged between 18 and 75 years old involved, across 30 sites in North America, Europe, Asia and Australia.

Dr Fiona Dignan, Consultant Haematologist at Central Manchester University Hospitals NHS Foundation Trust, is the Principal Investigator of the study. She said:

We are delighted to have found out that we have recruited the first patient in the UK, and only the second in Europe, to the new study.

“The main purpose of this research is to see if ENTO can improve chronic GVHD symptoms in participants who are taking a class of steroid hormones called corticosteroids. It also aims to investigate if the medication can reduce the amount of corticosteroids taken; be used as another treatment option for Chronic GVHD; can lessen the severity of chronic GVHD symptoms over time, and that it is safe and tolerable.

“Chronic GVHD is a frustrating and painful condition for a patient. Not only have they undergone major surgery in the form of a transplant but they then have to medicate and control the condition too. It can lead to issues affecting different parts of the body including thick and bumpy skin, hard and brittle nails, chronic diarrhoea, difficulty swallowing, bloating and stomach cramps, scarring of the liver, dry and painful eyes, shortness of breath and difficulty moving. We hope to be able to contribute towards research findings that will go on to support, and ultimately improve, the lives of our patients in the future.”

It is a randomised, double blind, placebo-controlled study. Placebo-controlled means that patients may be taking a tablet with no medicine in it but that looks like ENTO. Randomised means the study treatment that patients take will be chosen by chance, like flipping a coin. Double-blind means that both the study participants and their doctors will not know what study medication (ENTO or placebo) they are taking.

To find out more about the study, visit the Clinical Trials website or email: research.adultoncology@cmft.nhs.uk

^{[1] http://www.cancerresearchuk.org/about-cancer/coping-with-cancer/coping-physically/gvhd/about-graft-versus-host-disease#tWTFaCLGbcjIqw9d.99}