Research proposal from GOSH

One of the key priorities in the recently launched Aplastic Anaemia Trust's Research Strategy is to improve baseline understanding of rare bone marrow failures in child and adult populations. The research proposal from Great Ormond Street Hospital team and colleagues from York and Manchester Universities is to 'Establish a UK Paediatric bone marrow failure registry and biobank'. Over a period of three years, researchers will aim to obtain laboratory, clinical and quality of life data of all children diagnosed with bone marrow failure within a specified time, to determine how many children are being diagnosed, with what specific bone marrow failure diseases, what treatments they’re receiving and the quality of life following treatment. The biobank will enable future research into paediatric aplastic anaemia.

Assessment process

An independent Research Advisory Panel comprising of top haematologists from across the UK and an aplastic anaemia survivor was formed to assess proposals. 

According to a panel member:

These are rare diseases and having a national registry is pivotal to understanding how common these diseases are in the UK, the spectrum of disease and how they are managed. In addition Quality of Life assessment with a novel data capture tool (RUDY) will ensure maximal data is achieved as it is patient driven.

Why is this so important?

The AAT's Board of Trustees are delighted to have agreed to fund this project as the essential first step for key research in understanding childhood incidence of the disease in the UK, to measure the quality of life of children affected and ultimately, paving the way to significant improvements to children’s diagnosis, treatment and care.

Dr Sujith Samarasinghe, consultant paediatric haematologist at Great Ormond Street who will be leading the project, said:

This is a major milestone to set up the first paediatric aplastic anaemia registry in the UK, along with the first national paediatric bone marrow failure biobank. This is brilliant news.