The AAT, thanks to its generous supporters, has over the past few years provided King’s College Hospital researchers with significant funding, to establish a registry for Aplastic Anaemia and rare Bone Marrow Failure patients treated at King’s.

This database was set up in 2012 and finished recruiting in March 2017. It provided an invaluable insight into the incident rates of acquired and inherited Aplastic Anaemia, while also determining the prevalence of hMDS/AA overlap for these patients. It’s enabled King’s College experts to plan service requirements for the future.

What information was included in the registry?

• Essential baseline demographics
• Patient blood and bone marrow results
• Details of treatment given and response to treatment

How has the registry been used?

The register has been used by the King's research team to discover some substantial findings:

• the database could be linked to the current research findings on patients, enabling the latest results of research in Aplastic Anaemia to be summarised and published
• it allowed the researchers to support a joint study with the National Institute of Health (NIH) USA, to produce the largest study of inherited heterozygous RTEL1mutations in patients with AA and MDS/myeloid neoplasms
• analysing samples from patients in the register, enabled the first report on the finding of acquired somatic mutations in a large series of AA patients and showed an association with the risk of later development of MDS/AML. The findings of this report were published in the Blood

Key findings

By March 2017, the registry had recruited nearly 500 patients and determined that more adults presenting with AA, had inherited AA than previously thought (in fact, nearly 20%). Some 43% of patients presented with acquired AA, and nearly 38% were found to have hypoplastic MDS/overlap (hMDS/AA). Just over 22% were confirmed to have severe or very severe AA.

What's next?

Our funded research has flagged the need for a better understanding of aplastic anaemia nationally.

An ongoing database with all the past and current patients of Aplastic Anaemia would be a huge step forward for both researchers and clinicians. It would include data on the treatments received and outcomes of the treatment(s) delivered. However, crucially it would also provide the incident rates and prevalence of Aplastic Anaemia in the UK, for which there is currently no record.

We are making a start into paediatric AA/ rare BMF incidence and prevalence having recently awarded a research grant to Great Ormond Street Hospital research team. You can read more about it here. 

Unfortunately, statutory funders at the current point in time do not consider funding a national adult AA/rare BMF registry a priority. We need to seek funding from different sources. This is why funds raised by our supporters are so crucial.

Please donate, become a regular giver and help us plan for a better future for everyone affected by aplastic anaemia and allied rare bone marrow failures.