Evaluating Multidisciplinary Bone Marrow Failure Syndrome Care – a prospective observational clinical trial offering comprehensive diagnostic genomic evaluation, multidisciplinary case review and multidisciplinary clinical care

2020-2023 (Grant): Evaluating Multidisciplinary Bone Marrow Failure Syndrome Care – a prospective observational clinical trial offering comprehensive diagnostic genomic evaluation, multidisciplinary case review and multidisciplinary clinical care.

One of the most important initial steps in treating patients with bone marrow failure is getting the right diagnosis. In patients that are suspected to have an inherited bone marrow failure syndrome, complex genetic testing is required to reach the correct diagnosis. The EMBRACE clinical trial aims to measure the impact of providing Australian patients with bone marrow failure an accurate genetic diagnosis, up-to-date and comprehensive information about the disease, and access to treatment and clinical trials. The impact will be measured by evaluating patient outcomes, quality of life and health economic benefits for those receiving care under the EMBRACE study.

By ensuring that Australian patients have access to both the diagnostic and treatment expertise they need, it is hoped that EMBRACE will improve patient outcomes and understanding of these rare disorders.