Aplastic Anaemia Registry (AAR) and the Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry

2015-ongoing: The Australian Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry, Transfusion Research Unit, Monash University.

The Aplastic Anaemia Registry (AAR), established in 2012, is a collaboration between the Transfusion Research Unit in Monash University’s Department of Public Health and Preventive Medicine and partner hospitals, clinicians and patients. Information regarding patient background, treatment and subsequent outcomes are critical to understanding diseases and developing new treatments. This information is hard to obtain in rare diseases like Aplastic Anaemia and inherited Bone Marrow Failure Syndromes. Disease registries like the Aplastic Anaemia Registry provide a method of obtaining this information from individual hospitals and combining them together for research purposes. Maddie Riewoldt’s Vision has provided financial support for the Aplastic Anaemia Registry since 2016. The registry also provides infrastructure and data support for a number of clinical trials, including the DIAAMOND clinical trial and the inherited bone marrow failure and related disorders trial (IBMDx).

The AAR scope was subsequently expanded to include inherited bone marrow failure syndromes in light of the overlapping clinical features, laboratory investigations and treatment options available to both groups (Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry). The aim of the registry is to better define the incidence of Bone Marrow Failure Syndromes in Australia. It provides information on the range of diagnoses and treatment strategies being employed, documents the specific genetic causes that underlie inherited Bone Marrow Failure Syndromes, explores factors influencing clinical outcomes, clarifies optimal clinical management and informs and inspires future research in this area. As of December 2023, 385 patients have been registered from 33 active sites.