RecruitingACTRN12623000461695

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

Sponsor

Monash University

Enrollment

1,000 participants

Start Date

Jun 28, 2013

Study Type

Observational

Conditions

Hereditary Predisposition to Haematological Malignancy (HPHM)Aplastic Anaemia VEXAS Syndrome Bone Marrow Failure Syndromes

Summary

Aplastic Anaemia (AA) and Bone Marrow Failure Syndromes (BMFS) are rare diseases with significant morbidity and mortality. Currently there is little Australian data on the incidence of both acquired AA and BMFS, the therapies utilised or clinical outcomes. The relative rarity of this disease makes both the accrual of data and material to support scientific studies, and the establishment of high quality randomised prospective trials challenging. Furthermore, the existing literature concerning clinical outcomes of patients with AA may be biased by preferential reporting and publication of only good outcomes and/or serious or unusual events. The national registry for all Australian patients with AA and BMFS provides an important community resource to address these issues.

Eligibility

Sex: Both males and females

Plain Language Summary

Simplified for easier understanding

Aplastic anaemia and other bone marrow failure syndromes (BMFS) are serious conditions where the bone marrow cannot make enough healthy blood cells. These rare diseases can be life-threatening, and there is currently very little Australian data on how many people are affected, what treatments are used, and what outcomes patients experience. The Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry (AAR) is a national database collecting information from all Australian patients with these conditions over time. By joining the registry, your treatment details, test results, and health outcomes will be recorded (in a privacy-protected way) so that researchers and doctors can better understand these diseases and improve care for future patients. Anyone with a confirmed diagnosis of aplastic anaemia or another bone marrow failure syndrome is eligible to join, including children. Blood relatives of people with inherited forms of these conditions may also be invited. There are no age restrictions. Joining simply involves giving your consent for your clinical information to be collected.

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Interventions

This is a clinical registry collecting information on adult and paediatric patients with a diagnosis of aplastic anaemia or any other acquired or inherited bone marrow failure syndrome. Data to be collected includes, but is not limited to: demographics, family history, co-morbidities, clinical features, full blood examination, bone marrow assessments, diagnosis, treatment received, treatment outcomes and overall survival. Information will be collected by review of medical records. Patients are not required to complete any questionnaires.