None listed
Conditions
Brief 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.
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 n
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.
Sponsors
Monash University
Eligibility
Sex/Gender
All
Healthy volunteers
No
Inclusion criteria
Adult and paediatric patients with diagnostic features of aplastic anaemia (AA) on bone marrow biopsy (pancytopenia with a hypocellular bone marrow, absence of an abnormal marrow infiltrate and no increase in reticulin). Patients with AA that is acquired or due to inherited factors are included on the registry. The AAR now includes relatives of participants with a diagnosed BMFS.
Exclusion criteria
Patients who have chosen to ‘opt-off’ the registry. Also other causes of bone marrow failure (e.g. following chemotherapy or B12 deficiency) have been excluded.
Outcome results
None listed