Research Proposals
De-identified registry data may be requested by completing a Data Access Form, and be accompanied by relevant ethics committee certification.
Requests from contributing clinicians, staff and academic organisations will be fulfilled without charge. However external requests will be considered on a case-by-case basis by the AAR Steering Committee and may incur a fee as per the AAR data Access Policy.
For questions please contact us at aar@monash.edu.
Publications and Presentations
Cumulative Hematologic Morbidity and Outcomes in Shwachman–Diamond Syndrome
Myers, K.C., Donadieu, J., Goldberg, B., Delafoy, M., Zeidler, C., Mellor-Heineke, S., Wan, Y., Zhu, X., Cesaro, S., Cipoli, M., Watanabe, K., Fox, L.F., Rutten, C., Kuijpers, T.W., Kattamis, A., Delaporta, P., Wartiovaara-Kautto, U., Kilpivaara, O., Bertuch, A.A., O’Farrell, C., Cotter, M., Dunlea, E., Reilly, C.R., Warren, A.J., Geddis, A., Weller, E., Shimamura, A.; N Eng J Med, 2026;395:1241-1244. DOI: 10.1056/NEJMc260500
The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry
Fox LC, McQuilten ZK, Firkin F, Fox V, Badoux X, Bajel A, Barbaro P, Cole-Sinclair MF, Forsyth C, Gibson J, Hiwase DK, Johnston A, Mills A, Roncolato F, Sutherland R, Szer J, Ting SB, Vilcassim S, Young L, Waters NA, Wood EM; Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry (AAR) Investigators. Best Pract Res Clin Haematol. 2023 Dec;36(4):101516. doi: 10.1016/j.beha.2023.101516
McQuilten Z, Heritier S, Fox L, Fox V, Young L, Blombery P, Cunningham I, Curnow J, Higgins A, Hiwase DK, Filshie R, Firkin F, Lacaze P, Mason K, Mills AK, Pepperell D, Patil S, Stevenson W, Szer J, Waters N, Wilson K, Ting S, Wood E. BMJ Open. 2024 Jan 18;14(1):e076246. doi: 10.1136/bmjopen-2023-076246.
Revisiting acquired aplastic anaemia: current concepts in diagnosis and management.
Clucas, D. B., Fox, L. C., Wood, E. M., Hong, F. S., Gibson, J., Bajel, A., Szer, J., Blombery, P., McQuilten, Z. K., Hiwase, D., Firkin, F., Cole-Sinclair, M. F., & Australian Aplastic Anaemia Registry Steering Committee (2019). Internal medicine journal, 49(2), 152–159. https://doi.org/10.1111/imj.14140
A/Prof Zoe McQuilten: Trial in Progress: Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve and Relapsed/Refractory Severe Aplastic Anaemia – the DiAAMOND-Ava-First and DiAAMOND-Ava-Next, Bayesian Optimal Phase II Trials. American Society of Hematology Annual Meeting. New Orleans, December 2022.
A/Prof Stephen Ting: Trial in Progress: Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve and Relapsed/Refractory Severe Aplastic Anaemia – the DiAAMOND-Ava-First and DiAAMOND-Ava-Next, Bayesian Optimal Phase II Trials. European School of Haematology EBMT Translational Research Conference. Paris, November 2022.
Prof Zoe McQuilten: Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve Severe Aplastic Anemia: Results of the Diaamond-Ava-First Trial. American Society of Hematology Annual Meeting. San Diego, December 2024
Dr Lucy Fox: Cellular and Cell-Free Genomic Characterization of Patients Receiving Avatrombopag and Immunosuppressive Therapy for Treatment-Naïve Severe Aplastic Anemia. American Society of Hematology Annual Meeting. San Diego, December 2024
Dr Nathanael Lucas: Review of Aplastic Anaemia, BMFS and Introducing the AAR to New Zealand. Auckland Hospital Blood Club, March 2025, and Christchurch Haematology Journal Club, February 2025
Dr Nathanael Lucas: The Aplastic Anaemia and Inherited Bone Marrow Failure Registry – Roll-out across New Zealand. HSANZ Annual NZ Branch Meeting, April 2025, Christchurch, April 2025
Prof Zoe McQuilten: DIAAMOND Ix: Efficacy and safety of avatrombopag in addition to immunosuppressive therapy for patients with previously untreated sAA. National Symposium on Bone Marrow Failure Syndromes, May 2025
Dr Nathanael Lucas: Expanding the Aplastic Anaemia and Other Bone Marrow Failure Registry (AAR) to New Zealand – Exploring epidemiology, availability of diagnostic testing, and genomic profiles. Australian and NZ Children’s Haematology Oncology (ANZCHOG) conference, August 2025
Dr Lucy Fox: Whole genome transcriptome sequencing in inherited bone marrow failure syndromes and related diseases – the IBMDx study. American Society of Hematology, December 2025
Dr Nathanael Lucas: Somatic 6p loss of heterozygosity and HLA-inactivation in aplastic anaemia: A scoping systematic review and meta-analysis. RCPA Pathology Update, March 2026
Vanessa Fox: Current status of the Aplastic Anaemia and Other Bone Marrow Failure Syndromes Registry (AAR). Blood, October 2026
Dr Nathanael Lucas: Developing the Aplastic Anaemia and Other Bone Marrow Failure Registry in Aotearoa New Zealand: Collaboration, data governance, and stakeholder engagement.
Blood, October 2026
