How comparable are patient outcomes in the “real-world” with populations studied in pivotal AML trials?

Ing Soo Tiong, Meaghan Wall, Ashish Bajel, Akash Kalro, Shaun Fleming, Andrew W. Roberts, Nisha Thiagarajah, Chong Chyn Chua, Maya Latimer, David Yeung, Paula Marlton, Amanda Johnston, Anoop Enjeti, Chun Yew Fong, Gavin Cull, Stephen Larsen, Glen Kennedy, Anthony Schwarer, David Kipp, Sundra RamanathanEmma Verner, Campbell Tiley, Edward Morris, Uwe Hahn, John Moore, John Taper, Duncan Purtill, Pauline Warburton, William Stevenson, Nicholas Murphy, Peter Tan, Ashanka Beligaswatte, Howard Mutsando, Mark Hertzberg, Jake Shortt, Ferenc Szabo, Karin Dunne, Andrew H. Wei

Research output: Contribution to journalArticlepeer-review


Despite an increasing desire to use historical cohorts as “synthetic” controls for new drug evaluation, limited data exist regarding the comparability of real-world outcomes to those in clinical trials. Governmental cancer data often lacks details on treatment, response, and molecular characterization of disease sub-groups. The Australasian Leukaemia and Lymphoma Group National Blood Cancer Registry (ALLG NBCR) includes source information on morphology, cytogenetics, flow cytometry, and molecular features linked to treatment received (including transplantation), response to treatment, relapse, and survival outcome. Using data from 942 AML patients enrolled between 2012–2018, we assessed age and disease-matched control and interventional populations from published randomized trials that led to the registration of midostaurin, gemtuzumab ozogamicin, CPX-351, oral azacitidine, and venetoclax. Our analyses highlight important differences in real-world outcomes compared to clinical trial populations, including variations in anthracycline type, cytarabine intensity and scheduling during consolidation, and the frequency of allogeneic hematopoietic cell transplantation in first remission. Although real-world outcomes were comparable to some published studies, notable differences were apparent in others. If historical datasets were used to assess the impact of novel therapies, this work underscores the need to assess diverse datasets to enable geographic differences in treatment outcomes to be accounted for.

Original languageEnglish
Article number54
Number of pages11
JournalBlood Cancer Journal
Publication statusE-pub ahead of print - 26 Mar 2024


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