A collaborative study between Monash University and the Australasian Leukaemia & Lymphoma Group (ALLG) has recently published significant results in the New England Journal of Medicine. This clinical trial, supported by the Medical Research Future Fund (MRFF), confirms that a novel drug combination of cyclophosphamide and cyclosporine can significantly improve treatment outcomes for leukemia patients after stem cell transplantation.

The study was led by Professor David Curtis, Director of Malignant Haematology Research at the Australian Centre for Blood Diseases at Monash University, and involved a 5-year phase 3 randomized controlled trial conducted at 8 hospitals in Australia and New Zealand. The trial included 134 patients aged 18-70, primarily those with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL).

Results showed that compared to traditional therapies, the new regimen increased the proportion of patients achieving three-year survival without graft-versus-host disease (GVHD) from 14.2% to 49.1%, and reduced the incidence of severe side effects from 32.4% to 19.7%. "This new therapy is simple and safe, setting a new standard for matched related donor transplantation," said Professor Curtis. "GVHD is the leading cause of death or lifelong illness in 20% of transplant patients, and the new regimen effectively addresses this challenge."

The study results were presented at the European Hematology Association Congress (EHA 2025) in Milan, Italy. The researchers noted that this lower-toxicity, more effective treatment regimen has the potential to become the new norm for global clinical management of hematopoietic stem cell transplantation, particularly for high-risk blood cancer patients.