Long-term Study Reveals Disease Risk Surpasses Remission Status in Determining Transplant Outcomes for AML Patients

Recent findings from a long-term follow-up of the ASAP trial, conducted across various universities and clinics in Germany, highlight that the overall survival of high-risk acute myeloid leukemia (AML) patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT) is predominantly influenced by the genetic risk of the disease rather than the remission status prior to transplant. Notably, the study found that inducing remission with standard salvage chemotherapy before allo-HSCT does not provide additional survival benefits.

The ASAP trial, which stands for 'as soon as possible,' challenged current treatment paradigms for AML by comparing the traditional approach of remission induction and salvage chemotherapy with an alternative strategy of immediate transplantation following intensified conditioning and less aggressive disease management. The initial results published in 2024 already questioned the necessity of aggressive pre-transplant chemotherapy.

In the recent long-term analysis, published in the journal Blood, these initial insights are reinforced. The data, with a median follow-up of 61 months, showed that there was no significant difference in overall survival between patients who underwent remission induction (with a 5-year overall survival of 47.5%) and those who proceeded directly to transplantation without prior remission induction (with a 5-year overall survival of 46.1%). This suggests that immediate transplantation can be as effective as traditional approaches, especially considering shorter hospital stays and fewer adverse events associated with intensive chemotherapy.

Importantly, the study emphasizes that the intrinsic biology of the AML, particularly its genetic risk as classified by the ELN 2022 criteria, plays a far more critical role in survival outcomes. For example, patients with favorable risk AML had 5-year survival rates of 66%, while those with adverse risk AML had only 34%. When genetic risk factors were combined with treatment strategies, outcomes favored tailored approaches for different genetic subgroups.

Prof. Dr. Johannes Schetelig, a co-author of the study, explained that these findings challenge the longstanding standard of giving all AML patients intensive chemotherapy before transplant, arguing that a more personalized approach based on disease genetics could improve patient outcomes. Patients with favorable or intermediate risk AML could benefit from watchful waiting or less aggressive treatments, even in the context of transplantation.

Furthermore, the study observes that patients who undergo immediate transplantation tend to spend less time in hospital and experience fewer treatment-related complications, making this approach potentially preferable in certain patient populations. However, since non-inferiority of immediate transplantation compared to the standard remains unproven, it has not yet replaced existing treatment protocols but can be considered more often.

Overall, these findings mark a significant step toward customizing AML treatment through precision medicine. They underscore that the biology of the disease, rather than the remission status achieved through chemotherapy, determines post-transplant outcomes. This could pave the way for new strategies incorporating novel bridging therapies and personalized post-transplant care, especially for patients with high-risk genetic profiles.

In conclusion, the long-term results from the ASAP trial suggest a paradigm shift in AML transplantation practices, emphasizing the importance of genetic risk factors over traditional remission induction strategies in influencing survival outcomes.

Source: https://medicalxpress.com/news/2025-10-term-analysis-disease-remission-status.html