All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.
The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy
Introducing
Now you can personalise
your AML Hub experience!
Bookmark content to read later
Select your specific areas of interest
View content recommended for you
Find out moreThe AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.
The AML Hub is an independent medical education platform, sponsored by Daiichi Sankyo, Jazz Pharmaceuticals, Johnson & Johnson, Kura Oncology, Roche, Syndax and Thermo Fisher, and has been supported through a grant from Bristol Myers Squibb. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.
Impact of disease stage and MRD treatment on outcomes in patients with NPM1-mutated AML proceeding to allo-HSCT
Bookmark this article
NPM1 mutations in patients with acute myeloid leukemia (AML) indicate a favorable prognosis without allogeneic hematopoietic stem cell transplantation (allo-HSCT).1 Equally, patients with NPM1-mutated AML and positive measurable residual disease (MRD) often experience superior outcomes with allo-HSCT compared with chemotherapy.1 While MRD positivity prior to allo-HSCT is an adverse predictor of survival, the impact of disease stage at the time of transplant and of treatment for molecular positivity on patient outcomes remains unclear.1
During the 50th Annual Meeting of the European Society for Blood and Marrow Transplantation, Bug presented results from a study investigating outcomes of patients with NPM1-mutated AML in relation to their disease stage at the time of allo-HSCT. We summarize the presentation below.
Study design1
-
A retrospective multicenter study
-
Patient survival outcomes at the following disease stages were compared:
-
-
Positive MRD in first complete remission (CR1) vs hematologic relapse
-
Persistent MRD vs relapsed MRD
-
Treated vs untreated molecular relapse
-
-
Study endpoints were event-free survival, overall survival, cumulative incidence of relapse, and cumulative incidence of non-relapse mortality.
Key findings1
-
A total of 227 patients were enrolled in the trial
-
-
Persistent MRD at CR1 (n = 46)
-
Relapsed MRD at CR1 (n = 45)
-
Primary refractory disease (n = 24)
-
Hematologic relapse (n = 112)
-
-
Survival outcomes for each patient subgroup are shown in Figure 1.
Figure 1. Survival outcomes for A all patients after 4 years, B hematologic/molecular remission status, C persistent MRD vs relapsed MRD, and D positive MRD at CR1 vs CR2*
CI NRM, cumulative incidence of non-relapse mortality; CIR, cumulative incidence of relapse; CR1, first complete remission; CR2, second complete remission; EFS, event-free survival; MRD, measurable residual disease; OS, overall survival.
*Adapted from Bug.1
†n = 227
‡n = 227
§n = 91
‖n = 118
- Multivariable analysis showed the following factors to be associated with inferior overall survival:
-
-
Untreated hematologic relapse (hazard ratio [HR] 3.91; p = 0.009)
-
Refractory relapse (HR 5.55; p < 0.001)
-
Primary refractory disease (HR 3.76; p = 0.016)
-
Unrelated human leukocyte antigen-mismatched donor (HR 2.13; p = 0.044)
-
|
Key learnings |
|
- Gesine Bug. Excellent outcome of patients proceeding to allogeneic hematopoietic cell transplantation with untreated molecular relapse of NPM1-mutated AML. Oral abstract #OS1-07. 50th Annual Meeting of the European Society for Blood and Marrow. April 15, 2024; Glasgow, UK.
Your opinion matters
28 votes - 50 days left ...
Related articles
Newsletter
Subscribe to get the best content related to AML delivered to your inbox