Increase in fetal haemoglobin from baseline to day 10 is a biomarker for response to decitabine in AML: Results of the AML21 randomized phase III trial of the EORTC Leukemia Group, Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA) and German MDS Study Group
In brief
A day-10 fetal hemoglobin rise more than doubles remission odds with decitabine
Among 135 older adults with acute myeloid leukemia treated with decitabine, a rise in fetal hemoglobin by day 10 was linked to 2.4 times the odds of remission or remission with incomplete blood count recovery. The rise was not linked to longer survival, so it may help identify early treatment response but does not show who will live longer.
- Journal
- British journal of haematology (Q1)
- Published
- 3 November 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Julia Stomper, Michal Kicinski, Tobias Ma, Thomas Meyer, Sylvain Chantepie, Richard Noppeney, et al.
- PMID
- 42977697
- DOI
- 10.1111/bjh.70874
Why clinicians should know about it
- Picked for Hematology (top studies of the week, 8 November 2026): Randomized AML trial evaluates an early response biomarker
Abstract
Lack of response or delayed response to therapy are major challenges in the use of DNA-hypomethylating agents (HMA) in patients with acute myeloid leukaemia (AML). Fetal haemoglobin (HbF) is a potential biomarker for response to HMA treatment but has only been studied in small single-centre studies. We investigated the association between HbF and treatment outcome in the randomized phase 3 European Organisation for Research and Treatment of Cancer (EORTC)-1301 trial AML21, which included 606 fit, older AML patients who received 10-day decitabine or '3 + 7' chemotherapy as induction. HbF was measured twice in the decitabine arm (n = 135) and at baseline in the 3 + 7 arm (n = 97). In the decitabine arm, HbF increase from baseline to day 10 was associated with increased odds of reaching complete remission (CR) or CR with incomplete count recovery (CRi) (odds ratio [OR] 2.40, 95% confidence interval [CI] 1.15-5.02, p = 0.020). The hazard ratio (HR) for death comparing patients with HbF increase to those without was 0.83 (95% CI 0.55-1.28, p = 0.40). Baseline HbF in the decitabine arm was not associated with CR/CRi (OR 0.89, 95% CI 0.45-1.75, p = 0.73) or overall survival (HR 0.86, 95% CI 0.58-1.28, p = 0.47). In conclusion, this large prospective study adds to the body of evidence that HbF is an easy-to-measure early biomarker for response to HMA treatment.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.