HCT improves LFS of elderly patients with AML in phase 3 study

In elderly patients with acute myeloid leukemia (AML), allogeneic hematopoietic cell transplantation (HCT) improves leukemia-free survival (LFS) according to a prospective randomized study.

The study was led by Dietger Niederwieser, MD, a professor of medicine at the University of Leipzig in Germany, and published in Haematologica.

Dr. Niederwieser and colleagues conducted the study to determine whether HCT improves survival outcomes elderly patients with AML in first complete remission (CR1) compared to conventional non-HCT consolidation therapy, an area that remains unclear.

In the study, newly diagnosed AML patients aged 60-75 years in CR1 with an HLA-identical related or 10/10 matched unrelated donor were randomized (2:1 ratio) to receive HCT or non-HCT consolidation according to institutional treatment protocols. The primary endpoint was restricted mean (RM)-LFS, defined as time from randomization to the first of the following three events: hematological relapse, initiation of additional anti-leukemic therapy, or death from any cause.

The results showed the primary endpoint RM-LFS up to five years (ie, the expected lifetime in CR1 on a time horizon of five years) was 24.5 months in the HCT and 15.6 months in the non-HCT arm. The difference in RM-LFS of +6.4 months at four years (P=0.04) increased to +8.9 months at five years (P=0.022) and +10.8 months at six years (P=0.019) favoring HCT.

“The only treatment able to improve long-term LFS is HCT as shown in our unbiased randomized intention-to-treat study,” the authors wrote. “The current study provides the rationale to increase the use of HCT in elderly patients (currently performed only in a small proportion).”

In terms of the secondary endpoint overall survival (OS), the restricted mean (RM)-OS up to five years (ie, the expected lifetime on a time horizon of five years) was 27.8 months in the HCT and 28.6 months in the non-HCT arm (P=0.85).

“The improved 5-year RM-LFS after HCT in comparison to non-HCT does not translate into a RM-OS benefit during the study period,” the authors noted.

Reference

Niederwieser D, Hasenclever D, Berdel Wel. Hematopoietic cell transplantation for older acute myeloid leukemia patients in first complete remission: results of a randomized phase III study. Haematologica. 2025;110(1):68-77. 2025. doi:10.3324/haematol.2024.285879