Page 80 - Haematologica Vol. 110 - January 2025

ARTICLE - HCT in elderly AML
CI: 2.1-17.0), when adjusting for stratification parameters, cytogenetic risk group and donor type (Online Supplemen- tary Table S5). No difference in LFS was observed between patients transplanted from related and unrelated donors (Online Supplementary Figure S7). Analyses of LFS as per protocol revealed similar results.
Online Supplementary Figure S8A depicts the dependence of the treatment effect in RM-LFS as a function of the time horizon. After an early disadvantage of HCT due to NRM in the
D. Niederwieser et al. first year, the benefit of HCT over non-HCT emerges starting
at about 36 months and reaches statistical significance after 48 months.
The study does not allow for a robust analysis of molecular AML subsets. The distribution of NPM1-mutated AML in both treatment groups was not significantly different (28.4 vs. 45.7%, respectively; P=0.12). Proportions of continuous CR, relapse and NRM among NPM1-mutated and NPM1 wild- type patients between HCT and non-HCT treatment groups
AB
CD
Figure 2. Outcome according to randomization to hematopoietic cell transplantation (HCT) or non-HCT analyzed following the intention to treat principles. Numbers below leukemia-free survival (LFS) and overall survival (OS) are number of patients at risk (number of patients censored). (A) LFS, (B) OS, (C) cumulative relapse incidence (RI) and (D) non-relapse mortality (NRM).
Table 2. Patient outcome.
HCT (95% CI)
Non-HCT (95% CI)
P
RM-LFS up to 5 years in months
24.5 (18.9-30.1)
15.6 (10.4-20.8)
0.02
RM-OS up to 5 years in months
27.7 (22.2-33.0)
28.5 (21.7-35.3)
0.85
LFS 5 years %
28.8 (20.4-40.6)
8.9 (3.1-25.7)
0.02*
OS 5 years %
31.3 (22.6-43.2)
27.1 (15.9-46.4)
0.16*
Cum. RI 5 years %
37.8 (27.2-48.4)
91.1 (80.7-100.0)
<0.0001
Cum. NRM 5 years %
33.4 (23.0-43.9)
0 NA
<0.0001#
HCT: hematopoetic cell transplantation; CI: confidence interval; RM-LFS: restricted mean leukemia-free survival; RM-OS: restricted mean overall survival; OS: overall survival; cum. RI: cumulative relapse incidence; Cum. NRM: cumultative non-relapse mortality; *derived from the Kaplan Meier rate estimates, #Fisher’s exact test (0 in 1 arm); NA: not available (0 events).
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