Abstract
Results from pivotal clinical trials evaluating the combination of intensive chemotherapy with targeted therapies in acute myeloid leukemia (AML) are expected soon. While overall survival (OS) or event-free survival (EFS) are the recommended endpoints, new criteria are needed for assessing the intrinsic effectiveness of new drugs. In this study, the salvage-free, transplantation-free survival (STFS) was measured from day 1 of induction chemotherapy to the date of induction failure or salvage therapy, allogeneic transplantation (allo-HSCT), relapse, or death from any cause, whichever occurs first and assessed in a cohort of 1966 AML patients from the DATAML registry treated with intensive chemotherapy and 100 acute promyelocytic leukemia (APL) patients treated with arsenic trioxide and all-trans retinoic acid. Validation cohorts from the ALFA group were used.
At five years, STFS was estimated at 14.6% (95% confidence interval [CI], 13.0-16.2; median, 5.5 months) whereas OS, EFS and relapse-free survival estimations were 40.4% (95% CI, 38.2-42.6; median, 30.6 months), 31.4% (95% CI, 29.4-33.5; median, 15.3 months) and 37.6% (95% CI, 35.2-40.0; median, 21.9 months), respectively. Relapse was the most common first STFS event, followed by allo-HSCT. STFS varied depending on molecular subgroups and gene-gene mutation interactions. In the APL cohort, STFS (83.6% at five years) was similar to OS (85.6%) illustrating the impact of an effective and low-toxicity first-line treatment curing patients without salvage therapy or allo-HSCT. In the prospective randomized ALFA-0701 trial, gemtuzumab ozogamycin significantly improved STFS.
STFS should complement other survival endpoints to accurately analyze the impact of new drugs in AML.
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