Long-term outcome of acute promyelocytic leukemia treated with all-trans-retinoic acid, arsenic trioxide, and gemtuzumab

Long-term outcome of acute promyelocytic leukemia treated with all-trans-retinoic acid, arsenic trioxide, and gemtuzumab
In the past couple of decades, acute promyelocytic leukemia (APML) treatment has been revolutionized by the successful use of all-trans-retinoic acid (ATRA) and arsenic trioxide (ATO) to the exclusion of cytotoxic chemotherapy. As this regimen is relatively novel, long-term outcomes have not yet been reported.
This is a follow up of 187 patients (age 10 and above) from 3 consecutive MD Anderson trials using ATRA, ATO, and one dose gemtuzumab-ozogamicin to high-risk patients and low risk patients with leucocytosis during induction. A priori primary outcomes were event-free survival (EFS), disease-free survival (DFS), and overall survival (OS). These trials were not randomized.
At 5 years, EFS, DFS, and OS were 87%, 99%, and 89% respectively for the low risk group and 81%, 89%, and 86% for the high risk group. Survival was lower for patients > 60 years of age.
Single centre trial and non-randomized.
These long term outcomes lend further support for ATRA and ATO as standard of care for APML.