Translational research outputs in acute promyelocytic leukemia
This year’s Jose Carreras Award was presented to Professor Francesco Lo Coco for his outstanding contributions to the research and treatment of AML, specifically of acute promyelocytic leukemia (APL). In the honorary lecture he gave an update on the progress that has been achieved in the treatment of APL due to translational research, and discussed future research challenges.
APL, comprising 10-15% of AML cases, is characterized by the PML-RARA protein, which is a target for specific treatment consisting of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), combined with anthracyclines. Over the past decades, the development of these treatments has transformed APL from a highly fatal to a highly curable disease.
Lo Coco discussed several clinical studies that led to the improvement of outcome. In the AIDA trial ATRA was combined with chemotherapy (idarubicin), followed by studies showing that deintensification of the AIDA protocol, specifically by decreasing the amount of anthracyclines, is feasible. Combination of the AIDA protocol with ATO further improved results in APL patients treated at frontline. In the APL0406 trial a chemo-free regimen of ATRA plus ATO improved outcome as compared to ATRA plus chemotherapy.
According to Lo Coco, future research should focus on early death, therapy resistance, long term survivors and quality of life. HARMONY, a big data project in hematology, offers good opportunities to study these issues. A novel oral ATO formulation may help improve quality of life (at EHA congress: abstract PF238).