6/28/23, 11:02 PM Lo-Coco 2013 Pathway Feedback Search Clinical Topics Home Studies Lo-Coco 2013 Lo-Coco 2013 Disease Disease Acute myeloid leukemia Acute promyelocytic leukemia Trial question What is the role of all-trans retinoic acid plus arsenic trioxide among patients with acute promyelocytic leukemia? Study design Multi-center Open label RCT Population Characteristics of study participants 51.0% female N = 156 49.0% male 156 patients (80 female, 76 male) Inclusion criteria: patients with acute promyelocytic leukemia classified as low-to-intermediate risk Key exclusion criteria: age < 18 and 71 years, uncontrolled life-threatening infections, severe non-controlled pulmonary or cardiac disease, other active malignancy at the time of study entry, pregnancy Interventions N=77 ATRA-arsenic trioxide (all-trans retinoic acid plus arsenic trioxide for induction and consolidation therapy) N=79 ATRA-chemotherapy (standard all-trans retinoic acid-idarubicin induction therapy followed by three cycles of consolidation therapy with all-trans retinoic acid plus chemotherapy and https://web.pathway.md/studies/recwVBXiC5sktrhy8 1/2 6/28/23, 11:02 PM Lo-Coco 2013 Pathway maintenance therapy with low-dose chemotherapy and all-trans retinoic acid) Primary outcome Event-free survival at 2 years 97.0 % 97 86 72.8 % 48.5 % Significant increase 24.3 % NNT = 9 0.0 % ATRA-arsenic trioxide ATRA-chemotherapy Significant increase in event-free survival at 2 years (97% vs. 86%; AD 11%, 95% CI 2 to 22) Secondary outcomes Significant increase in overall survival at 2 years (99% vs. 91%; AD 8%, 95% CI 1.25 to 14.75) No significant difference in disease-free survival at 2 years (97% vs. 90%; AD 7%, 95% CI -1.59 to 15.59) No significant difference in relapse at 2 years (1% vs. 6%; ARD -5, 95% CI -13.3 to 3.3) Safety outcomes No significant difference in kinetics of minimal residual disease. Significant difference in hematologic toxicity (26 episodes vs. 59 episodes) and grade 3 or 4 hepatic toxicity (63% vs. 6%). Conclusion In patients with acute promyelocytic leukemia classified as low-to-intermediate risk, ATRA-arsenic trioxide was superior to ATRA-chemotherapy with respect to a event-free survival at 2 years. Reference Lo-Coco F, Avvisati G, Vignetti M et al. Retinoic acid and arsenic trioxide for acute promyelocytic leukemia. N Engl J Med. 2013 Jul 11;369(2):111-21. Open reference URL https://web.pathway.md/studies/recwVBXiC5sktrhy8 2/2