Long-Term Follow-up of Survival, Complications, Arsenic Retention and Quality of Life in Patients with Newly Diagnosed Acute Promyelocytic Leukemia Treated with All-Trans Retinoic Acid/Arsenic Trioxide Combination Therapy

Objective: All-trans retinoic acid (ATRA)/arsenic trioxide (ATO) combination therapy for newly diagnosed acute promyelocytic leukemia (APL) has yielded high efficacy in clinical trials, but there are few studies focusing on the long-term follow-up of survival and complications. Methods: In this study, we followed up 217 patients with newly diagnosed APL treated with ATRA/ATO combination therapy between 2001 and 2010. Health assessment for long-term complications and quality of life was performed for 112 of these patients, meanwhile the arsenic retention in their plasma, urine, hair and nails was detected by inductively coupled argon plasma mass spectrometry (ICP-MS). Results: A total of 199 patients (91.7%) achieved complete remission (CR). The estimated 10-year event-free survival (EFS), overall survival (OS), disease-free survival (DFS) and cumulative incidence of relapse (CIR) were 78.0%, 86.3%, 87.0% and 12.0%, respectively. High white blood cell (WBC) count remained as the only unfavorable prognostic factor for remission duration and led to significant differences between low-to-intermediate and high-risk groups in EFS (82.1% vs. 63.9%, P=0.016), DFS (90.6% vs. 73.1%, P=0.008) and CIR (8.0% vs. 26.9%, P=0.003). For the 112 patients who received health assessment, there were no significant long-term complications associated with ATRA/ATO therapy, except for higher incidence of grade-1 liver dysfunction (15.2%) and hepatic steatosis (42.9%) compared to healthy controls (both P Conclusion: This study with long-term follow-up clearly demonstrated that ATRA/ATO in newly diagnosed APL patients was associated with long-term survival, particularly for patients with low-to-intermediate risk diseases, as well as few complications, minimal arsenic retention and good quality of life. Disclosures Zhao: National Natural Science Foundation of China (81300451): Research Funding. Chen: National High-tech R&D Program (863 Program) (2012AA02A505): Research Funding.