Time to improve health-related quality of life outcomes in patients with acute promyelocytic leukemia.

Time to improve health-related quality of life outcomes in patients with acute promyelocytic leukemia Major advances have been made in the treatment of patients with acute promyelocytic leukemia (APL) with the development of molecularly targeted therapies, first with the introduction of all-trans retinoic acid (ATRA) added to standard chemotherapy and more recently with the advent of arsenic trioxide (ATO). 1 Recent data from a pivotal phase 3 trial comparing standard ATRA plus chemotherapy vs ATRA-ATO 2 and subsequent follow-up data strongly suggest that patients with low-to intermediate-risk disease may be cured by targeted treatment alone and without chemotherapy. 3 However, the important progress made in the understanding of APL biology and the excellent clinical results have not been paralleled by a full understanding of the impact of the disease and its treatment on patients' lives. APL is a life-threatening condition and a medical emergency. Based on international guidelines, immediate start of treatment with ATRA is recommended even before the diagnosis is fully confirmed by molecular analysis. 4 Therefore, it is self-evident that major efforts have historically been devoted to saving the lives of these patients and improving treatment outcomes. Figure 1. Health-related quality of life of APL patients in complete molecular remission compared with general population. (A) Self-reported functional scales and global quality of life of APL patients in molecular complete remission compared with general population norms adjusted by age and gender. (B) Self-reported symptoms of APL patients in molecular complete remission compared with general population norms adjusted by age and gender. For functional and global quality of life (QoL) scales, higher scores indicate better outcomes; for symptom scales, higher scores indicate higher symptom severity. Analysis is based on 119 patients (61 ATRA plus ATO patients and 58 ATRA plus chemotherapy patients). Asterisk indicates at least a small clinically relevant difference. It is now time to also better understand how APL treatments affect patients' health-related quality of life (HRQOL) and how we can best help these patients to fully recover from the negative consequences of therapies. What are the main functional limitations or specific symptoms that clinicians should mostly pay attention to? This type of information, for example, could help to facilitate APL tailored-based supportive care programs. Although 3 recent APL randomized controlled trials have included patient-reported HRQOL as a formal end point of the study, 5-7 much remains to be learned about HRQOL. Typically, APL patients receive high-intensity and …