Significant advances have occurred in the diagnosis, treatment, and lo
ng-term outcome of patients with acute promyelocytic leukemia (APL). T
he purpose of this review is to describe the molecular genetics of thi
s disease, the use of all-trans retinoic acid (ATRA) in clinical trial
s of APL, and the clinical and basic research questions for future inv
estigation. Findings of clinical studies in mainland China using ATRA
as induction therapy for patients with APL concurrent with laboratory
characterization of the molecular changes in APL have led to worldwide
clinical trials of ATRA in the treatment of patients with APL. Major
advances in understanding the molecular biology and genetics of APL ha
ve occurred over the past 5 years. These findings have been translated
into novel treatment strategies using all-trans retinoic acid as a di
fferentiation agent in the induction phase of therapy resulting in imp
roved long-term outcome, reduced morbidity, and lower costs for patien
ts with APL. Advanced molecular techniques are being employed for diag
nosis and for monitoring of patient response to treatment.