Importance of the field: Acute promyelocytic leukemia (APL) represents a paradigm of therapeutic success in clinical hematology. Since the introduction of all-trans-retinoic-acid in the early 1980s, complete remission rates exceed 90% and the cure rate is > 70%. Notwithstanding, various questions concerning the management of APL remain unanswered.
Areas covered in this review: The aim of this article is to focus on still controversial issues in the management of APL, such as the role of arsenic trioxide as front-line therapy, the management of older unfit patients, the potential utility of gemtuzumab-ozogamycin and the effectiveness (if any) of maintenance therapy for patients in molecular remission. In addition, the possibility of reducing the intensity of post-remission therapy, which is associated with substantial morbidity in potentially cured patients, is discussed.
What the reader will gain: Current and future therapeutic options for the treatment of newly diagnosed and relapsed APL.
Take home message: To date, the therapy of APL is the most successful example of differentiation therapy and its scientific history can serve as a model for subsequent development of similar treatments in other leukemias and cancers. However, treatment strategies continue to evolve rapidly, with particular focus on minimizing the early and late effects of cytotoxic chemotherapy.