Institutional members access full text with Ovid®

Share this article on:

ATRA + ATO: has a new standard of care been established in low-risk acute promyelocytic leukaemia?

Breccia, Massimoa; Cicconi, Laurab,c; Lo-Coco, Francescob,c

Current Opinion in Hematology: March 2014 - Volume 21 - Issue 2 - p 95–101
doi: 10.1097/MOH.0000000000000023
MYELOID DISEASE: Edited by Martin S. Tallman

Purpose of review Arsenic trioxide (ATO) has been shown to be the most effective single agent in acute promyelocytic leukaemia (APL) and has been approved for the treatment of relapsed patients both in the US and Europe. The role of ATO in front-line therapy of APL is under investigation.

Recent findings Pilot studies using ATO with or without all-trans retinoic acid (ATRA) have been carried out in newly diagnosed APL patients with the aim to reduce the short and long-term toxic effects of chemotherapy and to improve clinical outcome. Especially in patients with non-high-risk APL, the ATRA + ATO approach allowed significant increase in event-free survival and overall survival rates compared to standard ATRA and chemotherapy. This has been demonstrated by pilot studies and, more recently, by a randomized comparative multi-centre study conducted in Italy and Germany.

Summary The ATO + ATRA strategy for APL may provide the first paradigm of acute leukaemia curability by targeted agents and without chemotherapy. However, longer follow-up of available studies and independent confirmation of the Italian–German findings are awaited to firmly establish this paradigm. Finally, extension of this approach to other patient categories such as high-risk, elderly and children will need to be explored in the near future.

aDepartment of Hematology, Sapienza University, Rome

bDepartment of Biomedicine and Prevention, University Tor Vergata

cLaboratory of Neuro-Oncohematology, Santa Lucia Foundation, Rome, Italy

Correspondence to Francesco Lo Coco, MD, Department of Biomedicine and Prevention, University Tor Vergata, Via Montpellier 1, 00133 Rome, Italy. Tel: +39 06 20903220; e-mail:

© 2014 Wolters Kluwer Health | Lippincott Williams & Wilkins