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Br J Haematol. 2016 Mar;172(6):841-54. doi: 10.1111/bjh.13890. Epub 2015 Dec 21.

Current standard treatment of adult acute promyelocytic leukaemia.

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Department of Biomedicine and Prevention, Tor Vergata University, Rome, Italy.
Laboratory of Neuro-Oncohaematology, Santa Lucia Foundation, Rome, Italy.
Department of Cellular Biotechnologies and Haematology, Sapienza University, Rome, Italy.


The outcome of patients with acute promyelocytic leukaemia (APL) has dramatically improved over the last two decades, due to the introduction of combined all-trans retinoic acid (ATRA) and chemotherapy regimens and, more recently, to the advent of arsenic trioxide (ATO). ATRA and anthracycline-based chemotherapy remains a widely used strategy, providing cure rates above 80%, but it is associated with risk of severe infections and occurrence of secondary leukaemias. ATO is the most effective single agent in APL and, used alone or in combination with ATRA or ATRA and reduced-intensity chemotherapy, results in greater efficacy with considerably less haematological toxicity. The toxic profile of ATO includes frequent, but manageable, QTc prolongation and increase of liver enzymes. Two large randomized studies have shown that ATRA + ATO is superior to ATRA + chemotherapy for newly diagnosed low-risk APL resulting in 2-4 year event-free survival rates above 90% and very few relapses. According to real world data, the spectacular progress in APL outcomes reported in clinical trials has not been paralleled by a significant improvement in early death rates, this remains the most challenging issue for the final cure of the disease.


PML/RARA; acute promyelocytic leukaemia; all-trans retinoic acid; arsenic trioxide

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