Retinoic acid and arsenic trioxide for acute promyelocytic leukemia

F. Lo-Coco, G. Avvisati, M. Vignetti, C. Thiede, S. M. Orlando, S. Iacobelli, F. Ferrara, P. Fazi, L. Cicconi, E. Di Bona, G. Specchia, S. Sica, M. Divona, A. Levis, W. Fiedler, E. Cerqui, M. Breccia, G. Fioritoni, H. R. Salih, M. CazzolaL. Melillo, A. M. Carella, C. H. Brandts, E. Morra, M. Von Lilienfeld-Toal, B. Hertenstein, M. Wattad, M. Lübbert, M. Hänel, N. Schmitz, H. Link, M. G. Kropp, A. Rambaldi, G. La Nasa, M. Luppi, F. Ciceri, O. Finizio, A. Venditti, F. Fabbiano, K. Döhner, M. Sauer, A. Ganser, S. Amadori, F. Mandelli, H. Döhner, G. Ehninger, R. F. Schlenk, U. Platzbecker

Research output: Contribution to journalArticlepeer-review


BACKGROUND: All-trans retinoic acid (ATRA) with chemotherapy is the standard of care for acute promyelocytic leukemia (APL), resulting in cure rates exceeding 80%. Pilot studies of treatment with arsenic trioxide with or without ATRA have shown high efficacy and reduced hematologic toxicity. METHODS: We conducted a phase 3, multicenter trial comparing ATRA plus chemotherapy with ATRA plus arsenic trioxide in patients with APL classified as low-to-intermediate risk (white-cell count, ≤10×109per liter). Patients were randomly assigned to receive either ATRA plus arsenic trioxide for induction and consolidation therapy or standard ATRA- idarubicin induction therapy followed by three cycles of consolidation therapy with ATRA plus chemotherapy and maintenance therapy with low-dose chemotherapy and ATRA. The study was designed as a noninferiority trial to show that the difference between the rates of event-free survival at 2 years in the two groups was not greater than 5%. RESULTS: Complete remission was achieved in all 77 patients in the ATRA-arsenic trioxide group who could be evaluated (100%) and in 75 of 79 patients in the ATRA-chemotherapy group (95%) (P = 0.12). The median follow-up was 34.4 months. Two-year event-free survival rates were 97% in the ATRA-arsenic trioxide group and 86% in the ATRA-chemotherapy group (95% confidence interval for the difference, 2 to 22 percentage points; P

Original languageEnglish
Pages (from-to)111-121
Number of pages11
JournalNew England Journal of Medicine
Issue number2
Publication statusPublished - 2013

ASJC Scopus subject areas

  • Medicine(all)


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