Abstract
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 language | English |
|---|---|
| Pages (from-to) | 111-121 |
| Number of pages | 11 |
| Journal | New England Journal of Medicine |
| Volume | 369 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - 2013 |
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ASJC Scopus subject areas
- Medicine(all)
Cite this
Retinoic acid and arsenic trioxide for acute promyelocytic leukemia. / Lo-Coco, F.; Avvisati, G.; Vignetti, M.; Thiede, C.; Orlando, S. M.; Iacobelli, S.; Ferrara, F.; Fazi, P.; Cicconi, L.; Di Bona, E.; Specchia, G.; Sica, S.; Divona, M.; Levis, A.; Fiedler, W.; Cerqui, E.; Breccia, M.; Fioritoni, G.; Salih, H. R.; Cazzola, M.; Melillo, L.; Carella, A. M.; Brandts, C. H.; Morra, E.; Von Lilienfeld-Toal, M.; Hertenstein, B.; Wattad, M.; Lübbert, M.; Hänel, M.; Schmitz, N.; Link, H.; Kropp, M. G.; Rambaldi, A.; La Nasa, G.; Luppi, M.; Ciceri, F.; Finizio, O.; Venditti, A.; Fabbiano, F.; Döhner, K.; Sauer, M.; Ganser, A.; Amadori, S.; Mandelli, F.; Döhner, H.; Ehninger, G.; Schlenk, R. F.; Platzbecker, U.
In: New England Journal of Medicine, Vol. 369, No. 2, 2013, p. 111-121.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Retinoic acid and arsenic trioxide for acute promyelocytic leukemia
AU - Lo-Coco, F.
AU - Avvisati, G.
AU - Vignetti, M.
AU - Thiede, C.
AU - Orlando, S. M.
AU - Iacobelli, S.
AU - Ferrara, F.
AU - Fazi, P.
AU - Cicconi, L.
AU - Di Bona, E.
AU - Specchia, G.
AU - Sica, S.
AU - Divona, M.
AU - Levis, A.
AU - Fiedler, W.
AU - Cerqui, E.
AU - Breccia, M.
AU - Fioritoni, G.
AU - Salih, H. R.
AU - Cazzola, M.
AU - Melillo, L.
AU - Carella, A. M.
AU - Brandts, C. H.
AU - Morra, E.
AU - Von Lilienfeld-Toal, M.
AU - Hertenstein, B.
AU - Wattad, M.
AU - Lübbert, M.
AU - Hänel, M.
AU - Schmitz, N.
AU - Link, H.
AU - Kropp, M. G.
AU - Rambaldi, A.
AU - La Nasa, G.
AU - Luppi, M.
AU - Ciceri, F.
AU - Finizio, O.
AU - Venditti, A.
AU - Fabbiano, F.
AU - Döhner, K.
AU - Sauer, M.
AU - Ganser, A.
AU - Amadori, S.
AU - Mandelli, F.
AU - Döhner, H.
AU - Ehninger, G.
AU - Schlenk, R. F.
AU - Platzbecker, U.
PY - 2013
Y1 - 2013
N2 - 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
AB - 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
UR - http://www.scopus.com/inward/record.url?scp=84880287051&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84880287051&partnerID=8YFLogxK
U2 - 10.1056/NEJMoa1300874
DO - 10.1056/NEJMoa1300874
M3 - Article
C2 - 23841729
AN - SCOPUS:84880287051
VL - 369
SP - 111
EP - 121
JO - New England Journal of Medicine
JF - New England Journal of Medicine
SN - 0028-4793
IS - 2
ER -