Phase III Trial in Acute Promyelocytic Leukemia Patients

Summary

Open label, randomised, phase III multicenter trial.

Timeline

Start

2007-08

Primary completion

2012-09

Completion

2019-10-17

Outcome

Met primary endpoint

paper Two-year event-free survival rates were 97% in the ATRA-arsenic trioxide group and 86% in the ATRA-chemotherapy group PMID 23841729 ↗

paper P<0.001 for noninferiority and P=0.02 for superiority of ATRA-arsenic trioxide PMID 23841729 ↗

Publications

• Efficace F, Mandelli F, Avvisati G, Cottone F, Ferrara F, Di Bona E, Specchia G, Breccia M, Levis A, Sica S, Finizio O, Kropp MG, Fioritoni G, Cerqui E, Vignetti M, Amadori S, Schlenk RF, Platzbecker U, Lo-Coco F. Randomized phase III trial of retinoic acid and arsenic trioxide versus retinoic acid and chemotherapy in patients with acute promyelocytic leukemia: health-related quality-of-life outcomes. J Clin Oncol. 2014 Oct 20;32(30):3406-12. doi: 10.1200/JCO.2014.55.3453. Epub 2014 Sep 22.
• Lo-Coco F, Avvisati G, Vignetti M, Thiede C, Orlando SM, 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 HR, Cazzola M, Melillo L, Carella AM, Brandts CH, Morra E, von Lilienfeld-Toal M, Hertenstein B, Wattad M, Lubbert M, Hanel M, Schmitz N, Link H, Kropp MG, Rambaldi A, La Nasa G, Luppi M, Ciceri F, Finizio O, Venditti A, Fabbiano F, Dohner K, Sauer M, Ganser A, Amadori S, Mandelli F, Dohner H, Ehninger G, Schlenk RF, Platzbecker U; Gruppo Italiano Malattie Ematologiche dell'Adulto; German-Austrian Acute Myeloid Leukemia Study Group; Study Alliance Leukemia. Retinoic acid and arsenic trioxide for acute promyelocytic leukemia. N Engl J Med. 2013 Jul 11;369(2):111-21. doi: 10.1056/NEJMoa1300874.

Drugs

Evaluation	Drug	Modality	Dose	Route
Comparator	Idarubicin	Small molecule	5 mg/m2	Intravenous
Comparator	Idarubicin	Small molecule	12 mg/m2	Intravenous
Comparator	MERCAPTOPURINE ANHYDROUS	Small molecule	50 mg/m2	Oral
Comparator	Methotrexate	Small molecule	15 mg/m2	Intramuscular
Subject	arsenic trioxide	Small molecule	0.15 mg/kg	Intravenous
Background	Tretinoin	Unknown	45 mg/m2	Oral

Indications