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Haematologica. 2019 Jan;104(1):113-119. doi: 10.3324/haematol.2018.188888. Epub 2018 Aug 3.

Gemtuzumab ozogamicin for de novo acute myeloid leukemia: final efficacy and safety updates from the open-label, phase III ALFA-0701 trial.

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Service d'Hématologie et Oncologie, Centre Hospitalier de Versailles, Le Chesnay, France.
Service d'Hématologie et de Thérapie Cellulaire, Hôpital Henri Mondor, Créteil, France.
Laboratoire de Cytogénétique, Centre Hospitalier de Versailles, France.
Hôpital Saint-Louis (AP-HP), Université Paris Diderot, France.
Service d'Hématologie Clinique et Thérapie Cellulaire, Centre Hospitalier Universitaire, Limoges, France.
Hematologie Clinique, Hôpital François Mitterrand, Centre Hospitalier Universitaire, Dijon, France.
Hôpital Saint-Antoine (AP-HP), Université Paris Pierre et Marie Curie, France.
Centre Hospitalier Lyon Sud, Hospices Civils de Lyon, Université Lyon 1, Pierre Benite, France.
Hôpital Avicenne (AP-HP), Université Paris 13, Bobigny, France.
Global Clinical Development Pfizer Inc., Paris, France.
Global Product Development, Pfizer Inc., Collegeville, PA, USA.
Global Product Development, Pfizer Inc., Groton, CT, USA.
Pfizer Oncology, Pfizer Inc., Paris, France.
Université Lille, INSERM, Centre Hospitalier Universitaire Lille, UMR-S 1172 - Jean-Pierre Aubert Center - Centre de Recherche, Lille, France.
Departement de Biostatistique, Hôpital Saint-Louis (AP-HP), Universite Paris Diderot, INSERM S 717, France.
Hopital Saint-Louis (AP-HP), Universite Paris Diderot, France.
Service d'Hématologie et Oncologie, Centre Hospitalier de Versailles, Université de Versailles Saint Quentin, Le Chesnay, France


The randomized, phase III ALFA-0701 trial showed that a reduced and fractionated dose of gemtuzumab ozogamicin added to standard front-line chemotherapy significantly improves event-free survival (EFS) in adults with de novo acute myeloid leukemia (AML). Here we report an independent review of EFS, final overall survival (OS), and additional safety results from ALFA-0701. Patients (n=271) aged 50-70 years with de novo AML were randomized to receive conventional front-line induction chemotherapy (3+7daunorubicin+cytarabine) with/without gemtuzumab ozogamicin 3 mg/m2 on days 1, 4, and 7 during induction. Patients in remission following induction therapy received 2 courses of consolidation therapy (daunorubicin+cytarabine) with/without gemtuzumab ozogamicin (3 mg/m2/day on day 1) according to their initial randomization. The primary end point was investigator-assessed EFS. Secondary end points included OS and safety. A blinded independent review confirmed the investigator-assessed EFS results [August 1, 2011; hazard ratio (HR) 0.66; 95% Confidence Interval (CI): 0.49-0.89; 2-sided P=0.006], corresponding to a 34% reduction in risk of events in the gemtuzumab ozogamicin versus control arm. Final OS at April 30, 2013 favored gemtuzumab ozogamicin but was not significant. No differences in early death rate were observed between arms. The main toxicity associated with gemtuzumab ozogamicin was prolonged thrombocytopenia. Veno-occlusive disease (including after transplant) was observed in 6 patients in the gemtuzumab ozogamicin arm and 2 in the control arm. In conclusion, gemtuzumab ozogamicin added to standard intensive chemotherapy has a favorable benefit/risk ratio. These results expand front-line treatment options for adult patients with previously untreated AML. (Trial registered at; identifier: 00927498).

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