Azacitidine, intensive chemotherapy or best supportive care in relapsed or refractory acute myeloid leukemia, a DATAML registry study

Abstract

We analyzed 526 consecutive acute myeloid leukemia patients refractory to or relapsing after chemotherapy. 270 patients received intensive salvage chemotherapy (IC), 97 azacitidine (AZA) and 159 best supportive care (BSC). Complete response was obtained in 37/19/0% (p = .0008). Allogeneic stem-cell transplantation (alloSCT) was performed in 39.3/10.3/0%. Median overall survival (OS) and 5-year OS were 8.2/9.6/2.2 months and 16/6/2% (p < .0001). Predictive factors of worse OS were post-myelodysplastic/chronic myelomonocytic leukemia, bone marrow blasts ≥20%, adverse cytogenetics, AZA cycle ≥2 and no alloSCT at R/R for AZA and age, performance status, white blood cell count and myelodysplasia-related changes for IC. The impact of treatment was time-dependent: adjusted hazard ratio for OS was in favor of AZA up to 1 month, was not different between 1 and 7 months, then was in favor of IC after 7 months. While AZA represents a therapeutic option for the oldest patients, it does not lead to long-term survivors.

Keywords:

• Acute myeloid leukemia
• primary induction failure
• refractory
• relapse
• azacitidine
• intensive chemotherapy

Acknowledgments

We would like to thank the data management unit of Toulouse University for its support enabling e-CRF. We thank Mrs Michelle GOMES for her support to the GREMS association.

Author contributions

NG, HL, AS, ACDG collected data; ST, TL, SDS, PB, PYD, AP, SB, CR treated patients, JBR, LL, IL, ED, AB, EK provided biological data; SB, AP, CR, PYD designed the research study; EB analyzed the data; SB, CR and PYD wrote the paper; all coauthors checked the final version of the paper.

Disclosure statement

ED is an advisor for Novartis and Astellas. AP has received grants from Roche, Celgene, Sunesis, Novartis, Jazz, AbbVie, Astellas and Daiichi-Sankyo and is an advisor for Roche, Celgene, Sunesis, Amgen, Novartis, Jazz, AbbVie, Astellas, Daiichi-Sankyo, Sanofi and Pfizer. CR has received research grants from Amgen, Novartis, Celgene, Jazz Pharmaceuticals, Agios, Chugai, Sunesis, Astellas and MaatPharma and is an advisor for AbbVie, Sunesis, Janssen, Jazz, Novartis, Celgene, Astellas, Daiichi-Sankyo, Macrogenics and Pfizer. PYD is an advisor for Daiichi-Sankyo and Astellas. SB is an advisor for Jazz, Daiichi-Sankyo, Sanofi and Astellas.