-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

634 Azacitidine Vs. Decitabine in Unfit Newly Diagnosed Acute Myeloid Leukemia Patients: Results from the Pethema Registry

Program: Oral and Poster Abstracts
Type: Oral
Session: 615. Acute Myeloid Leukemia: Commercially Available Therapy, excluding Transplantation: Commercially Available Therapy, excluding Transplantation II
Hematology Disease Topics & Pathways:
AML, Diseases, Elderly, Study Population, Clinically relevant, Myeloid Malignancies
Monday, December 7, 2020: 12:00 PM

Jorge Labrador1*, David Martínez-Cuadrón, MD2,3*, Adolfo de la Fuente4*, Rebeca Rodríguez-Veiga, MD2,5*, Josefina Serrano, MD6*, Mar Tormo, MD7, Jose Antonio Pérez-Simón, MD8*, Fernando Ramos9*, Teresa Bernal del Castillo, MD, PhD10*, Maria López-Pavía11*, Fernanda Trigo12*, Pilar Martinez Sanchez13*, Juan Ignacio Rodriguez-Gutierrez14*, Carlos Rodriguez15*, Cristina Gil16*, Daniel Garcia17*, Susana Vives, MD18*, Maria Angeles Foncillas19*, Manuel Perez Encinas, MD20*, Andrés Novo, MD21*, Isabel Recio22*, Gabriela Rodriguez-Macías, MD23*, Juan Miguel Bergua Burgues, MD24*, Victor Noriega Concepcion25*, Esperanza Lavilla26*, Alicia Roldán Pérez27*, Miguel A. Sanz, MD2,3,28 and Pau Montesinos, MD, PhD28,29,30*

1Hematology Department, Research Unit, Hospital Universitario de Burgos, Burgos, Spain
2CIBERONC Instituto de Salud Carlos III, Madrid, Spain
3Hematology and hemotherapy department, Hospital Universitari i Politècnic La Fe, Valencia, Spain
4MD Anderson Cancer Center, Madrid, Spain
5Hospital Universitari i Politécnic La Fe, Valencia, Spain
6Clinical Hematology, Hospital Universitario Reina Sofía, Córdoba, Spain
7Hospital Clínico Universitario de Valencia, Valencia, Spain
8Department of Hematology, Hospital Universitario Virgen del Rocio; Instituto de Biomedicina de Sevilla (IBIS)/CSIC/Universidad de Sevilla,Spain, Sevilla, Spain
9Hematology Department, Hospital Universitario de León, León, Spain, León, Spain
10Hospital Universitario Central Asturias, ISPA, IUOPA, Oviedo, Spain
11Hospital General Universitari de València, Hematology Service, Valencia, Spain
12Dept. of Clinical Hematology, Centro Hospitalar Universitário de São João, Porto, Portugal
13Clinical Hematology, Hospital Universitario 12 de Octubre, Madrid, Spain
14Hospital Universitario Basurto, Bilbao, Spain
15Hospital Universitario de Gran Canaria Doctor Negrín, Las Palmas de Gran Canaria, Spain
16Hematology Department, Hospital General Universitario de Alicante, Alicante, Spain
17Hematology Department, Hospital Sanitas La Zarzuela, Madrid, Spain
18Hematology Department, ICO - Hospital Germans Trias i Pujol, Badalona, Spain
19Hematology Department, Hospital Universitario Infanta Leonor, Madrid, Spain
20Hospital Clínico Universitario de Santiago de Compostela, Santiago de Compostela, Spain
21Hematology Department, Hospital Universitario Son Espases, Palma de Mallorca, Spain
22Hematology Deparment, Complejo Asistencial de Ávila, Avila, Spain
23Hematology and hemotherapy department, Hospital General Universitario Gregorio Marañón, Madrid, Spain
24Hematology and hemotherapy department, Hospital San Pedro de Alcántara, Cáceres, Spain
25Hospital Universitario de A Coruña, A CoruñA, ESP
26Hospital Universitario Lucus Augusti, Lugo, Spain
27Hematology and hemotherapy department, Hospital Universitario Infanta Sofía, San Sebastián de los Reyes, Spain
28Programa Español de Tratamientos en Hematologia, PETHEMA, Valencia, Spain
29CIBERONC, Instituto de Salud Carlos III, Madrid, Spain
30Hematology Department, Hospital Universitario La Fe de Valencia,, Valencia, Spain, Spain


Options to treat elderly patients with newly diagnosed AML include intensive, attenuated chemotherapy, hypomethylating agents (HMA) and supportive care (SC). HMA have proven their efficacy in DACO-016 (NCT00260832) and AML-001 (NCT01074047) clinical trials, with a median overall survival (OS) of 7.7 months (95%CI, 6.2 to 9.2) with decitabine (DEC) vs. 5.0 months (95%CI, 4.3 to 6.3) with therapy choice (TC), considered SC or low-dose Ara-C (LDAC). Median OS was 10.4 months with azacitidine (AZA) (95%CI, 8.0 to 12.7) vs. 6.5 months (95%CI, 5.0 to 8.6) with conventional care regimens (CCR), considered standard induction chemotherapy, LDAC or SC. However, there are few direct comparative data of AZA and DEC in the context of trials or real-life settings.


Here, we compared clinical outcomes between AZA and DEC in AML patients not eligible for intensive chemotherapy in the epidemiologic PETHEMA registry.


We included newly diagnosed AML patients treated with AZA (75 mg/m2/d IV or SC days 1–7) or DEC (20 mg/m2/d IV days 1–5) that were not eligible for intensive chemotherapy.

Responses were recorded using IWG 2003 criteria. Rates of Complete Response (CR), complete response with incomplete recovery (CRi) and OS were co-primary endpoints.


Between 2006 and 2019, 638 patients were included. 497 (78%) received AZA and 141 (22%) received DEC as per physician judgement.

Baseline characteristics were comparable in both groups (Table 1), except for bone marrow blasts count ≥ 30%, which was more frequent in DEC group (59.2% vs 77.1%, p<0.001).

The CR rate was 16.3% vs 20.6% (p = 0.23); composite CR (CR+CRi) was 18.5% vs 22% (p = 0.35), and the overall response rate (ORR, partial remission (PR) plus CR+CRi) was 29.2% vs 34.8% (p=0.20); for AZA vs DEC, respectively.

A significantly higher ORR to AZA was associated with ECOG <2 (33.9% vs. 12.4% in patients with ECOG ≥2, OR 0.22, 95% CI 0.10 – 0.49, p=0.000), de novo AML (35.3% vs. 21.9% in secondary AML; OR 0.38, 95% CI 0.20 – 0.71, p=0.002) and estimated glomerular filtrate rate ≥ 45 mL/min/1.73m2 (30.4% vs. 9.3% in patients with estimated glomerular filtrate rate ≥ 45 mL/min/1.73m2; OR 0.15, 95% CI 0.034 – 0.67, p=0.013); while bone marrow blast count < 50% was the only factor influencing ORR to DEC (43.7% vs. 25% in ≥ 50% bone marrow blasts, p=0.029).

With a median follow up of 12 months, median OS was 10.0 (95% CI 8.7 – 11.2) vs 8.0 (5.7– 10.2) months for AZA vs DEC, respectively (p = 0.46) (Figure 1). Median OS was 21 (17.8 – 24.1) vs 16 (12.6 – 19.3) vs 6 months (5.0 – 7.0) for patients who achieved CR/CRi vs PR vs no response (p<0.001) (Figure 2).

Additional subgroup analyses by baseline characteristics performed to compare AZA vs DEC revealed that patients ≥ 80 years did benefit for treatment with AZA, median OS of 8 vs. 4 m (p=0.042), as well as patients with WBC ≥ 10 x109/L (8 vs. 5 m, p=0.036), platelet count <20 x109/L (8 vs. 4 m, p=0.021) and those with estimated glomerular filtrate rate ≥ 45 mL/min/1.73m2 (10 vs. 5m, p=0.033).


This is a large retrospective comparison with long-term follow-up of clinical outcomes associated with AZA and DEC treatment for patients with AML patients not eligible for intensive chemotherapy. There were no significant differences in ORR, CR/CRi or OS between AZA and DEC. However, patients with WBC ≥ 10 x109/L, platelet count <20 x109/L and estimated glomerular filtrate rate ≥ 45 mL/min/1.73m2 could benefit from AZA in terms of OS.

Disclosures: Tormo: Janssen: Honoraria; Daiichi Sankyo: Honoraria; Servier: Honoraria; Roche: Membership on an entity's Board of Directors or advisory committees; Astellas: Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees; MSD: Honoraria. Ramos: Amgen: Consultancy, Other: travel grant ; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: travel grant , Research Funding; Novartis: Consultancy, Other: travel grant; Takeda: Consultancy, Other: travel grant ; Daiichi-Sankyo: Other: travel grant ; Merck-Sahrp & Dohme: Other: travel grant; Rovi: Other: travel grant; Roche: Other: travel grant ; Jannsen: Other: travel grant; Abbvie: Consultancy, Other: travel grant .

*signifies non-member of ASH