-Author name in bold denotes the presenting author
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2850 Use of Venetoclax in Patients with Relapsed or Refractory Acute Myeloid Leukemia: The Pethema Registry Experience

Program: Oral and Poster Abstracts
Session: 615. Acute Myeloid Leukemia: Commercially Available Therapy, excluding Transplantation: Poster III
Hematology Disease Topics & Pathways:
AML, Adult, Diseases, Non-Biological, Therapies, Combinations, Study Population, Myeloid Malignancies, Clinically relevant
Monday, December 7, 2020, 7:00 AM-3:30 PM

Jorge Labrador1*, Miriam Saiz-Rodríguez2*, Maria Dunia De Miguel, MD3*, María Belén Vidriales4*, Manuel Perez Encinas, MD5*, Maria Jose Sanchez, MD6*, Rebeca Cuello, MD7*, Alicia Roldán Pérez8*, Susana Vives, MD9*, Gonzalo Benzo Callejo10*, Mercedes Colorado Araujo11*, María García-Fortes12*, Maria Jose Sayas13*, Carmen Olivier14*, Isabel Recio15*, Diego Conde Royo16*, Alvaro Bienert Garcia, MD17*, Maria Vahi18*, Carmen Muñoz García, PhD19*, Cristina Seri, MD20*, Mar Tormo, MD21, Ferran Vall-llovera22*, Maria Angeles Foncillas23*, David Martínez-Cuadron, MD24*, Miguel A. Sanz, MD25,26,27 and Pau Montesinos, MD, PhD28,29,30*

1Hematology Department, Research Unit, Hospital Universitario de Burgos, Burgos, Spain
2Research Unit, FBIS-Hospital Universitario de Burgos, Burgos, Spain
3Hematology department, Hospital Universitario de Guadalajara, Guadalajara, Spain
4Hematology Service. University Hospital of Salamanca and IBSAL, Salamanca, Spain
5Hospital Clínico Universitario de Santiago de Compostela, Santiago de Compostela, Spain
6Hematology Department, Hospital Universitario Lucus Augusti, Lugo, Spain
7Hematology and hemotherapy department, Hospital Clínico Universitario de Valladolid, Valladolid, Spain
8Hematology and hemotherapy department, Hospital Universitario Infanta Sofía, San Sebastián de los Reyes, Spain
9Hematology Department, ICO - Hospital Germans Trias i Pujol, Badalona, Spain
10Hematology Deparment, Hospital Universitario La Princesa, Madrid, Spain
11Hospital U. Marqués de Valdecilla, Servicio de Hematología-Hemoterapia, Santander, Spain
12Hospital Universitario Virgen De La Victoria, MáLaga, ESP
13Hospital Universitario Doctor Peset, Valencia, Spain
14Hospital General de Segovia, Segovia, Spain
15Hematology Deparment, Complejo Asistencial de Ávila, Avila, Spain
16Hematology Deparment, Hospital Universitario Príncipe de Asturias, Alcalá de Henares, Spain
17Hematology Deparment, Hospital Universitario de Canarias, Santa Cruz de Tenerife, Spain
18Hospital Universitario Virgen De Valme, Sevilla, ESP
19Hematology Deparment, Hospital Universitario Virgen Macarena, Sevilla, Spain
20Hematology and hemotherapy department, Hospital Central de la Defensa Gómez Ulla, Madrid, Spain
21Hospital Clínico Universitario de Valencia, Valencia, Spain
22Hematology Deparment, Hospital Universitari Mutua Terrasa, Barcelona, Spain
23Hematology Department, Hospital Universitario Infanta Leonor, Madrid, Spain
24Hospital Universitari i Politècnic La Fe, Valencia, Spain
25Hospital Universitario la Fe, Hematology Department, Department of Medicine, University of Valencia, Valencia, Spain
26Hematology and hemotherapy department, Hospital Universitari i Politècnic La Fe, Valencia, Spain
27Centro de Investigación Biomédica en Red de Cáncer (CIBERONC), Instituto Carlos III, Madrid, Spain
28CIBERONC, Instituto de Salud Carlos III, Madrid, Spain
29Hematology Department, Hospital Universitario La Fe de Valencia,, Valencia, Spain, Spain
30Programa Español de Tratamientos en Hematologia, PETHEMA, Valencia, Spain


The prognosis of patients with recurrent or refractory acute myeloid leukemia (AML-RR) is very poor, especially if they are not candidates for allogeneic transplantation (allo-SCT) after a second complete response (CR).

Venetoclax, a potent and selective inhibitor of the antiapoptotic protein BCL-2, was approved by the FDA in combination with hypomethylating agents (HMAs) or low-dose cytarabine (LDAC) in patients with newly diagnosed AML of age ≥ 75 years, or who have comorbidities that preclude the use of intensive chemotherapy. However, the evidence in AML-RR patients is still scarce.

For this reason, the objective of our study is to retrospectively analyze the efficacy of the off-label use of venetoclax in patients with AML-RR.


We conducted a retrospective, multicenter, observational study of a cohort of patients with AML-RR who were treated with venetoclax in the hospitals of the PETHEMA group. We evaluated efficacy, CR/CRi rate and overall survival (OS). We performed a descriptive analysis. Overall survival (OS) was calculated using the Kaplan-Meier method.


A total of 41 patients were included, 25 men and 16 women, with a median age of 68 years (25 - 82 years) and an ECOG ≥ 2 at the beginning of the venetoclax treatment in 52% of the cases.

Seventy-five percent of patients had AML with myelodysplasia-related changes. 25 patients (61%) were at high risk according to the European Leukemia Net 2017.

Sixty-six percent of patients received ≥2 previous lines (range, 1-4), 29 patients (71%) received intensive first line chemotherapy, 10 (25%) received a previous transplant and 18 (44%) received previous treatment with HMA.

Venetoclax median treatment duration was 40 days, and it was administered in 54% with azacitidine, 34% with decitabine and 12% with LDAC.

In all, 11% of patients achieved CR/CRi. Only 10% of patients received subsequent salvage treatment. With a median follow-up time of 166 days (range, 21 - 311), 65% of the patients died. The median OS from diagnosis was 15 months (1 - 67 months) and the median from venetoclax initiation was 78 days (2 - 311 days). Those patients who achieved CR/CRi had higher OS (median not reached vs. 78 days, p= 0.048).

Regarding toxicity, it was the expected in these patients. Twenty-eight percent of the patients required discontinuation of treatment due to toxicity. Sixty percent of the patients were admitted at some time during treatment with venetoclax, mainly because of infections (53%), 12% because of bleeding and other causes in 15%.


Our real-life series depicts a marginal probability of CR/CRi and poor OS after venetoclax-based salvage. Patients treated with this regimen had very poor-risk features, and were heavily pre-treated, which could explain in part the observed poor outcomes. Although follow-up is still short, the small proportion of responders did not reach the median overall survival. Further studies will help to identify those patients potentially benefiting from venetoclax-based salvage regimens.

Disclosures: Sanchez: Amgem: Other: travel grants; Janssen: Other: travel grants; Roche: Other: travel grants; Abbvie: Other: travel grants; Celgene: Other: travel grants. Tormo: Roche: Membership on an entity's Board of Directors or advisory committees; Astellas: Membership on an entity's Board of Directors or advisory committees; Servier: Honoraria; Celgene: Honoraria, 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; Janssen: Honoraria; MSD: Honoraria; Daiichi Sankyo: Honoraria.

OffLabel Disclosure: The objective of our study is to retrospectively analyze the efficacy of the off-label use of venetoclax in patients with recurrent or refractory acute myeloid leukemia

*signifies non-member of ASH