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Posted by on Jul 31, 2019 in Leukemia | 0 comments

In a nutshell

This study aimed to investigate if venetoclax would enable patients with acute myeloid leukemia to overcome disease progression after hypomethylating agents.  

This study concluded that adding venetoclax to these patient's treatment may result in a substantial anti-cancer activity. 

Some background

Patients with acute myeloid leukemia (AML) who have disease progression after treatment with hypomethylating agents (HMA) have a poor prognosis. HMA are drugs that inhibit DNA methylation which prevents the spreading of cancerous cells. Decitabine (Dacogen) and azacytidine (Vidaza) are HMAs.

Venetoclax (Venclexta) is a chemotherapy used to treat chronic lymphocytic leukemia.  

It is unknown if the addition of venetoclax to AML patients who failed HMA might overcome disease progression. 

Methods & findings

This study involved 23 patients with AML. Patients that relapsed or were unresponsive to HMA were involved. Patients who received an allogenic hematopoietic stem cell transplant (allo-HSCT) were also involved. Patients were treated with venetoclax and HMA. 6 patients had AML that relapsed after HSCT. Patients were followed up for an average of 5.3 months.

No patients experienced tumor lysis syndrome (a complication of cancer treatment resulting from rapid cancer cell death) and side effects were manageable. The most common side effect was febrile neutropenia experienced by 78% of patients. Febrile neutropenia is a low level of white blood cells and fever symptoms.  

43% of patients achieved complete remission (all signs of cancer gone) or complete remission with incomplete blood count recovery. 74% of patients were alive after 6 months. The average overall survival in patients who achieved remission was 10.8 months.  

Patients who had a higher number of blasts (immature cells) in the bone marrow and blood had lower chances of complete remission. Patients with higher white blood cells had increased chances of mortality. 

The bottom line

This study concluded that the addition of venetoclax to patients with HMA-refractory AML may result in a substantial anti-leukemic activity.  

The fine print

This study had a very small number of patients a very short follow-up. Further, larger studies are needed.

Published By :

Annals of Hematology

Date :

Jun 11, 2019

Original Title :

Venetoclax in patients with acute myeloid leukemia refractory to hypomethylating agents-a multicenter historical prospective study.

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