A New Investigative Treatment Option for Acute Myeloid Leukemia Produces Positive Results

A new study has uncovered that using a hypomethylating agent in combination with a BCL2 inhibitor called Venetoclax may be advantageous as a therapeutic option for individuals with acute myeloid leukemia (AML) following myeloproliferative neoplasm (MPN). The results from this study were published in the journal Leukemia Research.


Polycythemia vera (PV), primary myelofibrosis (PMF), and essential thrombocythemia (ET), are all Philadelphia chromosome-negative MPNs. These conditions cause proliferation of the myeloid cells within the bone marrow. All of these conditions can lead to secondary AML. This is also called MPN-blast phase or MPN-BP. For these patients, clinical outcomes are extremely poor with a median survival of only 3-5 months. Fortunately, there is a curative option for this subset of patients. It is called hematopoietic stem cell transplantation.


The FDA has recently approved a new treatment for AML patients called Venetoclax. This therapy is to be used in concert with azacytidine, cytarabine, decitabine, or HMA. This approval was specifically for patients who were newly diagnosed, 75 years or older, or those who were not able to tolerate chemotherapy.

However, individuals with MPN-BP were not included in the study that lead to the approval of this new treatment. These trials were called VIALE-A and VIALE-C. This recent study has amended the previous study to include MPN-BP (8) and MPN-AP (1) patients to see if a similar therapy could work for them.

New Findings

This new study assessed whether this subset of patients could achieve complete response (CR) or an incomplete hematologic recovery, but an otherwise complete response (CRi).

Of those given a combination of azacitide or decitabine and Venetoclax, 3 patients achieved CR. Two of these individuals had previously had a disease relapse when only receiving HMA therapy. Thankfully, that means that prior HMA exposure does not matter for response to this therapy. Two other patients given this treatment combination achieved stable disease.

For 3 of the patients who had achieved either stable disease, CR, or CRi, this combination therapy was used to move the patients toward HSCT. Every single one of these patients were still alive at the median follow-up, 8.5 months after the initiation of the study.

Looking Forward

This study was the largest to ever examine this treatment for patients with MPN-BP. Its results are promising for this therapy as an option for this subset of patients. Of course, further research is needed. But these results could mean better outcomes for a group of patients who has yet to have an advantageous treatment option.

You can read more about this therapy here.

Follow us