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Advances in AML Research | Where Do Clinical Trials Fit In?

Advances in AML Research | Where Do Clinical Trials Fit In? from Patient Empowerment Network on Vimeo.

How do clinical trials advance acute myeloid leukemia (AML) research? Dr. Farhad Ravandi-Kashani discusses newer AML treatments that have changed the landscape of care and how patient trial participation moves research forward.

Dr. Farhad Ravandi-Kashani is professor of medicine and Chief of the Section of Developmental Therapeutics in the Department of Leukemia at The University of Texas MD Anderson Cancer Center in Houston, TX. Learn more about Dr. Ravandi-Kashani.

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Transcript:

Laura Beth:

When it comes to AML research and emerging treatment options, what are you excited about right now?  

Dr. Ravandi:

Well, I think one of the biggest, I would call, revolutionary changes in AML was the introduction of venetoclax (Venclexta), because AML is a disease of the older population.  

The median age is about 68, which means half of the patients are older than 68, and among the other half, majority are older than 55. And when you go beyond 60, 65, traditional chemotherapy is not well-tolerated. But the introduction of venetoclax plus azacitidine (Onureg) or decitabine (Dacogen), depending on the center, has really completely changed the management of older AML patients from a situation that it was dismal treatment to a situation that’s still not great, but it’s a lot better, as in we don’t cure the majority, but many people have a tolerable therapy and live with their disease for several months if not several years.  

And personally, I have treated a 97-year-old patient, and she did well for three years, so. Because of this drug, age is no longer as frightening as it used to be for advanced age. 

Laura Beth:

So, where do clinical trials fit in when it comes to choosing treatment?   

Dr. Ravandi:

So, the best drugs that we have available now, the venetoclax that I mentioned and all the other drugs that I mentioned, targeted drugs, came from clinical trials.  

If we don’t do clinical trials, we would be still doing the same treatments that we were doing in the 1970s and ‘80s.  

In fact, up until about seven or eight years ago, many places were still doing the same treatments that was developed in 1970s, which in the era of computers, and Apple, and everything else, it’s mindboggling that we should be doing something that we were doing in the ‘70s. So, clinical trials are important to move the field forward. They are at major academic centers, all the clinical trials are extremely well-vetted and scientifically vetted, as well as with institutional review boards, ethically vetted. So, patients can be sure that they’re not going to get anything less and potentially more than what they would normally get.  

MPN Treatment Decisions: Which Path is Best for You?

MPN Treatment Decisions: Which Path is Best for You? from Patient Empowerment Network on Vimeo.

Dr. Ruben Mesa provides an overview of available treatments and reviews important factors to consider when choosing a therapy for essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF).

Dr. Ruben Mesa is an international expert in the research and care of patients with myeloproliferative neoplasms (MPNs). He serves as director of UT Health San Antonio MD Anderson Cancer Center in San Antonio, Texas. More about this expert here.

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Transcript:

Dr. Ruben Mesa:

The treatment landscape for myeloproliferative neoplasms is changing very rapidly. And in a good way, it’s increasingly having many more options for patients with Myeloproliferative Neoplasms. But I would separate it, really, into two groups. First, there are those individuals with essential thrombocythemia and polycythemia vera.

These individuals, we have newer therapies, such as interferons, we have, potentially, use of JAK inhibitors, we have some experimental therapies, as well as prior therapies we’ve used and become accustomed to, including hydroxyurea, phlebotomy, and aspirin.

But we’re learning much more about how to use these therapies; how to combine them; what constitutes success with these therapies; what should constitute a change in terms of therapy.

And there are new therapies being developed in the future that will impact this group of individuals with earlier MPNs: ET and PV.

For patients with myelofibrosis, the treatment is evolving. Patients with Myelofibrosis are affected in different ways. It is, in some ways, a more problematic disease.

There is evolution of our most impactful therapy, of stem cell transplantation. We have a better sense of in which patients we should consider that treatment, and how that can be applied in the safest way. We also have more medical treatments. We just saw in 2019 the approval of Fedratinib as the second specific JAK inhibitor approved for patients with myelofibrosis.

We, additionally, now have, truly dozens of clinical trials of new therapies in development that are in clinical trials right now that might be helpful for patients with myelofibrosis who have either had Ruxolitinib, or have a suboptimal response to Ruxolitinib, or sometimes even newly diagnosed patients. But I would say the future is very bright.

So, it is key with a treatment to first understand what is the treatment, what is the dose, and what is the goal? Each of the treatments have different goals. Some of the goals are to decrease the likelihood of blood clots or bleeding.

And frequently, we assess whether we’re protecting against the blood clots or bleeding by bringing down elevated counts. Is the plate account high, and we’re trying to bring it into the normal range? Is the hematocrit high, and we’re trying to bring that to under 45%? Is the white blood cell count high? Have we lowered each of those? First, it’s around controlling blood counts if that is the goal, as well as trying to decrease at risk of blood clots or bleeding.

 Second, if patients have symptoms associated with their MPN, sometimes itching, sometimes symptoms associated with high courts, sometimes enlargement of the spleen, or symptoms associated with the spleen, have we reduced or nullified those symptoms? Have we shrunk the spleen if the spleen was enlarged?

And then, finally, we assess our goal by trying to be sure that patients are not progressing or getting worse on the disease. So, depending upon the treatment, we first asses what is our goal? Is it to improve counts? Is it to improve symptoms? Is it to shrink the spleen? And have we accomplished one, two, or all three of those goals? Or was only one those our goals to begin with? 

An Expert Summary of Current MPN Treatment Options

An Expert Summary of Current MPN Treatment Options from Patient Empowerment Network on Vimeo.

 MPN expert, Lindsey Lyle, provides an overview of therapies used to treat myelofibrosis (MF), polycythemia vera (PV) and essential thrombocythemia (ET).

Lindsey Lyle is a physician assistant at the University of Colorado Cancer Center, specializing in hematological malignancies with a subspecialty in myeloproliferative neoplasms (MPNs). More about this expert here.

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Transcript:

Lindsey:

To overview the treatment types for MPNs, we have a variety of different mechanisms in which we use, and clumping these three main MPNs together, we can kind of break it down into, first of all, cytoreductive therapy, which is nonspecific, but really just reduces the amount of cells the bone marrow is producing. And so, it’s really to control the blood counts. And, different types of cytoreductive therapy generally are – hydroxyurea is used probably the most commonly.

There are some other sorts of chemotherapy that may be used in different instances. We also have biological agents, such as interferons, that may be used in patients with MPNs. We then have JAK inhibitors, which there are two FDA-approved JAK inhibitors at this point for myelofibrosis, and one approved for polycythemia vera.

We also have a variety of novel agents in clinical trials. These may be inhibiting different pathways of the cellular production or different signaling pathways at the level of the stem cell, so there are a variety of those. We also use hypomethylating agents in some patients who maybe have higher-risk disease, mainly myelofibrosis, that really changes the way that the stem cells are produced in the bone marrow in order to control the cell counts and also symptoms.

So, there are a variety of therapeutic measures that are taken. Additionally, not necessarily medication-related, but phlebotomy, which is considered a therapy for polycythemia vera, is generally used in order to reduce red blood cell volume, and then, aspirin is commonly used, especially in polycythemia vera and essential thrombocythemia as a supportive care medication to reduce risk of complications from the disease.