What factors are considered when choosing an AML treatment approach? Dr. Ann-Kathrin Eisfeld explains how shared decision-making comes into play when deciding on a therapy and reviews the options available to treat AML.
Dr. Ann-Kathrin Eisfeld is Director of the Clara D. Bloomfield Center for Leukemia Outcomes Research at The Ohio State University and a member of the Leukemia Research Program at the OSUCCC – James. Learn more about Dr. Eisfeld.
With all the new tools that are available, what other factors do you consider when working with an AML patient to choose a treatment approach for them?
Dr. Eisfeld:
The most important aspects are what we call – and this is – I’m glad that you bring this question up because I feel you have to think of – and that was what we’ve been talking about – called disease-associated factors. This is everything in the leukemic cell. They – how does a leukemia looks like? How does the blast look like? What changes are there?
That’s the biggest part of what I would call patient-associated factors: the patient age, the patient performance status, actually the patient. In every – because I think, sometimes, we forget about it. But we just look at all the molecular testing.
But even if – for example, there would be a patient with a very good risk leukemia, where I think, “Oh, this leukemia should respond very well to an intensive chemotherapy.”
If the patient cannot tolerate chemotherapy or – and I see it more often than I would wish for patients who are young who have a great performance status, but they just cannot – they – their family reasons. Small children sometimes – they just cannot be away for so long. This all comes into consideration. So, it’s really important because we all work together as a team. And the right treatment for the leukemia might not be the right treatment for the patient.
And for most cases, however, I think, it will only work if one stands with a whole heart with both physicians, and patients, and family. Because it’s a long journey behind the care that’s being given. And so, this is a joint decision-making, and there are different options that can be done. Of course, I would not advise something where I would think there are no chances of success.
And so, this has to be an open discussion. But this is – it’s very often a very tough treatment to communicate that and see what are the goals of each patient? That will be most important for treatment and decision-making.
Katherine Banwell:
What types of AML treatment classes are currently available?
Dr. Eisfeld:
This is a very good question. The most classic treatment class is intensive chemotherapy. This is just because people might have heard the names. It is called 3 + 7 or 7 + 3, which refers to one weeklong impatient chemotherapy treatment. But you get one chemotherapy for seven days. And the first three days, you get a second treatment as well.
That’s why it’s called three in seven in here, but it’s a total of seven days. So, we have intensive chemotherapy. And there are different flavors of it. But this is usually the backbone. The second class is what I would call a targeted inhibitor. And here we can look at two different aspects. We have targeted inhibitors for a specific DNA mutation that are found. And specifically, one are called IDH or FLT3 mutations.
And these are pill forms that I usually by now combined with a third drop class which is called hypomethylating agents. And I will go through in a moment.
But these are pills that really only work in patients and carry that genetic change. They have very, very low toxicity and very high chances of working. So, that’s why this testing is so important to see if one is one of the 15 percent of AML patients carrying an IDH mutation – 15 percent isn’t low. And a similar rate carries a FLT3 mutation.
And then there is also going to target inhibitors. That is targeted because it is against what I would call a pathway. The gene that is commonly activated in acute leukemia – and this is called BCL-2 and the drug is called venetoclax (Venclexta).
This is now stormed through the acute myeloid leukemia world in just a few years ago and has been approved as a front-line treatment option for several patients, especially for those who are older. And we know that even patients who respond usually favorably to chemotherapy, some of those also respond well to venetoclax the Bcl-2 inhibitor. The benefit is that this treatment in many cases if it works, can be done as an outpatient in here and has very often lower complications.
It is actually has so good results that I – sometimes it seems too easy. So, we actually advise patients to still try to get – the first time they get the treatment, do it at a center where it’s done more commonly. Because it sometimes – don’t underestimated the power of a pill. And it’s still a very, very powerful drug. So, doing it in a controlled setting – because if cancer cells break down, they break down and can create all sorts of trouble.
So, that is really something – for several leukemias, it can be concerning. And again, now the treatment group would be called hypomethylating agents. The names are azacitidine (Vidaza) and decitabine (Dacogen). And they act in a very different way. They try to change the epigenetics like methylation patterns. And often, if it is an untargeted way of the tumor cells and they can be used alone.
Or very often by now in combination with the targeted inhibitors that I was just mentioning. These are infusions that can be done either over five, seven, or 10 days depending on the combination treatment. And for patients, as I mentioned before, that don’t respond well to many other options to those patients with a complex karyotype. This is, for example, a scenario where patients can just receive this as their only therapy.
Katherine Banwell:
What about stem cell transplant? You didn’t mention that.
Dr. Eisfeld:
Yes. That would be the next one. So, stem cell transplant always comes as an option, which I would call as a maintenance therapy. Again, two aspects. We have two different end goals.
First is get rid of some leukemia. Second is to make sure it stays away. And as soon as the leukemia is in complete remission, depending on the performance status – the agent. Again, in multiple different things. It’s not an easy decision.
At that time, there has to be a conversation. And that always involves a leukemia physician and a transplant physician very often. These are different providers that goes for the risks and benefits. Where the question is if I only continue to do chemotherapy – because it’s never only once. You would always have to repeat your chemotherapy. What is the likelihood that the leukemia comes back, and does it outweigh the risks that comes with the stem cell or bone marrow transplant that comes in here. But for many leukemias, especially for young patients and for patients with higher risks, this is the only chance of a cure. That is the most curative and only curative attempt for many leukemia attempts.
Katherine Banwell:
Where do clinical trials fit into the treatment plan?
Dr. Eisfeld:
That is the absolute backbone. We always have to think about that.
Everything – all the treatment options that I mentioned – have been clinical trials, just very, very short time – very few years ago. So, every patient that comes to a leukemia or a cancer center, clinical trials will be discussed if they’re available. Because they will provide a special opportunity to have even more fine-tuned treatments – either newer agents. And I think what is very important to mention is that all clinical trials that are available would give the option of the best standard of care.
And then the hope that a patient wouldn’t be getting any of the best standard of care options that are approved. The hope is that the new agent or added agent in many cases would even do better.
It’s also important that there’s a lot of additional monitoring during the trial. I think it can be seen in two ways as two parts of a coin. In one way, it may be additional visits to the hospital or additional blood draws that are necessary to be sure that the medications are safe, and that researchers and conditions can learn about it. But on the other hand, it also gives you this extra bit of being looked after and really getting checked in and out, making sure that all organs are functioning that everything is just going fine. And many patients appreciate this a lot. And they have this pair of extra eyes on them all the time.
Katherine Banwell:
Dr. Eisfeld,what therapies are available for AML patients who relapse or don’t respond to initial therapy? And is this treatment approach different from those who are newly diagnosed?
Dr. Eisfeld:
Most of the time, the treatments available at relapse are the same available at the first diagnosis. Just because we know now that, for example, if you have a molecular marker that, for example, is available, it would act with also relatively high chance of relapse upset. However, at relapse, the most important thing I personally would do is consider a clinical trial even stronger than in the first mindset.
Because it means that the leukemia outsmarted current treatments very often. So, usually what we would be doing is see if there is a targeted inhibitor or a cell mutation FLT3 or IDH, which I would personally always prefer to go in MLL rearrangement now for the new menin inhibitors where one would go with the same option as if it would have been their diagnosis. But if not to really consider clinical trials is a strong urge.
Katherine Banwell:
Should patients or should relapsed patients undergo genetic testing again? Is it necessary?
Dr. Eisfeld:
Yes. At any time. Yes. Because we know that the leukemia changes. And you just can think about it in the way is that the cells that are surviving treatment, they’ve become smart. There was so much poison. There was so much treatment put on them.
And the ones that survive might have a quiet additional chromosome change as additional gene changes. And even if a genetic change has not been present at time of diagnosis, the reason the cell has survived might have been that it has now one of these changes that came up on a later time during treatment or while the cell is hiding somewhere to come back.
https://powerfulpatients.org/pen/wp-content/uploads/ACTIVATED-AML-Resource-Guide.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2023-03-01 12:39:302023-04-20 10:54:28[ACT]IVATED AML Resource Guide en español
TP53-mutated acute myeloid leukemia (AML) treatment has some challenges. Dr. Naval Daver from the University of Texas MD Anderson Cancer Center shares his perspective. Learn about promising treatments on the horizon for this AML subgroup.
[ACT]IVATION TIP from Dr. Daver: “The TP53 mutation remains the most difficult subset of acute myeloid leukemia, there is hope on the horizon with new treatments such as CD47 antibodies and targeted therapies like APR that are being looked at, and also a strong consideration for allogeneic transplant in TP53, because this seems to be the only modality associated with a good chance of cure after achieving remission with one of the frontline therapies.”
Dr. Daver, what have we learned about TP53-mutated AML? And what is the takeaway for these patients?
Dr. Naval Daver:
TP53-mutated AML remains the most difficult molecular subset of all acute myeloid leukemia. Patients who have this mutation, unfortunately, do not respond well to any of the established standard care therapies, including intensive chemotherapy, the HMA alone, such as azacitidine (Onureg or Vidaza), decitabine (Dacogen) alone, or even HMA venetoclax (Venclexta) with all of these, we do see responses, especially with HMA venetoclax or intensive chemotherapy, we can see 15 to 55 percent remission rate, but the remission, very short lived, early relapses and the median overall survival across all of these currently available standards of cares are between six to 10 months.
So there has been an intense effort in the last six, seven years to develop TP53-directed therapies or therapies that will work regardless of TP53 mutation, and there are two drugs this time that are very promising and being evaluated as ongoing Phase II and Phase III studies.
One of them is an immunotherapy drug called magrolimab which seems to have very similar activity and probability with good response rates in TP53-mutated AML. This has been completed in a single arm phase 1B study in front line TP53-mutated AML where we saw close to 50 percent CR, CRI complete permission rates. And median survival was above 11 months in older unfit TP53, which is better than any survival we have seen in the past in this population.
The other study was with the oral care targeted therapy towards TP53, called APR, and this therapy was specifically designed to target the TP53 mutation, and this is being evaluated in the frontline setting in combination with a society in venetoclax. We hope that these regimens are these novel therapies, one or both of them will be able to at least incrementally improve their current outcomes in TP53.
The other area where we have really been doing a lot of research, and I think the data is suggesting, is that allogeneic transplant may work for separate, and we are routinely considering transplant in these patients in the frontline setting, once they are able to achieve remission.
My activation tip is the TP53 mutation remains the most difficult subset of acute myeloid leukemia, there is hope on the horizon with new treatments such as CD47 antibodies and targeted therapies like APR that are being looked at, and also a strong consideration for allogeneic transplant in TP53, because this seems to be the only modality associated with a good chance of cure after achieving remission with one of the frontline therapies.
https://powerfulpatients.org/pen/wp-content/uploads/Challenges-in-Treating-TP53-Mutated-AML-Hope-on-the-Horizon.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2023-02-01 16:06:552023-04-20 10:50:52Challenges in Treating TP53-Mutated AML, Hope on the Horizon
Dr. Naval Daver from the University of Texas MD Anderson Cancer Center discusses whether untreated acute myeloid leukemia (AML) can be treated with lower intensity chemotherapy.
[ACT]IVATION TIP from Dr. Daver: “Ask your physician and your oncologist when you’re talking with them about what all the newest therapies are and what would be specifically the best treatment for their specific leukemia with respect to the different mutations.”
Dr. Daver, what are we learning about patients with untreated AML who are ineligible for intensive chemotherapy? Will intensive chemotherapy, a thing of the past, in the near future?
Dr. Naval Daver:
There has been a major shift over the last four or five years towards using lower intensity combinations, such as azacitidine (Onureg or Vidaza) and venetoclax (Venclexta) and patients who are definitely about 75 and not fit for intensive induction. I don’t think anybody debates in that population, but even in patients 60 to 75 years away, you are borderline, and maybe we could give intensive induction chemotherapy and get patients to through it with support of care, antifungals, antibiotics by close monitoring, but we’re seeing similar remission rates with azacitidine (Vidaza), venetoclax (Venclexta), much less toxicity, less mortality, and especially the goal is to get a number of these patients to allogeneic stem cell transplant, which it is.
Then we feel that the lower intensity, better tolerated, smoother remission getting patients in a good condition an allogeneic transplant may be the way to go now, of course, to really make the standard of care, we have to look at this in a randomized fashion to make sure that what we believe is actually what the data is going to confirm, so there is an ongoing randomized study looking at the azacitidine and venetoclax intensity versus the traditional intensive chemotherapy called three plus seven in patients 18 to 65 years of age, and that…then you will, I think, give us a lot of information and data as for whether we can start for placing intensive chemotherapy for a large proportion or majority of AML patients, even those who are younger.
Today, I don’t think that in terms of chemotherapies are a thing of the past, I think those patients who are below 60 or even those who are 60 to 65, who are routinely doing intensive induction chemotherapy, one has to realize that the five-year survival for many molecular subsets are close to 50 to 60 percent with intensive induction chemotherapy, whereas with HMA venetoclax in the older unit, we’re looking at three to five-year survival rates of about 30 percent, so we have still not seen data and younger patients with Hamas to be convinced that this will replace intensive chemotherapy altogether, I think the signal suggests that there is a potential for it to do so, especially with the use of allergenic tensor as plan, which we’re using quite frequently and…or maintenance.
But that has not yet been established. So I would still say we do use intensive chemo in those who are young and fit, so my activation tip for this is that there has been a lot of progress in the lower intensity therapies over the last six or seven years.
A decade ago would not even be asking whether there’s anything that can replace intensive chemo today we do have data with HMA venetoclax that suggest that it may be as good as intensive chemo looking at the response rates MRD negativity, and especially with three drug combinations where adding targeted therapies to HMA venetoclax, those response rates and depth of response looking as good, if not better, than intensive chemo there are randomized studies ongoing that are going to be looking at intensive chemotherapy versus HMA venetoclax and if those show equivalents or superiority for HMA venetoclax, I think in the next five, six years there will be a huge shift towards less use of intense and chemotherapy in the frontline setting, but we’re not there yet.
https://powerfulpatients.org/pen/wp-content/uploads/A-Look-at-Lower-Intensity-Chemotherapy-in-Untreated-AML.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2023-02-01 15:34:362023-04-20 10:55:15A Look at Lower Intensity Chemotherapy in Untreated AML
Why are acute myeloid leukemia (AML) clinical trials so critical? Dr. Naval Daver from the University of Texas MD Anderson Cancer Center shares his perspective about clinical trials. Learn how clinical trials help both current and future AML patients.
[ACT]IVATION TIP from Dr. Daver: “Clinical trials are critical, both for the patients themselves to get access to what we call tomorrow’s medicine today as well as potentially to help move the entire field forward.”
Dr. Daver, what is the importance of clinical trial participation as it relates to breakthroughs in AML, and what advice do you have for AML patients considering a clinical trial?
Dr. Naval Daver: Clinical trials are critical for the progress that we have already seen an acute myeloid leukemia, the drugs that have been improved in the last six, seven years, including venetoclax (Venclexta), FLT3 inhibitors, midostaurin (Rydapt or Tauritmo), gilteritinib (Xospata), hopefully quizartinib other emerging targeted therapies…IDH1, IDH2 inhibitors, menin inhibitors, CD47 antibodies, we’ve learned about all of them and have got approvals and many of them through the ongoing clinical trials.
I think it’s very important for patients to realize that in most large academic centers, we will only participate in the clinical trial if we think it has the potential to improve the standard of care in the future. There’s very little incentive for academic investigators or clinical investigators, such as myself, we’re very, very busy to get involved in a trial if we don’t think that it has the potential to improve the outcome or change the nature of AML therapy in the future, so a lot of patients often ask me, Oh, I want the randomized or placebo arm. There is no real placebo alone in any AML study that I’m aware of, most of the studies will use standard of care, which is what you would’ve gotten wherever you were getting treatment at home, locally, community hospital versus a standard of care plus where the new drug will be added, whether it’s the FLT3 inhibitor, the CD47 antibody, the menin inhibitor
So there’s a good chance, 50 percent that you’re going to get standard of care plus that we think has the potential to improve the outcome, of course, you never know, that’s what you do, the trial, but we think based on the previous pre-clinical data to pass when the page to deliver this looks like it will improve the outcome for this molecular or site group versus standard of care, which is what you will have gotten.
So I think it’s important to realize that you will never get less on standard of care and any clinical trial, at least in the AML field, and at least in our experience that they understand.
Now, beyond that, there’s also a Phase I in two states, and those are the ones that we focus on quite a bit at MD Anderson, these are single arm studies, meaning everybody will get the investigational agent combo, so azacitidine (Onureg or Vidaza) and venetoclax (Venclexta), we were one of the first sites to work on and leave this study and all of our patients in 2015, 2016, we’re getting this regiment, it was not approved to much later in 2019, 2020, and for those three, four years, our patients, hundreds of patients were able to get that combination, which probably cured many, many more than would have been cured to the standard of care until, of course, I’ve got a pro four years later, but for an option, of course, you cannot wait four years, so I’m a huge believer in clinical trials, I think it’s really, really important, both for the patients themselves as well as for the field, for us to be able to move the entire AML field forward for the next decade, and I would very strongly consider looking at or discussing with your treating physician trial options, and then you can look at them on your own through clinicaltrials.gov, or other sites with leukema and lymphoma that give a lot of information on clinical trials.
So my activation tip related to this question is that I think clinical trials are critical, both for the patients themselves to get access to what we call tomorrow’s medicine today as well as potentially to help move the entire field forward, all of the clinical drug approvals in progress we have seen in AML in the last six, seven years have come through clinical trials that patients in the past have agreed to kindly participate and helped probably themselves by getting better medications and combinations, and definitely the field to move forward, so definitely a big proponent for clinical trials.
What acute myeloid leukemia (AML) treatments are available for high-risk patients? Dr. Naval Daver from the University of Texas MD Anderson Cancer Center discusses various mutations, potential for cure, and clinical trials. Learn about the outlook for high-risk AML treatments.
[ACT]IVATION TIP from Dr. Daver: “The best way to get up to these agents is to go on clinical trials and incorporate these therapies, both in the frontline setting as well as in the relapsed refractory setting.”
Dr. Daver, what treatment strategies are available for high-risk AML patients?
Dr. Naval Daver:
High-risk AML patients includes a group of a number of different mutations, and cytogenetic abnormalities, this includes TP53 mutation, as well as adverse cytogenetics, which includes chromosome 17, deletion 5, deletion 7, as well as complex carrier type. This entire group historically had a poor outcome and has had limited responses to traditional intensive chemo, even if we achieve responses there, usually short-lived.
We do have some patients where we are able to achieve remission with intensive chemo or with azacitidine-venetoclax (Vidaza-Venclexta) and transition and transmission them transplant with about 25 to 30 percent potentially achieving a long-term remission and possible cure.
But aside from that, there is very little potential to cure these patients with just traditional intensive chemo, venetoclax in this area, there has been developments with the emergence new class of immunotherapy drugs, called CD47 antibodies, the one that’s most advanced in this field is a drug called magrolimab, and we are evaluating the drugs such as magrolimab in combination with azacitidine as well as in combination with azacitidine-venetoclax and are seeing high remission rates, both in TP53 mutated and TP53 wild type.
So this pathway that works by activating a macrophages or the immune system to attack the tumor cells, seems to be in some way mutation agnostic with response rates being maintained even in the traditional high-risk subsets, especially such as TP53 and complex cytogenetics for some of the other high-risk groups such as MLL, we’re using targeted therapies like menin inhibitors, and these seem to work well in those patients who have these adverse cytogenetic molecular abnormalities, so there is progress, and we think that the CD47 antibody field and hopefully the main inhibitor feed will be able to improve outcomes in these traditionally molecular cytogenetic subsets.
My activation point related to this question is for high-risk mutations and cytogenetic commonalities such as TP53 complex carrier chromosome 17 MLL, best hope at this time is in clinical trials evaluating novel therapies such as CD47 antibodies and menin inhibitors. These are not yet FDA-approved, but based on emerging data from the ongoing Phase I, II studies, we think that there is a good chance they will be approved in the future.
However, this time, the best way to get up to these agents is to go on clinical trials and incorporate these therapies, both in the frontline setting as well as in the relapsed refractory setting.
https://powerfulpatients.org/pen/wp-content/uploads/A-Look-at-Treatment-Strategies-for-High-Risk-AML-Patients.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2023-02-01 15:10:362023-04-20 10:48:00A Look at Treatment Strategies for High-Risk AML Patients
How have acute myeloid leukemia (AML) treatment approaches expanded for older and high-risk patients? Dr. Naval Daver from the University of Texas MD Anderson Cancer Center shares insight about additional treatment options. Learn about the potential for long-term cures for these patient groups.
[ACT]IVATION TIP from Dr. Daver: “There is a dramatic progress, especially in the treatment of older unfit AML with the approval of the azacitidine-venetoclax combination.”
Dr. Daver, for older and high-risk AML patients, how are the treatment approaches expanding?
Dr. Naval Daver:
In older and high-risk AML, the major approval has been the combination of azacitidine (Onureg or Vidaza) and venetoclax (Venclexta), which is a BCL-2 inhibitor, the regimen was evaluated in a large Phase III study called the VIALE study, where we looked at the standard of care for the last two decades for older unfit AML, which azacitidine alone versus the combination of azacitidine and venetoclax and this combination showed a three times higher remission rate, 75 versus 28 percent overall remission rate as well as an improvement in overall survival and long-term survivors.
So this has led to great progress with now remission rates of 75 percent achievable in older unfit AML and many of them being durable at three years with ongoing follow-up, so this has really opened the door for us to be able to treat patients up to 75, 80, 85 years of age with effective therapy given the three parts of these to achieve remission, which is usually associated with freedom from transfusion improvement, quality of life, improved energy, less time in the hospital, less infections.
The other progress now is coming from the use of targeted therapies as well in these populations, and even though the HMA venetoclax or azacitidine combination is doing very well.
We now have data, in fact, from the ASH 2022 December meeting that at three years, about 25 percent or so I would still remain alive with azacitidine was even or 8 percent, now it’s 25 percent. But, of course, we want to do much better than that, and so this is where we are incorporating the targeted therapies, the FLT3 inhibitors, the IDH1, IDH2 inhibitors, menin inhibitors, and immunotherapies onto the backbone of azacitidine-venetoclax, which we hope will further improve that long-term survival cure from 25 to hopefully 50 to 60 percent and beyond.
So a lot of progress, you know, going from less than 10 percent, a 30 percent survival, long-term, and I think in the next few years, even up to 50 percent with some of these new combinations. The activation tip related to this question is that there is a dramatic progress, especially in the treatment of older unfit AML with the approval of the azacitidine-venetoclax combination.
This regimen is now giving high remission rates, which approximate remission rates that are seen with traditional intensive chemotherapy without the mucositis and toxicities and better volatility, and we are now working to further improve the remission and the durability of this dominant of initial.
…potentially adding targeted therapy such as FLT3 inhibitors, IDH1, IDH2 menin inhibitors, and we think that potentially in the next decade, we could be achieving long-term cures in a large proportion of older unfit AML, which was something one could just dream of a decade ago.
What do newly diagnosed acute myeloid leukemia (AML) patients have for promising treatment options? Dr. Naval Daver from the University of Texas MD Anderson Cancer Center discusses progress in available treatments. Learn about therapies determined by key factors.
[ACT]IVATION TIP from Dr. Daver: “It’s very important to really consider all the available treatment options and if needed to seek consultation with an expert or academic center to get the most up-to-date treatments available for AML.“
Dr. Daver, for newly diagnosed AML patients, what are the latest and most promising available therapies?
Dr. Daver:
For newly diagnosed AML at this time, the most promising agents include targeted therapies and BCL-2 inhibitor treatments, these are non-chemotherapeutic drugs, and we’ve seen great progress in the application of these as well as recent FDA approvals.
So one of these is an agent called venetoclax (Venclexta), which is a BCL-2 inhibitor and venetoclax in combination with hypomethylating agents such as azacitidine (Onureg or Vidaza) has shown a response to close to 75 percent.
And the nice thing is that this regimen can be given and patients who are older than 70, 75 years of age, and even those who are having comorbidities are not fit for traditional intensive chemotherapy with similar response rates, so this has been approved in the last couple of years for the frontline treatment of AML, and we’ve been using this combination of venetoclax and azacitidine quite frequently with high efficacy in this patient population, the other new agents that have shown breakthroughs in AML are the targeted therapies, these include FLT3 inhibitors that target the FLT3 mutation and these have shown good activity, but the single agents with gilteritinib (Xospata) being approved in the relapsed refractory setting as a single agent where gilteritinib showed a response rate of about 50 percent as a single oral targeted therapy in relapsed FLT3-mutated AML, which is actually better than the response rate with high-dose combination more where the response rate is only about 25 to 30 percent.
So, gilteritinib is now approved, and it’s now moving and being evaluated in frontline setting other FLT3 inhibitors like lestaurtinib (CEP-701), actually just recently completed frontline studies showing improved outcome when lestaurtinib added to intensive chemo versus just intensive chemo in FLT3 in AML. And we hope and think there’s a good chance lestaurtinib will be approved in the near future.
And also IDH inhibitors have been approved both in the relapsed setting, frontline setting, and now we even have a third group of targeted therapy is called the menin inhibitors, they target MLL rearrangement and NPM1 mutations, which are seen in about 15 percent to 20 percent of the AML, so there’s been a lot of progress.
All of this in the last seven years, six, seven years with multiple targeted therapies, with multiple inhibitor-based treatments, showing progress in AML and then also recently, the concept of maintenance therapy, this is something we used for the last couple of decades in a acute lymphoblastic leukemia and multiple myeloma and in lymphoma.
But we had not had clear data in AML, but the recent study using oral formulation of a azacitidine in CC486 has shown the maintenance in patients who complete an induction consolidation and could not go to allogeneic stem cell transplant for one reason or the other was important and improve both overall survival and relapse-free survival, and so this is the first time now we have an FDA-approved and standard use of maintenance therapy after the traditional induction consolidation, so even changing the general paradigm of AML therapy.
So a lot has changed in the last seven to eight years in the treatment of acute myeloid leukemia, and this is very exciting.
And the activation tip related, this question is that there are multiple new targeted and low intensity therapeutic options available for patients with acute myeloid leukemia, and in our institution, in my opinion, even older patients are eligible for some form of therapy or the other…very few patients, if any, today, are being sent to hospitals or palliative care without treatment.
So it’s very important to really consider all the available treatment options and if needed to seek consultation with an expert or academic center to get the most up-to-date treatments available for AML.
https://powerfulpatients.org/pen/wp-content/uploads/What-Promising-AML-Treatments-Are-Available-for-Newly-Diagnosed-Patients.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2023-02-01 14:39:442023-05-22 10:37:42What Promising AML Treatments Are Available for Newly Diagnosed Patients?
How are acute myeloid leukemia (AML) patients assessed for intensive chemotherapy? Dr. Catherine Lai from Penn Medicine explains eligibility criteria. Learn factors that impact patient eligibility and treatment options for AML patients who are categorized as ineligible for intensive chemotherapy.
[ACT]IVATION TIP from Dr. Lai: “Talk with your physician about how they will determine whether or not you are fit or unfit for intensive chemotherapy.“
Okay, Dr. Lai, what are we learning about patients with untreated AML who are ineligible for intensive chemotherapy?
Dr. Catherine Lai:
To define ineligible for intensive chemotherapy, I think that that is a moving target because historically, we would define patients as eligible for intensive or less intensive chemotherapy based on an age cut-off. And as the population is becoming more fit and is also getting older, what I would like to say is that we should use physiologic age, not chronologic age to determine who is eligible for intensive chemotherapy, and that is…in terms of how that is assessed, that is not uniformly done.
But, in general, it takes into account how active a patient is and what they’re able to do on a day-to-day basis, so mostly their physical function, we also take into consideration their cognitive function as well, but to a lesser extent.
So, for patients who are ineligible for intensive chemotherapy, the standard practice would be the combination of azacitidine (Onureg or Vidaza) or decitabine (Dacogen), both of which are hypomethylating agents in combination with venetoclax (Venclexta), and that combination has really changed the landscape in terms of how we treat patients, it can be given as an outpatient, so it’s much better tolerated and has fewer side effects compared to intensive chemotherapy.
So the activation tip here is to talk with your physician about how they will determine whether or not you are fit or unfit for intensive chemotherapy.
https://powerfulpatients.org/pen/wp-content/uploads/Assessing-Untreated-AML-Patients-Who-Are-Ineligible-for-Intensive-Chemotherapy.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2023-02-01 13:28:252023-04-20 10:50:00Assessing Untreated AML Patients Who Are Ineligible for Intensive Chemotherapy
How does low-intensity AML therapy differ from high-intensity AML therapy? Expert Dr. Ellen Ritchie provides a comparison of the administration methods, side effects and reviews which AML patients low-intensity and high-intensity therapy are right for.
Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.
You mentioned earlier, Dr. Ritchie, low-intensity therapy. Could you tell us about the types of treatment options?
Dr. Ritchie:
So, I’ll go – high-intensity therapy or intense chemotherapy always has to be given really in a hospital. And if you don’t start it – if you can start certain intensive chemotherapies, like daunorubicin and cytarabine (Vyxeos), which is also intensive, in the outpatient setting, but by day seven or eight, you end up in the hospital. And in intensive chemotherapies, you lose your hair, there’s GI toxicities, you’re at high risk of developing infections and you need a lot of transfusion. And for even young people, it’s a difficult therapy for which you’re in the hospital, and 90-some percent of patients are on IV antibiotics.
So, it’s intensive chemotherapy because it has to be given in a hospital setting and requires intensive supportive care. Low-intensity therapy can be given in the outpatient setting. So, at the present time you can get a drug like azacitidine (Vidaza), for example, which is an injection that you get seven days in a row.
Unfortunately, you have to come to the doctor’s office every day for those injections, but once you’ve had the injection, you can go home. Combined with venetoclax (Venclexta) which is an oral agent. So, an oral agent can be given at home.
You need close supervision in the physician’s office when you’re on this type of therapy, but you don’t need the constant support that you need if you are getting intensive chemotherapy. So, it can be done, in the comfort really of your home and with your family. You will have to come in and have transfusions potentially as an outpatient, nearly everyone does. And there’s always the risk that you develop a fever and if you do, you have to come into the hospital for IV antibiotics.
But in general, low-intensity means not so much support needed in a hospitalized setting, and the tolerability of this particular chemotherapy in the outpatient setting.
https://powerfulpatients.org/pen/wp-content/uploads/What-Is-Low-Intensity-AML-Therapy-1.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2021-08-25 13:52:472021-08-25 13:52:47What Is Low-Intensity AML Therapy?
Dr. Daniel Pollyea, an AML specialist, dispels common myths around the causes and symptoms of AML and shares advice so that you can identify credible resources for information. Download the Program Guide here.
Dr. Daniel A. Pollyea is Clinical Director of Leukemia Services in the Division of Medical Oncology, Hematologic Malignancies and Blood and Marrow Transplant at University of Colorado Cancer Center.
I’m Ross Reynolds. Today we’re gonna be debunking some common misconceptions about the causes and symptoms of AML.
And joining me is Dr. Daniel Pollyea. Dr. Pollyea, could you introduce yourself?
Dr. Pollyea:
Yeah. Hi. Good morning, everyone. I’m Dan Pollyea. I’m an Associate Professor of Medicine here at the University of Colorado, where I am the Clinical Director of Leukemia Service.
Ross:
I wanna emphasize to you that this program is not a substitute for medical advice, so be sure to consult your healthcare team when it comes to solid information about it. But you will get some background that I think you’re gonna find useful. And you might have some questions as we go along.
Dr. Pollyea, let’s start out with the basics. What are the causes of AML?
Dr. Pollyea:
Yeah. So, Acute Myeloid Leukemia, it’s a disease, a cancer of the bone marrow.
And it’s the result of an accumulation of mutation and chromosomal abnormalities that affect the DNA of a precursor cell in the bone marrow, otherwise known as a stem cell.
And those abnormalities accumulate until that cell can no longer properly mature, and it also can’t properly die. And so, a cell like that just makes copy after copy after copy of a cell until it crowds out the whole bone marrow with these sorta useless, immature cells.
And the end result of that is the failure of the bone marrow, which causes all of the problems associated with this disease. So, biologically, that’s sort of what happens to make this disease occur.
Ross:
What are some of the myths that you hear from patients that come in and they say, “Oh, this must’ve caused my AML,” but you have to tell them that’s not so?
Dr. Pollyea:
Right. So, I mean, this is one of the most frustrating issues for patients and their families after diagnosis. I mean, it’s a rare disease, only about 30,000 cases a year in the United States. And so, trying to associate a rare disease with external or environmental factors is difficult to impossible. So, although there are a variety of exposures that probably contribute to this disease, we have very little understanding of what those exposures typically are or how that all works.
So, there’s a few things that we know pretty well; large doses of radiation, either associated with like industrial accidents like the Chernobyl disaster, or some of the radiation therapies that patients receive for other types of cancer. Other types of chemotherapy that are used to cure other cancers can contribute to this disease in later years.
We know that there are certain precursor conditions that can evolve to AML, so a person with myelodysplastic syndrome, for instance, has a fairly high chance of someday evolving to develop Acute Myeloid Leukemia. But beyond these sort of a few associations, there isn’t a whole lot that’s known or proven.
Ross:
Now there is radiation associated with X-rays, and some people think that X-rays can cause AML. Is that true?
Dr. Pollyea:
Yeah.
So, I mean, I think a priori no because millions of people get X-rays every day, and only 30,000 people a year get AML. So, clearly it’s not a simple association between getting an X-ray and developing AML. But I think that there is an unknown interaction between environmental exposures and a person’s individual genetic makeup that makes a person more or less susceptible to developing something like AML with respect to exposure to the environment or X-rays and things.
So, while you cannot say that getting an X-ray will lead to AML, certainly there are some people who are more sensitive to the damage that’s done by something like an X-ray. And so, the best course of action is to be cautious and judicious about your exposure to these things, but not to not get these things when they are medically necessary.
So, that’s the challenging balance.
Ross:
Here’s something else we’ve heard, that weed killers can be a risk factor for AML. Is that true?
Dr. Pollyea:
I mean, I think there’s a lot coming out now about weed killers and their association with other types of cancers. Again, I go back to the limitation we have in that in only 30,000 people a year in the United States get AML. Millions of people are exposed to weed killers.
We’re statistically never going to be able to make a clear association. I think that there are certainly some risks for some people. Whether you’re that person who’s more susceptible to developing leukemia or any other cancer because of exposure to a weed killer is impossible to know.
So, like all of these things, I think the advice we have is you have to live your life. You have to do your best to sort of avoid things that you can avoid that you think would be… Or that may cause problems. But not to let those things prevent you from living a normal life.
I know that’s not a satisfying answer, but at the moment that’s the best answer we have.
Ross:
Is formaldehyde exposure another risk factor for AML?
Dr. Pollyea:
Yeah. We think that it is, and kind of along the lines of benzene. But, again, we think that those studies that have shown those types of association show it in very high amounts, amounts that most people in this country would not be exposed to. But I do think, or we do think that there is something to that, to formaldehyde somehow contributing to this.
Ross:
What’s the difference between a risk factor for AML and a cause of AML?
Dr. Pollyea:
Yeah. So, I think risk factors by definition are things that may contribute to AML. And a risk factor for AML by that definition could be walking down the street and having some exposure to radiation from the sun. A cause of AML is something that is a much more solid sort of well-understood factor.
Like I said before, having myelodysplastic syndrome, there is a high chance that that can evolve to Acute Myeloid Leukemia. And if that happens then the MDS, the myelodysplastic syndrome, could be considered or would be considered the cause of your AML. So, very, very different in terms of the amount of evidence that goes into making those determinations
Ross:
Is there a genetic component to this? Can this run in a family?
Dr. Pollyea:
Yeah. So, this is a disease of the genome.
So, I mean, in a lot of respects it is a genetic disease. But the question is very different when you ask is this an inherited genetic disease? Is this disease due to a gene that I inherited from a parent or could pass along to a child?
For many, many years, the answer from the medical community was, “No.” This was not considered to be a disease that clustered in families or that could be inherited. We now know that that’s not necessarily the case. There are some very rare cases where this does seem to travel in families or cluster in families. And we’re now beginning to understand who those people are and what those genes are.
But the vast majority of people with this disease did not inherit a gene to contribute to it and cannot pass this along to a child. This is a random, spontaneous event that occurred within one person’s own body and is not traveling within family. So, we’re learning more and more about this, but really, the vast majority of this is not an inherited genetic condition.
Ross:
You’ve mentioned gene mutations. What mutates a gene? What causes that to happen that could lead down the line to AML?
Dr. Pollyea:
Yeah. Yeah. That’s a great question. Most of the time we do not know the answer to that. These gene mutations occur spontaneously, randomly, and we don’t understand why they happen when they do happen.
And I know that’s, again, not a satisfying answer. It’s very frustrating, particularly patients come in, and, “I’ve lived a healthy lifestyle. I’ve done everything right. I exercise. I eat right. How could this have happened?”
These are things that for the most part are out of the control of a person. These aren’t impacted by your diet or your activity levels, what you eat or don’t eat, what you do or don’t do. That’s a real frustration. In the end, in almost all cases we don’t know or understand why these gene mutations or these, I call them mistakes in the body, occur when they occur. We don’t understand them.
And, Dr. Pollyea, someone asked if benzene can be a risk factor for AML.
Dr. Pollyea:
Yeah. So, benzene is one of the sort of rare environmental exposure associations that we do have clear associations with AML.
But the level of benzene that a person would need to be exposed to is really something that hasn’t been seen in this country in a very long time.
We’d be talking about like an industrial accident type exposure in almost all cases, so being exposed to a cleaning solution or some other fairly minor exposure to benzene, we don’t think is enough, in most cases, to prompt this disease. But benzene in very high doses, like an industrial accident, yes, that is something that we understand can certainly contribute or cause AML.
Ross:
Autoimmune diseases, such as arthritis, can they increase the risk of AML?
Dr. Pollyea:
Oh, boy. That is a really interesting one. So, there are papers in the literature that do support those associations. And I know in my own practice I certainly see that trend. So, I do think that there is something there. There is a proven association between autoimmune conditions and myelodysplastic syndrome, which I said before can be a clear precursor condition to AML. So, certainly, that is an association that is a possibility.
It can be a little difficult to tease out whether it’s those diseases that are associated with ultimately developing AML, or the treatments that people get for some of those autoimmune diseases. Those treatments can modulate the immune system in certain ways that may, in fact, contribute or drive the disease. So, that’s a difficult thing to tease out.
But in general terms, yes, I think there are some associations. Now not by a long shot everyone with an autoimmune disease gets AML. It’s a teeny, tiny fraction. But I think there is an association there.
Ross:
How easy is it to diagnose AML?
Dr. Pollyea:
Well, I mean, I think there’s very clear diagnostic criteria for AML. But I guess that doesn’t really answer the question. And we certainly have patients who come to us after many months of frustration without a clear diagnosis.
So, those scenarios can play out. Many times AML’s a very dramatic presentation, so people get very, very sick very, very quickly with extraordinarily high white blood cell counts and suppression of all the other blood counts that come from the bone marrow like red blood cells and platelets.
In those cases it’s pretty clear that there is a type of acute leukemia going on. There can be some difficulty distinguishing Acute Myeloid from Acute Lymphoblastic Leukemia; those are sort of like cousins, but very different and treated differently. So, it kinda runs the gamut. I mean, it can be pretty clear, but it’s sometimes missed, so yeah.
Ross:
This is a great lead-in to my next question, which is about the symptoms of AML. What should be the warning signs that this might be something you need to get looked at?
Dr. Pollyea:
Right. So, at presentation, the main symptoms are reflective of the fact that the bone marrow, the organ that makes all the cells of the blood, has failed.
So, that can cause severe anemia. Signs of anemia: a white sort of appearance, feeling dizzy or lightheaded when standing, short of breath, weak, tired, fatigue. Those are all pretty clear presenting symptoms for AML. Because the bone marrow also is responsible for making platelets that clot the blood, some people will present with a bleeding complication, or a very subtle rash made up of these particular red dots. We call that a petechial rash. And that rash can come on when the platelet count gets very low.
Sometimes a person will present with an infection or infections that don’t go away or don’t clear because of decrease in white blood cells, the infection-fighting cells of the bone marrow. Those are made in the bone marrow and can fail in the setting of this disease. So, those are the most common symptoms at presentation, symptoms that are reflective of bone marrow failure.
Ross:
You mentioned that sometimes the presentation could be very dramatic, and it sounds like the symptoms are very severe, very quickly. Is that always the case? Is that often the case?
Dr. Pollyea:
That is the case in, I would say, a minority of times. That’s usually the case. It’s more often seen in younger patients with AML. Typically, older patients with AML have a more smoldering course and a much less dramatic presentation, although this sort of very dramatic and dangerous presentation can happen in older patients, but it’s probably something like a third of the time that those very dramatic and medical emergency presentations occur.
Ross:
How important is early diagnosis?
Dr. Pollyea:
Well, I mean, it’s crucial. I mean, in particular in those cases where it’s a very dramatic and proliferative diagnosis, or presentation. A quick diagnosis and recognition of this condition is very important because the sooner a person starts effective treatment the better the ultimate outcome is.
I would say in general terms that applies to all AML patients, but certainly there’s some degrees of variation. So, there’s some AML patients that when I hear about their case on the phone from a referring doctor, it’s appropriate to see them next week in the clinic.
So, it’s not always a medical emergency, but we would never, even in those next-week-in-the-clinic patients, this isn’t something that can wait for weeks or certainly months. This is something that needs to be addressed fairly quickly.
Ross:
What are the best ways to manage those symptoms?
Dr. Pollyea:
Right. So, I mean, at presentation, all those symptoms, the best way to manage those are to start treatment as quickly as possible. So, impacting the underlying cause of this disease is the most important and critical factor to getting a person feeling better because all of these problems stem from the disease in the bone marrow, and so everything else that you do to sort of help a person’s symptoms are Band-Aids when you’re not talking about getting to the root cause.
So, that’s at presentation. Now once we start treatment, there are many potential side effects to any number of treatments. And it all is dependent on what treatment you’re getting and other things about you that will make this a significant problem in some cases. And in that setting, we do have ways that we can aggressively manage a person’s side effects.
Ross:
Can you manage all of the symptoms? Or can people still be experiencing symptoms even after they’re in treatment?
Dr. Pollyea:
Absolutely. So, a person with this disease, depending on how long they’ve had it and some of the features, may not be feeling back to their baseline self for potentially weeks or months after treatment starts in the best-case scenario. So, that can be very frustrating, but a person needs to sort of be able to continue to have a good outlook and stay positive.
Because we are able in many cases to make a big impact on this disease and return a person to their pre-disease quality of life.
Ross:
What are some of the myths that you hear, Dr. Pollyea, about the treatment? Some things that people come in to you saying they think that it helps, but there’s no science to back that up?
Dr. Pollyea:
So, myths about treatment, so many people have a lot of preconceived notions about the intensity of a therapy that they’re going to be asked to withstand. And although sometimes we do treat this disease very intensively, that’s not always the case, and now we have some very effective lower-intensity regimens that can be used in a variety of different scenarios.
There are a lot of people who have a lot of preconceived notions about a stem-cell transplant or a bone-marrow transplant and whether or not they would be eligible for this based on maybe what they’ve heard from friends or family, or what they’ve seen in the internet.
And those are often incorrect. And so, keeping an open mind about treatment options, and discussing those in detail with your doctor are really, really important.
Ross:
You mentioned sometimes it presents in young people, sometimes in older people. What’s sort of typical?
Dr. Pollyea:
This is a disease of predominantly older patients, so the median age of presentation is 68. So, that means that over half of the patients are over 68 years old at diagnosis. So, while this does happen, can happen in younger patients, that’s really an unusual situation. This disease is, like I said, it is predominantly a disease of older patients.
Ross:
There are some patients who I understand think that supplements can deal with the symptoms of AML. Is that accurate?
Dr. Pollyea:
You know, I mean, I think the supplement question is always a challenge. A lot of these supplements, or most of these supplements have never been tested with the rigor of treatments that we’re accustomed to in the medical establishment.
That being said, I won’t deny that some of the supplements can help patients based on what patients’ experiences are and what they tell me. I think what’s really important is just be very open and honest with your doctor about the supplements that you’re taking or want to take to ensure that there are no sort of unanticipated interactions with treatments.
Because I think most doctors are very open to having their patients care for themselves in the ways that they’ve become accustomed to, and they know their bodies very well, and we’re very open to that. But there are sometimes that a drug or a supplement might have a bad interaction with the treatment.
And so, a good example in my practice is antioxidants. So, there’s a lot of literature, a lot of interest in antioxidants as cancer-prevention treatment.
And a lot of that is not well-established, but still I don’t see much harm. But when it comes time to treating a cancer, that’s a very different situation. When we give a patient treatment to try to kill the cancer cells, many times we’re trying to provoke oxidation. That’s part of how these drugs and these treatments work.
So, if you’re taking those treatments, but also at the same time taking antioxidants, there’s the potential you could sort of be cutting your therapy off at the knees, fighting it with one hand behind your back. So, for the period of time when my patients are getting an active treatment, I ask that they don’t take it antioxidant.
And they can resume that in the future in the hopes of preventing another cancer. But the time to prevent with an antioxidant isn’t appropriate when you’re dealing with an active cancer. So, that’s just one example.
Ross:
Fatigue could be a symptom of AML, but there are a lot of causes of fatigue.
How do you differentiate between something that really could be AML and something that isn’t?
Dr. Pollyea:
Yeah. That’s a challenge because I think these are, as I said, older patients. And older patients have a lot of other medical problems. And older people get fatigued, just that’s unfortunately part of the normal aging process. So, we would usually make an assumption that a person’s fatigue and diagnosis is due to the leukemia, the anemia as a result of the leukemia.
But as we successfully treat a patient if they are responding based on their numbers and other objective criteria, but the fatigue is not improving then I think that’s where we would start to look at other contributing factors, and there can be many, so having an open mind at that point is important.
But at the beginning, this is such a monster of a disease, it’s so overwhelming, I think the focus is usually on assumption that the fatigue is due to the disease or to a treatment associated with this disease.
Ross:
This question: is loss of appetite a symptom of AML?
Dr. Pollyea:
Yeah. I definitely see that, hear that, so sometimes people come in and they say that. Sometimes it may not be a loss of appetite, but an extreme weight loss, so a lot of different types of cancer, including AML, can cause that, just basically unintentional weight loss.
A person’s not trying to lose weight. They’re eating what they think is their normal amount and they’re losing tremendous amounts of weight. So, those are both potential presenting symptoms with AML. And loss of appetite, unfortunately, can be associated with some of the treatments for this disease. And taste changes, things not tasting good, can all contribute to that as well, so those are all challenges that our patients face.
Ross:
How important is to get a second opinion? I mean, are all doctors like you pretty much on the same page when it comes to symptoms and treatment?
Dr. Pollyea:
So, this is a challenge. So, the answer to the second question first is unfortunately, no. A lot of this hasn’t quite been standardized. And some doctors, oncologists, cancer doctors, they’ll predominantly be treating the things that are common: colon cancer, breast cancer, prostate cancer. And they will probably only have a few cases of acute leukemia a year.
And so, their approach to this is going to be different than somebody who spends all day seeing patients with AML and thinking about AML.
So, a second opinion is a very nice thing to be able to do. The problem with this disease is that most times it doesn’t afford that opportunity. So, with other conditions you have some time to go out, read about it, talk to some different doctors, get a good plan together.
With AML, often that’s not a possibility. A person is so urgently sick that you have to sorta deal with the resources where you are. The best recommendation I have there, if you do find yourself in a situation where there’s not a lot of expertise is to ask your doctor to just call somebody in the region or email somebody in the region who may have that expertise.
And most doctors all over the country have that sort of resource or partner that they will go to and talk the case through with them, and maybe a transfer to one of those high-volume centers is appropriate.
And maybe that’s not a possibility or appropriate, but maybe you would benefit from just talking… Maybe your doctor would benefit from talking this through. But in cases where it’s not such a dramatic presentation, then yeah, for sure, I think a second opinion can be appropriate. But this isn’t something that can be sort of drawn out for long period of time.
Ross:
You know, when you find out something like this, your tendency might be to jump on the web and start searching for AML. How do you vet those sources that you look at? How do you figure out that their – what would be a sign that they’re bogus sources?
Dr. Pollyea:
Yeah. I mean, I think this field is so rapidly changing and the treatment that we have, that I would, for the most part, assume that what you’re finding on the web is not relevant and is not an up-to-date resource. So, the resources that I listed, the NCCN, UpToDate, the Leukemia & Lymphoma Society, I should mention.
A very important resource that has up-to-date information, and they have even phone numbers for patients and their families to call to get connected with the proper people in a particular city, so that is a really important resource. But I’d be really, really cautious about what you find on the internet because things are changing so fast in this field. There’s a lot of outdated and misinformation on the internet.
Ross:
Well, then there’s outright scams. One of the things you mentioned before we went on is be cautious if someone’s asking you to put money upfront, or if it’s a nonmedical facility. What are some things that people should watch out for?
Dr. Pollyea:
Yeah. So, one of the things that is so important in our area is clinical trials and participating in clinical trials. Patients who opt to do this and receive experimental therapies can sometimes get the treatment of the future, get a drug that’s not currently available through the FDA, but may have a lot of promise.
And this is the way that we fight this disease. We’ve recently had an onslaught of approvals for AML and that’s because the patients being willing to participate in sanctioned clinical trials. So, participating in a sanctioned clinical trial is crucial, and it’s always a recommendation of all leukemia doctors.
When you participate in a conventional clinical trial, you’re asked to sign a consent form that explains what you’re doing and why. There is a confirmation that this has been vetted by an institution’s regulatory board that is prioritizing the safety and well-being of you, the patient. This has been approved by the FDA as a clinical trial. Nobody would ever ask you to pay money. That’s not ethical to participate in a clinical trial. Insurance covers whatever standard of care. And the clinical trial covers anything that isn’t.
So, if you find yourself in a situation where you’re not being asked to sign a consent form, where a clinical trial has not been reviewed by a regulatory board, where your doctor is not a leukemia specialist, where the FDA has not sanctioned the treatment, all of those are alarm signs.
Because there are people out there that are preying on patients in a desperate situation, a very difficult time in their life, and giving them sort of false hope and leading them down paths that are not legitimate.
One easy thing to do to sorta check to see if a clinical trial is legitimate is to go onto clinicaltrials.gov.
This is a resource set up by our national healthcare system that now feeds in every legitimate clinical trial from all over the world, needs to be registered on clinicaltrials.gov. So, if you can’t find your clinical trial on clinicaltrials.gov, I would have a lot skepticism and caution about that.
Ross:
Like what advice do you have for people when they’re first diagnosed? What are the first things they should try to do?
Dr. Pollyea:
Yeah. I mean, that reaction is totally normal and natural. I mean, many times these people are perfectly healthy or have been perfectly healthy, and this news is a complete shock.
And so, it is normal and appropriate to have some period of grieving for the healthy life that you are losing. But I would also, while giving yourself that time to grieve, first, draw on your support system, your family, your friends. Allow them to help you. Accept that assistance that they have. And to be optimistic because we are getting so much better at treating this disease.
I had mentioned before, there has been an onslaught of approvals for drugs in this area the likes of which hasn’t been seen in decades. We have new tools and weapons in our arsenal that we couldn’t have dreamed of even a few years ago.
We in our community are very excited and hopeful about the future and we hope that that will translate ultimately to patients, but being depressed or being down, being scared, all of that is normal.
All of that is expected. Anyone would feel like that. Allowing yourself to have those feelings and emotions is important, as long as it doesn’t get in the way of doing what you need to do to fight this disease.
Ross:
It sounds like you’re hopeful about new treatments for the disease. How about a cure? What’s the science? What’s the medical science say about that? Are we getting any closer to that?
Dr. Pollyea:
We are getting closer to curing this in more cases. So, like I mentioned before, as bad as this is, we can already cure some subsets of patients. There’s one type of Acute Myeloid Leukemia called Acute Promyelocytic Leukemia, APL. It’s an uncommon form of AML, less than 10 percent.
But we can cure close to 99 percent of people with APL. And APL, 15 years ago, was universally the worst form of acute leukemia to get. So, that dramatic 180 that we’ve seen in APL, we are hoping to translate into other forms of AML.
Some other forms of AML have cure rates as high as 50 percent, 60 percent, 70 percent in the right setting. Sometimes we can cure patients with a stem cell transplant fairly reliably. So, we are very, very hopeful about our ability to continue to make progress and cure more and more and more of these patients. That’s the future that we see.
Ross:
Dr. Pollyea, thank you so much. And thank you so much for ending on such a positive note. We really appreciate it. And thank you for joining us for this program today.
To learn more about AML and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Ross Reynolds. Thanks for joining us.
https://powerfulpatients.org/pen/wp-content/uploads/Pollyea-1.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2019-06-05 15:38:122022-09-08 12:04:58Fact or Fiction? AML Causes & Symptoms
When it comes to online Acute Myeloid Leukemia (AML) resources, how do you separate fact from fiction? During this LIVE webinar, Dr. Jessica Altman, Director of Acute Leukemia Program at the Robert H. Lurie Comprehensive Cancer Center, will share her expertise on treatments and side effects associated with AML.
Watch online on Monday, July 1 at3 PM Eastern (2PM Central, 12PM Pacific) for a 30-minute virtual webinar. Send questions in advance to question@powerfulpatients.org.
Fact or Fiction? AMLis brought to you by the Patient Empowerment Network. We thank Astellas Pharma US, Inc., Celgene Corporation and Daiichi Sankyo for their support.
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https://powerfulpatients.org/pen/wp-content/uploads/AML-May-15-1-1.png450810PEN Editorial Staffhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngPEN Editorial Staff2019-05-28 15:36:112019-06-07 14:16:44Fact or Fiction? AML Treatment and Side Effects
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