Transcript for:
Low Risk vs. High Risk MDS

this is a pre-recorded presentation so the presenter will not be taking any questions however all questions asked during the live presentation along with answers are included at the end of this presentation to learn more about our upcoming patient and family conferences in your area please visit AAA MDS org slash conferences to view other recorded presentations or to register for other live online learning events please visit a MDS org slash learn welcome to our live webinar titled low risk vs. high risk MDS thank you for joining us today my name is Lee Clark and I'll be moderating the presentation today as we get started I would like to thank the generous support of Celgene Takeda and Jaz pharmaceuticals as well as our patients families and caregivers for providing support for the webinar program today today's presenter is dr. Luskin she received her MD from the University of Pennsylvania in 2019 completed residency training and internal medicine at Brigham and women women's Hospital followed by a fellowship and the hematology oncology at the University of Pennsylvania she joined Dana Farber as a member of the adult leukemia program in 2016 with that said it is my pleasure to welcome dr. Luskin thank you for that kind introduction and the opportunity to speak to this group today speaking the patient's of families is by far my favorite audience and I hope that this webinar will be helpful for for the patients and families listed in understanding the diagnosis of my oldest prop my love dysplastic syndrome or MDS my task is to help understand the difference between low risk and high risk MDS and really understand risk in general in this disease and that's what I will hopefully leave with you you all with under better understanding with today in order to talk about risk in my oldest classic syndromes or MDS it's important that we have a good foundation understanding the diagnosis in general and that is where I'll begin today in my presentation I frequently find that patients go to their doctor for a problem of low blood counts and there are tools that they have a diagnosis called myelodysplastic syndrome which is a mouthful even difficult for leukemia doctor to say out loud most patients and families say I've never even heard of that and I have no idea what that means for me the technical definition for myelodysplastic syndrome is on this slide it's often described as a chronic myeloid lineage hematologic neoplasm characterized by ineffective clonal hematopoiesis and risked a progression to AML or acute myeloid leukemia the diagnosis characterized by site apenas and the presence of an MDS defining abnormality all of that is a very confusing set of words I like to break down into plain English what is mildest plastic syndrome my oldest plastic syndromes are cancers of a particular part of the blood system the bone marrow is where blood is made and when we talk about blood cancers and bone marrow cancers we're talking about one thing there are different types of blood and bone marrow cancers that affect different parts of the blood system this particular diagnosis affects something called the myeloid lineage particular type of blood cells main characteristic that all patients with myelodysplastic syndrome have are low blood counts which we often call Saito penis this is because the bone marrow or the blood factory doesn't produce blood effectively and so patients have one or more low blood counts we talk about blood counts we refer to three main types of cells the blood that circulates in our body is actually made up of three different types of cells one are the red blood cells these carry oxygen the other another one is called are called platelets these are a cell that helps our blood clot and then we have white blood cells and the main most common white blood cell is a blood cell called a neutrophil and these are the most important blood cells that help us fight infection most people are evaluated for and then diagnosed with mildest classic syndrome when they have one or more low blood counts or Saito piñas the most common is the presence of low red cells which is called anemia but patients may have low blood cells and one or more and including all three however low blood counts can be from many different causes and therefore evaluation is undertaken to find out the cause of the low blood counts to understand why the body is not making those blood cells very well and when the bone marrow biopsies so to make the diagnosis the patient generally undergoes a bone marrow biopsy to look at the bone marrow factory to find out why it is not producing blood cells and certain features in that bone marrow biopsy can tell the doctor that you have a mildest plastic syndrome a cancer of this particular part of the bone marrow one sign of the bone marrow cancer is something called dysplasia this means that the cells look abnormal under the microscope another another piece of evidence that can show that there is mildest plastic syndrome is that the chromosomes or the the where the genes are carried in the xcel are abnormal or there are too many blasts and these are the blasts are the most immature cells in the bone marrow and there were when there are too many this is a sign that there they have become cancerous and these are the aggressive and image for cancer cells so in summary a diagnosis of my oldest classic syndrome is made when the patient has low blood counts and one or more abnormalities in a bone marrow to suggest that the m.d. low blood counts are related to MDS who gets MDS MDS is primarily a diagnosis as made in older adults the median age at diagnosis is in the mid 70s and it's relatively rare with about somewhere in the range of 10 to 40,000 cases diagnosed each year however we're not sure if that number is accurate because many patients are likely undiagnosed or not fully evaluated and these cases are not always reported to national registries because it is not always considered a traditional cancer age is a major risk factor for this diagnosis and most people have no other risk factor other than their age so there are some some exposures that can lead to a higher risk of developing MDS the most common which is being exposed to chemotherapy or radiation for a prior diagnosis other causes of MDS or additional risk factors include certain environmental toxins and there are some cases where mild is plastic syndrome it runs in families or is related to a prior diagnosis of aplastic anemia once the diagnosis of MDS is given the natural next question is what does it mean to me this is particularly important because many patients when they present have no symptoms often a blood count has been checked for another reason I often meet patients who had a blood count checked for prior to a surgery or prior or for another reason and the low blood counts were entirely asymptomatic some patients have had mild have had some symptoms that led to the evaluation those symptoms are typically related to the low blood counts so remember red cells are one type of blood cell or those are low patients are in car we call anemic and that may result in fatigue or shortness of breath low platelets which is called thrombocytopenia may be associated with easy bruising or bleeding and low white blood cells particularly low nutritionals which is often referred to as neutropenia may lead to easy predisposition to infections more likely to get an infection so in terms of symptoms many patients of MDS have no symptoms when they're diagnosed and may have no symptoms for a long period of time other patients do have symptoms at diagnosis or develop them later in the course of their disease there's differences at the time of presentation so a presentation when a patient is first diagnosed in terms of how the patient feels and then for every person the what what's going to happen in the future may be different and so after understanding what MDS means at the time of diagnosis another common question is what is going to happen in the future what am I looking forward to in my life how does it MDS going to affect affect me when I make a diagnosis of MDS I tell my patients that we have to consider the risk for two major problems or complications in the future I remember this by remembering the ABCs the first thing I want to consider is how likely is the MDS to progress to a different more aggressive type of blood cancer called acute myeloid leukemia the second thing I have to consider is where the patient's blood counts our diagnosis and how likely they are to express progressive bone marrow bone marrow failure meaning the bone marrow cannot make blood counts and so having lower red cells white cells and platelets and the C is to remind us that this in all patients is technically a cancer though in some patients is relatively asymptomatic and requires no treatment none other patients requires more traditional cancer treatment Oh in summary MDS and AZ which are technically the mildest plastic syndromes because everybody's MDS or everybody syndrome is different in regard to the symptoms they have when they're diagnosed with regard to how abnormal their blood counts and which blood counts are abnormal and the third that is different between each patient is the risk of the disease worsening of progression that a B in the ABCs in terms of risk of developing leukemia and risk of the bone marrow working the bone marrow progressively failing making fewer white blood cells red blood cells and platelets in order to provide guidance about that risk how how is the MDS likely to affect me the patient and the physician must understand both the disease the MDS as well as the patient who has the disease so we're going to talk more about how we predict the risk of the complications I've mentioned and then in making treatment recommendations the patient and the doctor together need to consider the disease risk the context of the patient as a whole what the what the other medical problems are at play what other treatments they need for other medical conditions and what their goals of their treatment are now we get to the portion of the presentation where we talk more specifically about understanding MDS risk and so the goal is to understand the patient's disease so that estimates of complet of the risk of complications can be provided we don't stage or predict risk and MDS like we do with other types of cancers many patients are asked what stage do you have stage 1 2 3 4 and those are stages that are used in other types of cancers that occur in organs of the body that are just in one place for instance lung or the colon and blood cancer is blood is everywhere so we have to develop different risk or staging systems so in the MDS community we've developed risk scores that combine disease features of the MDS there are several different risk scores but almost all of them includes three major features one is the number of types of cell counts that are low white blood cell red cells and platelets and how low are those blood counts whether or not there are increased blasts or the immature or aggressive cells and what the chromosomes or the genetic features of the MDS are these scores again help predict how long yeah how long the person can live with the MDS and the risk of developing the more aggressive blood cancer or acute leukemia the risk scores that are used most commonly or and are often reviewed by patients or something called the IPSS or the IPS s R stands for international prognostic scoring system there are other similar scores that are used and based on institution and practitioner preference your doctor may use a different scoring system that is very similar to the ones discussed here those scoring systems can scoring systems can include other factors such as age whether the patient needs transfusions and other medical information so we use those three features the blood counts the presence or absence of extra blasts and the genetic features and calculate a score for each by combining the features of each of those categories and with that can assign a patient a risk stratification whether they are low intermediate or high based on their total scoring and these scores help us predict how the disease is likely to behave whether it's likely to be changed very slowly and not change significantly over years or whether the disease is likely to change and become impact the patient's health in the near future I always emphasize that these scores are merely tools they help us give our best educated guess on how a patient's disease will progress but any one person's disease may behave differently than predicted and that there is no guarantees for anywhere any patient about how their disease will behave there is a MDS based on whether it is low risk or high risk are very different diseases low risk MDS does not change for many months to many can up to up to many years whereas high risk MDS is expected to become aggressive within the order of weeks to a couple of months and behaves much more similarly to the diagnosis of acute myeloid leukemia high-risk MDS the average life expectancies in months to a couple of years where low earthkam Diaz is able you are able to live with for many many years without treatments many patients be evaluated for MDS also are getting more sophisticated testing called next-generation sequencing where the doctors are looking for other mutate other genetic abnormalities in their disease and using that to help provide guidance on how the disease will behave these sequencing platforms are very new and we are still learning how to use the information provided and they are not formally in many of the risk stratification systems your doctor may tell you that the information provided affects their prediction one way or another if you've gotten information about mutations or you've seen the results but not discuss them encourage you to talk to your doctor about how it affects their assessment of your disease this is just an example of efforts by researchers to understand how these different mutations might affect how disease at any particular risk behaves a patient with MDS is not just MDS every person comes with a medical history and and other features and so when I evaluate a patient with MDS I want to know everything else there is to know about them I want to know what other medical problems they have and it's very common with patients with MDS to have other medical problems because the average age of diagnosis is in the 60s and 70s some of these diagnoses are completely unrelated to the MDS but there are also situations where the MDS has led to other problems becoming worse such as lung or heart problems and then we also want to know how strong the patient is we use a term called frailty or performance status these are terms that we use just to understand how much how much strength of the patient have how likely are they be to be able to tolerate treatment when I approached the part of my visit with my patients where I talk about their treatment I talked to them about understanding them and there are characteristics their medical problems their strengths what they're able to do each day and I combined that with my understanding of their MDS what the risk of their MDS is and using those two things combined to make the best treatment recommendation and really individualize that treatment I think about treatment I think that all pages of MDS should receive careful evaluation for symptoms and they should receive supportive care for symptoms and consequences of low blood counts with the goal of helping the patient feel better and make sure that the MDS affects quality of life as little as possible and then there is a consideration of whether or not there should be additional treatment things like chemotherapy and bone marrow transplant which we'll talk about in the next several slides these came up these approaches are typically considered for patients who are younger and very healthy and in whom there's a high risk disease the disease is likely to affect the patient's health and symptoms in the near future the goal of these treatments are to impact the natural history of the disease and in some cases we try to cure the MDS focusing on supportive care we often talk about how to manage the low blood counts the most common low blood count is red low red cells or anemia and many patients with MDS received something called erythropoietin Withrow poises stimulating agent or an ESA these are injections that provide a supplement to the body's natural hormone called erythropoietin that normally tells the bone marrow to make red blood cells this can help the bone marrow work slightly better and prevent or reduce the need for red blood cell transfusions these injections work best for people who are not needing transfusions already or needing very infrequent transfusions they are not generally effective enough to prevent a patient from the new transfusions if there are wordy requiring them frequently some people ask me about the risk of getting too many transfusions one risk of too many transfusions is extra iron in the body it is not well known whether taking medicines to help the body get rid of extra iron is very helpful or not but this may be something that your doctor discusses with you there's also a drug called lenalidomide that or the brand name is Revlimid that is useful for a particular type of MDS with a chromosome abnormality called del 5q remember chromosome was the term I use for the genetic abnormality of the MDS this particular subtype of MDS responds very well to this drug many patients who take this drug in that particular subtype have a very beneficial response to this drug with an improvement in their hemoglobin on average of about five points and this drug can work for several several years in this group of patients it can be used in other patients with low-risk MDS but is much less effective and not used quite as commonly but it's an option from for some patients and may be discussed with you it's less common but many patients of MDS also have low white blood cells or neutrophils and low platelets or which is called Samba cytokine iya patients with low platelets there is a predisposition there's a tendency for easy bruising and bleeding and this can be managed with transfusions there is our medicines that can help the body when you form blood clots these met these medicines called antivirals can help the body hold on to the blood clots they make for longer and there is research currently happening about the use of injections to help the bone marrow make more platelets these injections are have been developed for other blood conditions and there may be a benefit in MDS as well and your doctor may speak to you about that low white blood cells or infection fighting cells generally attempt to monitor the patient carefully and treat any infections aggressively should they develop this is not a type of blood cell that can be transfused and so we have to manage infections in other ways le-let's and red cells can receive there are transfusions for those types of blood cells but there are not we do not have the ability typically to transfuse white blood cells deserves some comment that we are increasingly understanding that MDS makes patients feel unwell fatigue is common symptom that's even above just the low blood counts patients who have only mild anemia or who have anemia that is treated often still tell us that they feel less well than they did before their MDS we're currently trying to understand that area of wreaths at area of this disease better that we can learn how to address it right now we focus on recommending that we attend to all the other parts of Health and that affects quality of life and energy things like sleep and exercise nutrition mental health imagine all the other blood other medical problems that might affect how a patient feels when I refer to disease modifying therapies now we're talking about about treatments that try and change the the MDS try and stop it from progressing rather than just treating the symptoms in general we recommend disease modifying therapy for patients who have high risks so they have been felt by their doctor to have high risk disease often by one of the scoring systems I mentioned or for another reason sometimes that's because of the clinical trajectory meaning that they've seen change over time and they can see that things are changing faster than they would have predicted or perhaps one of the mutations in the newer sequencing has made the doctors too concerned about the patient being high risk so there are different ways to be assigned high risk that's important mostly to understand why your doctor thinks that you're high risk and if there is a concern that the disease may be caused progress or cause symptoms in near future we often recommend disease modifying therapy in patients who we think are strong enough to tolerate the treatment and not induce unnecessary side effects why don't we use these treatments for low-risk patients even in healthy and strong low risk patients it's because we have to always weigh the risk of benef the risk of side effects with the possibility of benefit and in low-risk MDS many lower CMDs often doesn't cause any any symptoms and doesn't have any risk for many many years we don't want to add side effects to a patient's life before they before they need it common and the really the only approved chemotherapy for MDS is a class of agent called hypo methylating agents and there are two in this drug class one is called ease aside a Dean or VISAA and the other is called this side of being our dakichan these are chemotherapy drugs and they're given with a goal of improving blood counts and preventing the development of acute myeloid leukemia these drugs are not curative they don't cure MDS and their goal though is to slow down the progression of the disease these drugs when they work have to be continued indefinitely to maintain the response that it's achieved when the drug has stopped the response is generally lost in a couple of weeks to a couple of months how likely is the treatment to work we don't have a test a diagnosis to tell any one person whether or not they are going to respond or not and so we offer this treatment to anybody who meets the our criteria of being high-risk and being strong enough for the treatment I quote my patients and approximately 50% chance of having improvement in the blood counts so some improvement in their disease and about a quarter of patients have we call a complete remission meaning their blood counts improve and when we look in the bone marrow the bone marrow the Blood Factory looks completely normal as well that doesn't mean the MDS has gone completely it's just been reduced to a low enough level that we cannot see it with our tools we'll be at the blood and the bone marrow underneath the microscope these drugs a society and decided being these chemotherapy drugs have been shown to improve survival how long patients can live in high-risk patients and this can in the patients who respond the can respond improve survival for months to several years in general these chemotherapy drugs are well tolerated the main side effects are making the blood counts worse before they actually become better the goal of the treatment is to make blood counts better but can lower the blood counts before the drugs work and ultimately make them better so that can feel counterintuitive to patients as they're just starting therapy that they're starting therapy for low blood counts and the chemotherapy can make their their blood counts worse before they ultimately improve after cellular cycles that's important to know that the response can take several cycles and the cycles each lasts for approximately one month and so we when barking out a treatment course I encourage my patients to stick with it for several months before deciding along with me whether it's beneficial to continue there's no age cutoff for who can get these chemotherapy drugs it's more important to know how strong the patient is rather than age and I've given this drug to patients of all ages who have high-risk disease and they're otherwise generally healthy and and strong you might often hear ask asked which drug is better the age acidity and decided me there are very similar medicines they have never been compared directly to each other and likely that will never be done and most most clinicians in our field feel that they're relatively similar and so when I see patients coming from other providers I tell them that I feel comfortable with either one that has been selected by their doctor chemotherapy is given for about one week either side of Dean standard treatment to seven days and it can be given IV or by an injection to the skin the side of beam is usually given for five days through the IV so in some what cases is given for longer ten days most important thing first up from for monitoring after treatment is that the main side effect is worsening of blood counts and so it's important to be proactive about monitoring blood counts after being treated as the blood counts are expected to decrease and the patient may need transfusions even if they did require them before and we have to be monitored very carefully for infections because the immune system may be reduced to an even lower level the three minutes given out patient has given every approximately four weeks finally high-risk patients these an intervention called transplant is often considered a low genetic means from somebody else stem cell transplant means replacing the bone marrow with stem cells from another person and building up a new bone marrow and blood system this procedure al genetic metaphoric stem cell transplant is the only way to cure MDS and this is considered in young patients who have high-risk MDS and we know that this this treatment can cure MDS but it is also a high risk treatment and so we know that it is beneficial only in those who are high-risk because otherwise the risk of the treatment is higher than the risk of the disease historically this was not an option for most patients with MDS because this is a disease of older adults and their 60s and 70s and we used to only do transplants with we call mildly of conditioning meaning very strong chemotherapy that would not be tolerated but over the last several decades advancements have made in doing transplants at our reduced intensity meaning gentler in terms of the chemotherapy that is used to allow us to treat more patients with MDS with this curative treatment we're increasingly understanding that aids itself shouldn't be a barrier to transplant and it's important that all patients who are who are who are healthy with not very many medical problems who are up into their 70s be considered for transplant and at least offered a consultation to find out if it's the right treatment for them so we are seeing and increasingly encouraging stem cell transplant referrals for adults of MDS who are otherwise healthy this is a slide showing the number of transplants for instance in patients who are 70 and older by by indication and the MDS line is in in blue you can see that in the last ten years the transplants being done for older adults with MDS is increasing as this intervention is becoming better recognized as a therapy for a subgroup of patients important if you think this applies to you that you ask your doctor if it's something that should be considered and if you're not at the transplant center consider asking for a referral to the nearest transplant center in your area and finally it's important to recognize the role of clinical trials we need better therapies for MDS we need more treatments than the ones I mentioned and we need better therapies and potentially therapies that could be appropriate for patients earlier in their disease so consider finding out if there are clinical trials at your Center I don't know if it's right for you as the only way for us to improve treatments for for current and future patients is to understand the disease better and try new therapies that are being developed by our scientists and researchers in summary if you or your loved one has been diagnosed with a mild as plastic syndrome important that you understand your MDS which may be different than the MDS in somebody else that's you've encountered every MDS is different and understanding your characteristics and risks is important it's also important that your physician to understand you your other other medical conditions strong you are what are you doing on a daily basis and also what you're hoping for what you're willing to - what you're willing to undertake in terms of accomplishing certain goals and so treatment should be selective between you and your doctor based on your disease risk your personal characteristics and what what you want you want you and your family are hoping to get with your treatment and as you've defined the treatment that you're going to pursue it's always important that you understand what your goals are are you doing a treatment to make your symptoms better because you are hoping this will help you live longer with your condition or you try and secure your disease it's important that everybody be on the same page about what a particular treatment recommendation is going to accomplish understand what the plan is for monitoring after treatments particularly if new treatments might affect your blood counts or symptoms how you're going to reach your doctor urgently or emergently what things can be done for for supportive care how to managing symptoms of the disease or the treatments ask questions ask about clinical trials this is about your health and in the more information you have the better you are able to navigate things that are new for you I think everybody that I work with here at dana-farber who have helped me learn a lot about MDS and take care of my patients with MDS and certainly want to thank my patients who as I've taken care of them have taught me a lot about what it's like to live with MDS and how I can better treat them and future patients and patients that I meet through webinars like this thank you very much I look forward to a few minutes of answering questions great thank you so much doctor in Luskin we've had some questions that have come in our first question is when should low risk MDS patients actively begin treatment that's a very good question because I focused a lot about my risk assessment in this talk for patients who are newly diagnosed but after a patient with newly diagnosed low-risk MDS what happens over time and over or in the future so for a low-risk MDS is really important to establish a monitoring plan with your physician to monitor your blood counts look for changes look for any sign of the MDS is changing and at that point if there's a sign of the MDS is becoming more aggressive to consider treatment at that point all right thank you so much a question comes from a caregiver hit his father was diagnosed with low risk MDS and November of 18 he man has dropped to a five patient has been transfused on with 20 bags from November through February last April he was prescribed full minified which he takes two tablets a day before bed and receives injections of ebo this has helped with raising the hemoglobin to a nine point five the question is will the hemoglobin always go up and down even though the patient is consuming medication daily and is receiving Ipoh o I believe the the questioners the caregiver father was on I think the little mind is what you said which is very similar to the drug level of mine that I mentioned in my slide it's in the same group of same type of drug so he's getting treatment to help the bone marrow trying to make blood more on its own but it sounds like the patients still requiring transfusions to have enough blood on a daily basis and so if transfusions are still needed usually transfusions are temporary fix and so how long the patient's blood will remain high as our transfusion is different for different people and so how often the patient will need transfusions is different so the the blood counts will be variable and ongoing monitoring is important additionally for patients who respond to drugs like little amide and Ipoh injections those responses tend to to last only a couple of months to a year or two and they eventually lose effectiveness and that's when patients often if they weren't before require transfusions and once transfusions are quite frequent frequent it may not make sense to continue those medications if they are no longer working exactly when those medications should be stopped is really not something that I can answer over this webinar but something to discuss with your doctor thank you very much can you speak about trisomy 8 and MDS and is there a higher risk for developing AML I would refer the questioners for either themselves or their loved one to talk about their their risk profile comprehensively with their doctor the risk is based on a combination of things like the low blood counts whether there are extra blasts and the genetics the trisomy 8 in particular is in the is not a particularly high risk for AML but you have to take it in context of all the other features thank you this patient has been on Aaron s since 2005 on twice a month and they know it will work until it doesn't work but is there a scientific guess as to how long it normally works on hemoglobin stays around ten six weeks without it the patient is usually down between a seven and eight let's say that the response of the of the writer is already much longer than it typically works so I hope it continues to work for much much longer right thank you the next question is the term pre leukemia has stood in this patient's away and many others who were veterans with MDS is cancer now the recognized term a number of years ago when this patient was diagnosed with cancer but the oncologists opinion was that MDS is a cancer not a malignant cancer this is a problem I actually tried to address in my consult because there are a lot of terms that get used by different providers and our intent as a community is not to confuse our patients some of the terms are older than others but get used in different settings I don't personally like to use the term pre leukemia because it doesn't emphasize the fact that MDS itself is a condition that needs to be assessed carefully and managed even though it can evolve into leukemia which is where the term comes from the malignancy is another term for cancer and so both MDS and acute leukemia are both blood cancers what's important to understanding you know the word cancer is a very general term and just like I tell patients there are different types of skin cancers that are have different implications different blood cancers some of them are slow growing and don't affect the patient's condition a day to day and others are symptomatic and require urgent therapy so what's important to know is what your particular cancer means for you malignancy as I mentioned was and it's another word for cancer and so no not really I think non-malignant cancer was the term that was used I think is can be a bit confusing thank you causes that causes appear to be more genetic rather than environmental is this the case we need to know more about why most most people develop MDS as mentioned there are some patients MDS we have a known risk prior chemotherapy or radiation for another cancer for instance but for those groups of patients where they're they don't have any particular occupational or personal exposures we are still trying to understand how much is due to genetics and how much is due to our environment I think we're increasingly recognizing that all cancers not just blood cancers are affected by other aspects of our health and our environment so I'm hoping by the time I did this webinar in a few years I can answer that question better thank you this patient was diagnosed at 50 with five - - has been taking Revlimid and is curious about the long-term side effects of the drug and also by being diagnosed sore early in life does this increase their chances of developing AML or other related MDS issues so there are we believe no drug is without side effects but in general the Revlimid the lenalidomide we feel is safe to take as as long as it's beneficial to the to the patient and hopefully the patient has had success if they are still taking the drug in terms of being diagnosed at a young age and risk for complications and AML the the risk is not different with age the difference is that at age 50 we hope that there will be many years the patient is young and so as we are around four more years there is more of a chance for the disease to change and become more aggressive so I encourage her to be monitored closely by her by her by her team and any signs of any changes think about other other therapies for her disease you know our next question is why is blood oxygen saturation not included and the decision about when to give or to offer a blood cell transfusion it turns out that patients even when they're very anemic typically have a normal blood oxygen saturation and so if the patient has a low blood oxygen saturation they should be other causes of loped of low oxygen should be you know carefully considered in terms of other medical problems such as heart and lung and its although the blood carries oxygen the oxygen saturation is not directly related to the to the red cell number thank you can the concept of MRD Mehta mom residual disease be defined for mdf it is still a area of research and contention is not yet used in our day-to-day practice as we develop better therapies for MDS we hope to get better we call remission meaning shrink the the disease to lower levels and then we'll need better tools to measure that it is not as developed for MD Athens's for other blood cancers because it is very difficult for us to separate out the cancer cells from normal cells when we take samples so there's a area of investigation thank you can you give your thoughts on blue powder cept I think as a community we're excited for any new drug that's available for this condition because unfortunately the list that I went through in this presentation was shorts I think this powder SEP will have a benefit for a small subset of low risk patients but it is has been studied in a small group of patients with low risk and a particular feature called rinse it or Oblast which is a particular feature of MDS and so I think for those for whom is appropriate I'll be looking forward to having that as an additional tool to offer but won't help the majority of patients with MDS thank you are you aware of any clinical trials for decadent to be given in pill form there where there are there's definitely interest in developing oral therapies for MDS including oral type of methylation agents whether there's a particular trial in your area I would encourage you to ask your doctor to help you look into that um I'm going to piece together this question I hope I'm going to answer it right on the question is regarding I'll toggle is transplant for MDS is that something that is considered so the answer to that is no the term mythologist transplant means giving yourself your own your own stem cells back to August transplants are used in individuals like lymphoma on multiple myeloma and it's used in those conditions they get very high dose chemotherapy to treat their disease and you get your own stem cells back to this help the bone marrow recover in this case the problem is with the MDS really is a disease that starts in the stem cells so given your own stem cells back wouldn't help solve the problem which is why we don't do we call auto or ecologist transplants for MDS thank you as time goes on will patients need additional bone marrow biopsies or or will the oncologists have everything they need to know just the regular blood tests the very good question and the two things I will say it's important to have a bone marrow biopsy at diagnosis often patients will be presumed to have MDS based on low blood counts but no bone marrow biopsy has been done and I encourage a full evaluation at diagnosis to allow full characterization of the disease and risk stratification as we've talked about I do recommend Ament follow-up bone marrow biopsies I don't recommend a specific schedule I typically recommend patients be monitored by bloodwork every at an interval that's appropriate for their disease generally more frequently if there have higher risk disease and less frequently with low-risk disease and if there is a persistent or a change in blood counts that is significant that is at the right time to do another bone marrow biopsy to see if there have been changes in the bone marrow thank you we'll all high risk MDS patients develop AML no not all high-risk MDS patients are at risk for both development of AML and the other way that the high-risk high risk MDS can progress is continuing work continued worsening of blood counts but these these risk tools are are just tools and there are sometimes low-risk patients whose disease disease behaves more aggressively in quote high-risk patients who have very stable disease for a period of a long period of time so nothing is absolute and these tools are best ability they provide us with the best tools we have to try and understand what's going to happen but there's a lot in medicine we still don't understand thank you this question comes from a child of a mom or their mom had MDS and would like to know is there a connection and should this person be concerned about developing MDS in most cases we don't in most cases MDS is not familial may not run not running in families so we are starting to recognize that in some cases there is a predisposition or a tendency and families for patients to develop MDS and AML at a higher frequency so without knowing if you're particularly if you have more than one family member in your in your in your close family that has MDS or AML I encourage you to speak to your doctor about a theme genetic counseling and if the the writer has concerns I would encourage them to talk to their doctor about speaking that kind of evaluation thank you the next question is this patient have had HIV for 32 years was diagnosed at 57 with mild MDS requiring Aaron s every 2 weeks for the past two years are you seeing more long-term survivors with HIV developing MDS that's a very good question I'm not / - not familiar specifically with the connection between MDS and HIV but I think that's something that would be important to look into as we have more long-term survivors thank you and our last question what is the risk of AML developing for a low-risk MDS patient that has deletion 5q and deletion 20 q [Music] the risk of developing a ill health IML for that for that scenario for low-risk a for a low-risk MDS is and is in depends on the timeframe you look at but generally considered in the single digits well thank you so much dr. Lufkin for the wonderful and very informative presentation today on behalf of the aplastic anemia and MDS international foundation I'd like to thank everyone for joining us today making us your resource of choice on bone marrow failure diseases if we weren't able to answer your question or you'd like to send a question for us to get answered please do so by emailing us at help h ELP at AAA MDS org so we're able to get your question answered as a reminder after I'm done speaking a post event survey will appear on your screen please to take time to complete the survey again thank you for joining us remember learning is hope this concludes today's program