Treating Acute Promyelocytic Leukemia (AML-M3) Recorded on: February 7, 2014 Lloyd E. Damon, M.D. Director of Hematology and Blood and Marrow Transplantation UCSF Medical Center Nancy White Gamble APL Survivor Please remember the opinions expressed on Patient Power are not necessarily the views of UCSF Medical Center, its medical staff or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That s how you ll get care that s most appropriate for you. What is acute promyelocytic leukemia, APL? We'll find out what you should know about it, particularly if you live in the Latino community. It's all coming up next on Patient Power. Hello and welcome to Patient Power. I'm Andrew Schorr. This program is sponsored by UCSF Medical Center. There are many cancers and among the cancers are leukemias, and among the cancers are chronic leukemias and also acute leukemias. And one rare acute leukemia is called APL, or acute promyelocytic leukemia. We're here to talk about that with someone who has lived it, lived through treatment and is here to help others. Joining us is Nancy White Gamble from the Napa Valley of California, north of San Francisco. We're going to meet her doctor in just a second. But, Nancy, you have survived this. It was with terrible bleeding, unexplained symptoms that went on for a while before you got the diagnosis of something you never heard of, right? That's correct. And how bad was the bleeding when it happened? I know you'd had a cough for a while, you'd had fatigue, you'd had blood spots on your leg. And then I know you were going home from the doctor, general practitioner after you had had a blood test, and what happened while you were driving in that car that day? Well, it's a day I'll never forget. It changed my life. After being seen for about a month for what
was diagnosed as a viral syndrome of some sort, the fatigue, loss of appetite, cough, just sore throat, chronic, was getting worse. And the day that I went to my HMO doctor, I wanted him to do further blood tests to see how I was really doing, because I felt that this viral syndrome that I had been seen for weeks was really not a viral syndrome. I felt in my heart there was something much more. And I had been brushing my teeth the morning of that office visit, and I had thrown a blood clot, and I thought that was very unusual. So when I spoke with him and said, you know, we need to do further blood tests to see how I'm really doing. I said, I have a feeling this is more than viral syndrome. Could I possibly even have cancer? And he reassured me not to worry about that. I left after doing the blood work, was driving home to where you know my regular outside primary doctor was. I got ahold of him because while driving my mouth was filling up with blood. The gums were bleeding profusely. As I drove to his office, and he started packing my mouth with gauze he got the blood work and said, Nancy, you are going to have to be admitted to the hospital immediately. At that point, no one was aware of what it was going to be really diagnosed as. Okay. Let me just skip ahead a little. I know you went to the emergency room. Yes. Your husband took you carefully. The emergency room doctor told you we think you have leukemia. Yeah. And then you started hearing when you met with an oncologist and had a bone marrow biopsy so they could look further, the results came back. It came back with this rare leukemia, APL, which I'm sure you'd never heard of, right? I had never heard of it. It was all just a nightmare that, in fact, I was even hearing the word leukemia, let alone this acute promyelocytic leukemia. Of course. And so that's something very rare, and appropriately your doctor there in Napa Valley said you really need to go to a center that specializes in this sort of acute care, and that brought you to UCSF Medical Center. And you had many, many weeks of treatment. And I understand because your condition was kind of diagnosed later it was touch-and-go for a while, wasn't it? There was one point, I know I read, that you thought you might not survive. Well, yes. About two and a half weeks into the treatment, I developed some brain hemorrhages on both sides of my brain. And at that point, I knew enough about medicine that this was very serious, and I actually was praying to God that he wouldn't take me and I needed a miracle. It was waiting for the moment to happen, and I didn't know when that was going to be, but I knew I 2
was close. Well, thank goodness it didn't. And so you were there many weeks. You went through a lot of treatment, and you had really many treatments after that, even as an outpatient. But we're five years later now, and you are a survivor. And I understand you have partly Latino heritage, and one of the things you learned is that there is a higher likelihood of this rare cancer in Latinos. That's absolutely true. For some unknown reason, the Latinos are about nine times more likely to get acute promyelocytic leukemia, and trying to bring the awareness to the Latino population that certain symptoms such as the ones that I had, flu-like symptoms, loss of appetite, chronic kind of sore throat, feeling under the weather like something is, you know, a bad flu, a bad cold, but more than that developing unusual spots on my legs such as kind of a dark freckle pigmentation, bruising on my stomach and then, of course, the bleeding of the gums. And you All may be signs. Right. And a leukemia that's different from others let's meet the doctor who really helped steer your care, who I know you feel I think maybe played a key role in saving your life, and that's Dr. Lloyd Damon. Dr. Damon is the director of adult hematology and blood and marrow transplantation at the UCSF Medical Center. Dr. Damon, I know it makes you feel great that you can help, your team can, someone who was so sick, and now here five years later is here feeling pretty good and able to talk about it. Yes. It's a great honor, and it's really gratifying to take care of such ill patients with a life-threatening illness, turn that around and possibly invoke cure. Right. So, Dr. Damon, APL, as I mentioned, there are chronic leukemias and acute ones. Some people are familiar with AML. Is this a type of AML? Is that what it is? Yes. It's a subtype of AML that represents about roughly 5 to 6 percent of the total. And I said in talking with Nancy that there's some predisposition that's higher in the Latino community. How much higher is it? There have been a number of studies, but one from southern California indicates about nine times greater chance that APL will be in a Latino individual as opposed to a non-latino. 3
Do we know why? Do not know why. The presumption is there's some inherited factor that predisposes people of Latino ancestry, but that really has not been established clearly. We don't really know if there are environmental factors or other factors involved. Now, when APL is appropriately diagnosed, is it survivable? What's the prognosis for someone with APL? Well, there are two parts of that question. The first is surviving the first month and getting into remission and then what happens afterwards. Compared to other forms of AML, there's a higher risk of bleeding and, in particular, bleeding into the brain in the first month of treatment. And it's understood why that is, based on the type of leukemia, and modern therapies have really improved that and made serious life-threatening bleeding less likely. The good news is and this is really great news is once you're in remission the cure rate is over 90 percent. It's really excellent. Wow. Okay. So Nancy had these unusual symptoms for quite a while. And why is it difficult for doctors in the community she was going to doctors near her who were not cancer doctors but just general doctors why is it difficult to be recognized? And what's a message maybe we could get out so that this could be recognized earlier because from what I hear you're saying that has a lot to do with survival? Right. Early recognition and diagnosis is really critical. And one of the problems is the symptoms are often a bit nonspecific and often mistaken for things like a viral illness or something else. And what's really important is not just seeing the patient and hearing what's happening but getting a blood count and then paying attention to the blood count. And when physicians see a blood count in which there's a very high white count, one of the things they need to think about is acute leukemia. And what's very different in acute promyelocytic leukemia is that the classical white count is actually low, not normal but actually low as opposed to being high in the other AMLs. And so many physicians don't really recognize that as being a feature of acute leukemia. Wow. Okay. So you mention that it's important to get early diagnosis and treatment, and you use this term "modern treatment." I would imagine that's something that at a major cancer center like at UCSF you deal with all the time, you're very familiar with, maybe even were involved certainly in clinical trials for modern therapy. So tell me, it would seem like when somebody is diagnosed with a rare condition like this they do need to get to a center such as yours where you have experience with it. 4
That's true. There are only a handful of centers that routinely treat acute leukemia. It's a rare disease, actually, and so experience means a lot. And then we have an uncommon subtype of a rare disease, so that adds to the experiential level of community hospitals to deal with that, which is not great. And then what's very interesting about acute promyelocytic leukemia we've learned we cannot treat it the same way as other AMLs, and, in fact chemotherapy, although it's the backbone, the therapy needs to be supplemented with a vitamin A derivative called all-trans retinoic acid. And now, believe it or not, part of the recipe is also arsenic-based therapy. So we're dealing with chemotherapy, a vitamin derivative and arsenic as the treatment for this with curative intent. And yeah, curative intent, and you said that there are many people who may well be cured if they can have modern therapy and have it early enough. So that's a good news story. Absolutely. And again it's one of the more curable cancers in humans right now one of the most curable. It's just like childhood ALL. We're really at that level of dealing with this disease. Now, one of the issues with all-trans retinoic acid and arsenic is they have very peculiar side effects that one needs to be aware of and dealing with prospectively to try and prevent or deal with in real time because some of the side effects can actually in and of themselves be life-threatening, and this is where the experience comes in. So centers that treat a lot of acute leukemia should be the centers that are treating patients with acute promyelocytic leukemia. Right. And, Nancy, I know you had your very intensive treatment at UCSF, and then with a doctor who had been trained by Dr. Damon you were able to be closer to home but monitored carefully. And I think as you had treatment daily for a while you had EKGs every day, right? I did. That was protocol for the arsenic treatment. There was a level of arsenic that they wanted to monitor and make sure it wasn't doing any kind of damage to my heart. So every day for 10 weeks, I would have an EKG to make sure my QTc level was not elevated. If it was, that meant that they would hold the arsenic for that day and then retest me to see if I would proceed on with it. Well, of course, we're all delighted it's worked out. So, Nancy, you decided to give back. You and your husband I know have helped establish an endowment at UCSF, so there can be knowledge developed and training for physicians related to this. Why did you decide to do that? Well, it was very clear to me surviving the ordeal of almost dying from the brain hemorrhage and getting through the treatment successfully that my purpose was to give back and to support Dr. Damon's leadership in his doctors in the future, and this endowment focuses on the training of future doctors, fellows in blood-related cancers. And I was able to meet my first fellow under the Nancy White Gamble Hematologic Malignancies 5
Endowment Fund just about a month ago, and I was very proud to see someone following in the footsteps of Dr. Damon and giving hope to patients like me when they needed it most that they could survive this treatment and be cured of this illness as I have been. Wow. That's great. Now, if someone else wanted to help contribute, is there a way they can do that? Yes, there is. It is my endowment that is called the Nancy White Gamble Hematologic Malignancies Endowment Fund at UCSF hematologic malignancies training program. They can gift by check to UCSF Foundation Fund, Number S, as in Sam, 0561, PO Box 45339, San Francisco, California 94145-0339. Okay. Well, I hope there are other people who can support that. So, Nancy, I know you're very grateful. Is there anything you want to say to Dr. Damon publicly here about the care that he and his team gave you? Well, the care that I received at UCSF by him saved my life. It was a team effort my faith, God, Dr. Damon, my husband, my mother. Dr. Damon gave me the hope. When I asked him point blank, Am I going to die? And at that point when I needed that hope to continue on and not give up, he said, No, you are not going to die. And hearing it from him because I had put my faith in him from the very beginning when I walked in those doors, I knew if he said I wasn't going to die that I needed to put every effort into living, and five years later I'm cured and I'm alive. All right, Nancy, well said. And, Dr. Damon, I know that does your heart good. It's what medicine is all about, isn't it? Absolutely. And Nancy's endowment fund is one way she's trying to make a contribution, which is incredibly important to education, but I also have to point out she comes and allows me to interview her in front of second-year medical students to tell her story and make teaching points about acute leukemia and acute promyelocytic leukemia in particular. And so physicians who will not be hematologists are benefitting from her story and her give-back to the community. So it's a tremendous thing. I'm so proud of her and really support all of her efforts in this regard. Wow. Thank you. Thank you, Dr. Damon. Well, this story of course for our listeners is about understanding a rare illness, understanding if some of these symptoms develop, particularly if you are of Latino heritage, you get it checked out. And it's a question for your doctor to answer, Could it be APL? And if it could be, you want to get 6
to a center such as UCSF to get the best care and get it early, so you have the chance, as Dr. Damon said, of really being part of a happy story in cancer where you can be cured. Thank you so much for being with us, Nancy, all the best to you. And Dr. Lloyd Damon, thank you for all you do, and hopefully there are younger doctors who are coming up who will be better informed now through all your efforts and that APL can continue to be a positive story and that you can conquer other cancers as well, all the best to you. Thank you for being with us. Thank you, Andrew. Yeah, thank you very much. Okay. Thank you to our listeners. And I'm Andrew Schorr. Remember, knowledge can be the best medicine of all. Please remember the opinions expressed on Patient Power are not necessarily the views of UCSF Medical Center, its medical staff or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That s how you ll get care that s most appropriate for you. 7