Monday, May 10, 2010 - Noon
Mazen Hanna, MD
Staff Cardiologist, Tomsich Family Department of Cardiovascular Medicine
Amyloidosis is a rare disease that is due to amyloid protein build up in the organs. The cause is unknown and is thought to result from many factors. Treatments are available to help manage symptoms and even control the production of amyloid protein. Since it is a disease that affects multiple organs/systems in the body, it requires much expertise and multidisciplinary approach. Dr. Mazen Hanna, Cardiologist and Dr. Frederic Reu, Oncologist answer questions about amyloidosis.
Cleveland_Clinic_Host: Welcome to our Online Health Chat with Drs. Mazen Hanna and Frederic Reu. We are thrilled to have them here today for this chat. Thank you for joining us Drs Hanna and Reu. Let’s begin with the questions.
Speaker_-_Dr__Mazen_Hanna: I am happy to be here today
Dr__Frederic_Reu: I am also happy to be here to answer questions.
General Amyloidosis Questions
muriel: Concerning ILE 122 Since this type of cardiac amyloidosis affects approximately 4 - 5 per cent of African American males at the age range of 50-65, how can we get the word out to the world? This would get amyloidosis out of the world of rare diseases and having an African American President and more African American's in government could possibly help us to spread awareness and raise research money towards a cure for all of the amyloidoses.
Speaker_-_Dr__Mazen_Hanna: We could try things such as Amyloid Awareness month - and also there are amyloid support groups that can also hold more public education about this. You make a good point - we need to raise awareness.
ga_peach: Approximately how many cases of AL Amyloidosis do you treat annually at the Cleveland Clinic?
Dr__Frederic_Reu: We see 40 - 50 new cases and treat 100 - 120 different individual cases yearly at Cleveland Clinic.
sarah123: My dad lives far away in Nebraska and was diagnosed with amyloidosis, They want to put him on chemo but I am nervous for him and I don’t know his doctors. If he came to the Cleveland Clinic, how long would he need to stay for treatment? Is that realistic to be treated at Cleveland Clinic for amyloidosis or should I just go with the doc out there even though I do not have faith in him?
Dr__Frederic_Reu: The most important question to answer is does he have light chain amyloidosis. If not - he should not have chemo. We can arrange a consultation and periodic follow ups there while your dad gets his treatment locally.
carik: My dad has AMYLOIDOSIS he hasn’t had any chemo. he’s 70 years old can you tell me what stages there are for this disease?
Dr__Frederic_Reu: It is not defined in stages but rather in number of organs involved and severity of organ involvement.
eclipsefarm_2: With treatment will blood vessel fragility resolve?
Dr__Frederic_Reu: It can - but just like all organs involved, the main goal is to prevent progression. The chance of achieving organ improvement is between 30 and maximally 50 percent.
jim: I have a close friend who had amyloidosis about 5 years ago. He went through chemo and had a bone marrow transplant. The disease damaged his kidneys. Yesterday he was complaining that he was short of breath after we took a walk. He also said he could feel palpitations. Could this be his amyloidosis? Is amyloidosis cured with the chemo and bone marrow transplant or is it just kept at bay to come back at a later time?
Speaker_-_Dr__Mazen_Hanna: First of all, these symptoms could represent heart involvement but would have to be evaluated.
Dr__Frederic_Reu: Chemo and bone marrow transplant does not cure amyloidosis but it can lead to long lasting remissions (measured in many years).
Emily: You state "the chance of achieving organ improvement is between 30 and maximally 50 percent" is this statistic affected by or independent of the pretreatment extent of organ damage?
Dr__Frederic_Reu: Yes - if there is scarring this cannot heal.
smith_v: Can you talk about diet and amyloidosis – is there anything someone should avoid if they have the condition?
Speaker_-_Dr__Mazen_Hanna: No - however if you have congestive heart failure from amyloid - you should restrict the sodium in your diet.
SecureHarbor: Do you correspond with the experts in Boston, such as Dr. Falk Cardiologist who has 20 plus years dealing with amyloidosis? Dr. Lever at the CC introduced us to him after he diagnosed my husband with a heart biopsy in 2008
Speaker_-_Dr__Mazen_Hanna: Yes we do communicate with experts at all facilities when indicated
Amyloidosis affecting organs: liver, kidney, lungs
ATTR-Journey: I obtained a liver transplant for my ATTR, alanine-60 variant, within about a year of real symptom onset. I have no peripheral neuropathy but it seems obvious I probably have autonomic neuropathy (GI symptoms, orthostatic hypotension). Cardiac involvement had started (ventricle wall slightly thicker and EF of 55%). I received my LT within the past year
Speaker_-_Dr__Mazen_Hanna: Unfortunately there are times when the progression of the amyloid can occur despite the liver transplant. Continual follow up of the heart would be necessary.
ATTR-Journey: Are persons who have had LT and whose heart condition degrades toward heart failure a potential candidate for a heart transplant (in other words is it possible to have a heart transplant after a liver transplant if heart failure started to occur)?
Speaker_-_Dr__Mazen_Hanna: Yes and this would be evaluated on a case by case basis.
jenniferK: My cousin was just diagnosed with Pulmonary Nodular Amyloidosis - can you talk about that? what is it and what does it mean?
Dr__Frederic_Reu: Pulmonary amyloidosis occurs most frequently with light chain amyloidosis. It only requires treatment if it causes symptoms. The treatment is directed against the clonal plasma cells.
QB786: My Father is a 76yr old and was diagnosed with lung cancer and amyloidosis. He has been on chemo for a year and I wonder if he is getting the right treatment. He was pretty active before all of this and he does not feel very good – very tired. In and out of heart failure. What should we do?
Dr__Frederic_Reu: We would need to see him - his fatigue may be due to the lung cancer and the treatment for lung cancer.
skarab: If one has AL amyloidosis manifesting in the kidneys, if a kidney transplant would be an option, wouldn't the Amy eventually recur in the new kidney?
Dr__Frederic_Reu: That is a concern and we only consider kidneys for transplant if the amyloidosis is in a complete hematological remission.
footballfan12:I was diagnosed with primary pulmonary amyloidosis and I am now taking a combination of chemotherapy and steroids. I am feeling a lot better but wonder what the success rate is with chemotherapy and amyloidosis
Dr__Frederic_Reu: Your physician should have an idea of how well the drugs are working based on blood and urine studies. If the light chains can be suppressed long term, the risk of progression in the lungs is low.
flrlady: My husband was just told that he has Amyloidosis He was diagnosed with Carcinoid Cancer last August and they took 18" of his small bowel. He has been losing weight for along time. Last they found Amyloidosis in his stomach. Do you think he needs to have biopsy in other places is this normal for this to progress this way – I do really not understand the diagnosis – is cancer the same as amyloidosis in the stomach?
Dr__Frederic_Reu: The need for biopsy is determined by symptoms. The type of amyloid needs to be determined. Cancer is not the same as amyloid but some cancers can cause amyloid. Cancers of the antibody producing cells cause light chain amyloidosis. With other cancers general inflammation can lead to inflammatory amyloid, which is not cancer.
Amyloidosis and Heart
jonathan: Can you talk about amyloidosis and heart transplant? If you have heart failure are you a candidate for heart failure?
Speaker_-_Dr__Mazen_Hanna: It depends on the degree of heart failure and the extent of other organ dysfunction. We have done heart transplants on patients with transthyretin amyloidosis. We have established a protocol for patients with light chain amyloidosis.
SecureHarbor: My husband has primary cardiac amyloidosis; received stem cell transplant hd melaphan 05.2008 with 65% response and has continued 16 months on dex and revlimid as his hear worsens; what are the options with possibly a heart pump as done in 2004 at the university of maryland
Speaker_-_Dr__Mazen_Hanna: We have never used an LVAD for someone with amyloidosis at the Cleveland Clinic - but we would be happy to evaluate him.
SecureHarbor: has anyone at any facility that you are aware; done a LVAD for cardiac amyloid; even if the amyloid is not in complete response?
Speaker_-_Dr__Mazen_Hanna: There have been isolated case reports. But -we have not done any at the Cleveland Clinic.
890ngbj: My dad has been having shortness of breath and had an MRI – they are now going to put him through a heart biopsy to rule out amyloidosis? Is that likely enlarged heart = amyloidosis? Isn’t that really rare? Anyway – can you tell me more about the heart biopsy – what does it involve and is that the only way to diagnose this condition?
Speaker_-_Dr__Mazen_Hanna: Amyloidosis typically does not cause an enlarged heart rather a thickened heart of normal chamber size. There are certain MRI characteristics that highly suggest amyloidosis.
Speaker_-_Dr__Mazen_Hanna: A heart biopsy is done by people with special training and is overall a very safe procedure with a low complication rate.
Speaker_-_Dr__Mazen_Hanna: Confirmation of cardiac amyloid as well as the type of amyloid can be confirmed by biopsy.
mickey45: What is the life expectancy if you have cardiac amyloidosis?
Speaker_-_Dr__Mazen_Hanna: It depends on the type of amyloidosis and the extent of organ involvement.
kellicari: Is amyloidosis the same as cardiomyopathy and if not, how do they tell the difference between cardiomyopathy and amyloidosis?
Speaker_-_Dr__Mazen_Hanna: Cardiomyopathy is a general term for any abnormality in the heart muscle. Amyloidosis causes a type of cardiomyopathy called restrictive cardiomyopathy where the heart is stiff and cannot fill well.
Speaker_-_Dr__Mazen_Hanna: In order to determine if amyloid is the cause of a cardiomyopathy one ultimately needs a heart biopsy.
sueS: Question for Dr. Mazen Hanna who is having a live Chat Monday May 10 about AMYLOIDOSIS. As facilitator of OH, MI, Philly and Phoenix AMYLOIDOSIS SUPPORT GROUPS, I e-mailed Dr. Hanna's chat to those groups. Many with Familial Amyloidosis have asked if FAMY will be included in the chat? Thanks, Sue.
Dr__Frederic_Reu: Familial Amyloidosis questions will be included in this chat - please feel free to submit your questions.
SHHS: Hi - I'm a 66 year old male with ATTR Med30 and I'm finishing my two year clinical trial with BMC this coming August. I have not received a liver transplant. I do have cardiac involvement with an ef of 70%. Would I be a candidate for a heart/liver transplant?
Speaker_-_Dr__Mazen_Hanna: We would need to perform a formal evaluation as it would be difficult to say whether you would need both a heart and liver transplant vs. liver alone.
domino: Does the Cleveland Clinic have any clinical trials for Familial Amyloidosis patients?
Dr__Frederic_Reu: We do not at this time. You can search on http://www.clinicaltrials.gov/ to see what available trials are.
Dr__Frederic_Reu: There is study with FOLDRX for patients with age related and TTR mutated cardiac amyloidosis open in Chicago.
kristen: A cousin of mine about 56 years old passed away from amyloidosis. I would appreciate any information you may be able to provide on this subject. I am interested in knowing how far away a cure might be and if it is hereditary.
Speaker_-_Dr__Mazen_Hanna: There are some types of amyloidosis that are hereditary and some that are not. There is no cure at this time however early diagnosis can lead to effective treatment in certain cases.
Dr__Frederic_Reu: It would be important to find out if this was a genetic amyloidosis type. If so, genetic counseling of first degree relatives would be advised.
lusby: From Lusby: is there any way to slow the progression of neuropathy caused by familial amyloid?
Speaker_-_Dr__Mazen_Hanna: FOLDRX actually showed statistically significant slowing of progression of neuropathy from familial amyloidosis.
ATTR-Journey: I am being treated at the Jacksonville Mayo campus. Are the Mayo facilities inter-related as to knowledge and expertise at treating ATTR FA? Or, should one pick a specific campus such as in Cleveland?
Speaker_-_Dr__Mazen_Hanna: We cannot speak to the Mayo Campus. Ask your physician in Jacksonville.
pljk567: My brother died at the beginning of the year from amyloidosis. My wife wants me to get tested? Should I and if so – what tests should I take to see if I have it?
Speaker_-_Dr__Mazen_Hanna: It depends on what type of amyloidosis your brother had. If it is the hereditary type, then yes you should get tested for the mutation (that is a blood test). If it is the light chain type, then genetic testing would not be necessary.
Light Chain Amyloidosis
SecureHarbor: what is your protocol for light chain amyloidosis? I asked about the heart pump earlier? Did you receive that question?
Dr__Frederic_Reu: We want to see a good hematologic response with prediction of long term hematologic remission since the transplanted heart would otherwise be affected by amyloidosis again.
Emily: I have AL Amy, with renal involvement. My total cholesterol is about 300. Should I be taking a drug to control cholesterol?
Dr__Frederic_Reu: Yes - it may even help the amyloid related vascular disease.
Emily: I have AL Amy. Why do I get purpura around my eyes? What causes this symptom of AL Amy?
Dr__Frederic_Reu: Fragility of the blood vessels with subsequent bleed into the subcutaneous tissues.
sberry: for patients w/ AL and Light Chain MM how closely does the reduction of SFLC correlate to the level of amyloid production? If you can control the MM do you control the AL?
Dr__Frederic_Reu: This question has not been evaluated in a way that we can quantify amyloid deposition but a recent study showed that a 90% reduction in serum free light chains correlated with amyloid related outcomes.
mary_jm: Can you talk about stem cell therapy and the treatment of amyloidosis. I have been looking at the boards online and there was a woman whose mom was helped with stem cell therapy for her amyloidosis. Is that a current treatment or do you have to fit into a research program?
Dr__Frederic_Reu: High dose chemotherapy followed by autologous (your own stem cells) stem cell transplant is a standard treatment for light chain amyloidosis.
Dr__Frederic_Reu: Other types of stem cell therapy are experimental.
Emily: In ASCT, what protocol do you typically use for AL amyloidosis with good organ function?
Dr__Frederic_Reu: Induction with velcade and dexamethasone followed by high dose melphalan (200 mg/m2) prior to ASCT.
JWP43: What is the primary difference between Multiple Myeloma and Amyloidosis?
Dr__Frederic_Reu: Multiple myeloma causes 4 symptoms summarized by the word CRAB - C = hypercalcemia (high calcium in blood); R = renal dysfunction (kidney); A= anemia (low hemoglobin blood count); B = bone disease related to localized or generalized bone resorption.
Dr__Frederic_Reu: AL Amyloid is characterized by deposition of light chains in various organs. These light chains are produced by the same cells that can cause multiple myeloma.
sharon1: Have you treated anyone with Multiple Myeloma that also had Cardiac Amyloidosis? What is the treatment and I wonder if the treatment would affect the amyloidosis?
Dr__Frederic_Reu: The most promising treatment for both diseases uses velcade and dexamethasone plus or minus low doses of conventional chemotherapy. It is most potent for both types of diseases.
ga_peach: Which typically comes first… myeloma or amyloidosis?
Dr__Frederic_Reu: Can go either way.
eclipsefarm_2: I have MM and Amyloidosis and have had excellent results with Velcade and Dex.I do not have any organ involvement and am scheduled for a stem cell transplant. Is there any information how people respond with these newer drugs without having a transplant?
Dr__Frederic_Reu: First of all, you must have some organ involvement - otherwise amyloidosis would not be the diagnosis. If all organ functions are well, that is all the better.
Dr__Frederic_Reu: We do not have data on long term results with velcade and dexamethasone but if you tolerate the drugs without side effects, one reasonable option would be to collect stem cells but continue on therapy as long as it works and as long as you tolerate it.
ga_peach: Do you know the newer version of velcade will become available?
Dr__Frederic_Reu: The drug is called carfilzomib and we have participated in clinical studies that are now closed. The results are submitted to the FDA and I anticipate approval within one to two years.
ATTR-Journey: Are new drugs being developed to get rid of existing amyloid in AF patients who have had liver transplants?
Dr__Frederic_Reu: Yes - drugs are developed with the goal to prevent deposition and remove amyloid. Only FOLDRX is in clinical trial at this point in the United States.
Emily: Why does Velcade/dexamethasone cause bloating? What exactly is happening...ascites or perhaps stomach distention? I do have leg/foot edema as well.
Dr__Frederic_Reu: There can be multiple reasons. Velcade can cause autonomic neuropathy. The anti-nausea medication given before velcade can cause constipation leading to bloating.
Dr__Frederic_Reu: Dexamethasone can also cause bloating.
Dr__Frederic_Reu: They should not directly cause ascites but dexamethasone can cause fluid accumulation. If you are concerned about ascites, this should be evaluated by your physician.
ATTR-Journey: Is there any estimate when the FOLDRX study will be complete?
Speaker_-_Dr__Mazen_Hanna: This study is anticipated to be completed in December of 2012 - you can go to www.clinicaltrials.gov for more information on FOLDRX.
SecureHarbor: Do I understand you correctly: You are saying there is a clinical trial with drugs that will remove amyloid deposits?
Dr__Frederic_Reu: Yes - although FOLDRX primarily tries to prevent amyloid deposition there is home that it will help remove amyloid from tissues.
skarab: If one has Al Amy in the kidneys and is currently on dialysis, even though the kidneys are still functioning, would one be a candidate for the Foldrx in hopes of eliminating amyloid deposits in the kidneys?
Dr__Frederic_Reu: No FOLDRX has not been studied for light chain removal. The treatment should be directed at the clonal plasma cells. There is currently no medication in clinical trials that aims at removing light chain amyloid deposits.
Emily: Many thanks for you time in answering my questions. It is so helpful to get additional information and relieves the stress of this disease. Thank you.
Speaker_-_Dr__Mazen_Hanna: Thank you for your comments.
Cleveland_Clinic_Host: I'm sorry to say that our time with Drs. Mazen Hanna and Frederic Reu are now over. Thank you again Drs. for taking the time to answer our questions about Amyloidosis.
Speaker_-_Dr__Mazen_Hanna: Thanks for your questions today. We would be happy to see you for an appointment.
Dr__Frederic_Reu: We hope we could be of some help today.
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