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Alpha-1 is a genetic disease of the liver and lungs. Welcome to Questions and Answers, asked by members of the Alpha Support Mailing Lists (Lung and Liver), and responded to by Dr. Jack Lieberman, Dr. Sandy Sandhaus and Sandy Brandley, Executive Director of the Alpha-1 Association and other members replying from the Alpha-1 Mailing Lists.

General Questions:

1. What’s meant by half life and what happens if get “too much” prolastin?
2. What percentage of MZ Alphas have lung problems, how many have liver problems, how many have both?
3. Debby asks: If your liver function tests are always high, will you get Cirrhosis?
4. Debby asks: How often do Alphas with COPD have bronchitis? Or is it ongoingall the time?
5. Robin asks: I was recently told that my hemoglobin was a little high at 18. My resting O2 sats are usually around 94% so, at what point does the increased red blood cells become problematic?
6. Jay asks: I have a particular question with respect to Phenotyping. Mine came up at 4.5 with a Pi Z. Does that mean ZZ or Null Z?
7. Dotty wrote: Which brings me to my question: Has anyone else received a Financial Assistance Application from BD/Express Scripts?
8. Debbie asks: What percentage of MZ Alphas have lung problems, how many have liver problems, how many have both?
9. How about ZZs?
10. If your liver function tests are always high, will you get cirrhosis?
11. Debbie asks: How often do Alphas with COPD have bronchitis? Or is it ongoing all the time?
12. Debbie asks:  I have heard specialists say differing things about the need for protein in your diet. One said eat a very low protein diet, others said to eat a high protein diet. How will I know which is right?

1. What’s meant by half life and what happens if get “too much” prolastin?
   Dr. Jack answers:
             The half life of a substance injected into the human body is the time in which half of the injected substance is removed or lost from the body.
             Regarding too much AAT: The AAT level normally rises in the blood in response to infections, cancer, pregnancy and/or estrogenic medication. High levels of AAT don’t last very long since, once it combines with elastase, it is quickly eliminated from the body. The extremely high level  resulting from a Prolastin infusion is transient, therefore requiring repeat infusions at weekly intervals to maintain a protective level.
             Thus high levels of AAT that might be detected in people with no AAT deficiency, but with one of the illnesses like cancer, in effect, is similar to the high sedimentation rate of blood and usually indicates the presence of an illness or pregnancy, etc. I know of no ill effect resulting “from” the high levels.
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2. What percentage of MZ Alphas have lung problems, how many have liver problems, how many have both?
   Dr. Jack answers:
             Only an infinitesimal number of MZs who don’t smoke get lung disease. If you do smoke heavily, your chances of getting lung disease are significant, but an actual percentage has never been determined. Getting liver disease is a possibility, but an actual percentage is also not known it is small. How about ZZs? Again actual percentages are not known for non-smokers. Smokers are about 100% for getting lung disease? Liver disease is about 15%.
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3. Debby asks: If your liver function tests are always high, will you get Cirrhosis?
   Dr. Jack answers:
             There must be a reason for your liver tests to “always be high.” One would need to analyze which tests are high. The fact that you have pain over your liver area is disturbing and is reason for you to have an intensive evaluation of your liver via liver scan and possibly even an eventual biopsy. This could be gall-bladder or liver disease. You should be seen by a Gastroenterologist interested in the liver.
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4. Debby asks: How often do Alphas with COPD have bronchitis? Or is it ongoingall the time?
   Dr. Jack answers:
             If an Alpha has frequent cough and sputum production, that person most likely has chronic bronchitis. About 2/3 of the Alphas with COPD have chronic bronchitis as well as emphysema.
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5. Robin asks: I was recently told that my hemoglobin was a little high at 18. However Prof. Lomas said I didn’t have polycythemia and, therefore, he didn’t need to get his leeches out :) My resting O2 sats are usually around 94% so, at what point does the increased red blood cells become problematic?
   Dr. Jack answers:
             Usually the hematocrit is the measurement that reveals when polycythemia is present. A value of greater than 50% is the indication of polycythemia.  A Hemoglobin of 18.3 is somewhat high (17.2 is the upper limit of normal), but with your O2 Sat of 94% you shouldn’t be polycythemic. Ask your doctor what your hematocrit value is to be sure.
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6. Jay asks: I have a particular question with respect to Phenotyping, etc. I FINALLY got my blood tested through Salt Lake City (after simply asking the phlebotomist to draw an extra tube of my own blood and mailing it myself and having the results sent doctor—since there was no medical necessity to do the test). Anyway, I got the results today. It said the reference range was 32.4 uM and that they do a double test and a phenotype on those less than 11 uM and/or less than 45% of the reference value. Mine came up at 4.5 with a Pi Z. Even though I have always known I was probably a “ZZ,” this test result just frightens me a little more than  before. In trying to determine the > percentage which a lot of folks discuss AAT levels in, am I  to divide 4.5 by 11 (=40.9%) or by 32.4 (=13.9%). For some reason, I was expecting to see Pi ZZ, not just Pi Z. Does that mean ZZ or Null Z? I know I could research and figure it out but I am more> than a little frustrated right now.
   Dr. Sandy answers:
             PiZ means you are ZZ  Your level is approximately 13.9% of normal. That’s pretty usual for a ZZ (or PiZ).
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7. Dotty wrote: Which brings me to my question: Has anyone else received a Financial Assistance Application from BD/Express Scripts? This really bothers me. I’m wondering if they have a legal right to request your salary and other personal information? I have never had to fill this out before and why should I, if I have insurance. I’m wondering if Bayer is trying to find out if I can afford the amount my insurance company refuses.
   Sandy Brandley answers:
             Yes, this is legal, in fact, it is a Medicare requirement that in order to have the co-pay waived an application confirming financial need is to be on file with the provider. It is illegal to waive the co-pay without substantiating financial need. There have been several lawsuits over this issue in the recent past and judgements with substantial fines have been levied.
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8. Debbie asks: What percentage of MZ Alphas have lung problems, how many have liver problems, how many have both?
   Dr Jack answers:
             Only an infinitessimal number of MZs who don’t smoke get lung disease. If you do smoke heavily, your chances of getting lung disease are significant, but an actual percentage has never been determined. Getting liver disease is a possibility, but an actual percentage is also not known; it is small.
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9. Question: How about ZZs?
   Dr Jack answers:
   Again actual percentages are not known for non-smokers. Smokers are about 100% for getting lung disease. Liver disease is about 15%.
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10. Question: If your liver function tests are always high, will you get cirrhosis?
    Dr. Jack answers:
              There must be a reason for your liver tests to “always be high.” One would need to analyze which tests are high. The fact that you have pain over your liver area is disturbing and is reason for you to have an intensive evaluation of your liver via liver scan and possibly even an eventual biopsy. This could be gall-bladder or liver disease. You should be seen by a Gastroenterologist interested in the liver.
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11. Debbie asks: How often do Alphas with COPD have bronchitis? Or is it ongoing all the time?
    Dr. Jack answers:
              If an Alpha has frequent cough and sputum production, that person most likely has chronic bronchitis. About 2/3 of the Alphas with COPD have chronic bronchitis as well as emphysema.
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12. Debbie asks:  I have heard specialists say differing things about the need for protein in your diet. One said eat a very low protein diet, others said to eat a high protein diet. How will I know which is right?
    Dr. Jack answers:
              Obviously moderation in everything you eat is the correct answer. Protein is a necessary component of one’s diet. Ignore any fad diet.
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Prolastin Questions:

Dr Jack states:
          The results of our Questionnaire Survey of ZZ patients indicates that most patients on this List take their Prolastin Infusions “weekly.” If taken every two weeks, one must be certain that double the weekly dose is prescribed. see http://www.alpha2alpha.org/ChestArticle.htm.

1. Debby asks: Bronchiolitis Obliterans Syndrome after (Heart) Lung Transplant.   Am I wrong in interpreting this as a possible case FOR continuing Prolastin even after transplant, something UAB has told me they see no need for?
2. Patricia asks: Is anyone keeping track of numbers on prolastin and the progression of the disease?
3. Patricia asks: Are there clear guidelines for when a doctor will prescribe prolastin for a patient and if so, what are they?
4. Patricia asks: Does anyone have any information on the number of Canadians on Prolastin and if they were effected by the shortages?
5. Patricia asks: If you start prolastin, when would your doctor recommend you stop or is it entirely a patient decision to go on or off it?
6. Bill in Va. asks: Since transplants DO get Prolastin infusions too, I take it I’d still want to retain my “Bayer Direct Registration Number” in an inactive file should I be prescribed to go back on infusions at a later date?
7. Joe asks: It’s my understanding that Bayer produces approximately 500 lots per month.  A lot is defined as an average person’s monthly requirement. Is this correct?  If this is incorrect, please give me the correct number?
8. Beth asks: Due to the possible Prolastin shortage this winter, many people are advocating “shorting” yourself 1 bottle per infusion to build up a “stockpile.” Since my dosage is only 3 bottles to begin with, this would be a significant drop. Do you feel it is advisable to take less product now (and hope to STAY healthy) in order to have some on hand for use during a possible health crisis?

Dieting

1. Debby asks: Bronchiolitis Obliterans Syndrome after (Heart) Lung Transplant.   Am I wrong in interpreting this as a possible case FOR continuing Prolastin even after transplant, something UAB has told me they see no need for?
   Dr. Jack answers:
             You are likely correct in your interpretation, but then Bronchiolitis Obliterans Syndrome should be more common in Alphas undergoing transplant than in non Alphas. I haven’t seen such data, though it must be available by now.
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2. Patricia asks: Is anyone keeping track of numbers on prolastin and the progression of the disease?
   Dr Jack answers:
             There was an NIH Register set up in the USA that kept track of Alpha patients, both on and off of Prolastin. Their report was published a year ago and showed some reduction in mortality and in the rate of loss of the FEV1 measurement.
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3. Patricia asks: Are there clear guidelines for when a doctor will prescribe prolastin for a patient and if so, what are they?
   Dr Jack answers:
             Definite guidelines were set up, including 1) any patients on Prolastin must have stopped smoking, 2) there must be definite evidence of lung dysfunction before being started on Prolastin, to avoid wasting the medication on someone who may never develop lung disease; usually an Alpha who has never smoked. 3) It was also suggested that those with terminal lung disease not be treated, but this “rule” is apparently not being followed rigidly if a severely ill patient requests “augmentation” therapy with Prolastin.
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4. Patricia asks: Does anyone have any information on the number of Canadians on Prolastin and if they were effected by the shortages?
   Dr Jack answers:
             In a recent survey of our Alpha-1 List members there were 3-5 from Canada. I don’t have any definite information about the number of Alphas in Canada, or their experience with Prolastin. I feel certain that others on this List will be able to provide further information on this point. I do not believe that the Canadian Government supports Prolastin therapy, but some Canadians do receive Prolastin on a private basis.
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5. Patricia asks: If you start prolastin, when would your doctor recommend you stop or is it entirely a patient decision to go on or off it?
   Dr Jack answers:
             Once you start Prolastin, you will be on it for the rest of your life. There may be other improved methods for augmenting your Alpha-1 levels in blood and/or lungs; thus, a different procedure may arise to reach the same goal.
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Bill in Va. asks: Since transplants DO get Prolastin infusions too, I take it I’d still want to retain my “Bayer Direct Registration Number” in an inactive file should I be prescribed to go back on infusions at a later date?
Cathy (AlphaNet Rep) replies:
          That’s what I would do. That way it would make things go quicker for you if you would decide to go back on.  Hope you get your lungs soon, Bill! Take care, Cathy
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6. Joe asks: It’s my understanding that Bayer produces approximately 500 lots per month.  A lot is defined as an average person’s monthly requirement. Is this correct?  If this is incorrect, please give me the correct number?
   Sandy Brantley answers:
             To help give a perspective of the products produced by plasma here are some statistics:   Out of 100 gallons of plasma there may be 120 grams of Prolastin (1 patient for 28 weeks), 1500 grams of IVIG (50 treatments), 800 vials of Albumin, and 90,000 units of Factor VIII. Therefore markets need to be found for all of the products not just the Prolastin and IVIG which are in short supply. I’m offering this only as a way to look at the issue of “supply and demand.”
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7. Beth asks: Due to the possible Prolastin shortage this winter, many people are advocating “shorting” yourself 1 bottle per infusion to build up a “stockpile.” Since my dosage is only 3 bottles to begin with, this would be a significant drop. Do you feel it is advisable to take less product now (and hope to STAY healthy) in order to have some on hand for use during a possible health crisis?
   Dr. Sandy answers:
             There is no right or wrong way to manage your Prolastin during a time of shortage. I’ll give my opinion and in a subsequent post, I will enter a copy of the official statement of the Medical and Scientific Advisory Committee of the Foundation  I would (recommend) saving a stockpile of 2 to 3 weeks worth of Prolastin for times of lung infection or worsened breathing. I’m not sure I’d make that stockpile by cutting back by a vial or two over many doses. My suggestion would be to identify some weeks that your feeling good enough to skip that week’s dose entirely, and put that aside. Take your usual dose for a couple more weeks, then do it again. My only rationale for this is that if every dose you take is low, it is possible that the Prolastin is having no effect whatsoever and so it would be the equivalent of taking no drug for a prolonged period. Since we don’t really know, in a scientific sense, what the “right” dose of Prolastin really is, you can see that this is all just speculation. Don’t forget to monitor the expiration dates of the vials you put aside and exchange them for vials with later expiration dates as they arrive. I’d like to mention that the shortage is likely to start in the Spring rather   than Winter, since it will take several weeks to months for a plant shut down to have an effect on supplies.
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Dieting:

1. Debby asks: I have heard specialists say differing things about the need for protein in your diet. One said eat a very low protein diet, others said to eat a high protein diet. How will I know which is right?
   Dr. Jack answers:
             Obviously moderation in everything you eat is the correct answer. Protein is a necessary component of one’s diet. Ignore any fad diet.

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Updated November 19, 2007