...and now for the sort of questions to consider asking your doctor for you case:
1) How many previous aplastic anemia patients has she treated? What were the treatments? Does she have experience in transplants, or making the decision when to bring a patient into transplant. Does she work directly with any Centers of Excellence (e.g. NIH, The Cleveland Clinic, Fred Hutchinson) when treating aplastic anemia patients?
2) A broad discussion of treatment options. I'd ask her 'off the cuff' type questions on the '5 points of potential disagreement' above such as "how do you feel about eltrombopag, would you recommend trying to get into a clinical trail with ATG/CsA/Eltrombopag?". "What is your view on complementary prophylactic drug treatement?". "What will be the test procedure for monitoring my Cyclosporine trough?" The point here isn't so much what her opinions are, but that she
has opinions. If she looks dumbfounded, if she hasn't heard of eltrombopag or doesn't know what a cyclosporine trough is, that is a huge sign she is absolutely not qualified to treat aplastic anemia, and if she is a good doctor and decent person, her next step will be to refer you to someone who is qualified.
3) I agree with Steve that "looks like" aplastic anemia as she said is in no way good enough. Aplastic anemia is a "diagnosis of exclusion". In other words it is properly diagnosed when the blood counts are broadly speaking all low (pancytopenia), the marrow is hypcellular,
and there are no cytogenetic or other abnormalities. Regarding your marrow, there are some specific questions to ask there:
a) What percentage is my marrow cellularity? (i.e. how hypocellular is it for my age)
b) Has the possibility of MDS reasonably been ruled out with cytogenetic testing of my marrow?
c) Has the possibility of PNH reasonably ruled been out via FISH testing of my marrow?
d) Is the blast count in my marrow within normal range.
.....also, be sure to ask for the full results of your bone marrow biopsy, which should contain all of the above. But first I'd ask her the specific questions above to be sure she's on the ball.
4) At what point would you recommend we "pull the trigger" and start treatment? Considering you are not now in the hospital (I assume), you are doing much better than many of us at diagnosis. Perhaps you only have a mild or moderate case of aplastic anemia. That would be good. But as a very helpful person from AAMDS once told me, there is a frustration for mild to moderate aplastic anemia, in that there really aren't proven treatments for that. I believe in such a case, often it is just "watch and wait", and perhaps the condition will stay stable, perhaps it'll resolve itself, perhaps it will worsen.
5) By the point above I mean specifically the blood counts - ANC, Hgb, Plt. What are those at for you now? I assume above transfusion levels?
6) Do my absolute lymphocyte count and reticulocyte count confer a good prognosis for ATG treatment? Again, if she doesn't know significance of this question, she has no business treating aplastic anemia and needs to refer you elsewhere. Specifically, NIH has found a strong correlation between a high lymphocyte count, a high reticulocyte count, and a good response to ATG. This is because your lymphocytes are the class of white blood cells which contain the rogue cells presumed to be causing the immune-mediated attack which is the root of aplastic anemia. If there are a lot of them (or at least a normal amount, you can check this on your CBC), then the ATG has a target to zero in on and kill. And your reticulocytes are the baby blood cells. If you have a normal amount of them, even if you are profoundly anemic, it suggests your marrow is still functional enough to respond to the anemia. Try not to get down if either of these are low though. I had a retic count of 0 at diagnosis, yet I still responded well. The initial point here is to make sure the doctor is right to treat your condition.
7) What are your procedures for handling the risk of analphylactic shock and oother risks associated with ATG treatment? You had asked about how ATG/CsA is administered. The ATG will be probably a week or so in the hospital, with the first four days getting the infusion (once each day). The infusion is set to run 4 hours a day, but if your body objects to that (my body did), they can slow that down considerably, so long as each of the 4 infusions are administered with a given 24-hour period. In short, ATG can be challenging, or you could sale through it. Part of the mystery of this thing.
8) What are the steps you take to prevent serum sickness post-ATG? Administration of a steroid, probably prednisone on a very high dose for a week or two, and then slowly tapered, should definitely be big part of her answer here. In case you hadn't heard about the serum sickness, it a very common but unfortunately often very unpleasant delayed reaction (allergic?) to the ATG. I got it, and it wasn't fun, but it passed.
9) How often will you be checking on me post-treatment? Under what conditions should I get myself to your office or an emergency room in between our appointments? Typically I believe she'd have you come in about 3 times a week at first to monitor your counts. But also, especially if you have a very low ANC (~500 or less), she should tell you to contact them immediately at any sign of infection, or with a fever > 100.5 F.
....this is probably very overwhelming, it was for me. I am sorry to see anyone have to go through this. But with aplastic anemia, even when the prognosis seems bad, in a way the prognosis is good on the long term. This is because modern medicine (not just any modern medicine, but those with expertise in aplastic anemia specifically) is improving greatly at keeping even those with very severe aplastic anemia alive for extended periods, and more promising treatments are being discovered. And even when all else fails and your marrow is hopelessly terminally empty or diseased, that is paradoxically when the greatest hope for a true and permanent cure really kicks in, because hematologists are getting better and better at giving you a new lease on life through transplant - be that a sibling, match-unrelated donor, partially matched donor, cord blood, or some combination thereof. Survival rates are rising and graft versus host incidence is falling.
One more thing, AAMDS suggests if you are to read one article on the current "gold standard" for treating aplastic anemia, read this one:
http://www.bloodjournal.org/content/...o-checked=true