[KMac]

So I've said a lot of broad philosophical stuff about seeking aplastic anemia treatment, now I'll get to the specifics, starting with areas where to my understanding the best experts may disagree, and then list the questions I'd ask your doctor were I in your shoes. Here are the areas where I think there may be disagreement (hence for you and your intuition to help decide):

1) Sometimes, instead of ATG/CsA, it is recommended the patient go immediately into transplant. Generally, if you are under 40 and have a sibling donor, I believe transplant is the preferred option, but boundaries between which way to go are not solid rock walls in any way. I for example was over 40 at diagnosis, yet my siblings were tested, and had one of them matched I would have went the transplant route.

2) There is a promising new treatment for aplastic anemia which again NIH has discovered called eltrombopag. The FDA has approved it as a salvage treatment (i.e. if the first treatment does not work and transplant is not an option). However, NIH is currently conducting trials including eltrombopag along with ATG/CsA. They cannot provide specifics as the trial is not complete, but in presentations they state that they are very excited about the results to date, and eltrombopag may revolutionize aplastic anemia treatment. Unlike ATG/CsA which just supresses your immune system (but often that is enough in itself), eltrombopag also actually stimulates the marrow to grow and produce healthy blood cells. So perhaps you would have the opportunity to decide if you'd like to take part in this trial.

3) Here's a controversial one - Johns Hopkins offers a treatment called High Dose Cyclophosmamide. To my understanding, it's a massive chemotheraphy dose to wipe out nearly all your bone marrow, leaving just the most basic pluripotent stem-cells (i.e. those from which all other blood cells can develop). The idea is to 'reboot' your hematologic system, with whatever defect which caused the aplastic anemia being destroyed in the process. Anecdotally there are cases of excellent sustained recovery, but NIH is strongly convinced this isn't a good treatment path based on their analysis, due to high toxicity, fatality, and sometimes poor recovery. This is a prime example of where the best experts, who I trust are well-meaning and qualified, still profoundly disagree (I personally cast my vote with the NIH treatment path).

4) Prophylactic treatments, when to use them, and which ones? Specifically antivirals, antibiotics, antifungals, and a drug called neupogen which can temporarily boost your white blood count to prevent infection. It is very common for hematologists to use some or all of the above, for some time period, to protect the patient from life-threatening complications due to low white blood count, while they are waiting for the true treatment to work.

5) When do you "pull the trigger"? By this I mean whatever the treatment, at what point do you apply it, and at what point do you stop applying it or taper it? How low does the hemoglobin and platelets get before you transfuse? How low does the ANC (neutrophils, a type of white blood) get before you give neupogen? How long do you keep giving it? Do you give prophylactic antivirals, antibiotics, & antifungals (and if so which ones), or do you only apply these in case of infection? What is a proper cyclosporine trough level for a given patient? I've heard vigorous debate on some of these questions. As my doctor said to me, everything can come at a cost, as these treatments often have side effects.