[Matthew42]

From the research I've done, I can say that Danazol does wonders for some people with aplastic anemia and pure red cell aplasia. In fact, responders sometimes have "complete" responses. And roughly 40% of responders never relapse, while others will have need to be on and off the drug for the rest of their lives.

I did read where taking too high of a dose can be problematic. I think 300-400 mg/day is about right for most people with AA and PRCA. It also takes about 3-9 months for to achieve an initial response, although there have been studies done showing that it can take 1 year or more. Interestingly, a few responses occurred at almost 2 years in people AA with very short telomeres. I could be wrong, but it's generally older people who would have short telomeres.

But, of course, Danazol doesn't work for everyone: it seems to work best in those with short telomeres, as said above (accelerated aging genetics affecting bone marrow, lungs liver, etc.), or those who are female who haven't responded to IST therapy (it has worked very well on males but not nearly as often). I'm just repeating what I've read in studies.

My mother's doctor is against all treatment like Danazol except the standard h-ATG/cyclosporine/Promacta. When that doesn't work, it's all Campath - or nothing. Rabbit ATG was not advised for my mother because of a high chance of bone marrow cytogenetic changes, along with the fact that Rabbit-ATG is not likely to work.

It is very uncommon, but ATG can take two years to kick in. In fact, Dr. Young included 2 years for his end-mark for response times because it does happen rarely. There was a former member on here who believed she had responded to ATG two years later. Or, the disease may have "burnt out", which is not a nonsensical idea. Some doctors are of the opinion that AA can burn out in a person who continues to have it for years without incident.

AA is not an autoimmune disease like lupus and MS -- it has a unique pathology. With other autoimmune diseases, you don't hear of immune-mediated burnout, but you do with AA. Again, the disease is highly mysterious, leaving you putting your hands up in the air like a madman.

Lastly, every one is different with this disease. Some can go 30-40 years without a problem, while others relapse frequently. Some take a a few years to find the right treatment (after a few failed ones) and then do rather well afterwards. Who knows?