They are some in the benign hematology world who say that Danazol is underused in the treatment of severe aplastic anemia; in fact, women who have short telomeres with SAA are very much strong candidates for Danazol as a first-line treatment. It can be combined with an immunosuppressant, but not necessary for this subset of patients.
In one study, 70% of women enrolled achieved a complete response with Danazol for SAA after failing with treatment for ATG + cyclosporine. I was shocked by the number of "complete responses" (not partial responses). Men, however, did not respond nearly as well, although a few of them had complete responses. Partial response rates were not given, however.
If patients relapse a few years later, they can start taking Danazol again. It is true that the relapse rate is higher than those who respond well to only immunosuppressant therapy.
I read a few other studies concerning Danazol. It only works well for a certain subset of SAA patients. Again, it's mainly used with adults with SAA with short telomeres or "suboptimal" chromosomes. Without PNH-clone increase is preferable, but not necessary.
I never knew any of this until recently.
