[Greg H]

Treatment

What treatment we turn to next will depend on the results of the biopsy.

If I've suddenly developed some scary abnormality, like Monosomy 7, or a big blast count, that would bump me into a higher-risk category and we'd probably be looking toward something like Vidaza with an eye toward transplant.

But, if I remain in my current INT-1, lower risk category, Dr. Olnes said, we're in "a bit of a gray area."

ESAs. If My natural EPO level was low, we could try Procrit or Aranesp to try and stimulate more red blood cell production. I, in fact, had one dose of Aranesp early on in my MDS experience. But Aranesp works best for folks with EPO under 100, and is generally not prescribed for folks with EPO over 500. The two times mine has been tested, I clocked in a 1550 and 1080. Dr. Olnes said it's highly unlikely to have slid all the way down to 100, and is likely even higher now than it was before.

Vidaza. He's not a fan of Vidaza for low-risk folks, because much of the research has shown it's most effective in higher-risk patients. It's also used as a bridge to transplant by my transplant center, so we wouldn't want to be doing Vidaza unless we were, in fact, moving toward transplant, and that's not something I expect to do as long as I am lower-risk and have a manageable transfusion requirement. I haven't decided whether I think every two weeks is "manageable." I know that many folks would consider that interval a blessing, because they have even more frequent transfusions.

Revlimid. Though Revlimid, or lenalidomide, is generally used for folks with the deletion 5q chromosomal abnormality, Dr. Olnes said the drug seems to be effective in 30-40% of MDSers who do not have that particular abnormality. That's a bit higher than the percentages I've seen, which have been more like 25%. Revlimid typically lowers platelet counts, which can make it a problem for some folks. My platelets bounce back and forth across the bottom of the reference range, and Dr. Olnes said he doubted Revlimid would depress them enough to cause a problem. So Revlimid is an option. Getting insurance to pay for it may be difficult, but there are a variety of clinical trials of Revlimid in non-5q folks that I might be able to access.

HDAC Inhibitors. Valproic Acid and other drugs in its class have been trialed among low-risk MDSers. Used alone (as a "monotherapy"), Dr. Olnes said, they haven't provided results that were all that impressive. But I've seen studies in which HDAC Inhibitors have produced improvement in about 25% of patients. Unfortunately, most of the current trials seem to be aimed at higher-risk patients, with combinations of Vidaza or Dacogen with an HDAC inhibitor.

Ezatiostat. I haven't spoken with Dr. Olnes about this drug, which has been the subject of trials in both intravenous and oral formulations. Kind of like the other options, it seems to produce improvement in 20-25% of folks with MDS. There aren't any trials currently recruiting, but I'm interested in learning more about this one.

Other options? We won't be making any decisions about treatment until the results of the bone marrow biopsy are in. But it's never too early to get started on the research. If you have suggestions, ideas, or links to good research, either about the options listed above or others I've missed, I'm all ears.

One of the things I have noticed is that most of the drugs being investigated seem to help 20-25% of patients. If you believe, as I do, that MDS is not one thing, but really a collection of related diseases, then this makes perfect sense. Some of the new drugs help one kind of MDS; some help another, but the trials include all types, because they are just getting started. Unfortunately, there's not much data to go on so far to figure out which patients are helped by which drugs. My reading so far suggests that Revlimid and the HDAC inhibitors seem more active amongst folks with mostly red cell problems, while ezatiostat seems more active on platelets and neutrophils.


And Campath?

The obvious question: "Do you regret participating in the Campath trial?"

Not for a minute. If you looked at the odds -- age under 60, trisomy 8, HLA-DR15 positive -- I had a high probability of response to immunosuppressive therapy. If it was going to work, it needed to be tried sooner rather than later, to prevent additional chromosomal damage. My platelets and my neutrophils were both trending down prior to enrolling in the trial.

Give the same scenario, I'd do it again in a heartbeat.

It may not have worked out for me, but there's no question that, for some folks, MDS is an immune system problem. The research is there. And maybe my participation in the trial will help the folks at NIH ultimately figure out exactly which patients are the right patients for IST and Campath.

I would do that trial again -- and will do other trials when appropriate to my disease profile. We aren't going to whip this disease by being timid.