If you are a patient with myelodysplastic syndromes, I’m sure your physician will suggest at some point that you participate in a clinical trial.

That’s an important topic that, in my opinion, needs careful consideration.

I gave the topic deep and serious thought and chose not to participate in clinical trials until my disease became acute.

At the time, I understood the state of knowledge about myelodysplastic syndromes (MDS) and its natural course well enough to believe that not much was known, particularly about my variant with two uncommon chromosomal abnormalities.

I figured that since I felt well enough with a disease that would ultimately prove fatal I might as well let it run its course for as long as I could stand to do nothing.

Nevertheless, I did agree to being enrolled in the international database of MDS patients so I knew that at least the basic facts about my disease would contribute to patient care in the future.

From the time of my MDS diagnosis, I was offered the opportunity to receive treatment and to participate in clinical trials. However, I chose a different path: supportive care.

My doctors concurred in this course of inaction, eventually rarely offering me the option of participating in clinical trials.

My disease remained stable for 16 years, which was a remarkably mild course for MDS.

The year before my disease became acute, my chromosomal abnormalities disappeared. At that time, I was offered the opportunity to participate in a clinical trial of Dacogen (decitabine) in combination with all-trans retinoic acid, or tretinoin (Vesanoid). I still felt well enough and did not think treatment would improve my quality of life. So I declined, again.

Eventually my disease became acute. I converted from MDS to secondary acute myeloid leukemia, or sAML (If you read Dr. Bowen’s column about the revisions to the IPSS you may have noticed that only around one-third of MDS patients convert to AML).

While the approved therapies for AML are often quite successful with primary AML, those same treatments rarely have any lasting effect for MDS patients. Typically MDS recurs within a year of treatment with standard AML drugs. Normally, a patient with sAML only remains alive for 1.5 to 2 years.

At that point, I considered my options.

I finally felt I was sick enough to warrant treatment. It might have been a good thing to start treatment sooner. However, for me it was important to know that my death was not far away before I was willing to sign up for a clinical trial.

None of the treatments for MDS offer a cure, with the possible exception of a stem cell transplant, but even that doesn’t work well if your disease cannot be driven into some kind of remission beforehand. Achieving remission (however temporary) is not easy to do with MDS; for many people with MDS, it does not happen.

I felt that if my doctors could find a matched donor for a stem cell transplant that would be the best treatment for me.

I knew that I had to do some type of chemotherapy or I wouldn’t even last the six months or more it can take to find a donor, if you are lucky enough to find one.

I also had become transfusion-dependent.

When my doctor re-offered me the same study I had refused a year earlier, I gave it some serious thought.

Doctors are experts in what is known about a disease and its treatments. Patients, on the other hand, have a great deal of information from their own bodies that the doctor can’t know.

Over the course of the 18 years I have had MDS, I have had five different hematologists at the same institution.

I have found that having good communication with your doctor is often hard work (see my related column), but I listen to my doctors and trust what they say.

Nevertheless, in the end, you and I, the patients, make the final decision, and we have to live with its consequences, good and bad.

I also thought that if no patients agreed to participate in clinical trials we would learn nothing new about MDS. By participating, I would be helping the people who come after me. Perhaps one day they will understand the disease well enough to cure it.

I came to the conclusion that participating in the study was a good intermediate measure for me. To use an FDA-approved treatment for MDS with another approved drug that might improve its action seemed like plausibly good science to me.

I formally requested that they try to locate a matched donor for a transplant and began the clinical trial.

Did it work? We don’t know yet. The Phase 1 trial is closed, the data is being analyzed.

Did you decide to participate in a clinical trial? I am interested in your experience.