At what point in your MPN diagnosis did you join a clinical trial? What trial (drug) were you in?
I was diagnosed with PV in 2001. Only on phlebotomy and 81mg aspirin until 2007 when I began hydroxyurea because I turned 60 in 2006 and was now “high risk.” In 2009 my spleen was enlarging. I had been hearing that Interferon was best taken in the early stage of MF and might reduce myelofibrosis. My hematologist advised me he would be having a trial with Pegasys (Interferon) and I should wait for that. I actually waited until 2012 when the trial became available. I was now showing signs through bone marrow biopsies of secondary MF.
Did you feel as though you received accurate information getting into the trial?
Yes, I had been reading about Interferon treatment and knew I wanted to try it. Perhaps I didn’t know how quickly I could be removed from the trial because of my worsening symptoms.
What didn’t you know going into the trial that would be useful for others to know or questions they might ask?
It would have helped to know at what point you may be pulled from the trial and what happens after you are removed. Also, I did not know that the FDA can shut a trial down at any point due to adverse reactions to few patients.
How many clinical trials have you participated in?
I am currently on my 3rd clinical trial. (First trial wasn’t improving my symptoms, second trial was shut down by the FDA and the pharma company decided not to proceed , and the third trial I have begun my 4th year.)
Were you able to get the drugs after the trial(s) if you were doing well?
I can continue with the drug I am currently on as long as I continue to have benefit and as long as there is funding for this drug by the pharma company, and that it doesn’t get shut down prior to FDA approval
What advice would you impart to those who are skeptical about clinical trials who are not doing well on available treatments?
What do you have to lose? If you are currently not well, you certainly may have good results and feel better. If you don’t show benefit you will be removed to try something else. We do not know the future after taking these drugs. That is a gamble we must take.
Would you participate in other trials knowing what you know now?
If I was not feeling well and I met the qualifications, I would try again.
Marylin C. has been living with her diagnosis for many years. She has graciously volunteered for trials not only for her own reasons, but for the sake of MPN patients and their futures.