The role of the patient advocate

An advocate can look out for your patients when they cannot look out for themselves.

There is a lot of talk these days about health insurance, medical quality, medical expense and the delivery of health care. However, part of the picture is still incomplete, and that is the role of the patient advocate. Physicians have seen what happens when a patient has an inadequate advocate, an angry advocate or no advocate at all.

The other day, I experienced a situation that illustrated for me the importance of a practical, knowledgeable patient advocate, one who is really making a difference in a patient’s care. I had just come from the American Society of Hematology meeting in New Orleans, where I found out about the COMFORT-I trial and the drug INCB18424 (Incyte). Initial results were quite promising for a disease that has never had any effective therapy.

Arthur Topilow

Srdan Verstovsek, MD, PhD, of The University of Texas M.D. Anderson Cancer Center, said that INCB18424 provided “durable and previously unachievable clinical benefits in patients with myelofibrosis with or without JAK2 activating mutations. It was equally gratifying to see significant clinical benefits in patients with advanced polycythemia vera and essential thrombocythemia, including normalization of blood counts, normalization of hematocrit without the need for phlebotomy, rapid and durable reductions in enlarged spleens, as well as rapid and durable reductions in symptoms, particularly pruritus.”

I wrote an e-mail to my friend Nina, who was my first piano teacher back in grade school. She knew that I was aware of her daughter Ronnie’s myelofibrosis diagnosis. Ronnie was now experiencing symptomatology from the disease. I referred her to the article about Dr. Verstovsek’s results in myelofibrosis patients.

Dear Nina –

This is pretty impressive stuff. Maybe Ronnie can be the lucky one to respond to the drug. It’s probably worth a trip to Texas after some correspondence. Ronnie’s doctor should be the one to make the phone call to Texas and clear the way.

Be in touch,

Arthur

I got a reply from Ronnie’s sister Jann, which pleasantly surprised me. She wrote:

Hi Arthur –

Believe it or not, Ronnie’s in the process of enrolling in this study! Because Ronnie started having some difficult symptoms, I called M.D. Anderson a few months ago to see what research was going on. The doctor I spoke to thought she’d be a great candidate for this particular study and referred her to a study group at Mt. Sinai. We’re just hoping she gets the medicine and not a placebo. Of course, we’re also hoping the medicine is effective and reduces her spleen size and lessens the bone pain she’s having now. We’ll definitely let you know how it goes.

Thanks again for thinking of her,

Jann

I called Nina to talk things over. After receiving permission from both sister Jann and mother Nina, I sent an e-mail to Ronnie and asked her to write a short summary of her illness. What she wrote struck a chord with me. This is the poignant response that she sent me:

Hi Arthur-

Thanks for thinking of me during your seminars. I am glad I am already working toward the Incyte COMFORT-1 trial. Now I only have to hope I get the medicine — not placebo.

My history is as follows: I was diagnosed in 1993 (at the age of 38) during a routine physical. Sloan-Kettering gave me a 5-year life expectancy unless I got a bone marrow transplant, which at that time had a 50% mortality rate. I had no symptoms, so clearly I was not going to choose that path.

That was when my sister took over as my advocate. She read, researched and contacted anyone and everyone who might be able to slow the process or come up with a cure.

Subsequently, I went to 10 additional doctors — including one who suggested I go to Germany for an experimental procedure. I went there for consultation with my sister Jann, who was seven months pregnant, accompanying me, along with her five-year-old and our husbands. Needless to say, I didn’t go for the procedure.

I had no symptoms until 2006, when I began to see my waistline disappearing as a result of my spleen enlarging. I started to have pain in my spleen if I exerted myself in the least and I had a drop in my normal energy.

Only during the past18 months have I started to get serious bone pain and a major drop in energy. Strangely, my spleen stopped hurting, although it is the same size.

I did a short stint on hydroxyurea earlier this year. Initially, I had a 15% drop in my spleen size, but it came back and became even bigger. I went up to 2,000 mg before it made me too sick to stay on it. Since it was not helping, there was no reason to endure it. I also did not know it was a chemotherapy drug until Jann brought it to my attention.

Jann continued all these years keeping tabs on everything that emerged regarding my illness. She made contact with M.D. Anderson when she saw the information about the COMFORT-I trial; she spoke to the doctor on my behalf, and with her guidance, I am now working with a doctor at Mt. Sinai to (hopefully) participate in the trial.

She found out about a symposium in New York, and we went to get an education on the progress and options for this disease.

Facing mortality at 38 years old was completely overwhelming to me. I was pretty much frozen. I could not even bring myself to read about the disease, treatments, etc., because it was so upsetting. I was unable to function. I simply could not absorb what was happening to me.

I kept my focus on my work and handed over my health care to my sister. She has been my rock and my guidance, as well as my absolute best friend. I have made her my medical proxy in my will, as I know I can trust her to do the right things regarding my health and well being.

I have seen my sister act as strongly on behalf of my mother and father. She saved my father’s life by finding out a serious side effect of one of his medications and actually brought it to the attention of the doctors. She was right, and he recovered.

I am glad you are writing something regarding this topic. Friends and family should not “mind their own business.” There is so much data out there that if you are lucky enough to have somebody like Jann in your life that is so devoted to keeping informed on everyone’s well being, it is really a gift.

I do not mind if you use names. I am proud of the amazing family I am a part of.

Thanks for your interest and again I appreciate you thinking of me.

Best Regards,

Ronni

Don’t you wish that all our patients had an advocate like this!

Arthur Topilow, MD, FACP, is in private practice at Atlantic Hematology & Oncology in Manasquan, N.J.

For more information:

• Verstovsek S. #756. Presented at: 51st ASH Annual Meeting and Exposition; Dec. 5-8, 2009; New Orleans.