The latest developments in research and treatment of MPNs
Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms
The Department of Leukemia has established the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms (MPNs) as a distinct operational unit within the Leukemia Department and Leukemia Outpatient Center. The goal of the Clinical Research Center for MPNs is to develop novel effective therapies for MPNs that will significantly improve patient's outcomes and quality of life, provide exceptional patient care and ultimately cure MPNs.
The Hanns A. Pielenz Clinical Research Center for MPNs has become the largest in the world for referrals of MPN patients and clinical research. Medications that have become the global standard of care for myelofibrosis (JAK1/2 inhibitor ruxolitinib) were developed at the Clinical Research Center for MPNs. We see more than 250 new patients with MPNs annually, far more than any other clinical center in the world, and are continuously engaged in clinical research studies to develop novel therapies for our MPN patients.
The main goals of the Hanns A. Pielenz Clinical Research Center for MPNs include:
- Conduct clinical trials to evaluate novel agents for patients with MPNs and provide exceptional patient care. For a comprehensive overview of novel MPN medications in clinical development at the Clinical Research Center of MPNs, please visit "Current MPN Research".
- Maintain a centralized tissue bank and database for collection and storage of patient specimens, which are valuable resources to conduct basic and translational research in MPNs
- Educate patients, caregivers, phycisians and the community at large about the latest findings regarding research and treatments in MPNs. We periodically publish the newsletter MPN Focus where we highlight the latest progress in MPN clinical research. To review the MPN Focus newsletters, please visit "MPN Awareness and Education".
During a routine checkup about 20 years ago, my primary care doctor noticed that my blood platelet count was going up. After meeting with a hematologist near my home in Dallas, I was diagnosed with essential thrombocytosis. That meant my body was making excess platelets for an unknown reason.
For about nine years, I took anagrelide daily to manage the essential thrombocytosis. I continued cycling, traveling and enjoying retirement. I just needed to be careful and avoid injuries or cuts since they could result in excessive bleeding.
But during a cycling trip, I noticed I was extremely fatigued. As an athlete and avid cyclist, I didn’t understand why I was feeling sluggish and having difficulty keeping pace. I felt the need to take a nap after each ride.
Not wanting to give up things I loved, I went to the doctor.
My bloodwork showed my hemoglobin level was very low, so my doctor suggested I make an appointment at MD Anderson for a bone marrow biopsy.
Helping myself and others through clinical trials
On my first trip to MD Anderson in July 2009, I met with leukemia specialist Hagop Kantarjian, M.D. The bone marrow biopsy results showed that the essential thrombocytosis had progressed to myelofibrosis, a condition that can sometimes develop into acute myeloid leukemia.
At the time, treatment options were slim. Instead of taking a maintenance medication like revlimid or undergoing a bone marrow transplant, I decided to focus on exploring clinical trial options.
Over the course of two years, I traveled to MD Anderson every three months for monitoring and enrolled in several clinical trials. I figured if my treatment could help somebody else, I was willing to do that. I had to discontinue the trials that didn’t work or made me sick, but I kept trying. In 2011, I met with hematologist-oncologist Srdan Verstovsek, M.D., to explore other treatment options.
Dr. Verstovsek started me on a clinical trial with the JAK inhibitor drug momelotinib. For about five years, my body responded well, and I felt free to enjoy my life again. But the drug was no longer working as effectively less than a year before the end of the clinical trial. I started needing blood transfusions because my red blood cell count was very low.. That’s when Verstovsek, along with stem cell transplant specialist Uday Popat, M.D., suggested that I consider an allogeneic stem cell transplant. I would need a donor.
Popat and Verstovsek’s team worked together and found that luckily, my younger sister was a perfect match to be my stem cell donor. Now, I had a big decision to make.
Weighing the pros and cons of a stem cell transplant
I didn’t know what would happen afterward if I got a stem cell transplant. At the time, I was 69 and functioning. I was still cycling and traveling, but it was becoming more difficult. Did I want to stay the course and hope that another drug would come along that might help? Or did I want to take a chance to be in full remission?
I started researching. I spoke to people who had stem cell transplants to better understand the struggles and successes. I also looked the support staff who would provide post-transplant care. I found MD Anderson’s staff to be far and away the best. In the end, I decided that a stem cell transplant was best for me.
My stem cell transplant and recovery
My care team worked together closely before, during and after my stem cell transplant, and ensured I got the supportive and rehabilitative care I needed. Nutrition played a big part in my recovery. I was put on a non-restrictive diet and was surprised that MD Anderson’s hospital food was so tasty and nutritious!
Initially, I had concerns about being away from home for the stem cell transplant. While that did require preplanning and organizing, being close to the hospital brought me and my wife a sense of security in case of an emergency. It also made the after-care checkups over the next few months much more convenient.
The transplant was successful, but it was a very difficult process. I lost a lot of weight in the first month due to stomach sensitivity and mouth sores. I also had to be very careful to avoid germs because my body couldn’t ward off infection.
Three years later, I finally feel like I’m getting back to my normal self, and I can’t praise MD Anderson enough. From the beginning to the end of my stem cell transplant, everyone was terrific. I am so grateful to be back to doing the things I love.
My advice for others considering a stem cell transplant
When I talk with anyone considering a stem cell transplant, I advise them to consider who is going to support them during and after the procedure. As a patient with a compromised immune system, you must be vigilant about your health as your body heals. My wife was invaluable to the process, and my sister stayed with us in Houston for four months. The patient can’t do everything alone.
It’s also important to stay positive and believe that things will get better. If you lose your positive attitude and your emotional wellbeing, your recovery is in jeopardy. It’s hard to do, but it’s important.
And finally, I tell people to remember that every case is different. You can talk to a lot of people about their stem cell transplant experiences. But in the end, you have to decide what is best for you.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.
In the spring of 2017, my husband Bill and I were getting ready to travel and do some charitable work. We’d finished raising our children and had been very active in our grandchildren’s lives, so we were looking forward to some time for ourselves.
Then, Bill was diagnosed with myelofibrosis. And all of our plans came to a grinding halt.
Fortunately, we knew what to do. One of our daughters was born with Down syndrome, and we’d learned very quickly that you can’t count on the best medical professionals to drop into your lap and perform miracles. You have to do your part, too. So, any time a challenge comes along, we make it our mission to find the right people, decide on a solution and work together for the best possible outcome.
We ended up choosing MD Anderson for Bill’s myelofibrosis treatment. Here are three reasons why.
1. Knowledgeable physicians with incomparable expertise in myelofibrosis treatment
I’ve worked in health care for about 30 years. So, I was already familiar with MD Anderson’s reputation. We’ve also known several people who chose MD Anderson for their cancer care. After our trip around the country in 2017, we realized MD Anderson was right for us, too.
After Bill’s diagnosis, we met with myelofibrosis specialists in Chicago, Indianapolis, New York City, Phoenix, Rochester and Seattle before coming to Houston. Ultimately, we chose MD Anderson because of its doctors’ expertise. Srdan Verstovsek, M.D., Ph.D., — or “Dr. V,” as he asked us to call him — was the most knowledgeable physician on myelofibrosis we had ever met.
Dr. Verstovsek was not only aware of all of the clinical trials happening internationally; he also knew who was conducting them, if they were still accepting patients and what the status was of every single one. He was phenomenally informed. And he went through the spreadsheet of clinical trials we’d compiled to decide on the best plan for Bill.
We were elated that he paid such careful attention to our needs and wants. We had just spent the better part of a year traveling the country, going from expert to expert. And instead of the medical jargon, general statements, discouraging news and poor prognoses we’d encountered at other places, MD Anderson gave us hope, encouragement and a PLAN! Finally, we had found the expert we were seeking.
2. A team approach to myelofibrosis treatment and care
Another thing that impressed us about MD Anderson was that we had medical professionals from so many specialties working together to care for my husband — and all of them understood the unique challenges of stem cell transplant patients.
During my husband’s procedure, for instance, he started developing a bacterial infection. We were completely unaware of it, but Bill’s daily bloodwork alerted the infectious disease specialists that trouble was brewing.
As it turns out, they were already watching for it. So, Harrys Torres, M.D., ordered Bill’s central line removed late one night and began an antibiotic treatment immediately. As a result, the infection never ever gained a foothold, and it didn’t impact our lives. But it did make us much more aware of the value of having a true team behind us.
We were also grateful for the expert guidance of MD Anderson’s rehabilitation staff. Our outpatient physical therapist, Rhodora Fontillas, was particularly wonderful as Bill recovered from the transplant. She was funny, focused, well-informed and a wonderful coach.
Rhodora also turned out to be a godsend when Bill contracted a serious fungal infection in June 2019. His recovery from that was even tougher than from the stem cell transplant. But Rhodora was a fantastic guide in rebuilding his lungs and muscle tone — because she has experience in both areas.
3. Personalized attention and every type of support
One of the most surprising — and meaningful — interactions at MD Anderson was with the chaplain. She created a special prayer just for Bill. And when the stem cells arrived from the donor, the chaplain stood on one side of him, while a nurse stood on the other side, holding the bag of cells. They reached across Bill and held hands, while I stood at the foot of the bed as the chaplain read the prayer aloud. It was a very emotional and uplifting moment.
Bill’s stem cell transplant marked a new beginning for us. A wonderful 21-year-old woman had literally given a part of herself to save Bill's life, and we were -- and are -- so incredibly grateful.
Looking back with gratitude and forward with hope
Now, we are looking forward to the day that Bill is fully recovered from the infection he caught last summer, even after he’d recovered his strength and stamina post-transplant. Unfortunately, he was still vulnerable to infections, because his immune system had not yet fully recovered. And it was a very serious illness, so he was hospitalized for almost three months.
We’ll be glad when we are far enough down the road that we don’t need to even think about graft vs. host disease anymore. We’ll also be glad when Bill’s immune system is back within normal ranges, so we can truly live our lives the way we did before his diagnosis. Until then, we are working hard to ensure that each day is valued and that we are doing all we can to remain as healthy as possible. And we’re extremely grateful to MD Anderson for everything its doctors have done to get him this far.
Request an appointment at MD Anderson online or by calling 1-877-632-6789.
This is a very exciting time because many new medications are being developed to treat MPNs.
Clinical Research Center for MPNs Team
Role of the Physician Assistant
Lindsey Lyle discusses the physician assistant's role in treating MPN patients
The Importance of Second Opinions
Dianne Robinson discusses the importance of getting a second opinion.
Rusty's Story: Coping with Myelofibrosis Diagnosis
Rusty Howard discusses his experience as a patient with myelofibrosis
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