4. Allogeneic Stem Cell Transplant – Hematopoietic stem cell transplant is the only curative treatment for MPNs. Given the high-risk nature of this procedure and potential for complications, it’s typically reserved for intermediate or high-risk myelofibrosis patients only. It’s a rigorous procedure, which requires a consultation with a stem cell transplant specialist to evaluate if it’s the right treatment approach for you. The evaluation typically includes age, functional status, presence of other diseases, health of major organ systems, psychological status, the availability of a caregiver, and other considerations. If you have intermediate or high-risk MF, ask your healthcare team if a transplant consultation is appropriate.
Julianne’s Story
At 55, Julianne was surprised by a heart attack. Fortunately, she received prompt treatment and recovered from the event.
“Although no one wants to have a heart attack, I was grateful that it wasn’t more serious, and felt like I got a second chance at life,” remembered Julianne. “I was determined to make the most of it.”
However, during the next several years, Julianne felt constantly fatigued and complained of feeling itchy all the time. She took antihistamines for the itchiness and initially thought the tiredness was a lingering result of the heart attack. Finally, at age 58, a bone marrow biopsy confirmed a diagnosis of ET.
Once she began seeing a hematologist and receiving treatment, which included a daily aspirin and hydroxyrea, her symptoms improved.
“I was so relieved to find out that my fatigue wasn’t something I just had to live with,” said Julianne. “And the itchiness, which was driving me crazy, was completely gone. I felt like I got my life back, again.”
Unfortunately, about eight years later, Julianne began experiencing new symptoms. She started to feel full quickly when eating and developed stomachaches after meals. Julianne also suffered from nightly fevers and sweats. Eventually, she started losing weight and became alarmed. She quickly made an appointment with her hematologist.
“My hematologist recommended a repeat bone marrow exam to see if my disease had progressed to myelofibrosis,” recalled Julianne. “I never knew that my disease could change like that, but I learned that MPNs can transform over time.”
Julianne was diagnosed with myelofibrosis (MF) and began a new therapy. Thankfully, she improved quickly and has been able to effectively manage her symptoms for many years. Of course, she is regularly monitored by her healthcare team to keep symptoms in check and monitor disease progression.
1Tefferi, A. The history of myeloproliferative disorders: before and after Dameshek. Leukemia. 2008;22(1):3-13.
2Lange et al. JAK2 p.V617F allele burden in myeloproliferative neoplasms one month after allogenic stem cell transplantation significantly predicts outcome and risk of relapse. Haematologica. 2013 98(5):72-8 Epub 2013 Jan 8.
3Austin SK, Lambert JR, et al. The JAK2 V617F mutation and thrombosis. Br J Haematol. 2008; 143(3):307; or to estimate risk of evolution to post ET/PV myelofibrosis. Alvarex-Larran, et al. JAK2 V617F; monitoring in polycythemia vera and essential thrombocythemia: clinical usefulness for predicting myelofibrotic transformation and thrombotic events. Am J Hematol. 2014;89(5):517.
www.mpn-qol.org
Chapter 2:
Monitoring Your Mpn Disease
Monitoring Your MPN Disease
Being diagnosed with a rare blood cancer is life changing for both you and your loved ones. It can be a confusing and emotional time as you begin to deal with your “new reality.” In fact, newly diagnosed patients often compare the emotional experience to the five stages of grief – denial, anger, bargaining, depression and acceptance. As difficult as it may seem, working through these stages can help you deal with your diagnosis.
Coping with illness is an ongoing process, filled with twists and turns and unknown paths. Learning as much as possible about your disease, working with a specialist, and getting support are the best ways to navigate this journey.
It’s also very important to accurately monitor your disease, which will help you advocate for yourself, stay on track with treatments, and improve outcomes. Although there are no known cures for MPN, many people who have ET, PV or MF can effectively manage symptoms and enjoy longevity with proper monitoring and treatment.
Finding a Specialist
The first step in getting quality care is to find a hematologist specializing in MPNs. Because MPNs are rare diseases with unique symptoms and treatment options, it’s essential to find a specialist who understands the needs of MPN patients. However, the rarity of MPNs can also make it more challenging to find a specialist. Your primary healthcare provider may be able to refer you to an MPN specialist, but be prepared to do some of your own research.
Some additional places to find a specialist include:
• The American Society of Hematology, which offers a convenient search tool to help you find treatment facilities by region and specialty. Visit: http://www.hematology.org/Patients/FAH.aspx, and go to “Find a Specialist.”
• The MPN Forum, which provides a list of patient-recommended hematologists who specialize in MPNs. Visit: https://mpnforum.com/list-hem/.
Questions to Ask Your Hematologist
• How many MPN patients do you treat now?
• What is my exact diagnosis?
• Did I test positive for any mutations? What kind of genetic testing can you offer me?
• What is my plan for managing this in the short and long term?
• What treatments am I a good candidate for, and why?
• What are the side effects of my treatment options?
• What kind of lifestyle changes should I make to deal with symptoms?
• If you are of childbearing age, how should I manage my family planning?
• How should I tell my immediate family about how this will affect my life and theirs?
• What kinds of clinical trials, if any, am I qualified for?
Source: MPN Research Foundation
Understanding The Complete Blood Count
One of the two major components used to monitor MPN is the complete blood count. The body is primarily made up of water and cells. While some cells stay in place to form tissues and organs, such as muscles or the heart, others may circulate around within the blood. The major blood cells include red blood cells, white blood cells and platelets. Since blood cells play a critical role in MPN progression and treatment, these cells are routinely monitored in MPN patients.
Specifically, changes in