treatments. In 2007, an international internet-based study of 1,179 patients was conducted for this purpose. The goal of the study was to look beyond blood counts and gene mutations, to learn what MPN patients actually experience. Questions centered around how the disease was impacting day-to-day life for patients. This Landmark Study found that symptoms were often debilitating and occurred frequently among respondents.
Specifically, the study uncovered the following:
Symptom % of respondents who experienced this symptom
Fatigue >80%
Pruritis (itchiness) 52.2%
Night sweats 49.2%
Bone pain 43.9%
Fever 13.7%
Weight loss 13%
In 2011, a unique symptom assessment tool, the MPN Symptom Assessment Form (MPN SAF), was developed (see Chapter 2), allowing for sensitive and standardized symptom evaluations for those living with MPN. The results of this international validation study, involving 402 patients, again revealed that MPN-associated symptoms were severe and frequent.
Like the earlier study, patients noted that fatigue was the most common symptom (93%) and was often debilitating. Additional symptoms were both physical and psychological in nature, including:
Decreased quality of life 84%
Insomnia 65%
Sad mood 65%
Sexuality problems 58%
Night sweats 56%
Bone pain 49%
Headache 48%
Cough 46%
Abdominal pain 46%
Weight loss 35%
Fevers 20%
Within the disease spectrum, those diagnosed with MF experienced the most symptoms.
Since these studies were conducted, a shorter version of the symptom assessment tool was developed to make it quicker and easier to use for both patients and physicians. This 10-item MPN Total Symptom Score (TSS) form is now routinely used to assess MPN symptom burden and is part of the recently developed National Cancer Care Network (NCCN) guidelines (see http://www.nccn.org). The impact on symptom burden, as measured by the MPN TSS, is used to gauge how well treatments are working and help catch and manage early progression of the disease.
Special Topics In MPN
When you are diagnosed with any form of MPN, there are some associated health issues you should be aware of, including:
1. Thrombotic events (blood clots) – A serious complication of MPN is the possibility of a blood clot in a major artery to the brain (stroke), heart (heart attack) or lungs (pulmonary embolism) or other areas. This risk for thrombotic events increases with other factors, such as age, blood pressure, obesity, smoking and a prior history of these events. If a thrombotic event occurs, several new considerations will be discussed between you and your healthcare team.
For instance, if you were not receiving treatment for your MPN previously, a thrombotic event will likely trigger a course of treatment. Remember, having a history of blood clots places you at a high risk for another thrombotic event in the future.
2. Bleeding – Although it may seem counterintuitive to what we’ve discussed about MPNs and the risk of blood clotting, abnormal bleeding can also occur with MPNs. This occurs most often in those with ET, who have very high platelet counts. As the number of platelets increase, they may “consume” a factor required for blood clotting. This is called the Von Willebrand Factor, and it can lead to an increased risk for bleeding. Sometimes healthcare providers will do a blood test to check the “Von Willebrand factor activity level” to help evaluate the risk of bleeding. The results may impact treatment decisions, such as avoiding aspirin therapy, or change a decision to undergo elective surgical procedures.
3. Surgery – Before you plan any surgical procedure, it’s important for MPN patients to have a thoughtful, thorough discussion with your surgeon and hematologist regarding your risk for blood clots or bleeding. It may be best to delay a procedure until your disease has been optimized to decrease your risk. Often, surgical procedures will require the use of anti-coagulants post operatively, and again, the risks and benefits should be discussed with your healthcare team.
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.
