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patients to feel abdominal discomfort, pain under the left ribs and/or early feelings of fullness.

      The tests used to diagnose MF are similar to those used in ET and PV, including:

      • A blood test to evaluate blood counts (red blood cells, white blood cells and platelets)

      • Molecular tests (done on blood or with a bone marrow biopsy)

      • Bone marrow biopsy, which is essential to diagnose MF. The bone marrow is viewed under a microscope to see cellular changes consistent with MF, as well as to determine how much scarring or “fibrosis” is present. This will be graded on a scale of 1 to 3, with 3 being the most fibrotic or scarred bone marrow.

      Other factors can be considered when making a diagnosis of MF, such as an enlarged spleen, changes in appearance of blood cells under a microscope, or changes in routine blood tests, such as higher LDH levels.

      World Health Organization (WHO) Criteria for Myelofibrosis:

      A diagnosis of MF requires meeting all three major criteria, and at least one minor criterion.

Major Criteria	Minor Criterion
Presence of megakaryocytic proliferation and atypia, accompanied by:• Fibrosis• Not meeting WHO criteria for other blood diseases• Presence of JAK2, CALR, or MPL mutation or other marker	Presence of at least one of the following, confirmed in two consecutive tests:• Low red blood cells not attributed to another condition• High white blood cell count• An enlarged spleen• Changes in appearance of cells under a microscope that are consistent with MF

      How is MF treated?

      The only curative treatment for MF is an allogeneic stem cell transplant, which involves transferring the stem cells from a healthy person (the donor) to the patient’s body after high-intensity chemotherapy or radiation. This can be a high-risk procedure. The intense doses of chemotherapy or radiation are used to inactivate the immune system to reduce the chance of cell graft rejection and enable donor cells to travel to the bone marrow. However, because this treatment temporarily shuts down the immune system, there can be adverse reactions.

      In the last decade, targeted therapy with Janus kinase (JAK) inhibitors has become available for treatment. This therapy can improve symptoms by inhibiting or slowing down the overproduction of blood cells, thereby decreasing the size of the spleen. This treatment may also prolong a patient’s life.

      Many other medical therapies are often used to treat MF, depending on a patient’s symptoms and the treating physician. For example, agents such as erythropoietin can be used to stimulate red blood cell count. Patients may also receive transfusions, steroids, or immunomodulating agents.

      In addition, many clinical trials are ongoing for patients with MF, and you are encouraged to talk to your healthcare team about which trials may be available to you.

      The MPN Symptom Burden

      As an MPN patient, you may experience a wide range of symptoms which can impact your quality of life (QOL). The symptoms associated with MPN, and the severity of those symptoms, can differ greatly from one person to another. Keeping track of how symptoms affect you day-to-day, ideally with a symptom diary (see Chapter 6), is the best way to monitor and treat the disease. That way, you can discuss any changes in symptoms with your healthcare team at each visit. Understanding your individual symptoms is very important to managing the disease and reducing its effects.

      To help us better understand this burden of symptoms, as well as how physicians evaluate and manage the disease, the MPN QOL group was formed. The group is a collaborative effort between UT Health – San Antonio, Mayo Clinic Arizona, Arizona State University and the University of Arizona. They are dedicated to advancing our knowledge of MPN symptom burden, quality of life, and interventions to improve MPN patient wellness.

      Obviously, having a better understanding of the variety of symptoms patients with MPN experience is important for developing effective 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

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