Patients with myelofibrosis have a number of anemia- and non–anemia-related symptoms that can substantially impact their quality of life (QOL), but there is considerable patient variability. Measurement of QOL is important to track and address disease-related effects on patient symptoms and their activities of daily living.

There are both anemia- and non–anemia-related factors that affect QOL in patients with myelofibrosis. With respect to non–anemia-related factors, a whole series of potential symptoms may be present that are related to the spleen, including early satiety, nausea, upper quadrant pain, urinary frequency, bowel disturbances, and pain from splenic infarction. These symptoms are often accompanied by constitutional symptoms such as fatigue, fevers, chills, weakness, and malaise.

For some patients with myelofibrosis, their main symptoms are primarily related to their anemia and, in more severe cases, to their anemia and thrombocytopenia, in which case the bone marrow becomes fibrotic, and hematopoiesis is limited. These patients are transfusion dependent, and they experience anemia- and disease-related fatigue, as well as access to care–related QOL decrements. For example, they may have to go to the doctor’s office multiple times each week for transfusions or treatment for recurrent epistaxis.

The symptoms that are associated with myelofibrosis are different than what is seen with myelodysplastic syndromes, sometimes because of spleen-related symptoms and also because these patients are in a heightened catabolic state. Not only might they have cytopenias, but they can also have low albumin levels or anasarca secondary to clots or poor nutrition, which may also lead to orthopnea or paroxysmal nocturnal dyspnea. These compounding effects can greatly impact patient QOL.

We can measure QOL with the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF), which we refer to as the MPN-SAF Total Symptom Score (MPN-SAF TSS), but it is important to remember that there is substantial variability in the measurement of QOL. A person’s impression of their QOL and how they articulate their experience can vary considerably, so, when assessing patient-reported outcomes over time, you have to be cognizant that a person’s impression of their QOL and how they articulate that is subjective and personal, and it will vary among patients.

It is also important to consider how we talk to patients about their disease experience and what types of support they have. Fortunately, there is an increasing number and variety of opportunities for people to get the support they need, including measures such as nutritional support, psychological counseling, physical therapy and rehabilitation, and emotional support. All of these are important adjunctive measures for patients with myelofibrosis, particularly those with disabling muscle, joint, and bone pain and poor exercise tolerance. Also, some centers do a really good job of incorporating alternative interventions such as meditation, acupuncture, or Ayurveda. Finally, exploring the microbiome has gained a lot of attention recently, and interventions aimed at altering the microbiome are scientifically thought provoking but not yet ready for widespread clinical use.