Ask the Experts – Maureen Thyne – Myelofibrosis

Oncology Nurse Advisor (ONA): How is patient risk assessed in myelofibrosis?

Ms Thyne: Myelofibrosis is a very heterogeneous disease. At initial diagnosis, risk factors can be evaluated using the International Prognostic Scoring System (IPSS) for myelofibrosis, which looks at age older than 65, hemoglobin less than 10, white blood cell count (WBC) greater than 25,000, the presence of peripheral blasts, and constitutional symptoms (such as unintentional weight loss, fever, or excessive sweating). Based on the number of risk factors, patients are stratified as low, intermediate-1, intermediate-2, or high risk. Similarly, for patients who were previously diagnosed and whose risk needs to be re-assessed, the Dynamic International Prognostic Scoring System (DIPSS) and DIPPS-plus can be utilized.

ONA: What are the goals of treatment of myelofibrosis?

Ms Thyne: Myelofibrosis is a variable disease, and each patient’s case and care plan are different. We understand that at this time, stem cell transplant is the only opportunity for cure, but there are several medications that can help slow the progression of the disease and/or improve symptoms. Some supportive care measures, like transfusion, may also be utilized. Understanding your patient’s goals, whether that is for aggressive treatment or symptom management or somewhere in between, can help you set a care plan that best suits their specific situation.

ONA: What role does ruxolitinib play in the treatment of myelofibrosis? What other treatment options are considered for patients with myelofibrosis?^4,5

Ms Thyne: Ruxolitinib is the only medication specifically approved for patients with intermediate or high risk myelofibrosis, and it is used to help improve blood counts, symptoms, and quality of life. Some patients also have an improvement in overall survival. It is an oral medicine, taken twice daily, and requires regular doctor visits to have blood tests and physical exams. Other measures used to treat myelofibrosis include supportive measures, like blood transfusions, or cytoreductive therapies, like hydroxyurea, or even chemotherapy. Stem cell transplant is also an available treatment for certain patients, and at this time is the only treatment that can lead to cure. There are also many clinical trials available for patients in an ongoing effort to improve our treatment algorithm. This is a rare disease, with somewhat limited treatments, so more options are always welcome!

ONA: How do you assess and manage symptom burden in patients with myelofibrosis?

Ms Thyne: Having an open line of communication with your patient is the best way to continually assess their symptom burden. Sometimes asking your patient to keep a symptom journal, or occasionally complete a self-assessment (like the MPN-10, which is available online), can help them remember the symptoms and patterns during your visit. I also welcome the spouse/family member/friends of the patient to weigh in ― sometimes they notice new things or changes that the patient doesn’t. Ultimately, encouraging the patient to keep you informed of their symptoms is most important.

ONA: What does the future treatment landscape for myelofibrosis look like?

Ms Thyne: Myelofibrosis is a rare disease that remains difficult to treat. Over the past decade or so, however, it has benefitted from increased attention from the medical and scientific communities. We understand that it is meaningful to help improve patients’ symptoms even if it doesn’t ultimately fix the disease entirely. There has also been a steady stream of clinical trials investigating new uses for known medications as well as new compounds, and hopefully we will find a good treatment option for all our patients in the near future.