Michael J. Mauro, MD, the leader of the Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center, discusses the risks of disease progression in myelofibrosis.

Although disease progression presents differently across the spectrum of myelofibrosis, the threat of transformation to acute myeloid leukemia (AML) remains a significant challenge, Mauro says. The treatment plan of patients who progress to accelerated or blast phase myelofibrosis often needs to be altered in anticipation of AML transformation; however, some approaches will continue patients on their current therapy, such as ruxolitinib (Jakafi), to prevent anti-inflammatory escape or continue clinical benefit, Mauro explains. Often, surveillance for progression in myelofibrosis can identify features of leukemic transformation, such as new mutations in driver genes and sudden changes in allelic fractions. 

Additionally, patients with progressive myelofibrosis may experience more profound cytopenias that can indicate more aggressive disease or poor disease control, Mauro says. Other signs of disease progression include splenomegaly, thrombosis, inflammation, generalized organ dysfunction, weight loss, and hypoalbuminemia, Mauro concludes. 

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Michael J. Mauro, MD, discusses the risks of disease progression in myelofibrosis.  



Dr. Mauro on the Risks of Disease Progression in Myelofibrosis 

Michael J. Mauro, MD, the leader of the Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center, discusses considerations for early treatment initiation in myelofibrosis.

Disease progression occurs on a spectrum in myelofibrosis, ranging from asymptomatic disease to radiographic disease progression or pathologic disease progression, Mauro explains. However, progression is most often driven by symptom burden, Mauro adds. 

Ruxolitinib (Jakafi), the mainstay of treatment in myelofibrosis, is associated with symptom management and quality of life (QOL) improvement, Mauro says. However, patients without significant symptom burden, which would be indicative of treatment initiation, could still be treated early, Mauro explains. Early utilization of chronic maintenance treatment can reduce splenomegaly and improve blood counts, as well as potentially alter the natural biology of the disease, Mauro says. As such, patients who have some critical mass of symptoms in which treatment is warranted should be treated early, Mauro concludes. 

Michael J. Mauro, MD, discusses considerations for early treatment initiation in myelofibrosis.  



Dr. Mauro on Considerations for Early Treatment Initiation in Myelofibrosis 

Michael J. Mauro, MD, the leader of the Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center, discusses managing symptom burden in patients with myelofibrosis.

The management of symptom burden in patients with myelofibrosis often starts with symptom assessment, according to Mauro. Standard tools are utilized in clinical trials and have been validated and added to the National Comprehensive Cancer Network treatment guidelines; these tools can aid in identifying some of the symptoms that patients may experience, Mauro says. These tools are helpful, as certain symptoms experienced by patients with myelofibrosis can be difficult to discern from other chronic illnesses, Mauro notes. 

Taking inventory of symptoms is the first step in disease management, followed by prioritization and determining which symptoms are the most bothersome for patients, according to Mauro; this can be a part of a broader discussion with the patient when determining treatment goals, Mauro notes. Lastly, it is important to look at all available therapy options to decide which will be the most beneficial strategy for each patient, Mauro concludes.

Michael J. Mauro, MD, discusses managing symptom burden in patients with myelofibrosis. 


Dr. Mauro on Managing Symptom Burden in Myelofibrosis

discusses treatment discontinuation for patients with chronic myeloid leukemia (CML)

hematologist and leader of the Myeloproliferative Neoplasms Program, Leukemia Service at Memorial Sloan Kettering Cancer Center, 

 can offer a “functional cure,” for some CML patients, 

aid Mauro.  After a period of treatment, some patients may be able to discontinue TKI therapy.

	If patients have a good response to therapy without any degree of relapse, lower-risk disease at presentation, and a period of time in a deep molecular remission, they may be a candidate for treatment discontinuation.  On average

 about half of the patients that fit that criteria may be able to stop TKI therepy without having significant relapse.

View more from the 2015 Congress on Hematologic Malignancies

Michael Mauro, MD, hematologist and leader of the Myeloproliferative Neoplasms Program, Leukemia Service at Memorial Sloan Kettering Cancer Center, discusses treatment discontinuation for patients with chronic myeloid leukemia.

Michael Mauro, MD

Articles

Dr. Mauro on the Risks of Disease Progression in Myelofibrosis

Dr. Mauro on Considerations for Early Treatment Initiation in Myelofibrosis

Dr. Mauro on Managing Symptom Burden in Myelofibrosis

Dr. Michael Mauro on Treatment Discontinuation for CML