Expert Insights into the Treatment of Myelofibrosis - Episode 4

Clinical Scenario: Patient with Primary Myelofibrosis

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Dr John Mascarenhas presents a patient case scenario of a 68-year-old woman with a common presentation of primary myelofibrosis.

John Mascarenhas, MD: Let’s transition to our first patient case. This is a 68-year-old woman who presented to her primary doctor with complaints of fatigue, increased bone pain, and abdominal discomfort that had evolved over the prior 2 months. Her past medical history was notable for hypertension and hyperlipidemia. On examination, she had a spleen that was palpable 4 cm below the left costal margin. Her laboratory results showed that her hemoglobin was 8.3 g/dL, her platelet count was 47,000 per mm3, and her white blood cell count was 23,000 per mm3. She didn’t have blasts on the peripheral blood smear, but she had some early myeloid forms.

A bone marrow biopsy was done, which showed a hypercellular bone marrow, about 80%. There was an increase in atypical megakaryocytes that were clustered. She had bone marrow fibrosis grade 3 out of 3. There was no increase in blasts by immunohistochemical staining, and her aspirate is dry. Someone checked a JAK2 V617F, which was 35%, but NGS [next-generation sequencing] wasn’t done on this patient. The diagnosis of primary myelofibrosis was provided and the patient so far is treatment naive.

This is a patient who presented with primary myelofibrosis, which is a common presentation. Patients will present with protean symptoms of bone pain, weight loss, usually fatigue, symptoms of anemia, and sometimes night sweats and fevers. Sometimes it presents without any discernible symptoms and can be found during routine laboratory evaluation for a screening procedure or something that’s routine in general health maintenance. It’s very variable in how patients present, and their courses are very variable. It’s a very heterogeneous group of patients. There isn’t a 1-size-fits-all description of what a patient with primary myelofibrosis looks like, but the symptoms can be quite varied.

In this patient, if you look at the clinical variables—Srdan went through them earlier—things that have independent prognostic significance include age, elevation of white blood cell count, the presence of peripheral blood blasts, systemic symptoms, and anemia, which is a very significant prognostic marker. By the Dynamic International Prognostic Scoring System [DIPSS], this patient would be intermediate-2. The DIPSS-Plus, which incorporates abnormal cytogenetics, red-cell transfusion dependence, and thrombocytopenia, would also be DIPSS-2. This patient would have somewhere between a 3- to 4-year median survival.

It’s important to pause here to remind the audience that it doesn’t tell the patient specifically what the duration of their life will be. As Srdan said, it helps segregate patients broadly into groups, because our treatment approach is a risk-adapted treatment approach. In this intermediate-2–risk patient with a survival of potentially less than 5 years, something like transplant would be a reasonable consideration unless there was something clearly contraindicated.

Transcript edited for clarity.