Identifying the Signs and Symptoms of Myelofibrosis
Expert panelists provide an overview of the signs and symptoms indicative of myelofibrosis and consider factors that help to inform prognostication.
Transcript:
John O. Mascarenhas, MD: Welcome to this Cancer Network® presentation titled, “Advances in the Field of Myelofibrosis.” I’m your host, John Mascarenhas. I’m the director of the adult leukemia program and the leader of clinical investigation within the myeloproliferative disorder program at Mount Sinai [Hospital] in New York [New York]. I’m joined by my friends and colleagues. Gabby Hobbs, please introduce yourself.
Gabriela S. Hobbs, MD: Hi, everybody. I’m thrilled to be here. My name is Gabby Hobbs. I’m the clinical director of the leukemia service at the Massachusetts General Hospital in Boston, and I treat patients with myeloproliferative neoplasms [MPNs] in clinic.
John O. Mascarenhas, MD: Welcome, Gabby. Thanks for joining us. Kristen?
Kristen M. Pettit, MD: Hi, everyone. I’m Kristen Pettit. I’m a clinical associate professor of medicine at the University of Michigan and focus on MPN clinical treatment and clinical research.
John O. Mascarenhas, MD: Thanks for joining us, Kristen. Andrew?
Andrew T. Kuykendall, MD: Hi, I’m Andrew Kuykendall. I’m an assistant member at Moffitt Cancer Center in Tampa, Florida, a clinical researcher focusing on myeloproliferative neoplasms.
John O. Mascarenhas, MD: Fantastic. I’m excited to have this panel for this discussion. We’re going to review 2 patient scenarios, share our insights, and discuss recent updates in the management of patients with myelofibrosis. Let’s start. The first question we’re going to ask is what are the main signs and symptoms of myelofibrosis? I’ll open this up to Andrew, who’s on the left side of my screen. How do patients present? What do they look like?
Andrew T. Kuykendall, MD: With myelofibrosis, the interesting thing is that they don’t have to present in 1 set way; patients present in a variety of standpoints. You have this classic patient with myeloproliferative neoplasm who presents with a lot of symptoms, called constitutional symptoms: fevers, chills, night sweats, bone pain, weight loss, and abdominal pain related to splenomegaly or organomegaly. The classic thought we have with patients with myelofibrosis is that they come in with some of these symptoms. Additionally, patients could present with abnormal lab counts, done by their primary care physician, with unexplained anemia or immature blood cells floating around. That might clue in physicians to do a more extensive work-up and to figure out exactly what’s going on. There isn’t 1 set way to present. Patients can present in a variety of ways.
John O. Mascarenhas, MD: Fantastic, agreed. It’s pretty heterogeneous how patients present and behave with these diseases. In today’s discussion, we’re going to make it clinically relevant but also bring in emerging data to make it evidence based. There were some abstracts at recent meetings, like ASCO [American Society of Clinical Oncology Annual Meeting], elucidating or identifying clinical features and symptom burden. I’m wondering if Kristen or Gabby wants to comment on the association between thrombocytopenia and symptoms.
Kristen M. Pettit, MD: Sure, we both probably could. Not surprisingly, anemia and thrombocytopenia have been shown to be associated with several aspects of myelofibrosis and worse overall survival for patients with higher risk of leukemic transformation. Thrombocytopenia is also associated with worse symptom burden, which isn’t surprising given what we see clinically in our patients on a day-to-day basis.
John O. Mascarenhas, MD: Excellent. Gabby, anything you want to add in terms of the relationship between cytopenias and symptoms?
Gabriela S. Hobbs, MD: One question I get all the time from patients is, “If my counts look better, why don’t I feel better? If my counts are controlled, why are my symptoms still present?” One thing we know in clinical practice and from some of the abstracts highlighted is that blood counts don’t tell the whole story. It’s interesting to think about patients who aren’t transfusion dependent but still have tremendous fatigue. There’s a lot that we need to understand in terms of what cytokines are driving those symptoms and how we make those better. Is it that we need to target the anemia so that they’re not short of breath and they have symptoms of anemia resolved? Or is something else contributing to those symptoms? We don’t have great measures, other than the MPN Symptom Assessment Form, which is a subjective way of making it less subjective. But the burden of symptoms is out of proportion to what’s going on from a CDC [Centers for Disease Control and Prevention] perspective.
John O. Mascarenhas, MD: Absolutely. Sometimes, if you get a patient’s hemoglobin up to a respectable number and they continue to complain about fatigue and the anemia-related symptoms, it’s clearly not all about blood cancer. The more recent analyses that link isolated thrombocytopenia or severe thrombocytopenia with certain anemia-related symptoms like fatigue or even spleen-related symptoms. The idea is that thrombocytopenia might be a more predictive laboratory feature that associates with the symptom burden. It’s fascinating and tells us that there are hematologic surrogates for advanced disease, and thrombocytopenia is 1 of them. There’s a lot of focus on anemia these days, trying to alleviate anemia with novel therapies, whether they’re active in ligand traps or novel JAK inhibitors that have ACVR1 inhibition. Some recent studies have also pointed out that there’s a lot of health care resource utilization when patients are anemic, particularly transfusion dependent. A lot of that is beyond actual transfusions. They spend a lot more time in the hospital, and those patients are defining a vulnerable and symptom burden and health care resource utilization–burdened population that’s in need of effective therapy. Anemia, thrombocytopenia, and their effects on the disease state and the burden of disease, both from a symptom standpoint and a health care resource utilization standpoint.
Transcript edited for clarity.
