Criteria and assessments that can be used to help clinicians select treatment and monitor patients on therapy for myelofibrosis.
Pankit Vachhani, MD: For every patient I see with myelofibrosis, I make sure I do a risk stratification at the time of diagnosis and then subsequently follow-up using DIPSS [Dynamic International Prognostic Scoring System] or DIPSS Plus risk stratification schemas, so I can track their progress and see how they are doing. That is a crucial component: risk stratifying them. The second is symptom assessment. Frequently, our patients give a picture of being asymptomatic, or we don’t assess symptoms thoroughly enough to elucidate every symptom they may have. If one uses a symptom assessment form, for example the MPN-SAF TSS [Myeloproliferative Neoplasm-Symptom Assessment Form Total Symptom Score], one may find more patient symptoms than what would appear initially. It’s also important to get a rough estimation of all their symptoms and the severity. Those are 2 crucial things: No. 1, risk stratifying, and No. 2, identifying their symptoms and burden. In addition, I find it useful to obtain an ultrasound or a CT-guided assessment of their spleen length, especially in patients for whom I’m not able to palpate their spleen thoroughly for different anatomical reasons. Once I have done that, my treatment paradigm is further elucidated based on what their risk stratification schema is, and what their symptoms are. I target each of these components at the same time.
What is the best approach for monitoring response to therapy? For every patient I see, I obtain a CBC [complete blood count], a symptom assessment at every visit, and possibly a radiological assessment of their spleen size every few months. This is crucial. We want to keep track of how the patient is doing in terms of their symptoms and also take a look at their blood counts to see if they are having any signs of progression, stability, or adverse events from their treatments. In the event one were to see, for example, a drop in platelet count or hemoglobin level, or a rise in white blood cell count, or an increase or new emergence of peripheral blood blasts, or for example, in the event of having worsening splenomegaly or new symptoms that emerge on stable doses of JAK inhibitors, one may be worried about a potential resistance or relapse/refractoriness to therapy. That is when the consideration for switching therapy to something else comes about. That something else could be in the form of hypomethylating agents or intensive chemotherapy, should the patient have had a definite progression of their underlying myelofibrosis, or to an alternative JAK inhibitor.
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