Myelofibrosis Caregiver Interview Series: Monitoring Myelofibrosis Treatment Effectiveness

Monitoring treatment response in myelofibrosis requires a balanced assessment of patient-reported symptoms, objective scoring tools, hematologic parameters, and spleen size. In part 1 of this Myelofibrosis Caregiver Interview Series, Fadi Haddad, MD, outlines a practical, real-world approach to evaluating therapeutic effectiveness and determining when to adjust management strategies.

TRANSCRIPT

Tim F. How do you monitor treatment effectiveness over time?

Fadi Haddad, MD: Once I start a treatment for myelofibrosis, I rely on several parameters to determine if the treatment is working, yes or no. First, I rely on the patient's symptoms and this could be assessed subjectively whenever I meet the patient in the clinic. I ask them, "Hey, how have you been for the past two weeks or since the last visit? Do you feel any improvement in your symptoms?" And usually those are like fatigue, night sweat, weight loss. So patients are usually able to tell me, "Oh, it's the same. Oh, I feel slightly better. I'm more energetic." But in parallel, I also rely on objective scoring system that we use in the clinic where patients fills a checklist and I have a score at the end. And this score, I can track it over time and understand if a patient is doing better from a symptom perspective, yes or no.

As long as the patient is deriving benefit, I continue with the same treatment. If I feel that patient is getting worse, the symptoms are not improving after certain time on therapy, then I may discuss with the patient changing to a different drug or shifting strategies. I also monitor the blood tests over time, the red cells, the white cells, and the platelets. And if I see that these numbers are improving, maybe the anemia getting better, the platelets getting better over time, even if they are not completely normal yet, then this is indicative to me that the treatment is working. If on the other hand, the numbers are the same, maybe getting worse, then this may tell me that the treatment is not really working or it's maybe toxic and this could guide me to maybe change the treatment. I rely also on the spleen size. Now, not all the patients have an enlarged spleen, but some patients do have an enlarged spleen that can cause abdominal pain, that can cause early society, difficulty when they eat.

And once we start a treatment, after sometimes patients do feel some improvement, they feel their spleen size is shrinking, they can eat better, they have less pain, then this is indicative for me that the treatment is actually working. Finally, I always tell my patient that it may take some time before they see the effect of the medication. It's not like we start treatment today and tomorrow you're going to feel better, but also the experience of each patient is very individualized and very different than others.

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