MPN Workshop of the Carolinas 2025 | Time without transfusion reliance: quantifying transfusion independence in myelofibrosis

Anemia is a clear burden for patients with myelofibrosis, less oxygen-carrying capacity, and all the impact that has on activities of daily living, you know, cardiovascular health, on and on. When someone is needing transfusions, in addition to that severe decrease in oxygen-carrying capacity. There is a real impact of undergoing transfusions. Each transfusion, both is a significant investment of time and hassle. You know, let’s say the patient sees you in clinic on Wednesday, they need a transfusion. You’re able to arrange for that maybe Wednesday, maybe Thursday, they have to get cross-matched. That’s gonna take X number of hours. They may need to come back to the center to get the second unit, if the center didn’t have enough room to give them both units. They get diuretics that might give them additional side effects. After they receive it, they may not feel well for a couple days. So each transfusion is this multi-day arc that really is a burden to patients. Additionally, when someone is needing even intermittent transfusions, their hemoglobin is really doing this. It’s going up, they get transfused, but then that kind of drifts down, you know, until they’re really anemic, and then they kind of go up again, and then they drift down. So they spend a lot of time really being more anemic than is possible. 

So this analysis of time without transfusion really shows, you know, what is the duration of that period which we want. So one, a stable hemoglobin that doesn’t need transfusion. So they’re not doing this, so they have a much more even keel in terms of how they’re feeling. And two, their burden of heavily being tied to the healthcare system is much less. Because if their hemoglobin is stable at a hemoglobin of 9 grams per deciliter, they’re not needing to come in. It’s not going to take four days. Again, you may be able to check that blood count at home, and if they feel well, they don’t need to come into the clinic at all. So that difference is huge. So again, we look at it as another lens. How do we quantify really period or weeks of time that the individual really is having much less burden of healthcare, feeling better, and probably has an impact in terms of less cardiovascular risk burden, and mortality by truly being too anemic for longer periods of time. You know, we have data that individuals that achieve transfusion independence from momelotinib had a better survival than those that didn’t. You know, now the mechanism of the improvement in survival, none of us know for certain. But one of it is possible that, again, the cardiovascular burden of having your hemoglobin going up and down like a yo-yo, you know, is real, and that stability has value and that analysis I think is one lens on how do we quantify that.

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