ASH 2025 | Biomarkers in ITP: understanding a patient’s immune phenotype and guiding therapeutic selection

Biomarkers have sort of been a very challenging area that have not led to much success in ITP in terms of helping us understand more about a patient’s specific underlying immune phenotype in ITP, whether they’re more platelet autoantibody focused in terms of their disease, more T-cell focused, right? We recognize that there are many different sort of underlying immune phenotypes that may respond better to certain therapies, but we don’t actually have a way right now of saying which patients are which in clinical practice. We did do a study a number of years ago that showed patients that have normal or only slightly elevated endogenous thrombopoietin levels did respond better to thrombopoietin receptor agonists. But again, most people are not going to be able or not going to want to check a thrombopoietin level necessarily before starting a patient on treatment because it’s a send-out test in most places. So we recognize that. So this is an area of ongoing investigation, and it’s an area of interest. But unfortunately, right now, it’s therapeutic selection based on shared decision-making, discussion with the patient, discussion about the mode of administration, how frequently the patient has to take the medicine, the potential side effects, and recognizing that there may be certain aspects of the patient, like, for example, if they have a strong thromboembolic history that might guide you toward one choice of medicine or another.

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