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SOHO 2026 | High-risk CCUS as a distinct disease entity: implications for clinical trials
Zhuoer Xie, MD, MS, Moffitt Cancer Center, Tampa, FL, discusses findings from a recent study showing that patients with high-risk clonal cytopenia of undetermined significance (CCUS) have clinical features comparable with lower-risk myelodysplastic syndromes (MDS). Dr Xie highlights that, despite similar disease-related event rates, patients with high-risk CCUS are often excluded from clinical trials. She emphasizes that clinical trial eligibility should be guided by biological risk rather than diagnostic labels, viewing CCUS, MDS, and acute myeloid leukemia (AML) as a disease continuum. This interview took place at the 14th Annual Meeting of the Society of Hematologic Oncology (SOHO 2026) in Houston, TX.
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Transcript
So this is another very good question. So I would say yes to the clinical trial infrastructure, but I’d be more careful about the word entity. Is that, you know, what we found is that when we stratify patients with the CCUS by molecular risk rather than by the morphology, high-risk CCUS overlaps with lower-risk MDS in outcomes. But the reality is the awkward part is because the only thing separating those two groups is dysplasia...
So this is another very good question. So I would say yes to the clinical trial infrastructure, but I’d be more careful about the word entity. Is that, you know, what we found is that when we stratify patients with the CCUS by molecular risk rather than by the morphology, high-risk CCUS overlaps with lower-risk MDS in outcomes. But the reality is the awkward part is because the only thing separating those two groups is dysplasia. Morphology leaves the high-risk patients in a diagnostic gap where neither watchful waiting nor intervention is optimized, where clinical trials largely exclude them. So these are not the patients who are stable. They have disease-related events like infection, transfusion dependency, cardiovascular disease, leukemia transformation, similar to patients with a lower-risk MDS. So right now, the label determines the access more than measured risk does. It restricts the patients and slows innovation. So my argument to our sponsors and the regulators is straightforward, is that the eligibility should follow the risk and not the diagnostic label. But I don’t think we need a new WHO category to fix this. I think CHIP, CCUS, MDS, AML is a continuum. In our study, well, high-risk CCUS behaves very much like a lower-risk MDS. The survival is not statistically significant, but it might be limited by the power or the sample size. I’d say it is a continuum. So calling it a new disease entity is a classification move, though opening the clinical trial is the management move. That is the one that actually helps our patients. Thank you.
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