EHA 2023 | Approved and emerging agents to manage anemia and thrombocytopenia in myelofibrosis

Cytopenias in myelofibrosis are a difficult problem, particularly in those individuals with primary myelofibrosis. Over time, we’ve learned that these cytopenic myelofibrosis patients even have some important biological differences – they tend to be more likely to be non-JAK2, they tend to have more primary myelofibrosis, and we know that cytopenias are adverse prognostically. Now we fortunately have an evolving range of options.

First, pacritinib, which has been approved in the United States but hopefully soon here in Europe, for individuals with significant thrombocytopenia – that’s been validated through multiple studies, improvements in spleen symptoms – and in the US, there’s an indication for anyone with a platelet count of less than 50 thousand. Additionally, there’s information that pacritinib inhibits ACBR1, can improve anemia maybe in up to a third of patients, so certainly has benefit in both.

Secondly we have momelotinib, which may become approved in the US even as early as the end of June of 2023. And there, improvements in spleen symptoms, but also anemia. Last year’s EHA congress, we presented data from the MOMENTUM study, which showed in the second line setting for patients with significant anemia, it was vastly superior to danazol for improvements in spleen symptoms and also in anemia.

So I think increasing options, future state, we’ve got new agents on the horizon, potentially luspatercept that can help to improve anemia, there’s anemia benefit with pelabresib and other combination approaches. So, hopefully more options for cytopenic patients with myelofibrosis.