Managing patients with myelofibrosis (MF) who have both low blood cell counts and enlarged spleens is a bit like balancing on a bongo board while rolling down the side of a mountain.
If you lean too much toward reducing spleen size, your blood counts could tumble further. If you tip toward stable blood counts, then reducing the spleen becomes less effective. Having to continually weigh these priorities, physicians face a constant challenge when managing patients with cytopenic MF.
“The outcome is the worst for these patients,” says Srdan Verstovsek, MD, United Energy Resources, Inc. Professor of Medicine and hematologist-oncologist at MD Anderson Cancer Center in Houston, “because the ability to decrease the spleen and perhaps improve quality of life is limited by worsening production of blood cells in bone marrow.”
Other factors that predict a poor prognosis and are associated with cytopenias include red blood cell transfusions (anemia) and bleeding risks (thrombocytopenia). “There is a critical unmet need to adequately manage this population,” says Dr Verstovsek.
What really adds to the urgency is that cytopenic patients are most in need of aggressive therapy because they have the poorest prognosis in MF. Both enlarged spleen size and cytopenias may be markers for suboptimal outcomes.¹ There really is no wiggle room for dosing decisions that could lessen the impact on spleen reduction among those with low blood counts.
Patients who present with splenomegaly and low blood counts represent a troubling percentage of those with MF. Up to 54% of patients present with thrombocytopenia and/or anemia at diagnosis² (with incidence rates increasing as disease progresses³). And Dr Verstovsek estimates ~80% of patients with MF have enlarged spleens.
Bottom line, cytopenias plus splenomegaly represents a volatile combination in MF that may reduce survival rates—a critical reason why management strategies should be vigilant of compromises.