Dr Gerds on Unmet Needs in Low-Risk Myelofibrosis

Aaron Gerds, MD, assistant professor, medicine, Hematology and Medical Oncology, deputy director, Clinical Research, Cleveland Clinic Taussig Cancer Institute, discusses unmet needs for patients with low-risk myelofibrosis, including needs observed during the prospective, observational MOST trial (NCT02953704), which collected disease progression data for patients with low- or intermediate-1–risk myelofibrosis.

Findings presented at the 2024 EHA Congress showed that 58.5% of patients with low- or intermediate-1–risk myelofibrosis enrolled in cohort A (n = 205) met at least 1 criterion for disease progression after a median follow-up of less than 53 months. Among patients who experienced progression in cohort A, 64 (53.3%) met 1 criterion for progression, 27 (22.5%) met 2 criteria, and 29 (24.2%) met at least 3 disease progression criteria. A hemoglobin level of less than 10 g/dL (47.5%) and a platelet count of less than 100 x 109/L (31.7%) marked the most common progression criteria met in this cohort.

When considering timelines for patients with low-risk myelofibrosis, time to progression is generally thought of in terms of years rather than months, Gerds says; however, findings showed the majority of patients in cohort A experienced disease progression during the study.

Although patients with low-risk myelofibrosis may initially present with mild symptoms or be relatively asymptomatic, the risk of disease progression remains, particularly within the first year following diagnosis, and these patients need to be closely monitored, Gerds explains. He emphasizes the need for close observation to detect early signs of progression and symptoms for patients with low-risk myelofibrosis.

Along with more vigilant surveillance, Gerds advocates for the development of therapies that can alter the disease progression course for patients with low-risk myelofibrosis. Given the proportion of patients whose disease progressed during the MOST trial, these findings point to the need for additional therapies that can alter the underlying disease, rather than simply managing symptoms, Gerds concludes.