SOHO State of the Art Updates and Next Questions | Early Intervention in Myelofibrosis: Where Are We and Does It Matter?

ABSTRACT

Historically, therapeutic clinical trials in myelofibrosis have predominantly focused on targeting patients with higher-risk disease who are at risk of increased morbidity and mortality. The endpoints have been designed to target regularly measured disease parameters that are of immediate pertinence to patient's welfare including splenic volume reduction and symptom reduction. These efforts have resulted in meaningful and measurable improvements in disease parameters in these high-risk study populations and multiple FDA approved agents. However, they have not tackled specific interventions that may be applied to patients with earlier or less advanced disease state. In this review, we summarize evidence from completed and ongoing clinical trials investigating different aspects of intervention targeted at less advanced disease and advocate for the merit of this approach.