Defining disease modification in myelofibrosis in the era of targeted therapy.

ABSTRACT: The development of targeted therapies for the treatment of myelofibrosis highlights a unique issue in a field that has historically relied on symptom relief, rather than survival benefit or modification of disease course, as key response criteria. There is, therefore, a need to understand what constitutes disease modification of myelofibrosis to advance appropriate drug development and therapeutic pathways. Here, the authors discuss recent clinical trial data of agents in development and dissect the potential for novel end points to act as disease modifying parameters. Using the rationale garnered from latest clinical and scientific evidence, the authors propose a definition of disease modification in myelofibrosis. With improved overall survival a critical outcome, alongside the normaliza

SUBMITTER: Pemmaraju N 

PROVIDER: S-EPMC9322520 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature