TY - JOUR
T1 - Defining disease modification in myelofibrosis in the era of targeted therapy
AU - Pemmaraju, Naveen
AU - Verstovsek, Srdan
AU - Mesa, Ruben
AU - Gupta, Vikas
AU - Garcia, Jacqueline S.
AU - Scandura, Joseph M.
AU - Oh, Stephen T.
AU - Passamonti, Francesco
AU - Döhner, Konstanze
AU - Mead, Adam J.
N1 - Publisher Copyright:
© 2022 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society.
PY - 2022/7/1
Y1 - 2022/7/1
N2 - 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 normalization of hematopoiesis and improvement in bone marrow fibrosis, there will be an increasing need for surrogate measures of survival for use in the early stages of trials. As such, the design of future clinical trials will require re-evaluation and updating to incorporate informative parameters and end points with standardized definitions and methodologies.
AB - 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 normalization of hematopoiesis and improvement in bone marrow fibrosis, there will be an increasing need for surrogate measures of survival for use in the early stages of trials. As such, the design of future clinical trials will require re-evaluation and updating to incorporate informative parameters and end points with standardized definitions and methodologies.
KW - bone marrow fibrosis
KW - disease modification
KW - myelofibrosis
KW - myelofibrosis pathophysiology
KW - targeted therapy
UR - http://www.scopus.com/inward/record.url?scp=85129204022&partnerID=8YFLogxK
U2 - 10.1002/cncr.34205
DO - 10.1002/cncr.34205
M3 - Review article
C2 - 35499819
AN - SCOPUS:85129204022
SN - 0008-543X
VL - 128
SP - 2420
EP - 2432
JO - Cancer
JF - Cancer
IS - 13
ER -