Defining disease modification in myelofibrosis in the era of targeted therapy

被引:38
|
作者
Pemmaraju, Naveen [1 ]
Verstovsek, Srdan [1 ]
Mesa, Ruben [2 ]
Gupta, Vikas [3 ]
Garcia, Jacqueline S. [4 ]
Scandura, Joseph M. [5 ,6 ]
Oh, Stephen T. [7 ]
Passamonti, Francesco [8 ]
Dohner, Konstanze [9 ]
Mead, Adam J. [10 ]
机构
[1] Univ Texas MD Anderson Canc Ctr, Dept Leukemia, Houston, TX 77030 USA
[2] UT Hlth San Antonio Canc Ctr, San Antonio, TX USA
[3] Univ Toronto, Princess Margaret Canc Ctr, Toronto, ON, Canada
[4] Dana Farber Canc Inst, Boston, MA 02115 USA
[5] Weill Cornell Med, Hematol Oncol, Dept Med, New York, NY USA
[6] New York Presbyterian Hosp, New York, NY USA
[7] Washington Univ, Sch Med, Dept Med, St Louis, MO 63110 USA
[8] Univ Insubria, Dept Med & Surg, Varese, Italy
[9] Univ Hosp, Dept Internal Med 3, Ulm, Germany
[10] Univ Oxford, Natl Inst Hlth Res, Weatherall Inst Mol Med, MRC,Mol Haematol Unit,Oxford Biomed Res Ctr, Oxford, England
关键词
bone marrow fibrosis; disease modification; myelofibrosis; myelofibrosis pathophysiology; targeted therapy; CHRONIC MYELOPROLIFERATIVE NEOPLASMS; POLYCYTHEMIA-VERA; RESPONSE CRITERIA; RUXOLITINIB; EFFICACY; SAFETY; COMBINATION; PROGENITOR; CONSENSUS; LEUKEMIA;
D O I
10.1002/cncr.34205
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
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.
引用
收藏
页码:2420 / 2432
页数:13
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