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Defining disease modification in myelofibrosis in the era of targeted therapy
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 const...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9322520/ https://www.ncbi.nlm.nih.gov/pubmed/35499819 http://dx.doi.org/10.1002/cncr.34205 |
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author | Pemmaraju, Naveen Verstovsek, Srdan Mesa, Ruben Gupta, Vikas Garcia, Jacqueline S. Scandura, Joseph M. Oh, Stephen T. Passamonti, Francesco Döhner, Konstanze Mead, Adam J. |
author_facet | Pemmaraju, Naveen Verstovsek, Srdan Mesa, Ruben Gupta, Vikas Garcia, Jacqueline S. Scandura, Joseph M. Oh, Stephen T. Passamonti, Francesco Döhner, Konstanze Mead, Adam J. |
author_sort | Pemmaraju, Naveen |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-9322520 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-93225202022-07-30 Defining disease modification in myelofibrosis in the era of targeted therapy Pemmaraju, Naveen Verstovsek, Srdan Mesa, Ruben Gupta, Vikas Garcia, Jacqueline S. Scandura, Joseph M. Oh, Stephen T. Passamonti, Francesco Döhner, Konstanze Mead, Adam J. Cancer Review Articles 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. John Wiley and Sons Inc. 2022-05-02 2022-07-01 /pmc/articles/PMC9322520/ /pubmed/35499819 http://dx.doi.org/10.1002/cncr.34205 Text en © 2022 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society https://creativecommons.org/licenses/by-nc/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. |
spellingShingle | Review Articles Pemmaraju, Naveen Verstovsek, Srdan Mesa, Ruben Gupta, Vikas Garcia, Jacqueline S. Scandura, Joseph M. Oh, Stephen T. Passamonti, Francesco Döhner, Konstanze Mead, Adam J. Defining disease modification in myelofibrosis in the era of targeted therapy |
title | Defining disease modification in myelofibrosis in the era of targeted therapy |
title_full | Defining disease modification in myelofibrosis in the era of targeted therapy |
title_fullStr | Defining disease modification in myelofibrosis in the era of targeted therapy |
title_full_unstemmed | Defining disease modification in myelofibrosis in the era of targeted therapy |
title_short | Defining disease modification in myelofibrosis in the era of targeted therapy |
title_sort | defining disease modification in myelofibrosis in the era of targeted therapy |
topic | Review Articles |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9322520/ https://www.ncbi.nlm.nih.gov/pubmed/35499819 http://dx.doi.org/10.1002/cncr.34205 |
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