Year: 2024
Type: article
Abstract: SUMMARY: While the current approach to precursor hematologic conditions is to "watch and wait," this may change with the development of therapies that are safe and extend survival or delay the onset of symptomatic disease. The goal of future therapies in precursor hematologic conditions is to improve survival and prevent or delay the development of symptomatic disease while maximizing safety. Clinical trial considerations in this field include identifying an appropriate at-risk population, safety assessments, dose selection, primary and secondary trial endpoints including surrogate endpoints, control arms, and quality-of-life metrics, all of which may enable more precise benefit-risk assessment.
Source: Blood Cancer Discovery
Authors: Irene M. Ghobrial, Nicole Gormley, Shaji K. Kumar, Maria-Victoria Mateos, P. Leif Bergsagel, Marta Chesi, Madhav V. Dhodapkar, Angela Dispenzieri, Rafael Fonseca, Gad Getz, Efstathios Kastritis, Sigurdur Y. Kristinsson, Jose Angel Martinez-Climent, Salomon Manier, Catherine R. Marinac, Francesco Maura, Gareth J. Morgan, Faith E. Davies, Omar Nadeem, Mario Nuvolone, Bruno Paiva, Elizabeth O'Donnell, Felipe Prosper, Urvi A. Shah, Romanos Sklavenitis-Pistofidis, Adam S. Sperling, George S. Vassiliou, Nikhil C. Munshi, Philip E. Castle, Kenneth C. Anderson, Jesus F. San Miguel
Institutions: Dana-Farber Cancer Institute, United States Food and Drug Administration, Mayo Clinic, Consejo Superior de Investigaciones Científicas, Universidad de Salamanca, Instituto de Investigación Biomédica de Salamanca, Centro de Investigación del Cáncer, Mayo Clinic in Arizona, Emory University, Winship Cancer Institute, Broad Institute, Harvard University, National and Kapodistrian University of Athens, University of Iceland, Navarre Institute of Health Research, Clinica Universidad de Navarra, Centro de Investigación Médica Aplicada, Inserm, Université de Lille, Centre Hospitalier Universitaire de Lille, University of Miami, Sylvester Comprehensive Cancer Center, NYU Langone Health, NYU Langone’s Laura and Isaac Perlmutter Cancer Center, New York University, University of Pavia, Policlinico San Matteo Fondazione, Centro de Investigación Biomédica en Red de Cáncer, Memorial Sloan Kettering Cancer Center, Wellcome/MRC Cambridge Stem Cell Institute, University of Cambridge, Wellcome Sanger Institute, National Cancer Institute
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: green
Funder: National Cancer Institute
OpenAlex ID: W4392473413