Round Table Discussion on Optimal Clinical Trial Design in Precursor Multiple Myeloma

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 NuvoloneBruno 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

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)146-152
Number of pages7
JournalBlood cancer discovery
Volume5
Issue number3
DOIs
Publication statusPublished - 1 May 2024

Bibliographical note

Publisher Copyright:
©2024 American Association for Cancer Research.

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