Scientific publications
Round Table Discussion on Optimal Clinical Trial Design in Precursor Multiple Myeloma
Irene M Ghobrial 1 , Nicole Gormley 2 , Shaji K Kumar 3 , Maria-Victoria Mateos 4 , P Leif Bergsagel 5 , Marta Chesi 5 , Madhav V Dhodapkar 6 , Angela Dispenzieri 3 , Rafael Fonseca 5 , Gad Getz 7 , Efstathios Kastritis 8 , Sigurdur Y Kristinsson 9 , Jose Angel Martinez-Climent 10 , Salomon Manier 11 , Catherine R Marinac 1 , Francesco Maura 12 , Gareth J Morgan 13 , Faith E Davies 13 , Omar Nadeem 1 , Mario Nuvolone 14 15 , Bruno Paiva 10 , Elizabeth O'Donnell 1 , Felipe Prosper 16 17 , Urvi A Shah 18 , Romanos Sklavenitis-Pistofidis 1 7 , Adam S Sperling 1 , George S Vassiliou 19 20 21 , Nikhil C Munshi 1 , Philip E Castle 22 , Kenneth C Anderson 1 , Jesus F San Miguel 10
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.
CITATION Blood Cancer Discov. 2024 Mar 4:OF1-OF7. doi: 10.1158/2643-3230.BCD-24-0022
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