Round table discussion on optimal clinical trial design in precursor multiple myeloma Journal Article
| Authors: | Ghobrial, I. M.; Gormley, N.; Kumar, S. K.; Mateos, M. V.; Bergsagel, P. L.; Chesi, M.; Dhodapkar, M. V.; Dispenzieri, A.; Fonseca, R.; Getz, G.; Kastritis, E.; Kristinsson, S. Y.; Martinez-Climent, J. A.; Manier, S.; Marinac, C. R.; Maura, F.; Morgan, G. J.; Davies, F. E.; Nadeem, O.; Nuvolone, M.; Paiva, B.; O’Donnell, E.; Prosper, F.; Shah, U. A.; Sklavenitis-Pistofidis, R.; Sperling, A. S.; Vassiliou, G. S.; Munshi, N. C.; Castle, P. E.; Anderson, K. C.; San Miguel, J. F. |
| Article Title: | Round table discussion on optimal clinical trial design in precursor multiple myeloma |
| 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. ©2024 American Association for Cancer Research. |
| Keywords: | cancer survival; overall survival; drug safety; treatment duration; clinical trials as topic; disease marker; cancer patient; research design; methodology; antineoplastic agent; progression free survival; quality of life; multiple myeloma; time; risk assessment; cancer regression; minimal residual disease; high risk population; retreatment; organ injury; randomized controlled trial (topic); clinical trial (topic); study design; procedures; humans; human; article; treatment free survival; biochemical progression free survival; diversity, equity and inclusion; precursor multiple myeloma |
| Journal Title: | Blood Cancer Discovery |
| Volume: | 5 |
| Issue: | 3 |
| ISSN: | 2643-3230 |
| Publisher: | American Association for Cancer Research |
| Date Published: | 2024-05-01 |
| Start Page: | 146 |
| End Page: | 152 |
| Language: | English |
| DOI: | 10.1158/2643-3230.Bcd-24-0022 |
| PUBMED: | 38441243 |
| PROVIDER: | scopus |
| PMCID: | PMC11061588 |
| DOI/URL: | |
| Notes: | Article -- Source: Scopus |
