251 Molecular Profiling guided treatment in refractory solid tumors: Practical impact and clinical responses. Experience of a single center

Value of Multiplatform Molecular Profiling (MMP) of Tumors in Clinical Practice

“…In the remaining 4 studies (involving a total of 127 evaluable patients), other outcome measures were used such as response rate (complete response [CR] rate plus partial response [PR] rate), disease control rate (CR plus PR plus stable disease [SD] rate), and 1-year survival rate. MMP-guided treatment was associated with clinical benefit in a considerable proportion of patients (20%-73%) in these 4 studies as well 16,18,20,21 . Although the studies were consistent in demonstrating that MMP provides relevant biomarker information, modifies treatment decisions, and facilitates extension of PFS [13][14][15][16][17][18][19][20][21][22][23] , they were limited by relatively modest cohort sizes, and some also by their retrospective design.…”

“…MMP-guided treatment was associated with clinical benefit in a considerable proportion of patients (20%-73%) in these 4 studies as well 16,18,20,21 . Although the studies were consistent in demonstrating that MMP provides relevant biomarker information, modifies treatment decisions, and facilitates extension of PFS [13][14][15][16][17][18][19][20][21][22][23] , they were limited by relatively modest cohort sizes, and some also by their retrospective design.…”

“…Three studies from the US, Australia, and Israel reported on changes in treatment decisions between empiric selection (pre-MMP) and MMP-guided selection 15,17,19 . The change rate was high and ranged from 74% to 100% of cases 17,19,21 . All the studies reported on clinical benefit.…”

“…Following this pivotal study, the CMI MMP approach was incorporated into clinical practice, and reports describing additional analyses focusing on the utility of this approach were published/presented. Table 2 summarizes the Von Hoff study as well as 10 subsequent studies conducted worldwide (US, Austria, Australia, Israel, Lebanon, and Egypt) [13][14][15][16][17][18][19][20][21][22][23] . Some of these studies investigated advanced disease in specific tumor types such as gastric/esophageal 14 , pancreatic/pancreaticobiliary 15,16 , breast 17 , and salivary glands cancer 18 , whereas other studies included a variety of refractory solid cancers including rare tumors [19][20][21][22][23] .…”

“…Table 2 summarizes the Von Hoff study as well as 10 subsequent studies conducted worldwide (US, Austria, Australia, Israel, Lebanon, and Egypt) [13][14][15][16][17][18][19][20][21][22][23] . Some of these studies investigated advanced disease in specific tumor types such as gastric/esophageal 14 , pancreatic/pancreaticobiliary 15,16 , breast 17 , and salivary glands cancer 18 , whereas other studies included a variety of refractory solid cancers including rare tumors [19][20][21][22][23] . Overall, 345 patients were evaluable in the 11 studies combined.…”