A multicenter phase II study of pazopanib in patients with advanced gastrointestinal stromal tumors (GIST) following failure of at least imatinib and sunitinib

Pazopanib in metastatic multiply treated progressive gastrointestinal stromal tumors: feasible and efficacious

“…One of them, is pazopanib, which is a potent VEGFR, PDGFR and kit inhibitor. Ganjoo et al in a small phase trial (13) showed that pazopanib was a well tolerated TKI with potential in progressive GIST with median PFS of 1.9 months and median OS of 10.7 months.…”

“…Commonly used in RCC (10) and soft tissue sarcomas (11), little was known of its activity against wild type KIT or drug resistant KIT mutations. However, a large phase 2 trial, PAZOGIST, and an earlier, smaller one by Ganjoo et al, evaluated the role of pazopanib in progressive GIST (post prior IM and sunitinib) and found a PFS benefit on comparison with best supportive care alone (12,13).…”

ACBI 2017 40TH Annual Conference

“…New diagnostic adjuncts such as SDHB immunohistochemistry 7 , has provided additional prognostic information and prediction of malignant risk but further biomarkers are needed. Furthermore there is a lack of effective treatments for malignant disease associated with SDH mutations 8,9 . This project aims to investigate mechanisms of SDH-associated tumourigenesis in order to i) identify biomarkers of malignant disease, ii) identify new methods to assess variant pathogenicity, iii) enable better genotype phenotype correlations and iv) to identify potential targeted therapies for this inherited neoplasia syndrome.…”

Cost-effectiveness of precision medicine in gastrointestinal stromal tumor and gastric adenocarcinoma

“…In regards to unresectable/metastatic GIST, other TKI's, such as sorafenib, dasatinib, nilotinib, ponatinib, and pazopanib, have been used in early phase trials and show some promise. If they are able to demonstrate favorable phase III results in the future, all of these agents have the possibility of being FDA approved, especially if they are shown to have activity in KIT wildtype GIST, where TKIs only produce modest response rates (78)(79)(80). All of these TKIs have been shown to have significant acquisition costs and treatment related costs related to rare but serious adverse effects.…”