Work

Metastatic and multiply relapsed SDH deficient GIST and paraganglioma displays clinical response to combined Poly ADP-Ribose Polymerase inhibition and Temozolomide

Year: 2022

Type: preprint

Abstract: Pediatric GIST (gastrointestinal stromal tumors) are mostly KIT/ PDGFRA-WT and harbor mutations in SDH (succinate dehydrogenase), making them TKI (tyrosine kinase inhibitor) resistant due to the absence of gain-of-function tyrosine kinase mutations. Due to rarity of GIST in children, there have been few clinical trials available for patients with advanced disease, resulting in limited treatment options and a lack of pediatric-specific consensus guidelines. Here, we report the case of a patient with progressive, metastatic SDHB-mutant GIST that displayed a significant clinical response with a combination of Olaparib and TMZ (temozolomide).

DOI: 10.22541/au.165544424.41884113/v1

Authors: Juan Vasquez, Charu Singh, Ranjit Bindra, Peter M. Glazer, Farzana Pashankar

Institution: Yale University

Language: English

Topic: Gastrointestinal Tumor Research and Treatment

Subfield: Gastroenterology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Funders: Doris Duke Charitable Foundation, Yale University, Yale Center for Clinical Investigation, Yale School of Medicine

Award: G3355976779

OpenAlex ID: W4283078190

Cites: 22

Cited by:

Related to: 10