Related Experiment Video

Updated: Apr 14, 2026

Robotic Duodenal Sleeve Resection for Gastrointestinal Stromal Tumor with Rare Exon 8 KIT Mutation Following Neoadjuvant Imatinib
06:43

Robotic Duodenal Sleeve Resection for Gastrointestinal Stromal Tumor with Rare Exon 8 KIT Mutation Following Neoadjuvant Imatinib

Published on: April 3, 2026

92

Regorafenib treatment for advanced, refractory gastrointestinal stromal tumor: a report of the UK managed access

Attila Kollàr1, Marco Maruzzo2, Christina Messiou2

  • 1Department of Medical Oncology, University Hospital Bern, Inselspital, 3010 Bern, Switzerland.

Clinical Sarcoma Research
|April 24, 2015
PubMed
Abstract

Insights

Regorafenib shows encouraging safety and activity in advanced gastrointestinal stromal tumors (GIST) patients resistant to prior therapies. This multi-targeted tyrosine kinase inhibitor (TKI) demonstrated notable disease control and acceptable tolerability in routine clinical practice.

Area of Science:

  • Oncology
  • Pharmacology
  • Gastrointestinal Cancer Research

Background:

  • Tyrosine kinase inhibitors (TKIs) have transformed gastrointestinal stromal tumor (GIST) treatment.
  • Acquired resistance to first and second-line therapies remains a significant clinical challenge in GIST management.
  • Regorafenib, an oral multi-targeted TKI, offers a potential therapeutic option for patients with advanced GIST who have progressed on prior treatments.

Purpose of the Study:

  • To assess the safety and activity of regorafenib in patients with advanced GIST within a Managed Access Program (MAP).
  • To evaluate response rates, toxicity, treatment duration, and survival outcomes in a real-world clinical setting.

Main Methods:

  • Retrospective review of consecutive advanced GIST patients treated with regorafenib after progression or intolerance to imatinib and sunitinib.
  • Assessment of treatment response using RECIST and Choi criteria.
  • Grading of toxicities according to CTCAE v4.0 criteria.

Main Results:

  • All 20 patients achieved at least stable disease, with 2 partial responses (11%) by RECIST and 7 (39%) by Choi criteria.
  • Median progression-free survival (PFS) was 9.4 months and median overall survival (OS) was 12.2 months.
  • 50% of patients experienced grade 3 toxicities, and 55% required dose reduction; 3 patients discontinued treatment due to adverse events.

Conclusions:

  • Regorafenib demonstrates encouraging activity and tolerability in routine clinical practice for advanced GIST.
  • Adverse events observed are consistent with previously reported trial data.
  • The study supports the use of regorafenib in heavily pretreated GIST patients.
Keywords:
ChoiGastrointestinal stromal tumorMutationRegorafenib

More Related Videos

Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor07:21

Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor

Published on: May 2, 2022

2.7K
Establishment and Characterization of Three Afatinib-resistant Lung Adenocarcinoma PC-9 Cell Lines Developed with Increasing Doses of Afatinib09:38

Establishment and Characterization of Three Afatinib-resistant Lung Adenocarcinoma PC-9 Cell Lines Developed with Increasing Doses of Afatinib

Published on: June 26, 2019

8.5K

Related Concept Videos

Treatment Resistent Cancers02:56

Treatment Resistent Cancers

1.5K
Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...
3.9K
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
9.2K
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers01:26

Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers

Receptor tyrosine kinase inhibitors (TKIs) and calcium channel blockers (CCBs) are two critical categories of drugs employed in the treatment of pulmonary artery hypertension (PAH). PAH is a disease that causes high blood pressure in the pulmonary arteries, resulting in chest pain, fatigue, and shortness of breath.
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
662
Drugs for Treatment of Ulcerative Colitis in IBD01:29

Drugs for Treatment of Ulcerative Colitis in IBD

Ulcerative colitis is a chronic inflammatory condition primarily affecting the colon and rectum. The primary drugs used in the treatment of ulcerative colitis are aminosalicylates. They exhibit anti-inflammatory and immunosuppressive properties. They modulate inflammatory mediators and inhibit the activity of nuclear factor κB (NF-κB). Aminosalicylates also reduce inflammation by inhibiting prostaglandin and leukotriene production and decreasing neutrophil chemotaxis and superoxide...
667