Cancer Res Treat.  2017 Apr;49(2):350-357. 10.4143/crt.2016.067.

Efficacy and Safety of Regorafenib in Korean Patients with Advanced Gastrointestinal Stromal Tumor after Failure of Imatinib and Sunitinib: A Multicenter Study Based on the Management Access Program

• Son MK
• Ryu MH
• Park JO
• Im SA
• Kim TY
• Lee SJ
• Ryoo BY
• Park SR
• Kang YK

• KMID: 2378107
• DOI: http://doi.org/10.4143/crt.2016.067

Abstract

PURPOSE
The aim of this study was to confirm the efficacy and safety of regorafenib for advanced gastrointestinal stromal tumors (GISTs) reported in the GRID phase III trial in Korean patients.
MATERIALS AND METHODS
Fifty-seven Korean patientswith advanced GISTwho experienced both imatinib and sunitinib failure were enrolled in the management access program between December 2012 and November 2013 and treated with regorafenib (160 mg orally once daily in a 3 weeks on/1 week off).
RESULTS
None of the patients achieved a complete or partial response while 25 patients (44%) showed stable disease for ≥ 12 weeks. With a median follow-up of 12.7 months (range, 0.2 to 27.6 months), the median progression-free survival and overall survival were 4.5 months (95% confidence interval [CI], 3.8 to 5.3) and 12.9 months (95% CI, 8.1 to 17.7), respectively. Interestingly, 15 patients (26%) experienced an exacerbation of their cancer-related symptoms (abdominal pain in eight and abdominal distension in five) during the rest period for regorafenib, but all were ameliorated upon the resumption of regorafenib. The most common grade 3 or 4 adverse event was a hand-foot skin reaction (25%). The regorafenib dose was reduced in 44 patients (77%) due to toxicity, which manifested mainly as a hand-foot skin reaction (n=31).
CONCLUSION
This study confirmed the efficacy and safety of regorafenib for advanced GIST after imatinib and sunitinib failure in Korean patients. Considering the exacerbation of the cancer-related symptoms observed during the rest periods, further exploration of the continuous dosing schedule of regorafenib is warranted in future clinical trials.

Keyword

Regorafenib; Gastrointestinal stromal tumors; Management access program; Failure to imatinib; Sunitinib

MeSH Terms

Appointments and Schedules
Disease-Free Survival
Follow-Up Studies
Gastrointestinal Stromal Tumors*
Humans
Imatinib Mesylate*
Skin
Imatinib Mesylate

Figure

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  Fig. 1. Progression-free survival (PFS) (A) and overall survival (OS) (B) for the entire study population.

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  Fig. 2. Progression-free survival (PFS) by liver metastasis (A), overall survival (OS) by liver metastasis (B), PFS by primary tumor genotype (C), and OS by primary tumor genotype (D). The p-values were obtained using the log-rank test.

Reference

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