This is an early access version, the complete PDF, HTML, and XML versions will be available soon.
Real-World Study of Patients with Metastatic Colorectal Cancer and Long-Term Response to Regorafenib in the USA
by
and
Richard D. Kim
1,*, 
Xiaoyun Pan
2,
Yiqiao Zhang
2,
Orsolya Lunacsek
2,
Federica Pisa
3,
Helene Ostojic
4 and
Marc Peeters
5 1
Department of Gastrointestinal Oncology, Moffitt Cancer Center, Tampa, FL 33612, USA
2
Real World Evidence Oncology, Bayer HealthCare Pharmaceuticals Inc., Whippany, NJ 07981, USA
3
Real World Evidence Oncology, Bayer AG, 13353 Berlin, Germany
4
Bayer Consumer Care AG, 4052 Basel, Switzerland
5
Center for Oncological Research, Antwerp University Hospital, 2650 Edegem, Belgium
*
Author to whom correspondence should be addressed.
Cancers 2025, 17(19), 3196; https://doi.org/10.3390/cancers17193196
Submission received: 27 June 2025
/
Revised: 17 September 2025
/
Accepted: 28 September 2025
/
Published: 30 September 2025
(This article belongs to the Section Cancer Metastasis)
Simple Summary
The research conducted in this study looked at treatment with regorafenib—a drug given to patients with advanced colon cancer that has spread to other parts of the body. The main objective was to determine which type of patients benefit from this drug for a long time, including demographics such as age and ethnicity, health status, and certain biological markers in their cancer cells. The study found some patients took regorafenib for at least four, five, or six months, and these patients had common characteristics, such as their health status, the side of the body where the colon cancer started, and whether disease had spread to the liver. Most had also been previously treated with the drug bevacizumab. The findings showed that patients can experience long-term benefit from regorafenib, and this information could help doctors predict who may have a better chance of responding well to this drug.
Abstract
Background: Several narrative reports document long-term responses to regorafenib treatment in patients with metastatic colorectal cancer (mCRC). However, no large-scale study has assessed long-term responses and there are no established predictors of potential long-term benefit. We carried out an observational study of characteristics of patients treated in real-world clinical practice in the USA using duration of treatment (DoT) as a surrogate for treatment response. Patients and Methods: This retrospective cohort study used a de-identified electronic health record-derived database and included patients aged ≥18 years with mCRC who initiated regorafenib monotherapy between 1 July 2013 and 30 June 2023. Patient cohorts were defined by DoT ≥4 months (LTR4), ≥5 months (LTR5), or ≥6 months (LTR6) and are not mutually exclusive. Results: Of 2444 patients who initiated regorafenib monotherapy during the study, those with long-term response were analyzed: 544 had LTR4 (22%), 367 had LTR5 (15%), and 250 had LTR6 (10%). Most patients with long-term responses had left-sided tumors (65–70%), Eastern Cooperative Oncology Group performance status of 0/1 (67–68%), and liver metastases (55–61%) and had received prior bevacizumab treatment (60–67%). The median age in each group was 66 years, and patients most frequently initiated regorafenib as third-line treatment (31–33%). Median time to regorafenib discontinuation was 6.0–9.3 months among long-term responders. Conclusion: Most patients with long-term responses to regorafenib had favorable performance status at treatment initiation, left-sided tumors, and liver metastases and had received prior bevacizumab treatment. The study highlights that patients in the real-world setting were able to tolerate and maintain long-term responses to regorafenib treatment.
