Cancer Chemotherapy and Pharmacology

, Volume 80, Issue 5, pp 945–954 | Cite as

Regorafenib in advanced hepatocellular carcinoma (HCC): considerations for treatment

  • Kyung Kim
  • Reena Jha
  • Petra A. Prins
  • Hongkun Wang
  • Monica Chacha
  • Marion L. Hartley
  • Aiwu Ruth He
Original Article



We report our institutional observations of ten patients with advanced hepatocellular carcinoma (HCC) (seven and three were Child–Pugh class A and B, respectively) who received compassionate regorafenib therapy between June 2016 and January 2017. These patients did not fit the rigid criteria of a clinical trial and represented the use of regorafenib in an everyday clinic situation.


Regorafenib (160 mg P.O. daily) was administered to patients on a 4-week cycle (3 weeks on, 1 week off) until disease progression (assessed using mRECIST criteria) or discontinuation secondary to toxicity (assessed using CTCAE criteria). Relevant clinical data were abstracted from patient medical records and reviewed retrospectively.


The median duration of patient treatment was 6.6 weeks, and the median time to disease progression was 12.5 weeks. Most common treatment emergent adverse events were fatigue, diarrhea, and hand–foot skin reaction. Elevated AST and ALT were the most commonly observed laboratory-assessed adverse events, which reached grade 3 status in the Child–Pugh class B patients only. We observed intolerance to regorafenib treatment in one patient who had previously received a liver transplant. We also saw lithium toxicity in one patient receiving long-term lithium treatment, suggesting a potential and unexpected drug–drug interaction with regorafenib.


Taken together, our observations indicate that regorafenib is beneficial in the treatment of patients with advanced HCC who progressed on or demonstrated intolerance to sorafenib therapy; however, careful selection and close monitoring of patients is necessary to maximize the benefit while minimizing the toxicities of regorafenib treatment.


Hepatocellular carcinoma HCC Regorafenib Observation Pretreated 


Compliance with ethical standards

Conflict of interest

Dr. Aiwu Ruth He serves as a speaker in Bayer’s speaker’s program. All other authors have no conflicts of interest to report. There is no disclosure of potential conflicts of interest among all authors.

Research involving human participants and/or animals

The study involves medical chart review of patients. The study is carried out under IRB-approved protocol with the titile of “Establishment of the High Quality Tumor Biobank and Clinical Database (2007- 345)”.

Informed consent

Informed consent is waived for this retrospective medical chart review.


No funding is obtained for this study.


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Copyright information

© Springer-Verlag GmbH Germany 2017

Authors and Affiliations

  1. 1.Georgetown University HospitalWashington, DCUSA
  2. 2.Department of RadiologyGeorgetown University HospitalWashington, DCUSA
  3. 3.The Ruesch Center for the Cure of Gastrointestinal CancersWashington, DCUSA
  4. 4.Georgetown Lombardi Comprehensive Cancer CenterWashington, DCUSA
  5. 5.Department of Biostatistics, Bioinformatics, and BiomathematicsWashington, DCUSA

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