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Close observation versus upfront treatment in hepatocellular carcinoma: are the exception points worth the risk?
  1. Danielle M Tholey1,
  2. Ben Hornung2,
  3. Charles K Enestvedt3,
  4. Yiyi Chen4,
  5. Willscott S Naugler1,
  6. Khashayar Farsad5,
  7. Nima Nabavizadeh6,
  8. Barry Schlansky1,
  9. Joseph Ahn1,
  10. Janice H Jou1
  1. 1 Department of Gastroenterology and Hepatology, Oregon Health and Science University, Portland, Oregon, USA
  2. 2 Department of Internal Medicine, Oregon Health and Science University, Portland, Oregon, USA
  3. 3 Department of Abdominal Transplantation Surgery, Oregon Health and Science University, Portland, Oregon, USA
  4. 4 Division of Biostatistics, Oregon Health and Science University, Portland, Oregon, USA
  5. 5 Department of Interventional Radiology, Oregon Health and Science University, Portland, Oregon, USA
  6. 6 Department of Radiation Oncology, Oregon Health and Science University, Portland, Oregon, USA
  1. Correspondence to Dr Janice H Jou; jou{at}ohsu.edu

Abstract

Introduction To assess the outcomes of immediate LDT versus observation strategies for T1 hepatocellular carcinoma (HCC) with respect to progression beyond Milan and survival.

Method T1 HCCs were retrospectively reviewed from a multidisciplinary tumour board database between September 2007 and May 2015. In the observation group, T1 lesions were observed until the tumour grew to meet T2 criteria (=2 cm). The treatment group consisted of T1 lesions treated at diagnosis with liver directed therapy (LDT). Kaplan-Meier plots were constructed for tumour progression beyond Milan and overall survival.

Results 87 patients (observation n=56; LDT n=31) were included in the study. A total of 22% (n=19) of patients progressed beyond Milan with no difference in progression between treatment and observation groups (19% vs 23%, p=0.49). Median time to progression beyond Milan was 16 months. Overall transplantation rate was 22% (observation group n=16; treatment group n=3, p=0.04). Median survival was 55 months with LDT versus 36 months in the observation group (p=0.22). In patients who progressed to T2 (n=60), longer time to T2 progression was a predictor of improved survival (HR=0.94, 95% CI 0.88 to 0.99, p=0.03).

Conclusions Immediate LDT of T1 lesions was not associated with increased risk of progression beyond Milan criteria when compared with an observation approach. Longer time to T2 progression was associated with increased survival and may be a surrogate for favourable tumour biology.

  • hepatocellular carcinoma
  • liver transplantation
  • orthotopic liver transplantation
  • hepatoma
  • liver cirrhosis

This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/

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Footnotes

  • Contributors DMT, JHJ, CKE and WSN generated the hypothesis and study design with guidance from JA and KF. DMT was the primary author of the manuscript with assistance by BH. Data collection was conducted by BH and DMT. YC conducted the statistical analyses and assisted with revision of the manuscript. JHJ and CKE assisted with study implementation, provided expertise in guiding each stage of the study and revised and edited the manuscript. Manuscript revision and data analysis were also performed by JA, KF, WSN, NN and BS. All authors provided final approval of the article prior to submission.

  • Competing interests None declared.

  • Ethics approval IRB.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Data sharing statement No additional data are available for this paper.