Simple Score to Select Sorafenib Treatment Candidates: A Report from the South American Liver Research Network

LEATHERS, JAMES S.; BALDERRAMO, DOMINGO; PRIETO, JHON E.; DIEHL, FERNANDO; GONZALEZ BALLERGA, ESTEBAN; FERREIRO, MELINA R.; CARRERA, ENRIQUE; BARREYRO, FERNANDO J.; FERRER, JAVIER DIAZ; SINGH, DUPINDER; DEBES, JOSE D.

San Francisco

Congreso; The Liver Meeting 2018; 2018

American Association for the Study of Liver Diseases

Background: There is limited knowledge about sorafenib use for hepatocellular carcinoma (HCC) in South America. In resource-limited settings, sorafenib may be the only option for treating HCC. Using a cohort from the South American Liver Research Network, we created a cost-effective prognostic scoring system to help identify patients likely to have a survival benefit on sorafenib treatment, using simple laboratory variables. Methods: We analyzed HCC cases treated with sorafenib from five countries within the South American Liver Research Network, between January 2010 and June 2017. The primary endpoint was overall survival (OS). In order to design the score, named PIB for Platelet-INR-Bilirubin (PIB), we assigned each patient in the sorafenib cohort, withavailable laboratory and survival data, one point for each of the following: 1) total bilirubin ≤ 3.0mg/dL, 2) platelets ≤ 250x 109/L, 3) INR ≤ 1.6. This yielded a hypothetical score range of 0 to 3. Cox proportional hazard regression and log-rank tests were used to assess the equality of survivor functions. A biostatistician was consulted for review of our data analysis. Statistical analysis was performed using STATA v14.2 (Statacorp, College Station, TX.) Results: Of 1336 evaluated patients, 127 were treated with sorafenib. The median age of individuals was 65 years (IQR 55-71) and 70% were male. Median survival was 8 months (IQR 2-17). Variables associated with survival were total bilirubin >/< 3mg/dl (2 vs.8.5 months, p = 0.001), INR >/< 1.6 IU (5 vs. 8 months, p =0.01), platelets >/< 250.000 mm3 (2 vs. 8 months, p = 0.01), Barcelona Clinic Liver Cancer (BCLC) Stage (A/B, 13 months; C/D, 6 months, p = 0.008) and presence of any sorafenib related side effects (11 vs. 2 months, p = 0.009). There were no patients with a PIB score of ?0?, five patients with a score of ?1?, 21 patients with a score of ?2? and 61 patients with a score of ?3?. Patients with a PIB score of 1 or 2 had a median survival of 2 months whereas patients with a PIB score of 3 had a median survival of 10.5 months. Conclusion: Platelets, INR, total bilirubin, BCLC stage and presence of side effects were associated with survival on sorafenib. The PIB score may be useful for the selection of sorafenib treatment candidates in resource-limited settings.