Cost Benefits, Comparable Survival Outcomes in HCC After Reduced Therapy Dosage


Patients with hepatocellular carcinoma (HCC) who took a reduced therapy dose had a lower cumulative pill cost and were less likely to discontinue treatment due to toxicities, according to a recent study.

The study also showed that lower doses did not have a significant effect on overall survival (OS).

Currently, the only Food and Drug Administration (FDA) approved first-line therapy for patients with advanced HCC is sorafenib (Nexavar, Bayer). Prior retrospective studies have indicated that using a sorafenib dosage escalation strategy may be beneficial to patients with HCC. However, further data is needed to determine influence on patient outcomes and costs.

Kim A Reiss, MD, Perelman Center for Advanced Medicine (Philadelphia, PA), and colleagues conducted a retrospective study to assess whether a reduced starting dosage of sorafenib affected OS or cost of treatment for patients with advanced HCC. A total of 4903 patients from the Veterans Health Administration were sampled, 3094 of whom (63%) received the standard dosage of 800 mg per day and the remaining 1809 patients (37%) received a reduced starting dosage.

Results of the study were published in the Journal of Clinical Oncology (online September 5, 2017; doi:10.1200/JCO.2017.73.8245).

On average, the reduced starting dosage for patients in that group was 367 mg per day. After 6 months, the average dosage remained lower in the reduced dosage group compared with the standard dosage group (573 vs 678 mg/day, respectively; P < .001).


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Despite both groups receiving a similar number of days of sorafenib therapy, patients in the reduced dosage group received fewer pills than those in the standard dosage group (median number of pills, 180 vs 276, respectively; P < .001), equating to a significantly lower total pill-related costs for the reduced dosage group (median pill-related costs, $5636 vs $8661; P < .001).

After matching 1675 patients from either group 1:1, researchers found that those who received reduced dosages were less likely to discontinue sorafenib therapy due to gastrointestinal adverse events (8.7% vs 10.8%, respectively; P = .047).

A subgroup analysis showed no significant difference in OS rates between the two groups.

The initiation of sorafenib therapy at reduced dosages was associated with reduced pill burden, reduced treatment costs, and a trend toward a decreased rate of discontinuing sorafenib because of adverse events,” researchers concluded.—Zachary Bessette