Dual Immunotherapy Received FDA Approval for Liver Cancer Treatment

On 10 March 2020, the US Food and Drug Administration (FDA) granted accelerated approval for the treatment of patients previously treated with sorafenib who had hepatocellular carcinoma (HCC), with the combination of the immunotherapy drugs nivolumab and ipilimumab (Opdivo and Yervoy). Note: Hepatocellular carcinoma (HepatoCellular Carcinoma = HCC) is the most common subtype of liver cancer and accounts for 75% of all liver cancers. The efficacy of this dual immunotherapy in liver cancer was investigated in CHECKMATE-040 Cohort 4, a multicenter, multicohort, open-label study that included patients with HCC whose disease had progressed on sorafenib or who could not tolerate sorafenib. A total of 49 patients received 1 mg / kg nivolumab with 3 mg / kg ipilimumab every 3 weeks for four doses, followed by single-agent nivolumab 240 mg every 2 weeks until disease progression or unacceptable adverse effects. The main efficacy outcome measures were overall response rate and duration of response.

• The overall response rate, including 4 complete responses and 12 partial responses, was 33%.
• Duration of response ranged from 4.6 to 30.5+ months, with 31% of responses lasting at least 24 months.

The most common (>20%) adverse effects with nivolumab in combination with ipilimumab were fatigue, diarrhea, rash, itching, nausea, musculoskeletal pain, pyrexia (fever), cough, decreased appetite, vomiting, abdominal pain, shortness of breath, upper respiratory tract infection, arthralgia, headache, hypothyroidism, weight loss and dizziness. - Related topic: What are the adverse effects of immunotherapy drugs, and how are they treated? For HCC, the recommended doses are 1 mg / kg nivolumab with concurrent 3 mg / kg ipilimumab every 3 weeks for the first 4 cycles, followed by nivolumab 240 mg every 2 weeks or 480 mg every 4 weeks. This FDA approval was granted as an "accelerated approval" based on overall response rate and duration of response. Continued approval for HCC may depend on verification and description of clinical benefit in confirmatory trials.