Compassionate use of sorafenib in patients with advanced renal cell cancer

In 2005, a phase III trial demonstrated a significant increase in progression-free survival in patients with renal cell cancer (RCC) treated with sorafenib versus placebo. While awaiting the full review by the US Federal Drug Administration, we initiated a treatment protocol as a mechanism for providing sorafenib to patients with advanced RCC but who were ineligible for other sorafenib clinical trials, also known as "compassionate use." In December 2005, sorafenib became commercially available, and this protocol was closed. Herein, we report our single-institution experience with this study.

Eligibility criteria included adults with advanced RCC with adequate organ function and performance status (PS). Treatment consisted of sorafenib 400 mg orally twice a day.

We enrolled 14 patients. The median age was 64 years, and PS was 2. All had metastatic RCC that had progressed after a median of 2 therapies. One patient (7%) had a partial response, and 3 (21%) had stable disease, for a clinical benefit rate of 29%. Severe toxicities included 1 patient with each of grade 4 thrombocytopenia, grade 3 warfarin-induced coagulopathy (drug-to-drug interaction), hypertension, diarrhea, anorexia, nausea, rash, and headache. Five subjects received concomitant radiation therapy without unexpected toxicities.

Sorafenib has modest activity in patients with advanced RCC who have progressed after multiple therapies.