Compassionate use of sorafenib in patients with advanced renal cell cancer.
BACKGROUND
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.


METHODS
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.


RESULTS
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.


CONCLUSIONS
Sorafenib has modest activity in patients with advanced RCC who have progressed after multiple therapies.
Laber
Damian A
DA
Division of Hematology and Medical Oncology, University of Louisville, J.G. Brown Cancer Center, Louisville, KY 40202, USA. damianla@ulh.org
Mushtaq
Mian
M
D000284:Administration, Oral; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D002291:Carcinoma, Papillary / Q000188:drug therapy / Q000473:pathology; D002292:Carcinoma, Renal Cell / Q000188:drug therapy* / Q000473:pathology; D005260:Female; D006801:Humans; D007211:Indoles / Q000627:therapeutic use*; D007680:Kidney Neoplasms / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D008875:Middle Aged; D011758:Pyrroles / Q000627:therapeutic use*; D000077210:Sunitinib; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D007211:Indoles; D011758:Pyrroles; D000077210:Sunitinib