Evaluation of Safety, Tolerability and Activity of Temsirolimus in Patients with Advanced or Metastatic Renal Cell Carcinoma (A/MRCC) in the Usual Health Care Setting

ABSTRACT Introduction Temsirolimus (TEMS), an i.v. mTOR inhibitor, is approved in the EU for the first-line treatment of patients with a/mRCC who have at least 3 of 6 prognostic risk factors. A pivotal study had demonstrated significantly increased overall survival with TEMS in poor risk patients compared to the former standard Interferon (10.9 vs. 7.3 mo; p = 0.0078). To better identify the safety profile and efficacy of TEMS during clinical routine, collection of data in a postapproval non-interventional trial seems to be adequate. Methods A registry for a/mRCC patients treated with TEMS was started in Germany in January 2008 (NCT00700258). Primary objective: evaluation of the safety profile of TEMS. Secondary objectives: tolerability and anti-tumor activity of TEMS; profile of patients; sequence of systemic therapies. Inclusion criteria: histologically confirmed a/mRCC treated with TEMS and written informed consent by the patient. Results 118 active study centers recruited 455 patients from Feb. 2008 to April 2012. Preliminary data are available for 430 patients: 68.7% male, median age 68 years, median Karnofsky index 80%. Histological subtype: 75.3% clear cell, 10.9% papillary and 2.6% chromophobe RCC. According to modified MSKCC criteria 96.0% of patients (n = 323) were classified as poor risk and 4.0% as intermediate risk patients. Adverse events (AE) and serious adverse events (SAE) defined as drug related were observed in 41.2% and 10.2%, respectively. Skin disorders, fatigue, nausea, diarrhea, peripheral edema, anemia and dyspnea of any grade were the most frequent AEs. Median progression-free survival for the total patient population was 151 days (d), for the subgroup of 1st line patients 162 d, for patients ≥ 65 yrs 155 d. Median overall survival for all patients was 381 d. Conclusions Patient population in the registry represents the expected pattern in a/mRCC regarding distribution of age, sex, and histology. Safety profile and clinical efficacy of TEMS in the usual health care setting confirm the phase-III data. In addition, for patients ≥ 65 years safety and clinical efficacy of TEMS are comparable to results of the overall study population. Disclosure U. Mueller: Employment by Pfizer. G. Krekeler: Employment by Pfizer. L. Bergmann: Advisory boards: Bayer, GSK, Novartis, Pfizer, Roche, Astellas; Honoraria: Novartis, Pfizer, Roche. D. Kalanovic: Employment by Pfizer. All other authors have declared no conflicts of interest.