Prospective Observational Study of Pazopanib in Patients with Advanced Renal Cell Carcinoma (PRINCIPAL Study)

The oncologist

article

Manuela Schmidinger¹  Chaitali Babanrao Pisal²  Yulia Hirschberg³  Luca Dezzani³  Qasim Ahmad³  Eric Jonasch⁴  Aristotelis Bamias⁵  Giuseppe Procopio⁶  Robert Hawkins⁷  Angel Rodriguez Sanchez⁸  Sergio Vázquez⁹  Narayanan Srihari^1,10  Haralabos Kalofonos^1,11  Petri Bono^1,12 

[1] Medical University of Vienna;Novartis Healthcare Private Limited;Novartis Pharmaceuticals Corporation, East Hanover;The University of Texas MD Anderson Cancer Center;National and Kapodistrian University of Athens Alexandra Hospital;Fondazione IRCCS Istituto Nazionale dei Tumori;The Christie Hospital and University of Manchester, United Kingdom;University Hospital of Leon;Hospital Universitario Lucus Augusti;Shrewsbury & Telford Hospitals NHS Trust, United Kingdom;University of Patras;Helsinki University Hospital and University of Helsinki

关键词: Renal cell carcinoma;    Pazopanib;    Real-world;    Observational;    Tyrosine kinase inhibitor;   

DOI  :  10.1634/theoncologist.2018-0787

学科分类：地质学

来源: AlphaMed Press Incorporated

【 摘 要 】

Background Real-world data are essential to accurately assessing efficacy and toxicity of approved agents in everyday practice. PRINCIPAL, a prospective, observational study, was designed to confirm the real-world safety and efficacy of pazopanib in patients with advanced renal cell carcinoma (RCC). Subjects, Materials, and Methods Patients with clear cell advanced/metastatic RCC and a clinical decision to initiate pazopanib treatment within 30 days of enrollment were eligible. Primary objectives included progression-free survival (PFS), overall survival (OS), objective response rate (ORR), relative dose intensity (RDI) and its effect on treatment outcomes, change in health-related quality of life (HRQoL), and safety. We also compared characteristics and outcomes of clinical-trial-eligible (CTE) patients, defined using COMPARZ trial eligibility criteria, with those of non-clinical-trial-eligible (NCTE) patients. Secondary study objectives were to evaluate clinical efficacy, safety, and RDI in patient subgroups. Results Six hundred fifty-seven patients were enrolled and received ≥1 dose of pazopanib. Median PFS and OS were 10.3 months (95% confidence interval [CI], 9.2–12.0) and 29.9 months (95% CI, 24.7 to not reached), respectively, and the ORR was 30.3%. HRQoL showed no or little deterioration over time. Treatment-related serious adverse events (AEs) and AEs of special interest occurred in 64 (9.7%), and 399 (60.7%) patients, respectively. More patients were classified NCTE than CTE (85.2% vs. 14.8%). Efficacy of pazopanib was similar between the two groups. Conclusion PRINCIPAL confirms the efficacy and safety of pazopanib in patients with advanced/metastatic RCC in a real-world clinical setting. Implications for Practice PRINCIPAL is the largest ( n  = 657) prospective, observational study of pazopanib in patients with advanced/metastatic renal cell carcinoma, to the authors’ knowledge. Consistent with clinical trial results that often contain specific patient types, the PRINCIPAL study demonstrated that the effectiveness and safety of pazopanib is similarly safe and effective in patients with advanced kidney cancer in a real-world clinical setting. The PRINCIPAL study showed that patients with advanced kidney cancer who are treated with first-line pazopanib generally do not show disease progression for approximately 10 months and generally survive for nearly 30 months.

【 授权许可】

CC BY|CC BY-NC