TY - JOUR
T1 - Prospective Observational Study of Pazopanib in Patients with Advanced Renal Cell Carcinoma (PRINCIPAL Study)
AU - on behalf of the PRINCIPAL Study Group
AU - Schmidinger, Manuela
AU - Bamias, Aristotelis
AU - Procopio, Giuseppe
AU - Hawkins, Robert
AU - Sanchez, Angel Rodriguez
AU - Vázquez, Sergio
AU - Srihari, Narayanan
AU - Kalofonos, Haralabos
AU - Bono, Petri
AU - Pisal, Chaitali Babanrao
AU - Hirschberg, Yulia
AU - Dezzani, Luca
AU - Ahmad, Qasim
AU - Jonasch, Eric
AU - Gimeno, Ramón Aldabó
AU - Herranz, Urbano Anido
AU - Ardavanis, Alexandros
AU - Ashraf, Safeer A.
AU - Bamias, Aristotelis
AU - Barone, Carlo
AU - Bella, Santiago R.
AU - Belz, Hanjo
AU - Companario, Esperanza Blanco
AU - Bolling, Claus
AU - Bono, Petri
AU - Bothe, Katrin
AU - Carteni, Giacomo
AU - Espinosa, Javier Cassinello
AU - Clausse, Marylene
AU - Confente, Caterina
AU - Coskun, Hasan
AU - Herrero, Guillermo Crespo
AU - Demey, Wim
AU - D'hondt, Randal
AU - Santasusana, Montserrat Domènech
AU - Doshi, Gury
AU - Elkiran, Emin
AU - Facchini, Gaetano
AU - Fein, Luis
AU - Calvo, Ovidio Fernandez
AU - Flaherty, Amber
AU - Fountzilas, George
AU - Fruehauf, John
AU - Díaz, Enrique Gallardo
AU - Garcia, Rosa
AU - Domínguez, Rocío García
AU - Ghosn, Marwan
AU - Milella, Michele
AU - Porta, Camillo
AU - Procopio, Giuseppe
PY - 2019/4
Y1 - 2019/4
N2 - 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.
AB - 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.
KW - Observational
KW - Pazopanib
KW - Real-world
KW - Renal cell carcinoma
KW - Tyrosine kinase inhibitor
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UR - http://www.scopus.com/inward/citedby.url?scp=85062990799&partnerID=8YFLogxK
U2 - 10.1634/theoncologist.2018-0787
DO - 10.1634/theoncologist.2018-0787
M3 - Article
C2 - 30867244
AN - SCOPUS:85062990799
VL - 24
SP - 491
EP - 497
JO - Oncologist
JF - Oncologist
SN - 1083-7159
IS - 4
ER -