Patients rank toxicity against progression free survival in second-line treatment of advanced renal cell carcinoma

Abstract

Background:

The aims of this study were to quantify and contrast patient preferences between second-line advanced renal cell carcinoma (RCC) medication profiles and their associated benefits and toxicities, and to help frame the doctor–patient discussion about selecting appropriate RCC therapies.

Research design and methods:

Adult residents of the US with a diagnosis of RCC completed a Web-enabled choice-format conjoint survey consisting of a series of 10 treatment-choice questions, each of which included a pair of hypothetical RCC medication profiles. Each profile was described by various medication attributes (features or outcomes) with varying levels. The attributes included efficacy (progression-free survival [PFS]), tolerability (fatigue, stomach problems, mucositis or stomatitis, hand–foot syndrome [HFS]), serious but rare adverse events (pneumonitis, hepatic impairment), and mode of administration. Treatment-choice questions were based on an experimental design with known statistical properties. Random-parameters logit regression was used to estimate relative preference weights for each attribute level. Benefit equivalent measures (additional months of PFS in exchange for toxicities) were also calculated.

Results:

Of the 272 patients who completed the survey, the majority were female (53%), white (92%), and had at least a college degree (66%). The mean age was 57 years (standard deviation: 10 years). Over the range of attributes and attribute levels included in the survey, PFS was the most important attribute, followed by fatigue, stomach problems, hepatic impairment, mucositis or stomatitis, HFS, pneumonitis, and mode of administration. To reduce severe fatigue to mild-to-moderate fatigue, patients on average would be willing to forego 4.4 months of PFS. To reduce hepatic impairment risk from 0.5% to 0.0%, patients on average would be willing to forego 1.0 month of PFS. The main study limitation was that patients answered hypothetical treatment-choice questions.

Conclusions:

This study provides information to physicians about patient priorities when reviewing and selecting RCC therapies with patients.

Keywords::

• Patient preferences
• Renal cell carcinoma
• Toxicities
• Trade-offs

Transparency

Declaration of funding

This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Declaration of financial/other relationships

Dr Wong has teaching and speaking interests and membership on advisory committees or review panels for Pfizer, Genentech, Bristol-Myers Squibb, and Novartis. Dr Hauber, Ms Mohamed, and Ms Yang have disclosed that they are employees of RTI Health Solutions, a company that received funding from Novartis to conduct this study. Drs Liu, Rogerio, and Garay have stock ownership in Novartis.

Acknowledgments

The authors would like to thank the Kidney Cancer Association for their assistance in locating RCC patients who were interested in participating in this study, and the patients who chose to participate in either the pilot study or the main study. The authors also would like to thank Paul Shannon and Stuart Yarr of RTI Health Solutions for programming the online survey and Vikram Kilambi and Ryan Ziemiecki of RTI Health Solutions for their assistance in analyzing the data.