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Patient-Centered Outcome Measures in Lung Cancer Trials

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Abstract

Background

Scientific communities focusing on cancer research have urged for the development of trials that address patient-centered outcome measures instead of solely focusing on cancer as a disease-centered process. This is important for a patient with lung cancer because of the rapid course of disease and generally poor prognosis. We set out to determine the characteristics and study objectives of the current clinical trials in pulmonary malignancies.

Methods

The United States National Institutes of Health clinical trial registry was searched on April 23rd 2015, for currently recruiting phase I, II, or III clinical trials in lung cancer. Trial characteristics and study objectives were extracted from the registry website.

Results

Of the 419 clinical trials included in this review, patient-centered outcome measures are investigated in a minority of the trials. Outcome measures as quality of life, functional capacity, and health care utilization are included in a small number of trials (20, 4, and 2 % respectively). Treatment completion is included in 1 % of the trials. Research goals are most frequently toxicity (78 %) and progression-free survival (76 %).

Conclusion

Patient-centered outcome measures are included in a minority of the currently recruiting clinical trials in pulmonary malignancies. If we do not investigate these outcome measures, it is not possible to increase our knowledge of the optimal treatment, as this should aim to optimize the patient’s wellbeing as well as the course of disease. One option could be to incorporate combinations of patient- and disease-centered endpoints, for instance by using overall treatment utility or quality-adjusted outcome measures.

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Abbreviations

FDA:

Food and Drug Administration

NIH:

National Institutes of Health

NSCLC:

Non-small cell lung cancer

PS:

Performance status

PROM:

Patient-related outcome measures

SCLC:

Small cell lung cancer

WHO:

World Health Organization

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Acknowledgments

Guarantor

Karlijn J. G. Schulkes.

Authors’ Contribution

K.J.G.S. made substantial contribution to conception and design, acquisition, analysis and interpretation of data, drafting the article, and final approval of the version to be published. C.N. made substantial contribution to acquisition, analysis and interpretation of data, drafting the article, and final approval of the version to be published. F.B. made substantial contribution to conception and revising the article critically for important intellectual content and final approval of the version to be published. M.E.H. made substantial contribution to conception and design, analysis and interpretation of data, revising the article critically for important intellectual content, and final approval of the version to be published. L.J.R.E. made substantial contribution to conception and design, revising the article critically for important intellectual content, and final approval of the version to be published.

Funding

This study was supported by the Aart Huisman Scholarship for research in geriatric oncology and the Cornelis Visser Foundation.

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Karlijn J. G. Schulkes.

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Conflict of interest

None.

Appendix

Appendix

Study objective

Classified as

Overall survival

Overall survival

Mortality at a particular time point during follow-up

 

Progression-free survival

Progression-free survival

Event-free survival

 

Disease-free survival

 

Time-to-progression

 

Duration of response

 

Toxicity

Toxicity

Safety

 

Feasibility

 

Maximum-tolerated dose

 

Response

Efficacy

Efficacy

 

Time-to-responde

 

Engraftment

 

Completion of planned treatment

Completion of treatment

Achieved dose intensity

 

Compliance to treatment

 

Pharmacokinetics

Pharmacological parameters

Pharmacodynamics

 

Health care utilization

Health care utilization

Health economics

 

Laboratory parameters

Biological parameters

Genetic parameters

 

Tumor biology

 

Quality of life

Quality of life

Care dependence

Functioning

Institutionalization

 

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Schulkes, K.J.G., Nguyen, C., van den Bos, F. et al. Patient-Centered Outcome Measures in Lung Cancer Trials. Lung 194, 647–652 (2016). https://doi.org/10.1007/s00408-016-9903-2

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  • DOI: https://doi.org/10.1007/s00408-016-9903-2

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