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
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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.
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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