Table 3.

Incorporating Clinical Outcome Assessment (COA) Measures in Clinical Trials: Impact of Patient-Reported Outcome (PRO) Measures on Study Findings and Implications (Examples)

Study Authors, YearRef	Type of Cancer	Drug/Treatment/Intervention	PROs Endpoint	Impact on Objective Measure	Impact on COAs and Broader Aspects	Overall Effect	Conclusions/Implications
Tannock et al, 199620	Prostate cancer	Adjuvant mitoxantrone with prednisolone	PE	–	Improve in pain	+	FDA approval for advanced hormone-refractory prostate cancer
LATITUDE trial (Fizazi et al, 2017)22	Prostate cancer	Abiraterone acetate	EE	Increased OS	Improvement in HRQoL	+	FDA approval for high-risk castration- sensitive prostate cancer
Verstovsek et al, 201222	Myelo-fibrosis	Ruxolitinib	SE	Reduction of spleen volume	Reduction in symptoms burden	+	Approval of ruxolitinib for treatment of myelofibrosis
Thornton et al, 200814	Breast cancer	Taxanes in adjuvant chemotherapy regimen	PE	–	Worse mental health outcome and double emotional recovery time	–	Monitoring depression in patients receiving taxanes
RTOG 0525 substudy (Armstrong et al, 2013)15	Brain tumor (GBM)	Dose-dense temozolomide treatment associated with radiotherapy	EE	No change in survival	Greater symptom burden	–	Standard dose of temozolomide
RTOG 0933 (Gondi et al, 2014)17	Brain metastases	HA-WBRT	SEa	–	Memory preservation and improved QoL	+	Evidence supporting HA-WBRT
EORTC-NCIC trial (Stupp et al, 2009)23	Brain tumor (GBM)	Adjuvant temozolomide with radiotherapy	SE	Increased survival	No change in QoL	+	Change in standard of care

Abbreviations: EE, exploratory endpoint; EORTC-NCIC, European Organisation for Research and Treatment of Cancer-National Cancer Institute of Canada; GBM, glioblastoma; HA-WBRT, hippocampal-sparing whole-brain radiotherapy; HRQoL, health-related quality of life; PE, primary endpoint; QoL, quality of life; Ref, reference; SE, secondary endpoint; WBRT, whole-brain radiotherapy.

^aWith performance outcome (PerfO: neurocognitive testing) as PE.