Patient-reported outcomes in randomised clinical trials of bladder cancer: an updated systematic review

Van Hemelrijck, Mieke; Sparano, Francesco; Josephs, Debra; Sprangers, Mirjam; Cottone, Francesco; Efficace, Fabio

doi:10.1186/s12894-019-0518-9

BMC Urology

Table 3 Comparison of PRO quality reporting over time in Bladder Cancer RCTs with PROs as a secondary outcome

From: Patient-reported outcomes in randomised clinical trials of bladder cancer: an updated systematic review

Methodological issue	Category	RCT with PROs (Jan.2004 –Mar. 2014) (n. 9), N. (%)	RCTs with PROs (Apr.2014 – Jun.2018) (n. 8), N. (%)	P-value
Title and abstract
The PRO should be identified as an outcome in the abstract	No	1 (11.1)	3 (37.5)	0.29
The PRO should be identified as an outcome in the abstract	Yes	8 (88.9)	5 (62.5)
Introduction, background, and objectives
The PRO hypothesis should be stated and specify the relevant PRO domain if applicable	No	5 (55.6)	5 (62.5)	1
	Yes	2 (22.2)	1 (12.5)
	N/A (if explorative)	2 (22.2)	2 (25)
Methods
Outcomes
The mode of administration of the PRO tool and the methods of collecting data should be described	No	7 (77.8)	5 (62.5)	0.62
	Yes	2 (22.2)	3 (37.5)
Electronic mode of PRO administration^a	No	1 (11.1)	2 (25)	1
	Yes	1 (11.1)	0 (0)
	N/A	7 (77.8)	6 (75)
The rationale for choice of the PRO instrument used should be provided	No	4 (44.4)	4 (50)	1
	Yes	5 (55.6)	4 (50)
Evidence of PRO instrument validity and reliability should be provided or cited	No	4 (44.4)	3 (37.5)	0.44
	Yes, for all PRO instruments	5 (55.6)	3 (37.5)
	Yes, only for some PRO instruments	0 (0)	2 (25)
The intended PRO data collection schedule should be provided	No	2 (22.2)	1 (12.5)	1
	Yes	7 (77.8)	7 (87.5)
PROs should be identified in the trial protocol post-hoc analyses	No	9 (100)	4 (50)	0.03^a
	Yes	0 (0)	4 (50)
The status of PRO as either a primary or secondary outcome should be stated	No	2 (22.2)	3 (37.5)	0.62
	Yes	7 (77.8)	5 (62.5)
Statistical methods
There should be evidence of appropriate statistical analysis and tests of statistical significance for each PRO hypothesis tested	No	0 (0)	2 (25)	0.223
	Yes	2 (22.2)	0 (0)
	N/A	7 (77.8)	6 (75)
The extent of missing data should be stated^b	No	8 (88.9)	2 (25)	0.015^a
The extent of missing data should be stated^b	Yes	1 (11.1)	6 (75)
Statistical approaches for dealing with missing data should be explicitly stated^b	No	9 (100)	6 (75)	0.206
	Yes	0 (0)	2 (25)
Results
Participant flow
A flow diagram or a description of the allocation of participants and those lost to follow-up should be provided for PROs specifically	No	7 (77.8)	5 (62.5)	0.62
	Yes	2 (22.2)	3 (37.5)
The reasons for missing data should be explained	No	8 (88.9)	5 (62.5)	0.294
The reasons for missing data should be explained	Yes	1 (11.1)	3 (37.5)
Baseline data
The study patients characteristics should be described including baseline PRO scores	No	6 (66.7)	3 (37.5)	0.347
	Yes	3 (33.3)	5 (62.5)
Outcomes and estimation
PRO outcomes also reported in a graphical format^a	No	5 (55.6)	6 (75)	0.62
PRO outcomes also reported in a graphical format^a	Yes	4 (44.4)	2 (25)
Discussion
Limitations
The limitations of the PRO components of the trial should be explicitly discussed	No	5 (55.6)	4 (50)	1
	Yes	4 (44.4)	4 (50)
Generalizability
Generalizability issues uniquely related to the PRO results should be discussed	No	5 (55.6)	6 (75)	0.62
	Yes	4 (44.4)	2 (25)
Interpretation
PROs are interpreted (Not only re-stated)^a	No	2 (22.2)	5 (62.5)	0.153
PROs are interpreted (Not only re-stated)^a	Yes	7 (77.8)	3 (37.5)
The clinical significance of the PRO findings should be discussed	No	6 (66.7)	6 (75)	1
	Yes	3 (33.3)	2 (25)
Methodology used to assess clinical significance is discussed^a	Anchor based (e.g., minimal important difference)	1 (11.1)	0 (0)	1
	Distribution based (e.g. effect size)	1 (11.1)	2 (25)
	Both	1 (11.1)	0 (0)
	Missing	6 (66.7)	6 (75)
The PRO results should be discussed in the context of the other clinical trial outcomes	No	2 (22.2)	1 (12.5)	1
	Yes	7 (77.8)	7 (87.5)

For descriptive purposes, subheadings of this table reflect those reported in the ISOQOL recommended standards [15]; however, rating of items was independent of location of the information within the manuscript
^aThese items were not included in the ISOQOL recommended standards [15] and in the calculation of the ISOQOL score but have been evaluated in our study and reported in this table to have a wider outlook on the level of reporting
^bThese items were originally combined in the ISOQOL recommended standards [15] but have been split in this report to better investigate possible discrepancies between documentation of PRO missing data (ie, reporting how many patients did not complete a given questionnaire at any given time point) versus actual reporting of statistical methods to address this issue. Also, we wanted to be consistent with items reported in the CONSORT PRO Extension [35] (ie, statistical approaches for dealing with missing data is reported as a stand-alone issue)

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ISSN: 1471-2490

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