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Table 4 Summary of the primary outcomes, weighted medians and outcomes with highest ranked weighted medians for the 25 nephrology trial primary outcomes. The six trials marked [**Primary outcome match**] are those where the primary outcome was either considered most important by the panel or was among those considered equally most important by the panel

From: Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want

Trial ID

Primary outcome

Number of responses to trial

Number of outcomes

Median ranking of primary [range]

Number of times primary was within respondents’ top 5 outcomes

Outcome(s) with highest ranked median

Median ranking of highest placed outcome [range]

Trial 1 [**Primary outcome match**]

End-stage renal disease or death from cardiovascular causes

11

4

1 [1-4]

11

End-stage renal disease or death from cardiovascular causes

1 [1-4]

  HCPs

 

5

 

1 [1-3]

4

 

1 [1-3]

  Patients

 

6

 

1.5 [1-4]

6

 

1.5 [1-4]

Trial 2

Mean change from the baseline haemoglobin level to the mean level during the evaluation period

11

3

2 [1-3]

11

Mean change from the baseline haemoglobin level to the mean level during the evaluation period

Proportion of patients in whom there was a response in haemoglobin levels

Proportion of patients receiving a transfusion

2 [1-3]

2 [1-3]

2 [1-3]

  HCPs

 

6

 

2 [1-3]

6

Mean change from the baseline haemoglobin level to the mean level during the evaluation period

1 [1-3]

Patients

 

5

 

1 [1-3]

4

1) Mean change from the baseline haemoglobin level to the mean level during the evaluation period

2) Proportion of patients in whom there was a response in haemoglobin levels 3) Proportion of patients receiving a transfusion

2 [1-3]

2 [1-3]

2 [1-3]

Trial 3

Occurrence of a major cardiovascular or renal event

10

9

4 [1-9]

8

1) Occurrence of a major cardiovascular or renal event

2) Need for dialysis or transplantation

3) Worsening of kidney function

4 [1-9]

4 [1-6]

4 [1-9]

  HCPs

 

5

 

3 [1-5]

5

All-cause mortality

1 [1-6]

  Patients

 

5

 

4 [3-9]

3

Need for dialysis or transplantation

1 [1-6]

Trial 4

Difference between treatment groups in the proportion of patients remaining on spironolactone at week 12

11

7

5 [1-7]

6

Difference between treatment groups in the change in systolic automated office blood pressure from baseline to week 12

2 [1-7]

  HCPs

 

5

 

5 [1-6]

3

Difference between treatment groups in the change in systolic automated office blood pressure from baseline to week 12

2 [1-5]

  Patients

 

6

 

5.5 [2-7]

3

Patient-reported outcomes as measured by the EQ-5D-5L questionnaire

1.5 [1-7]

Trial 5

Independence from dialysis at 90 days after random allocation to groups

10

5

3.5 [1-5]

10

Overall survival at the end of the study

1 [1-5]

  HCPs

 

5

 

3 [1-4]

5

Overall survival at the end of the study

1 [1-5]

  Patients

 

5

 

4 [1-5]

5

Overall survival at the end of the study

1 [1-5]

Trial 6 [**Primary outcome match**]

A composite of death from any cause by 28 days after randomisation and the presence of at least one organ failure at 7 days after randomization

10

7

1 [1-3]

10

A composite of death from any cause by 28 days after randomisation and the presence of at least one organ failure at 7 days after randomization

1 [1-3]

  HCPs

 

5

 

1 [1-1]

5

A composite of death from any cause by 28 days after randomisation and the presence of at least one organ failure at 7 days after randomization

1 [1-1]

  Patients

 

5

 

2 [1-3]

5

A composite of death from any cause by 28 days after randomisation and the presence of at least one organ failure at 7 days after randomization

2 [1-3]

Trial 7

A ranking of clinical outcomes, maximum relative change in creatinine, dialysis, and death, within 7 days of randomization

10

6

2.5 [1-5]

10

Death

2 [1-6]

  HCPs

 

5

 

2 [2-5]

5

Death

1 [1-4]

  Patients

 

5

 

3 [1-5]

5

Progression to higher stages of acute kidney injury

2 [1-6]

Trial 8 [**Primary outcome match**]

A further 20% decline in excretory renal function from baseline readings

10

3

1 [1-2]

10

A further 20% decline in excretory renal function from baseline readings

1 [1-2]

  HCPs

 

5

 

1 [1-2]

5

A further 20% decline in excretory renal function from baseline readings

1 [1-2]

  Patients

 

5

 

1 [1-1]

5

A further 20% decline in excretory renal function from baseline readings

1 [1-1]

Trial 9

Relapse-free survival based on the period of time until the first relapse

10

3

2 [1-3]

10

Relapse-free survival based on the period of time until the first relapse

Probability of progression-free survival based on the time until the progression to FRNS, SDNS or SRN

Relapse rate

2 [1-3]

2 [1-3]

2 [1-3]

  HCPs

 

5

 

2 [1-3]

5

Probability of progression-free survival based on the time until the progression to FRNS, SDNS or SRN

1 [1-3]

  Patients

 

5

 

1 [1-3]

5

Relapse-free survival based on the period of time until the first relapse

1 [1-3]

Trial 10

The change in UACR from baseline to the end of treatment

10

10

4 [2-9]

7

Proportion of participants in remission defined as a reversal of UACR to normoalbuminuria

3 [1-8]

  HCPs

 

5

 

4 [2-6]

4

Proportion of participants in remission defined as a reversal of UACR to normoalbuminuria

Proportion of participants who sustained remission after treatment

2 [1-8]

2 [1-5]

  Patients

 

5

 

5 [2-9]

3

Plasma aldosterone concentration

2 [2-7]

Trial 11

The change in log-transformed urine albumin-to-creatinine ratio from baseline to the end of treatment

10

9

3.5 [1-8]

9

eGFR

The remission rate from early-stage nephropathy to prenephropathy stage at the end of treatment

2 [1-6]

2 [1-6]

  HCPs

 

5

 

3 [1-4]

5

The progression rate from early-stage nephropathy to overt nephropathy during the treatment period

2 [1-3]

  Patients

 

5

 

5 [3-8]

4

eGFR

1 [1-2]

Trial 12 [**Primary outcome match**]

Percentage of patients with treatment success

10

5

1 [1-5]

10

Percentage of patients with treatment success

1 [1-5]

  HCPs

 

5

 

1 [1-5]

5

Percentage of patients with treatment success

1 [1-5]

  Patients

 

5

 

1 [1-5]

5

Percentage of patients with treatment success

1 [1,2,3,4,5]

Trial 13

Change in cGFR from baseline to month 12

10

7

3 [2-7]

9

Patient and graft survival

1 [1-4]

  HCPs

 

5

 

3 [2-3]

5

Patient and graft survival

1 [1-1]

  Patients

 

5

 

4 [3-7]

4

Incidence of AR

1 [1-4]

Trial 14

The rate of haemodialysis independence at 3 months after randomization

10

10

7 [1-10]

3

Overall survival

1.5 [1-7]

  HCPs

 

5

 

7 [1-8]

 

Overall survival

1 [1-5]

  Patients

 

5

 

8 [4-10]

 

Overall survival

2 [1-7]

Trial 15: primary 1 [**Primary outcome match**]

Loss of kidney function at 1 year

12

2

1 [1-2]

12

Loss of kidney function at 1 year

1 [1-2]

  HCPs

 

5

 

1 [1-1]

5

Loss of kidney function at 1 year

1 [1-1]

  Patients

 

7

 

2 [1-2]

7

Acute kidney injury within 30 days of surgery

1 [1-2]

Trial 15: primary 2

Acute kidney injury within 30 days of surgery

12

2

2 [1-2]

12

Loss of kidney function at 1 year

1 [1-2]

  HCPs

 

5

 

2 [2-2]

5

Loss of kidney function at 1 year

1 [1-1]

  Patients

 

7

 

1 [1-2]

7

Acute kidney injury within 30 days of surgery

1 [1-2]

Trial 16

eGFR over time

12

8

3 [1-8]

10

Blood pressure control

2.5 [1-8]

  HCPs

 

5

 

4 [1-8]

4

Blood pressure control

2 [1-3]

  Patients

 

7

 

3 [1-8]

6

Early recognition and diagnosis of CKD

2 [1-6]

Trial 17

Change from baseline to 24 weeks in UACR in the first morning urine sample

11

8

4 [1-7]

9

Serum cystatin C-derived eGFR

3 [1-8]

  HCPs

 

5

 

1 [1-5]

5

Change from baseline to 24 weeks in UACR in the first morning urine sample

1 [1-5]

  Patients

 

6

 

4.5 [2-7]

4

24-h total urine albumin excretion

2.5 [1-4]

Trial 18

Relative change in serum phosphate concentrations from baseline to end of treatment

12

3

1.5 [1-2]

12

Relative change in serum phosphate concentrations from baseline to end of treatment

1.5 [1-2]

  HCPs

 

5

 

1 [1-2]

5

Relative change in serum phosphate concentrations from baseline to end of treatment

1 [1-2]

  Patients

 

7

 

2 [1-2]

7

Changes in corrected PTH concentrations from baseline to end of treatment

Relative change in serum phosphate concentrations from baseline to end of treatment

2 [1-3]

2 [1-2]

Trial 19

Mean change in Hgb from baseline to end of treatment

12

4

1.5 [1-4]

12

Mean change in Hgb from baseline to end of treatment

1.5 [1-4]

  HCPs

 

5

 

1 [1-1]

5

Mean change in Hgb from baseline to end of treatment

1 [1-1]

  Patients

 

7

 

3 [1-4]

7

Mean intradialytic change from pre-haemodialysis to post-haemodialysis in serum iron, unsaturated iron-binding capacity (UIBC) and TSAT

Mean change in Hgb from baseline every 4 weeks

2 [1-4]

2 [1-4]

Trial 20

Measured GFR at 12 months

9

10

4 [1-9]

6

Rate of change of eGFR calculated from creatinine values at 0, 1, 3, 6, 9 and 12 months

1 [1-5]

  HCPs

 

5

 

4 [1-9]

4

Rate of change of eGFR calculated from creatinine values at 0, 1, 3, 6, 9 and 12 months

2 [1-5]

  Patients

 

4

 

5 [3-9]

2

Rate of change of eGFR calculated from creatinine values at 0, 1, 3, 6, 9 and 12 months

1 [1-1]

Trial 21

Left ventricular mass index

9

10

5 [1-7]

5

All-cause mortality and hospitalization for CV events

1 [1-2]

  HCPs

   

5 [4-7]

3

All-cause mortality and hospitalization for CV events

1 [1-2]

  Patients

   

5 [1-7]

2

1)

2) All-cause mortality and hospitalization for CV events Exercise capacity: New York Heart Association functional class, 6-min walk test

2 [1-2]

2 [1-7]

Trial 22

Percentage of subjects achieving an increase in Hb of ≥ 1.0 g/dL at any study point between baseline and End of Study or introduction or dose increase of ESA, blood transfusion or use of iron outside of protocol

9

5

3 [2-5]

5

1)

2) Mean change in Hb from baseline to end of week 6 (day 42) and end of week 8 (day 56)/end of study

Percent of subjects achieving a clinical response

2 [1-5]

2 [1-5]

  HCPs

 

5

 

3 [2-4]

5

Percent of subjects achieving a clinical response

1 [1-3]

  Patients

 

4

 

3.5 [3-5]

4

Mean change from baseline to highest Hb

1 [1-4]

Trial 23

Rate of s-K + decline in the first 48 h, using all post-baseline s-K + data

9

5

2 [1-3]

9

Rate of s-K + decline in the first 48 h, using all postbaseline s-K + data

Kidney function parameters

2 [1-3]

2 [1-4]

  HCPs

 

5

 

2 [1-3]

5

Rate of s-K + decline in the first 48 h, using all postbaseline s-K + data

Changes in s-K + at various time points after start of treatment

2 [1-3]

2 [1-3]

  Patients

 

4

 

3 [2-3]

4

Kidney function parameters

1 [1-1]

Trial 24

Proportion of CRR (complete renal remission) at 24 weeks

9

2

2 [1-2]

9

CRR rate at 48 weeks

1 [1-2]

  HCPs

 

5

 

2 [1-2]

5

CRR rate at 48 weeks

1 [1-2]

  Patients

 

4

 

1 [1-2]

4

Proportion of CRR (complete renal remission) at 24 weeks

1 [1-2]