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Table 2 Methodological attributes relevant to interpretation of study results from a clinical perspective for 95 randomized questions, by non-inferiority and superiority trials

From: Clinical significance in pediatric oncology randomized controlled treatment trials: a systematic review

Characteristic

Non-inferiority trials

(N = 13)

Superiority trials (N = 82)

n

%b

n

%b

Methods

 Expected magnitude of difference identified explicitly as the MCIDc

2

15.4

2

2.4

 Justification for MCIDa

  Clinical relevance

1

50.0

1

50.0

  Methodological

1

50.0

1

50.0

 Delta value

Stated as an absolute difference

13

100.0

78

95.1

 Margin (median, IQR)

− 0.10

− 0.10,0.10

0.12

0.10, 0.17

 Time-to-event

12

92.3

69

88.5

 Dichotomous outcome

1

7.7

9

11.5

 Stated as relative difference

0

0.0

7

8.5

  Margin (median, IQR)

N/A

0.63

0.60, 2.50

  Time-to-event

N/A

6

85.7

  Dichotomous outcome

N/A

1

14.3

  Stated as a percentage and ratio

0

0.0

4

4.9

 Anticipated control value stated

10

76.9

63

76.8

 Assumptions in the control group

6

46.2

12

14.6

 Stated

  Results from previous trial or systematic review

5

83.3

11

91.7

  Based on clinical expertise

1

16.7

1

8.3

 Type 1 error (α value)

 Stated

10

76.9

52

63.4

  0.20

0

0.0

1

1.9

  0.10

2

20.0

2

3.8

  0.05

8

80.0

49

94.2

 Sides

 Stated

12

92.3

40

48.8

  One-sided

10

83.3

29

72.5

  Two-sided

2

16.7

11

27.5

 Type 2 error (1 − β value)

 Stated

12

92.3

81

98.8

   < 80%

2

16.7

7

8.6

  80 to 85%

6

50.0

58

71.6

  85 to 90%

1

8.3

7

8.6

   ≥ 90%

3

25.0

9

11.1

Results

 Statistical significance of primary outcome reported via p value

13

100.0

66

80.5

 Confidence intervals/standard error for primary outcome reported

12

92.3

52

63.4

 Treatment effect stated

6

46.2

6

7.3

Discussion (and/or Results)

 Clinical importance of primary outcome discussed

10

76.9

11

13.4

  1. aMCID minimally clinically important difference, IQR interquartile range
  2. bPercentages may not sum to 100% due to rounding
  3. cAssumed to be the delta value from the sample size calculation