Literature Appraisal Effectiveness of Therapy

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Literature Appraisal

Effectiveness of Therapy

Measures of treatment effect

Statistical significance

Odds ratio

Relative risk

Absolute risk reduction

Number needed to treat

Measures of treatment effect

Control

Experiment

Outcome (death)

Yes a c

No b d

Total

200

Total in each group

100

200

100

After 1 year

Total in each group

Die

100

200

100

25

10

After 1 year

Total in each group

Die (Survive)

100

200

100

25 + (75)

10 + (90)

Risk & Relative Risk

After 1 year

What is the

Total in each group

Die (Survive) Risk?

(a proportion)

100

200

100

25 + (75)

10 + (90)

After 1 year

Total in each group

Die (Survive) Risk

100

200

100

25 + (75)

10 + (90)

After 1 year

Total in each group

Die (Survive) Risk

100

200

100

25 + (75)

10 + (90)

After 1 year

Total in each group

Die (Survive) Risk

100

200

100

25 + (75)

10 + (90)

After 1 year

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

25%

After 1 year

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

25%

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

25%

0.1

10%

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

25%

0.1

10%

100

200

100

Die (Survive) Risk

25 + (75) 0.25

25%

10 + (90) 0.1

10%

Risk ratio or Relative risk (RR) =

100

200

100

Die (Survive) Risk

25 + (75) 0.25

25%

10 + (90) 0.1

10%

Risk ratio or Relative risk (RR) =

100

200

100

Die (Survive) Risk

25 + (75) 0.25

25%

10 + (90) 0.1

10%

Risk ratio or Relative risk (RR) =

2.5

Odds & Odds Ratio

Odds

• The ratio between the amounts staked by parties in a bet, based on the expected probability either way.

• The balance of advantage or superiority.

100

200

100

Die

What about

(Survive) Risk Odds?

25 + (75)

10 + (90)

0.25

0.1

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

1to3

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

1to3

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

1to3

1to9

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

1to3

1to9

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

1to3

1to9

Odds

Ratio

(O.R.) =

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

1to3

1to9

Odds

Ratio

(O.R.) =

100

200

100

Die (Survive) Risk

25 + (75)

10 + (90)

0.25

0.1

Odds

1to3

1to9

Odds

Ratio

(O.R.) =

3

100

200

100

Die (Survive) Risk

25 + (75) 0.25

Odds

1to3

10 + (90) 0.1

1to9

Risk ratio Odds or Relative Ratio risk (RR) = (O.R.) =

2.5

3

Measures of treatment effect influence clinicians decisions

• Clinicians:

– more inclined to treat if the results are presented as relative risk

– less inclined to treat if the results are presented as absolute risk reduction

Forrow et al. Am J Med 1992;92:121

Absolute Risk Reduction

• Control group event rate (CER)

= Deaths / Controls

• Experiment group event rate (EER)

= Deaths / Treated

Absolute risk reduction (difference)

ARR = CER - EER

100

200

100

Die Risk

25

10

0.25

25%

0.1

10%

Absolute Risk

Reduction

(ARR) =

CER

EER

100

200

100

Die Risk

25

10

0.25

25%

0.1

10%

Absolute Risk

Reduction

(ARR) =

0.15

15%

Number Needed to Treat

NNT

Clinical value of measures of treatment effect

Number Needed To Treat

The odds ratio etc. not easy to understand, especially for patients.

• The number needed to treat (NNT) to prevent an adverse event is a more clinically relevant measure of the consequences of treatment

Sackett DL. EBM 1996; 1: 164-6

Sinclair JC. J Clin Epidemiol 1994; 47: 881-9

Number Need to Treat (NNT)

Out of 100 patients treated 10 died compared to 25 in the placebo group and 15 extra survived.

Therefore:

To get 1 more patient to survive,

6.7 (100/15) have to be treated.

100/15

1/ 0.15

NNT = 1/ ARR

100

200

100

Die Risk

25 0.25

25%

10 0.1

10%

Absolute Risk

Reduction (ARR) =

0.15

15%

NNT=

1/ARR=

1/0.15=

6.7

Q.E.D.

MAGPIE

Of the patients treated (5015) 40 fitted compared to 96 in the placebo group (5055)

In % Mg 0.8% vs Placebo 1.9%

Therefore: ARR 1.8 – 0.8 = 1.1%

(11 per 1000)

To get 1 more patient to survive,

91 (100/1.1) have to be treated. = NNT

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