!"#$%&'() +,'-'. /,',#0)1 !,'"$%.

2 3"&'-#('()'
uouglas 8. WhlLe, Mu, MAS
AssoclaLe rofessor
ueparLmenL of Crlucal Care Medlclne
unlverslLy of lusburgh School of Medlclne
4#)56-7 !"')6&'50,'
no poLenual CCls relevanL Lo Lhls Lalk
8,#0%"%$ 9:;,)(<,'
Aer Lhls sesslon, learners wlll be able Lo:
1. ldenufy approprlaLe sLausucal LesL for cerLaln Lypes of
varlables
2. CalculaLe sensluvlLy, speclclLy, absoluLe and relauve rlsk
reducuon, number needed Lo LreaL
3. Lxplaln how relauve rlsk reducuon can be a mlsleadlng
measure.
4. Lxplaln Lhe lmporLance of randomlzauon and bllndlng ln Lhe
conducL of experlmenLal sLudles.
+7=,' &> ?#0"#:6,'
Conunuous-
SysLollc blood pressure
WelghL
8lnary- Cnly 2 responses posslble
?es or no
urlne Loxlcology posluve for cocalne?

CaLegorlcal- muluple dlscreLe cholces
v/C scan resulLs: low, lnLermedlaLe, hlgh rlsk


!"#$%&'() +,'-'
@&50 >0",%A. -1, BCB -#:6,
D 3
E !
!"#$%#$
F G
'$#(
F
G
+,'- #))50#)7
H,%'"(<"-7: WhaL percenLage of people !"#$ #$&
'"(&)(& have a *+(",-& LesL? very sensluve LesLs
have few false negauve resulLs. (posluve ln dlsease =
lu)
H=,)"I)"-7: WhaL percenLage of people !"#$+.# #$&
'"(&)(& have a /&0),-& LesL? very speclc LesLs
have few false-posluve LesLs. (negauve ln healLh =
nlP)
H,%'"(<"-7
H,%'"(<"-7: WhaL percenLage
of people !"#$ #$& '"(&)(&
have a *+(",-& LesL? very
sensluve LesLs have few false
negauve resulLs. (posluve ln
dlsease = lu)
A/(A+C)
D 3
E !
!"#$%#$
F G
'$#(
F
G
H=,)"I)"-7
H=,)"I)"-7J Lhe percenLage of
people wlLhouL Lhe dlsease
have a negauve LesL.
(nlP= negauve ln healLh)
u/(u+8)
lalse posluve (Lhe 'lp
slde of speclclLy)= 8/(u
+8)
40 30
40 90
1"(&)(&
+ -
2&(# 3&(.4#
+
-
80 120
!"#$%&'() -,'-'
A bloLech company LouLs Lhelr L-ux" -a bedslde, non-lnvaslve
dlagnosuc LesL for L- as a sclenuc breakLhrough. ?our
lnsuLuuon sLudles 200 pauenLs who also undergo pulmonary
anglogram. 80 pauenLs have a L dlagnosed vla anglogram, of
whom 40 have a posluve L-ux. Among Lhose wlLh a negauve
anglogram, 30 have a posluve L-ux.
WhaL ls Lhe speclclLy of Lhe L-ux?
A. 0.66
8. 0.73
C. 0.23

H=,)"I)"-7
H=,)"I)"-7J Lhe
percenLage of people
wlLhouL Lhe dlsease have
a negauve LesL.
(nlP= negauve ln healLh)
u/(u+8)
lalse negauve= C/(A+C)
KL ML
KL NL
!"#$%#$
F G
'$#( )$#*+(
F
G
OL PBL
H-#('()#6 +,'-'
ln a recenL sLudy for a new sepsls drug, sub[ecLs
were randomlzed Lo recelve elLher Lhe drug or a
placebo:
morLallLy ln conLrol group: 43
MorLallLy ln lnLervenuon: 37

SLausucal LesL Lo compare proporuons:
-Chl-square
-p<0.001
H-#('()#6 +,'-'
ln a recenL sLudy for a new drug Lo LreaL hyperLenslve
crlsls, sub[ecLs were randomlzed Lo recelve elLher
Lhe drug or a placebo. 8 measured aL 2 hours:
Mean S8 ln conLrol group: 212mm Pg
Mean S8 ln lnLervenuon group : 171mm Pg

SLausucal LesL Lo compare conunuous ouLcomes:
--1-LesL
-p<0.0001
H-5A",' -& =0&<, -0,#-Q,%- ,R)#)7
9:',0<#(&%#6 '-5A",'
unconLrolled case serles, prospecuve cohorL
sLudles, cross-secuonal sLudles and case-conLrol
sLudles
Suscepuble Lo slgnlcanL confoundlng.

E&%>&5%A,0'J lacLors assoclaLed wlLh Lhe *3&'"5#+3
and causlng Lhe +.#5+6&7

SC=,0"Q,%-'
8andomlzed, conLrolled Lrlals

8andom asslgnmenL of sub[ecLs Lo Lhe
lnLervenuon and conLrol groups equally
dlsLrlbuLes confounders (known and unknown).
/E+'J 36"%A"%$
- ConLrols for blases LhaL may arlse from knowlng Lhe
LreaLmenL allocauon
- lmporLanL Lo bllnd Lhose evaluaung Lhe ouLcomes,
especlally lf Lhe ouLcome ls sub[ecuve
- ln double-bllnded 8C1s, dlerence ln ouLcome due
Lo elLher chance or Lhe LreaLmenL asslgnmenL.
/E+'J 4&66&TG5=
U non-dlerenual losL-Lo-follow up may resulL ln losL
power.

- ulerenual losL-Lo-follow up may resulL ln losL power
and blased resulLs.
S<"A,%), #:&5- +0,#-Q,%-'J
E&%)65'"&%'
U 1he gold sLandard sLudy deslgn for evaluaung Lhe emcacy
of LreaLmenLs ls Lhe randomlzed, conLrolled Lrlal (8C1).
- ln 8C1s, randomlzauon Lends Lo dlsLrlbuLe equally boLh
known and unknown confounders beLween Lhe Lwo
groups.

V,#'50,' &> ,W,)-
4&)5'
8lsk, rlsk rauos
ulsungulshlng relauve from absoluLe rlsks
ueLermlnlng number needed Lo LreaL"
V,#'50,' &> ,W,)-J
/"'X
/"'X Y Z &> =,&=6, T1& A,<,6&= -1, &5-)&Q,
Z &> =,&=6, #- 0"'X
D 3
E !
!"#$%#$
F G
')$%(,$-(
F
G
/"'XYD[DF3
.- (/$ ()$%(,$-(
0)1*23
V,#'50,' &> ,W,)-J
/"'X /#(&

/"'X 0#(& Y \D[DF3] 0"'X "% -0,#-Q,%- $0&5=
\E[EF!] 0"'X "% =6#),:& $0&5=

D 3
E !
!"#$%#$
F G
')$%(,$-(
F
G
/"'XYD[DF3
.- (/$ ()$%(,$-(
0)1*23
V,#'50,' &> ,W,)-J
0"'X 0#(&'[0,6#(<, 0"'X
8lsk rauo" ls Lhe same as Lhe relauve rlsk"
8elauve rlsk reducuon (888) = 1- rlsk rauo (always a
posluve number)

lf 88 0.74, Lhen
888 = 1 - 0.74 = 0.26
V,#'50,' &> ,W,)-J
/"'X
An example:
Conslder a hypoLheucal 8C1:
An 8C1 enrolls 400 pauenLs Lo recelve anubloucs
or placebo ln an eorL Lo decrease Lhe lncldence
of venulaLor assoclaLed pneumonla (vA).

V,#'50,' &> ,W,)-J
/"'X
89)6*4& :;<
10/200 ln Lhe anublouc group geL vA
13/200 ln Lhe placebo group geL vA

Croup AsslgnmenL 8lsk
Anubloucs 10/200 = 0.030
lacebo 13/200 = 0.073

V,#'50,' &> ,W,)-J
/"'X 0#(&'
8auos of Lhe 2 rlsks

8lsk 8auo = 0.030/0.073 = 0.67


V,#'50,' &> ,W,)-J
H-#('()#6 '"$%"I)#%), &> 0"'X 0#(&'
measured by Lhe 93 condence lnLerval
Lhe 93 condence lnLerval ls Lhe range of values noL
sLausucally dlerenL Lhan Lhe 88
lf lL lncludes 1.0, a plauslble ouLcome ls LhaL no dlerence ln
rlsk was noLed
wldLh of Lhe condence lnLerval ls mosL sensluve Lo Lhe
number of ouLcomes ln each group
V,#'50,' &> ,W,)-J
H-#('()#6 '"$%"I)#%), &> 0"'X 0#(&'
Lxample
88 = 0.67
(93 Cl 0.28 - 4.48]

+1, %5Q:,0 %,,A,A -& -0,#- \^^+]


Group Number in
each group
Mortality Risk Risk Ratio
Antibiotics 100 50 0.50 0.67
Placebo 100 75 0.75
1) How many people must be treated with abx to avoid 1 VAP?

A. 2
B. 4
C. 6
D. 10
+1, %5Q:,0 %,,A,A -& -0,#-
Definition: The number of patients who
must be treated with a therapy in order to
prevent one occurrence of the negative
outcome (e.g., VAP)

formula:
nn1= 1 / (#:'&65-, 0"'X A"W,0,%),)

ln Lhls example:
1 / (0.73-0.30) = 1/0.23 = 4