1.

An investigator wants to assess whether the use of a specific medication given to infants
born prematurely is associated with developmental delay. Fifty infants who were given the
medication and fifty comparison infants who were also born prematurely but not given the
medication will be selected for the analysis. Each infant will undergo extensive testing at
age 2 for various aspects of development. Identify the type of study proposed and indicate
its specific strengths and weaknesses.

Study: Prospective Cohort

Strengths: This type of study can assess the temporal relationship. Bias are not as big of
an issue in cohort studies as they are in case-control studies.

Weaknesses: This type of study is difficult if the chosen disease is rare, and confounders
may be an issue.

2. In the study of a new anti-hypertensive medication, systolic blood pressures are measu
treatment is administered). The data are as follows:

120 112 138 145 135 150 145 163
148 128 143 156 160 142 150

a. Compute the sample mean.

142.33
b. Compute the sample median.
145.00
c. Compute the sample standard deviation.
14.11
d. Compute the sample range.
51.00

3. Total cholesterol in children aged 10-15 is assumed to follow a normal distribution with
and a standard deviation of 22.4.

a. What proportion of children 10-15 years of age have total cholesterol between 180 an
0.171

b. What proportion of children 10-15 years of age would be classified as hyperlipidemic (

hyperlipidemia is defined as a total cholesterol level over 200)?
0.344

a. What proportion of children 10-15 years of age have total cholesterol between 180 an
0.171

b. What proportion of children 10-15 years of age would be classified as hyperlipidemic (

hyperlipidemia is defined as a total cholesterol level over 200)?
0.344

4. A national survey of graduate students is conducted to assess their consumption of c

following table summarizes the data.

a. What proportion of students drink decaffeinated coffee only?
17.4%
b. What proportion of coffee drinkers (caffeinated and decaffeinated) are female?
54.6%
c. What proportion of the females do not drink coffee?
12.7%

Drink Drink Caffeinated

Do not drink coffee Total
Decaffeinated Only Coffee

Male 145 94 365 604

Female 80 121 430 631
Total 225 215 795 1235

a. 215 / 1235 b. (121 + 430) / (215 + 795)

0.17408907 0.54554455

5. The following data are collected in a clinical trial evaluating a new compound designed to
healing in trauma patients. The new compound is compared against a placebo. After treatm
the new compound or placebo the extent of wound healing is measured and the data are sh

Suppose that clinicians feel that if the percent reduction in the size of the wound
50% then the treatment is a success.

Is there a difference in the extent of wound healing by treatment? (Hint: Are treatment and
healing independent?) Run the appropriate test at a 5% level of significance.
Reject since 14.25 > 4
a. Generate a 95% confidence interval for the percent success in patients receiving the new
(0.50, 0.67)
b. Generate a 95% confidence interval for the difference in the percent success between th
placebo
(0.11, 0.35)
c. Generate a 95% confidence interval for the relative risk of treatment success between tre
(1.25, 2.19)
d. Generate a 95% confidence interval for the odds ratio of treatment success between trea
(1.55, 4.31)
 Reject since 14.25 > 4
a. Generate a 95% confidence interval for the percent success in patients receiving the new
(0.50, 0.67)
b. Generate a 95% confidence interval for the difference in the percent success between th
placebo
(0.11, 0.35)
c. Generate a 95% confidence interval for the relative risk of treatment success between tre
(1.25, 2.19)
d. Generate a 95% confidence interval for the odds ratio of treatment success between trea
(1.55, 4.31)

Wound Healing: % Reduction in Size of Wound

Treatment None 1-25% 26-50% 51-75%

New Compound
5 9 9 14
(n=125)

Placebo (n=125) 9 8 9 59

Total 14 17 18 73

1 independent sample dichotomous

x n p z upper
23 125 0.184 1.96 0.25192897

2 independent sample dichotomous

x1 n1 x2 n2 p1
73 125 44 125 0.584

CI FOR RELATIVE RISK

95% RR
x1 n1 x2 n2 p1
73 125 44 125 0.584

CI FOR ODDS RATIO

95% OR
x1 n1 x2 n2 Z
73 125 44 125 1.96
6. The following data was collected in a clinical trial evaluating a new compound designe
trauma patients. The new compound is compared against a placebo. After treatment for
compound or placebo the extent of wound healing is measured and the data are shown b

Is there a difference in the extent of wound healing by treatment? (Hint: Are treatment a
healing independent?) Run the appropriate test at a 5% level of significance

Percent Wound Healing

Treatment 0-25% 26-50% 51-75% 76-100%

New Compound
15 37 32 41
(n=125)

Placebo (n=125) 36 45 34 10

Total 51 82 66 51

Step 1: H0: reatment and percent wound healing are independent of each other
H1: H0 is false
a = 0.05

2
(O - E )
Step 2: 2
E
Step 3: Reject H0 if X^2 > 7.81

Step 4: X^2 = 28.32

Step 5: Reject since 28.32 > 7.81

7. A study is conducted to compare mean cholesterol levels for individuals following a

at least 6 months to individuals following a conventional (low-fat, low calorie) diet for
data are summarized below:

Test if there is a significant difference in mean cholesterol levels between the diet pro
significance.
 7. A study is conducted to compare mean cholesterol levels for individuals following a
at least 6 months to individuals following a conventional (low-fat, low calorie) diet for
data are summarized below:

Test if there is a significant difference in mean cholesterol levels between the diet pro
significance.

Std Dev
Diet Program Sample Size Mean Cholesterol
Cholesterol

Low Carbohydrate 50 225.4 24.5

Conventional 75 203.8 21.6

2 samples difference in mean (continuous)

x bar 1 s1 n1 x bar 2
225.4 24.5 50 203.8

8. A study is conducted to compare 3 new appetite suppressants (A, B, C) to a placebo in

reduction. A total of 80 participants are involved and are randomly assigned to the comp
The outcome of interest is weight reduction, measured in pounds. The following data are
treatment.

Is there a significant difference in mean weight reduction among the 4 treatments? Use a
SST=1,889.

A B C Placebo
Mean (SD)
6.4 (4.1) 8.9 (4.0) 2.2 (3.9) 2.5 (4.3)
Weight Reduction

source of variation sums of squares df

2
S S B = n j(X j - X )
 2
between treatments S S B = n j(X j - X ) k- 1
625.2 3

2
error S S E = ( X - X j) N-k
1263.8 76
2
total S S T = (X - X ) N-1
1889 79

N= 80
k= 4
df1 = 3
df2 = 76

9. Suppose we want to design a new placebo-controlled trial to evaluate an experimenta

increase lung capacity. The primary outcome is peak expiratory flow rate, a continuous
liters per minute. The primary outcome will be measured after 6 months on treatment.
expect to lose 10% of the participants over the course of follow up. The mean peak exp
adults is 300 with a standard deviation of 50. How many subjects should be enrolled to e
detect a difference of 15 liters per minute with a two sided test and a=0.05?

|1 2| 15
ES= = =0 . 30 ,
50
Z 1 /2 + Z 1- 2
( ) (
2
1 . 96+0 . 84
ni = 2
=174.2.
ES
=2
0 . 30 )
Need n1=175 and n2=175 with complete outcome data.

N (number to enroll) * (% retained) = desired sample size

N (0.90) = 350
N = 350/0.90 = 389

Need to enroll N=389 to account for 10% attrition.

10. A clinical trial was conducted comparing a new compound designed to improve woun
patients to a placebo. After treatment for 5 days, 58% of the patients taking the new co
reduction in the size of their wound as compared to 44% in the placebo group. The trial
How many subjects would be required to detect the difference in proportions observed in
A two sided test is planned at a=0.05.

% power continuous (difference in proportions)

p - p 0 .0 5
E S p1 1 - p 0 0 0 .0 5 0 .1 1
E S p 0 ( 1 - p 0 ) 0 .2 6 ( 1 - 0 .2 6 ) 0 .1 1
p 0(1 - p 0 ) 0 .2 6 ( 1 - 0 .2 6 )
2
Z 1 - /2 Z 1 - 2 1 . 9 6 1 . 2 8 2 2 2
n Z 1 - / 2 Z 1 - 1 . 9 6 1 . 2 8 2 8 6 8 . 6
n
E S 0 .1 1 8 6 8 .6
E S 0 .1 1
|p1 - p0| = |difference in mean|
= SD Need n1 = 200 and n2 = 200 with c
Z (1- a/2) = 1.96 outcome data.
Z (1 - B) = 0.84 (for 80%)
= 1.282 (for 90%)
E = % (convert to decimal!!!)

* always round up n *

12. Suppose we want to design a new study to assess the implications of childhood obe
health outcomes in adulthood. In the study we will consider total cholesterol at age 50
cardiovascular disease by age 50 as the key outcomes. We will compare normal versu
overweight/obese children. Assume that the standard deviation in total cholesterol is 3

a. How many normal and overweight/obese children are required to ensure that a 95%
interval estimate for the difference in total cholesterol has a margin of error not exceed

376.5
2
Z 2 1 . 96( 35 )
n
Need n1 = 377 (normal
complete outcome data.
i = 2 =2 ( ) (
weight children)
E
and n2 = =
5 )
377 (overweight/obese childr

b. How many normal and overweight/obese children are required to ensure 80% power
4% difference in incident cardiovascular disease between groups? Assume that approx
of normal weight children will develop cardiovascular disease by age 50 and that 7% o
overweight/obese children will develop cardiovascular disease by age 50 and that a tw
with a=0.05 will be used.

 Need n1 = 377 (normal weight children) and n2 = 377 (overweight/obese childr
complete outcome data.

b. How many normal and overweight/obese children are required to ensure 80% power
4% difference in incident cardiovascular disease between groups? Assume that approx
of normal weight children will develop cardiovascular disease by age 50 and that 7% o
overweight/obese children will develop cardiovascular disease by age 50 and that a tw
with a=0.05 will be used.

Need n1 = 464 (normal weight children) and n2 = 464 (overweight/obese childr

complete outcome data.

c. How many normal and overweight/obese children should be enrolled to satisfy (a) an
assuming that 20% will be lost to follow-up over the course of the study?

A total of 928 children (464 * 2) will suffice for both parts (a) and (b).

N (number to enroll) * (% retained) = desired sample size

N (0.80) = 928
N = 928/0.80 = 1160

Need to enroll N = 1160 to account for 20% attrition.

13. An observational study is conducted to investigate the association between age a

serum cholesterol. The correlation is estimated at r = 0.35. The study involves n=12
participants and the mean (std dev) age is 44.3 (10.0) years with an age range of 35
and mean (std dev) total cholesterol is 202.8 (38.4).

a. Estimate the equation of the line that best describes the association between age
independent variable) and total serum cholesterol.

Y = Bo + B1 * X
Bo = 143.26
B1 = 1.344
Y = 143.26 + 1.344 X

b. Estimate the total serum cholesterol for a 50-year old person.

Y = 143.26 + 1.344 * 50
Y = 210.46

c. Estimate the total serum cholesterol for a 70-year old person.

Y = 143.26 + 1.344 * 40
Y = 197.02
 Y = 143.26 + 1.344 X

b. Estimate the total serum cholesterol for a 50-year old person.

Y = 143.26 + 1.344 * 50
Y = 210.46

c. Estimate the total serum cholesterol for a 70-year old person.

Y = 143.26 + 1.344 * 40
Y = 197.02
ation given to infants
who were given the
ly but not given the
extensive testing at
roposed and indicate

ias are not as big of

e, and confounders

pressures are measured at baseline (or the start of the study before any

Given, mean = 191

rmal distribution with a mean of 191
SD = 22.4

erol between 180 and 190?

3a) x
d as hyperlipidemic (Assume that P( x<180) 180
P( x<190) 190
 erol between 180 and 190?

d as hyperlipidemic (Assume that

3b)
P(x>200) 200

heir consumption of coffee. The

ed) are female?

c. 80 / 631
0.1267828843

ompound designed to improve wound

placebo. After treatment for 5 days with
d and the data are shown below. categorical or ordinal outcome
size of the wound is greater than 2
(O - E )
2
E
nt: Are treatment and the percent wound
cance.
O = observed
ents receiving the new compound. E = expected

success between the new compound and

success between treatments

success between treatments.

ents receiving the new compound.

success between the new compound and

success between treatments

success between treatments.

e of Wound
76-100% Total None

9 4.5673758865
46
10 9.4326241135
95
19 141
df = (5-1)(2-1)
X^2 =

Reject since 14.25 > 7.81

lower Answer
0.1160710259 (0.50, 0.67)

p2 z upper lower Answer

0.352 1.96 0.3523178213 0.11168218 (0.11, 0.35)

p1/p2
p2 z RR (relative risk)upper lower
0.352 1.96 1.6590909091 0.78639147 0.22614814

OR upper lower exp (upper) exp (lower)

2.5843531469 1.4605895476 0.4383609391 4.3084988 1.550164321
w compound designed to improve wound healing in
o. After treatment for 5 days with the new
the data are shown below.

Hint: Are treatment and the percent wound

nificance

Expected
Total 0-25% 26-50% 51-75%

25.5 41 33
125
25.5 41 33
125
250

df = (4-1)(2-1) =3 -->
dent of each other X^2 = 28.32

Reject since 28.32 > 7.81

ndividuals following a low carbohydrate diet for

, low calorie) diet for at least 6 months. The

between the diet programs using a 5% level of

ndividuals following a low carbohydrate diet for
, low calorie) diet for at least 6 months. The

between the diet programs using a 5% level of

Step 1. H0: u1 =
s2 n2 sp Z or t H1: u1 u2
21.6 75 22.7995310892 5.18905726
Step 2.

Step 3. Reject H0

Step 4. z = 5.19

Step 5. Reject H0

B, C) to a placebo in terms of their effects on weight

assigned to the comparison groups (in equal numbers).
he following data are observed after 3 months on

4 treatments? Use a 5% level of significance. Hint:

mean squares F

mean of means = 5

SSB = 20 (6.4 - 5) ^2 + 20 (8.9 - 5)^2 + 20 (2.2 - 5)^2 +

5)^2
 mean of means = 5
SSB / ( k - 1 ) ( MSB / MSE )
208.4 12.532362716 SSB = 20 (6.4 - 5) ^2 + 20 (8.9 - 5)^2 + 20 (2.2 - 5)^2 +
5)^2
SSE / ( N - k )
16.6289473684 Step 1: H0: All means are equal
H1: H0 is false
a = 0.05

Step 2: n j(X j X ) 2 /(k 1 )

F
(X X j ) 2 /(N k )
Step 3: Reject if F > 2.73

Step 4: F = 12.53

Step 5: Reject H0 since 12.53 > 2.73

luate an experimental medication to

w rate, a continuous variable measured in
onths on treatment. The investigators
The mean peak expiratory flow rate in
hould be enrolled to ensure 80% power to
a=0.05?
ned to improve wound healing in trauma
ts taking the new compound had a substantial
ebo group. The trial failed to show significance.
oportions observed in the trial with 80% power?

and n2 = 200 with complete

tions of childhood obesity on poor

cholesterol at age 50 and incident
ompare normal versus
total cholesterol is 35.

to ensure that a 95% confidence

in of error not exceeding 5 units?

eight/obese children) with

to ensure 80% power to detect a

Assume that approximately 3%
age 50 and that 7% of
age 50 and that a two sided test
eight/obese children) with

to ensure 80% power to detect a

Assume that approximately 3%
age 50 and that 7% of
age 50 and that a two sided test

eight/obese children) with

rolled to satisfy (a) and (b)

study?

nd (b).

iation between age and total

e study involves n=125
h an age range of 35 to 55 years,

ciation between age (as the

 120 Column1
148
112 Mean 142.33
128 Standard Erro 3.64
138 Median 145.00
143 Mode 145.00
145 Standard Devi 14.11
156 Sample Varia 199.10
135 Kurtosis 0.29
160 Skewness -0.72
150 Range 51.00
142 Minimum 112.00
145 Maximum 163.00
150 Sum 2135.00
163 Count 15.00

u SD normdist(true)
191 22.4 0.312
191 22.4 0.482
 0.171 (Subtracting 0.482 - 0.312)

191 22.4 0.656 0.344 since x> number, we have to substra

since x> number, we have to substract by 1 at the

inal outcome

df = (c - 1) (r - 1) H0: samples are independent of each other

Table 3 H1: H0 is false

Reject if X^2 is greater than df

 Expected
1-25% 26-50% 51-75% 76-100%

5.5460992908 5.8723404255 23.8156028369 6.1985815603

11.4539007092 12.1276595745 49.1843971631 12.8014184397

=4 --> Table 3
14.25 9.49

t since 14.25 > 7.81

exponential exponential Answer

2.1954597 1.2537614 (1.25, 2.19)

Answer
(1.55, 4.30)
 76-100%

25.5

25.5

Table 3
7.81
 Step 1. H0: u1 = u2 = 0.05
H1: u1 u2

Step 2. X1 - X 2
Z
1 1
Sp
n1 n2
Step 3. Reject H0 if z -1.96 or if z 1.96. (Table 1c)

Step 4. z = 5.19

Step 5. Reject H0 because z = 5.19, which is not -1.96 or 1.96

5)^2 + 20 (2.2 - 5)^2 + 20 (2.5 -

5)^2 + 20 (2.2 - 5)^2 + 20 (2.5 -

2.73
we have to substract by 1 at the end!!
bstract by 1 at the end!!

each other