Metodologia cercetrii tiinifice

Colectarea datelor
Tipuri de variabile
Strategii de msurare

Corin Badiu, 2015

Obiective
Stabilirea tipurilor de variabile

Surse IT de date
Identifica si localizeaz seturile corecte de date
In instituie
In afara instituiei

Strategii de msurare
Analiza, interpretarea si raportarea rezultatelor

Selecteaz programul adecvat: Excel, SPSS

Folosete programele pentru analize statistice simple si
prezentarea grafica a rezultatelor
Interpreteaz rezultatele

Tipuri de variabile
Variabile de
confuzie*

Predictor*

Rezultat

Modificatori ai efectului*
*Considerate general ca expunere la factori de risc

Tipuri de studii clinice

Studii fr variabile

Studii de caz, serii de cazuri, editoriale,

opinii / comentarii, rapoarte review

Studii cu o singura variabila

Studii descriptive

Studii cu 2 variabile

Experimente
Studii observaionale
Meta-analize si review-uri sistematice

Ierarhia tipurilor de studii clinice

Studii clinice
Descriptive

Analitice

Experimentale

Observationale

Cohorta

Caz-control

Cross-sectional

Variabile

Variabila predictor
(independenta)

Variabila rezultat
(dependenta)

Evidence Based Medicine

Metode de tratament susinute de dovezi clinice si de
cercetare.
Necesita integrarea celor mai bune dovezi din cercetare
pentru diagnostic si tratament cu experiena clinica.
Ia in considerare ce este optim pentru fiecare pacient
precum i preferinele acestuia.

Realitatea

Cercetarea asupra eficientei unui tratament face subiectul unui

numr mic de articole.

Evidence based medicine este considerat un concept ce

folosete baze de date inclusiv studii sistematice de caz
pentru a ghida intervenii terapeutice.

Dovezile trebuie evaluate intr-un context terapeutic efectiv, Ce

tip de intervenie capt sens pentru mine ca practician?

ntrebri clinice
Care este cea mai bun alegere terapeutic pentru pacientul meu?
Este acest program suficient de bine documentat teoretic?
Funcioneaz acest program terapeutic?
Ct timp dureaz acest program?
Ce urmeaz dup aceea?

Utilitatea clinicii!
Clinicienii

Sunt n linia I a asistentei medicale

Au expertiza clinica necesara

i cunosc bine pacienii

Sunt formai pentru gndire tiinifica

Sunt obinuii cu colectarea datelor

tiu sa identifice rezultate

Msurtori de baz
Linia de baz este o msurtoare a ratei de
rspuns in absena tratamentului

Linia de baz

Stabilete necesitatea tratamentului

Permite mbuntirea documentaiei
Ne permite s mbuntim studiul

Date de baz
Se creeaz un set de exemplare ale intelor
studiului
Pregtim o fi de nregistrare
Folosim msurtori conform criteriilor de referin

Strategii de Colectare a Datelor

ntotdeauna alegei mai mult dect o msurtoare
Verific acurateea datelor de baz
Selecteaz designul cercetrii clinice

Design de Cercetare Clinic

ABA design
Test, tratament, test
ABAB design
Test, tratament, test, tratament
Design serial n timp
Stabilete o linie de baz
ncepe tratamentul
Msoar rezultatele tratamentului
Design cu linii de baz multiple
Obine un numr de linii de baz diferite
Fiecare linie de baz trebuie s fie independent de altele
Trateaz doar o variabil odat

Instrumente de colectare a datelor

Cerine

Fiabilitate
Valide
Sensibilitate
Universale
Fr erori

Instrumente de colectare a datelor

Ce inseamn fiabilitate?
Instrumentul va putea msura cu aceeai acuratee:
n situaii diferite de testare?
Testeaz - retesteaz fiabilitatea

Experimentatori diferii?
Fiablitate Inter - operator

Instrumente de colectare a datelor

Este valid?
Instrumentul care
va fi folosit poate
msura datele pe
care le dorim?

Instrumente de colectare a datelor

Este Sensibil?
Instrumentul ar trebui sa fie la fel de sensibil indiferent
de prezenta sau nu a variabilei msurate i de cantitatea
acesteia (linearitate).
Trebuie sa se caracterizeze valorile:
Fals-pozitivi:
Subiectii sanatosi, prezentati ca bolnavi.

Fals-negativi:
Subiecii bolnavi, prezentai ca sanatosi.

Instrumente de colectare a datelor

Este universal?
Investigatorul ar trebui sa foloseasc un
instrument larg aplicat care sa ajute la
compararea datelor i diminuarea erorilor
de raportare, pentru a face posibila
compararea cu date din literatura.

Instrumente de colectare a datelor

Este fara erori?

Ar trebui sa nu existe diferene intre valoarea reala i valoarea pe

care un investigator o obine prin msurare altele dect
diferente date de variabilitatea de eantionare.

Metode de colectare
1. Colectare Randomizata (Simpla)
2. Colectare Sistematica
3. Colectare Stratificata
4. Colectare pe grupe / segmente
5. Colectare pe alte criterii
6. Alte tipuri de colectare

Variabile Calitative i Cantitative

Exemple de variabile calitative sunt ocupatia, sexul, status

marital, etc.
Variabilele care permit cuantificarea i care pot fi msurate
sunt considerate variabile cantitative. Exemple de variabile
cantitative sunt greutatea, naltimea, vrsta.
Variabilele cantitative pot fi clasificate ca discrete sau continue

Tipuri de variabile

1.

Variabile de tip categorii (e.g., Sex, Status Marital,

religie, naionalitate, profesie)

2.

Variabile de tip continuu (e.g., vrsta, venit,

greutate, inaltime)

3.

Variabile discrete (e.g., Numar de copii)

4.

Variabile Binare sau Dihotomice (e.g., ntrebri la

care se da raspuns prin Da sau Nu)

Scale de Date
1. Nominale: Aceste date nu reprezint o cantitate (e.g., Status Marital,
Sex)
2. Ordinale: Aceste date reprezint o serie ordonata de elemente interrelaionate (e.g., nivelul de educatie)
3. Interval: Aceste date reprezint o cantitate msurata pe un scala de
interval avnd un numr egal de uniti i un punct zero arbitrar.
(e.g.: Temperatura in Celsius)
4. Raport : Variabile, cum este greutatea pentru care putem compara
semnificatia uneia fata de cealalta (, 100 Kg este dublul a 50 Kg)

Variabile din protocol

TIPURI de VARIABLE

independente

dependente

intermediare

de confuzie

Variabila Independent
O caracteristica pentru care se face o
msurtoare i/sau observaie, care influeneaz
un eveniment sau rezultat (variabila dependenta).
NOTA
Variabila independenta nu este influenat de
eveniment sau rezultat, dar o poate determina
sau contribui la variaia sa.

Variabila Dependent
O variabil a crei valoare este dependent de
efectul altor variabile (variabila independenta)
in raportul de cauzalitate studiat.
Sinonime: variabila rezultat sau rspuns.

Variabile Intermediare
O variabila care apare intr-o relaie de cauzalitate de la o
variabila independenta la o variabila dependenta.
Sinonime: intervenie, mediere
NOTE
Produce variaii in variabila dependenta i variaz
datorita variabilei independente.

O asemenea variabila este asociata att cu variabila

dependenta cat i cu variabila independenta.

Variabile de confuzie
Un factor (care este el insusi determinant al
rezultatului), care perturba efectul aparent al unei
variabile de studiu asupra rezultatului.
NOTA
Un asemenea factor poate fi distribuit inegal intre
cei expui i neexpui i astfel influeneaz
magnitudinea aparenta i chiar direcia efectului.

Organizarea Datelor
1. Tabel de frecventa
2. Histograma de frecventa
3. Histograma de frecventa relativa
4. Histograma
5. Poligon de frecventa
6. Poligon de frecventa relativa
7. Grafic cu coloane
8. Grafic placinta
9. Tabel Trunchi cu ramuri
10.Box plot

Tabel de frecventa
S presupunem c suntem interesai n studierea numrului
de copii n familiile care triesc ntr-o comunitate.
Urmtoarele date au fost colectate pe baza unui eantion
aleatoriu de n = 30 de familii din comunitate.
2, 2, 5, 3, 0, 1, 3, 2, 3, 4, 1, 3, 4, 5, 7, 3, 2, 4, 1, 0, 5, 8, 6,
5, 4 , 2, 4, 4, 7, 6
Organizai aceste date ntr-un tabel de frecvene!

Tabel de frecventa
X=Nr. de copii

Frecventa

Frecventa relativa

2/30=0.067

3/30=0.100

5/30=0.167

5/30=0.167

6/30=0.200

4/30=0.133

2/30=0.067

2/30=0.067

1/30=0.033

Grafic de frecventa

Tabel de frecventa
Acum, s presupunem c avem nevoie s construim un tabel de
frecven similar pentru vrsta pacienilor cu probleme cardiace intr-o
clinica.
Urmtoarele date au fost colectate pe baza unui eantion aleatoriu de n =
30 pacieni care au mers la camera de gard a clinicii pentru probleme
cardiace.
Masuratorile sunt: 42, 38, 51, 53, 40, 68, 62, 36, 32, 45, 51, 67, 53, 59,
47, 63, 52, 64, 61, 43, 56, 58, 66, 54, 56, 52, 40, 55, 72, 69.

Tabel de frecventa
Grupe de varsta

Frecventa

Frecventa Relativa

32 -36.99

2/30=0.067

37- 41.99

3/30=0.100

42-46.99

4/30=0.134

47-51.99

3/30=0.100

52-56.99

8/30=0.267

57-61.99

3/30=0.100

62-66.99

4/30=0.134

67-72

3/30=0.100

Total

n=30

1.00

Msurarea tendinei centrale

Empiric
Pentru o distribuie normal de aproximativ
a) 68% din msurtori se ncadreaz n jurul valorii de o
deviaie standard faa de medie
b) 95% din msurtori se ncadreaz n termen de dou deviaii
standard n jurul mediei
c) 99,7% din msurtori se ncadreaz n termen de trei abateri
standard n jurul mediei

Interval de ncredere

Distribuie, Tendina centrala

Mean

Concepte necesare

Scale de msura
Distribuie, tendina centrala, variabilitate,
probabilitate
Prevalenta i incidenta bolilor
Rezultate ale bolilor (mortalitate, morbiditate)
Asocieri (corelaie sau covariana)
Impact asupra sanatatii (diferene de risc i rapoarte)
Sensibilitate, specificitate, valoare predictiva

Scale

Nominal - clasificarea calitativ de valoare egal:

sex, ras, culoare, ora
Ordinal - clasificarea calitativ, care poate fi ordonata
dupa rang: statutul socio-economic al familiilor

Interval - date numerice sau cantitative: pot fi

ordonate dupa rang i comparate: temperatura

Interval cu o valoare de referinta: de timp sau spaiu

Prevalena i Incidena
Prevalenta = probabilitatea bolii in
intreaga populatie, in orice moment
2% din populatie are diabet zaharat

Incidena=probabilitatea ca un subiect sa
dezvolte boala pe parcursul intervalului
0,2% sau 2 la 1000 de cazuri noi pe an

Sensibilitate, Specificitate
Pacienti
cu boala

sensibilitate
= a / (a+c)

specificitate
= d / (b+d)

Test
pozitiv
Test
negativ

Pacienti fara
boala

Valoare Predictiva

Pacienti
cu boala

Valoare predictiva
Pozitiva = a / ( a+b)
Test
pozitiv

Valoare predictiva
Negativa = d / (c+d)

Test
negativ

Pacienti fara
boala

Eroarea n trialuri clinice

Domeniile n care poate aprea eroare
Eroare sistematic n. . .
alocare
rspuns
evaluare

Eroarea n trialuri clinice

Eroarea de alocare sau susceptibilitate
Poate aparea atunci cand alocarea unui pacient la un grup de
studiu este influenata de cunoaterea de catre investigator a
tratamentului care urmeaz s fie primit.
Poate duce la grupuri de tratament cu prognostic diferit.

Eroarea n trialuri clinice

Eroarea de alocare sau susceptibilitate
Grupurile de tratament trebuie s aib prognostic similar,
care se realizeaz prin:
Randomizarea pacienilor
Evaluarea prospectiv a pacienilor
Definirea clara a criteriilor de includere i excludere

Randomizarea n trialuri clinice

Apare atunci cand pacientii sunt atribuiti pentru tratamente
prin intermediul unui mecanism care impiedica att
pacienii cat i cercettorul de la cunoaterea tratamentului
care se atribuie.

Beneficiile Randomizarii
Previne introducerea sistematic a erorii.
Diminueaz posibilitatea de eroare de alocare.
Echilibreaza factori de prognostic pentru grupurile
de tratament.
mbuntete validitatea testelor statistice utilizate
pentru a compara tratamentele.

Eroarea n trialuri clinice

Eroarea de rspuns i evaluare / Eroarea de inregistrare
Poate aparea atunci cand un pacient raporteaza un raspuns
de tratament sau atunci cnd un anchetator evalueaz
acest rspuns; fiecare persoan poate fi influenat de
cunoaterea tratamentului.
Un pacient sau un anchetator poate avea o idee
preconceput despre care tratament este mai bun.

Eroarea n trialuri clinice

Mascarea
Pentru a minimiza eroarea de raspuns si de evaluare /
de nregistrare
Single Blind (patient blinded): protejeaza pentru eroarea
de raspuns.
Double Blind (patient and investigator blinded): protejeaza
pentru eroarea de raspuns si de evaluare / de nregistrare.

Eroarea n trialuri clinice

Transfer bias
Occurs when patients are lost to follow-up.
Must be minimized.
Performance bias
Can occur with a single surgeon or with
multiple surgeons.

Confuzia - factori
Relationship between coffee and
pancreatic cancer, BUT
Smoking is a known risk factor for
pancreatic cancer
Smoking is associated with coffee
drinking but it is not a result of coffee
drinking.

Detectarea confuziei
If an association is observed between
coffee drinking and pancreatic cancer

Coffee actually causes pancreatic cancer,

or
The coffee drinking and pancreatic cancer
association is the result of confounding by
cigarette smoking.

Evaluarea confuziei
If you know something is a possible
confounder, in the data analysis use

Stratification, or
Adjustment

Fear the unknown!

Taxonomia designului de studii

Treatment vs. Observational
Prospective vs. Retrospective
Longitudinal vs. Cross-sectional
Randomized vs. Non-Randomized
Blinded/Masked or Not
Single-blind, Double blind, Unblinded

Tipuri de studii randomizate

Parallel Group
Sequential Trials
Group Sequential trials
Cross-over
Factorial Designs

Grup paralel
Randomize patients to one of k
treatments
Response

Measure at end of study

Delta or % change from baseline
Repeated measures
Function of multiple measures

Studiul Ideal - Gold Standard

Double blind
Randomized
Parallel groups

2 studii
Study 1

A U.S. study (2000) compared 469 patients with brain cancer

to 422 patients who did not have brain cancer. The patients
cell phone use was measured using a questionnaire. The two
groups use of cell phones was similar.

Study 2

An Australian study (1997) conducted a study with 200

transgenic mice. One hundred were exposed for two 30
minute periods a day to the same kind of microwaves with
roughly the same power as the kind transmitted from a cell
phone. The other 100 mice were not exposed. After 18
months, the brain tumor rate for the exposed mice was twice
as high as that for the unexposed mice.

Diferente?
How do the two studies differ?

Study 1

Study 2

?
Why do the results of different medical
studies sometimes disagree?

Could the second study be performed

on human beings?

Evaluarea faptelor
Suppose a friend recently diagnosed with
brain cancer was a frequent cell phone
user. Is this strong evidence that frequent
cell phone use increases the likelihood of
getting brain cancer?

Informal observations of this type are called

_____________ _____________.
You should rely on reputable research studies,
not anecdotes.

Cum putem obine informaii

Observational Study

The researcher observes values of the response and

explanatory variables for the sampled subjects without
imposing any treatments
Example:

Experiment

The researcher assigns experimental conditions (also

called treatments) to subjects (also called experimental
units) and then observes outcomes on the response
variable.
Treatments correspond to values of the explanatory
variable
Example:

Tipuri de studii observationale

Retrospective

Observational studies that look back in time

This is sometimes done to find risk factors for certain
diseases

Cross-Sectional

Observational studies that take a cross section of

the population at the current time

Prospective

Observational studies in which subjects are

followed into the future

Studii Experimentale /
Observaionale
In an observational study, there can always be
confusion variables affecting the results.
It is easier to adjust for confusion variables in an
experiment.
In general, we can study the effect of an explanatory
variable on a response variable more accurately with
an experiment than with an observational study.

Design al colectrii datelor pentru

studii observaionale
Simple Random Sampling (SRS)

A simple random sample of n subjects from a population is

one in which each possible sample of that size has the
_______ chance of being selected.

Colectarea datelor n studii

observaionale
Stratified Sampling

A stratified random sample divides the population into

separate groups, called ________, and then selects an SRS
from each stratum.

Colectarea datelor n studii

observaionale

Cluster Sampling

A cluster random sample can be used if the target population

naturally divides into groups, each of which is representative of the
entire target population. In this method, a SRS of groups (or strata)
is taken. Every member of the selected groups is put into the
sample.

Colectarea datelor n studii

observaionale
Systematic Sampling

A systematic sample selects every kth person from

the sample frame. The researcher randomly
selects a number between 1 and k in order to
know which person to select first, then selects
every kth person after this.

Avantaje ale colectrii

Simple Random Sampling (SRS)

It is the easiest most widespread form of sampling.

Each subject has an _______ chance to be in the
sample.
The sample enables us to determine how likely it is
that descriptive statistics (like the sample mean)
fall close to corresponding values for which we
would like to make inference (like the population
mean).

Eroarea n colectarea datelor

A sampling method is _________ if

The sample tends to favor some parts of the

population over others.
In other words, the results from the sample are not
representative of the population.

Obviously, __________ samples are our goal.

Tipuri de erori
Undercoverage

Occurs when a sampling frame leaves out some groups in

the population

Nonresponse bias

Occurs when some sampled subjects cannot be reached,

refuse to participate or fail to answer some questions

Response bias

Occurs when the subject gives an incorrect response or

when the question wording or the way the interviewer asks
the questions is confusing or misleading

Elemente pentru un studiu corect

Control group

Gives us something to compare against

Enables us to control the __________ _______
The placebo effect occurs when patients seem to improve
regardless of the treatment they receive.

Randomization

Eliminates ______ that can result when researchers assign

treatments to the subjects
Balances the group on variables that you know affect the
response
Balances the group on _________ variables that may be
unknown to you

Elemente pentru un studiu corect

Blinding

Increases reliability of the results

_________-blind: subjects do not know the
treatment assignment
_________-blind: neither the subjects nor those
in contact with the subjects know the treatment
assignment

Exemplu

A pharmaceutical company has developed a new drug for treating

high blood pressure. To determine the effectiveness of the drug,
the company conducted an experiment in which subjects with a
history of high blood pressure were treated with the new drug.

A later experiment randomly divided subjects with a history of

high blood pressure into two groups. Group A was treated with
the new drug as before. Group B received the most popular drug
on the market at that time. The subjects were unaware of which
treatment they received. 60% of the patients in Group A
improved, while 63% of the patients in Group B improved.

The __________ experiment is better because

Exemplu

To investigate whether antidepressants help smokers to quit smoking,

one study used 429 men and women who were 18 or older and had
smoked 15 cigarettes or more per day in the previous year. They were
all highly motivated to quit and in good health. They were assigned to
one of two groups: one group took an antidepressant called Zyban,
while the other group did not take anything. At the end of a year, the
study observed whether each subject had successfully abstained from
smoking.

Studii comparative randomizate

Randomization ensures that the groups of subjects
are similar in all respects before the treatments are
applied.
Using a control group for comparison ensures that
external influences operate equally on both groups.
If the groups are large enough, natural differences in
subjects will average out.
This means that there be little difference in the results
for the groups unless the treatments themselves
actually cause the difference.

Studii i erori

Types of studies:

Observational studies and experiments

Experiments control for lurking variables

Sampling designs:

SRS, stratified random samples and cluster samples

SRS is the preferred method

Potential sources of bias:

Undercoverage

Response bias

Nonresponse bias

Convenience sampling

Voluntary response sampling

Elements of good experiments:

Control group, randomization and blinding

Precauii
If a group is underrepresented in the sample, we cannot
make inference about it.
We must be careful when interpreting the results of
observational studies.
For comparison of several treatments to be valid, you must
apply all treatments to similar groups of experimental units.
Interesting questions are usually pretty tough to answer. This
is due in part to the fact that no single experiment or
observational study can determine causation.

Stop and Think!!!

Write the study!
Describe & classify the
variables.
Instruments for measure?
Bias?
Prepare to analyze data!