Clinicoepidemiological research designs in childhood allergic diseases

Hyeon-Jong Yang

doi:10.4168/aard.2016.4.2.91

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Yang: Clinicoepidemiological research designs in childhood allergic diseases

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Allergy, Asthma & Respiratory Disease 2016; 4(2): 91-99.

Published online: 31 March 2016

DOI: https://doi.org/10.4168/aard.2016.4.2.91

Clinicoepidemiological research designs in childhood allergic diseases

Hyeon-Jong Yang

Department of Pediatrics, Soonchunhyang University Hospital, Soonchunhyang University College of Medicine, Seoul, Korea.

Correspondence to: Hyeon-Jong Yang. Department of Pediatrics, Pediatric Allergy and Respiratory Center, Soonchunhyang University College of Medicine, 59 Daesagwan-ro, Yongsan-gu, Seoul 04401, Korea. Tel: +82-2-709-9390, Fax: +82-2-794-5471, Ilove902@hanmail.net

Received 27 January 2016 Revised 6 March 2016 Accepted 8 March 2016

(open-access):

This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/).

Abstract

Clinical epidemiology is defined as a method for investigating the distribution and determinants of diseases and for applying this knowledge in their prevention, and simply means application of epidemiological methods for medical research. In evidence-based medicine, randomized controlled trials (RCT) are the gold standard for assessing efficacy and safety of the intervention, while it is commonly impractical because of many limitations, such as ethical/legal problems and weak external-validity. High internal-validity of RCT permits to assess the direct efficacy of intervention without interference with bias and confounder; however, it has less generalizability or applicability to the real-life practice. Evidence-based practical guidelines are developed for patient management and decision making in real-life practice; paradoxically, the evidence of the guidelines does not come from real life, but from strict trial life. To overcome these limitations, pragmatic clinical trials for assessing the effectiveness of intervention in real-life practice or high-quality observational studies would be the best alternatives or could add more strong evidence. This article provides an overview of clinicoepidemiological research designs in the field of childhood allergic diseases and their strength/weakness.

Keywords: Child, Epidemiology, Hypersensitivity, Pragmatic clinical trial, Randomized controlled trial, Research design

Figures and Tables

Fig. 1

Algorithm for classification of clinical epidemiology.

Fig. 2

Classification of clinicoepidemiological research designs.

Table 1

Level of prevention in the childhood allergic diseases

Level	Definition	Targets	Example
Primary prevention	To prevent development of disease through risk-protection or health-promotion	Gene Environments Life-style Diet Medicine	Vaccination Smoke-free legislation
Secondary prevention	Screening and early detection of latent diseases and early treatment of disease-development Prevention of the disease-related complications and disabilities	Biomarker Therapy	Screening test Efficacy and effectiveness of therapy
Tertiary prevention	To control and minimize a disease-related disabilities	Quality of life Depression Function	Psychotherapy Rehabilitation

Table 2

Ideal study designs according to purpose of study

Purpose	Study design
To estimate prevalence	Cross-sectional
To estimate incidence	Cohort
To identify causal relationship	Cohort, case-control
To study of rare diseases	Case-control
To study of rare exposure	Cohort study
To study of multiple exposures	Case-control
To study of multiple end points	Cohort study
To prevent development of disease	High risk cohort or interventional cohort
To prevent progress of disease	Case cohort
To estimate efficacy of intervention in pure population	Randomized controlled trial
To estimate comparative effectiveness of intervention in real-world practice	Pragmatic clinical trial
To estimate effectiveness of intervention in community	Community study

Table 3

Strengths and weaknesses of observational study designs in clinical epidemiology

Study design	Outcomes	Strengths	Weaknesses
Epidemiological study
Cross-sectional	Prevalence	Time requirement: low	Causal relationship cannot be estimated
Cross-sectional	Correlation	Study-costs: low	Biases: high
Case-control	Odds ratio	Optimal design to study causal relationship in rare disease	Control can be turned to be case
		Ability to study multiple exposures	Not practical for study of rare exposure
		Time requirement: lower than cohort	Multiple outcomes cannot be measured
		Study-costs: lower than cohort	Selection and recall biases: high
		Study-costs: lower than cohort	Confounding: high
Cohort	Incidence	Optimal design to study causal relationship in rare exposure	Not practical for study of rare outcome
	Relative risk	Ability to study multiple endpoints and outcomes	Time requirement: high
	Hazard ratio	Ability to study variables which change over time	Study-costs: high
		Causal relationship most clearly be estimated	Selection and recall biases: relatively low
			Confounding: relatively low
			Loss to follow up bias:high
Clinical study
Observational study with drugs in case cohort	Effectiveness	Similar with epidemiological retrospective-cohort study (exposure = given drug)	Similar with epidemiological retrospective-cohort study

Table 4

Advantages and disadvantages of experimental study designs in clinical epidemiology

Heading	Randomized clinical trial	Pragmatic clinical trial	Interventional cohort	Epidemiological, experimental, study
Subjects	Patients in narrow population	Patients in real-life population	High-risk population	Community
Randomization	Yes	Yes	Possible	No
Control	Yes	Yes	Yes	No
Setting	Highly controlled	Pragmatic controlled	Population-based	Community-based
Setting	Specialized center	Usual practice	Population-based	Community-based
Inclusion/exclusion criteria	Many	Few	Some	NA
Adherence/compliance	Very good	Low	NA	NA
Therapy	Blinded	Blinded or open-labelled	Blinded, usually single	Open
Comparator	Placebo	Other type of real practice	Nonintervention	Before intervention
Outcome	Efficacy	Comparative effectiveness	Efficacy or effectiveness	Effectiveness
Study purpose	Hypothesis testing	Decision-making	Hypothesis testing	Hypothesis testing

NA, nonapplicable.

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ORCID iDs: Hyeon-Jong Yang
https://orcid.org/http://orcid.org/0000-0002-7287-4300
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