Pharmacoepidemiological safety studies in children: a systematic review

Osemeke U Osokogu; Julijana Dukanovic; Carmen Ferrajolo; Caitlin Dodd; Alexandra C Pacurariu; Wichor M Bramer; Geert 'tJong; Daniel Weibel; Miriam C J M Sturkenboom; Florentia Kaguelidou

doi:10.1002/pds.4041

Pharmacoepidemiological safety studies in children: a systematic review

Osemeke U Osokogu, Julijana Dukanovic, Carmen Ferrajolo, Caitlin Dodd, Alexandra C Pacurariu, Wichor M Bramer, Geert 'tJong, Daniel Weibel, Miriam C J M Sturkenboom, Florentia Kaguelidou

Research output: Contribution to journal › Review article › peer-review

Abstract

PURPOSE: In order to identify challenges in pediatric pharmacoepidemiological safety studies, we assessed the characteristics of such (published) studies.

METHODS: Relevant articles from inception to 2013 were retrieved from Embase and Medline. We sequentially screened titles, abstracts and full texts with independent validation. We systematically collected data regarding general information, study methods and results.

RESULTS: Out of 4825 unique articles, 268 full texts (5.6%) were retained; 147 (54.9%) pertained to drugs rather than vaccines. Considering the 268 studies, 202 (75.4%) concerned children and adolescents (2 to 11 years) and 14 (5.3%) included preterm newborns. Most studies originated from North America (154 [57.5%]) or Europe (92 [34.3%]). Only 47 studies (17.5%) were privately funded. The majority (174 [64.9%]) were cohort studies. Out of 268 studies, 196 (73.1%) collected data retrospectively; paper medical charts were the most common data source for the exposures (85 [31.7%]) and outcomes (122 [45.5%]). Only 3 (2.0%) drug-only studies investigated rarely used drugs. Considering all 268 studies, only 27 (10.1%) reported sample size or power calculation. Most (75 [51.0%]) drug-only studies corrected confounding by multivariate modeling unlike stratification in 66 (55.9%) vaccine-only studies. Considering 75 child-only studies without any statistically significant result, 41 (54.7%) did not discuss lack of power.

CONCLUSIONS: Although the field of pediatric pharmacoepidemiology is steadily developing evaluation seldom includes neonates, is mainly focused on few drug classes and safety outcomes and concerns mainly drug use in developed countries. Small study size is a specific challenge in pediatrics. Reporting should be improved. © 2016 The Authors. Pharmacoepidemiology and Drug Safety Published by John Wiley & Sons Ltd.

Original language	English
Pages (from-to)	861-70
Number of pages	10
Journal	Pharmacoepidemiology and Drug Safety
Volume	25
Issue number	8
DOIs	https://doi.org/10.1002/pds.4041
Publication status	Published - Aug 2016
Externally published	Yes

Keywords

Adolescent
Child
Confounding Factors, Epidemiologic
Data Collection/methods
Drug-Related Side Effects and Adverse Reactions/epidemiology
Epidemiologic Research Design
Humans
Infant, Newborn
Multivariate Analysis
Pediatrics
Pharmacoepidemiology/methods
Research Support as Topic/statistics & numerical data

Access to Document

10.1002/pds.4041Licence: CC BY-NC-ND

Cite this

@article{eb86c6704a384ce98d2b94478a2d09cb,

title = "Pharmacoepidemiological safety studies in children: a systematic review",

abstract = "PURPOSE: In order to identify challenges in pediatric pharmacoepidemiological safety studies, we assessed the characteristics of such (published) studies.METHODS: Relevant articles from inception to 2013 were retrieved from Embase and Medline. We sequentially screened titles, abstracts and full texts with independent validation. We systematically collected data regarding general information, study methods and results.RESULTS: Out of 4825 unique articles, 268 full texts (5.6%) were retained; 147 (54.9%) pertained to drugs rather than vaccines. Considering the 268 studies, 202 (75.4%) concerned children and adolescents (2 to 11 years) and 14 (5.3%) included preterm newborns. Most studies originated from North America (154 [57.5%]) or Europe (92 [34.3%]). Only 47 studies (17.5%) were privately funded. The majority (174 [64.9%]) were cohort studies. Out of 268 studies, 196 (73.1%) collected data retrospectively; paper medical charts were the most common data source for the exposures (85 [31.7%]) and outcomes (122 [45.5%]). Only 3 (2.0%) drug-only studies investigated rarely used drugs. Considering all 268 studies, only 27 (10.1%) reported sample size or power calculation. Most (75 [51.0%]) drug-only studies corrected confounding by multivariate modeling unlike stratification in 66 (55.9%) vaccine-only studies. Considering 75 child-only studies without any statistically significant result, 41 (54.7%) did not discuss lack of power.CONCLUSIONS: Although the field of pediatric pharmacoepidemiology is steadily developing evaluation seldom includes neonates, is mainly focused on few drug classes and safety outcomes and concerns mainly drug use in developed countries. Small study size is a specific challenge in pediatrics. Reporting should be improved. {\textcopyright} 2016 The Authors. Pharmacoepidemiology and Drug Safety Published by John Wiley & Sons Ltd.",

keywords = "Adolescent, Child, Confounding Factors, Epidemiologic, Data Collection/methods, Drug-Related Side Effects and Adverse Reactions/epidemiology, Epidemiologic Research Design, Humans, Infant, Newborn, Multivariate Analysis, Pediatrics, Pharmacoepidemiology/methods, Research Support as Topic/statistics & numerical data",

author = "Osokogu, {Osemeke U} and Julijana Dukanovic and Carmen Ferrajolo and Caitlin Dodd and Pacurariu, {Alexandra C} and Bramer, {Wichor M} and Geert 'tJong and Daniel Weibel and Sturkenboom, {Miriam C J M} and Florentia Kaguelidou",

note = "{\textcopyright} 2016 The Authors. Pharmacoepidemiology and Drug Safety Published by John Wiley & Sons Ltd.",

year = "2016",

month = aug,

doi = "10.1002/pds.4041",

language = "English",

volume = "25",

pages = "861--70",

journal = "Pharmacoepidemiology and Drug Safety",

issn = "1053-8569",

publisher = "John Wiley & Sons Inc.",

number = "8",

}

TY - JOUR

T1 - Pharmacoepidemiological safety studies in children

T2 - a systematic review

AU - Osokogu, Osemeke U

AU - Dukanovic, Julijana

AU - Ferrajolo, Carmen

AU - Dodd, Caitlin

AU - Pacurariu, Alexandra C

AU - Bramer, Wichor M

AU - 'tJong, Geert

AU - Weibel, Daniel

AU - Sturkenboom, Miriam C J M

AU - Kaguelidou, Florentia

PY - 2016/8

Y1 - 2016/8

N2 - PURPOSE: In order to identify challenges in pediatric pharmacoepidemiological safety studies, we assessed the characteristics of such (published) studies.METHODS: Relevant articles from inception to 2013 were retrieved from Embase and Medline. We sequentially screened titles, abstracts and full texts with independent validation. We systematically collected data regarding general information, study methods and results.RESULTS: Out of 4825 unique articles, 268 full texts (5.6%) were retained; 147 (54.9%) pertained to drugs rather than vaccines. Considering the 268 studies, 202 (75.4%) concerned children and adolescents (2 to 11 years) and 14 (5.3%) included preterm newborns. Most studies originated from North America (154 [57.5%]) or Europe (92 [34.3%]). Only 47 studies (17.5%) were privately funded. The majority (174 [64.9%]) were cohort studies. Out of 268 studies, 196 (73.1%) collected data retrospectively; paper medical charts were the most common data source for the exposures (85 [31.7%]) and outcomes (122 [45.5%]). Only 3 (2.0%) drug-only studies investigated rarely used drugs. Considering all 268 studies, only 27 (10.1%) reported sample size or power calculation. Most (75 [51.0%]) drug-only studies corrected confounding by multivariate modeling unlike stratification in 66 (55.9%) vaccine-only studies. Considering 75 child-only studies without any statistically significant result, 41 (54.7%) did not discuss lack of power.CONCLUSIONS: Although the field of pediatric pharmacoepidemiology is steadily developing evaluation seldom includes neonates, is mainly focused on few drug classes and safety outcomes and concerns mainly drug use in developed countries. Small study size is a specific challenge in pediatrics. Reporting should be improved. © 2016 The Authors. Pharmacoepidemiology and Drug Safety Published by John Wiley & Sons Ltd.

AB - PURPOSE: In order to identify challenges in pediatric pharmacoepidemiological safety studies, we assessed the characteristics of such (published) studies.METHODS: Relevant articles from inception to 2013 were retrieved from Embase and Medline. We sequentially screened titles, abstracts and full texts with independent validation. We systematically collected data regarding general information, study methods and results.RESULTS: Out of 4825 unique articles, 268 full texts (5.6%) were retained; 147 (54.9%) pertained to drugs rather than vaccines. Considering the 268 studies, 202 (75.4%) concerned children and adolescents (2 to 11 years) and 14 (5.3%) included preterm newborns. Most studies originated from North America (154 [57.5%]) or Europe (92 [34.3%]). Only 47 studies (17.5%) were privately funded. The majority (174 [64.9%]) were cohort studies. Out of 268 studies, 196 (73.1%) collected data retrospectively; paper medical charts were the most common data source for the exposures (85 [31.7%]) and outcomes (122 [45.5%]). Only 3 (2.0%) drug-only studies investigated rarely used drugs. Considering all 268 studies, only 27 (10.1%) reported sample size or power calculation. Most (75 [51.0%]) drug-only studies corrected confounding by multivariate modeling unlike stratification in 66 (55.9%) vaccine-only studies. Considering 75 child-only studies without any statistically significant result, 41 (54.7%) did not discuss lack of power.CONCLUSIONS: Although the field of pediatric pharmacoepidemiology is steadily developing evaluation seldom includes neonates, is mainly focused on few drug classes and safety outcomes and concerns mainly drug use in developed countries. Small study size is a specific challenge in pediatrics. Reporting should be improved. © 2016 The Authors. Pharmacoepidemiology and Drug Safety Published by John Wiley & Sons Ltd.

KW - Adolescent

KW - Child

KW - Confounding Factors, Epidemiologic

KW - Data Collection/methods

KW - Drug-Related Side Effects and Adverse Reactions/epidemiology

KW - Epidemiologic Research Design

KW - Humans

KW - Infant, Newborn

KW - Multivariate Analysis

KW - Pediatrics

KW - Pharmacoepidemiology/methods

KW - Research Support as Topic/statistics & numerical data

U2 - 10.1002/pds.4041

DO - 10.1002/pds.4041

M3 - Review article

C2 - 27255559

SN - 1053-8569

VL - 25

SP - 861

EP - 870

JO - Pharmacoepidemiology and Drug Safety

JF - Pharmacoepidemiology and Drug Safety

IS - 8

ER -

Pharmacoepidemiological safety studies in children: a systematic review

Abstract

Keywords

Access to Document

Fingerprint

Cite this