Recommendations on Off-Label Drug Use in Pediatric Guidelines

Min Meng; Min Meng; Min Meng; Min Meng; Qi Zhou; Qi Zhou; Wenjuan Lei; Min Tian; Ping Wang; Ping Wang; Yunlan Liu; Yajia Sun; Yaolong Chen; Yaolong Chen; Yaolong Chen; Yaolong Chen; Yaolong Chen; Yaolong Chen; Yaolong Chen; Yaolong Chen; Yaolong Chen; Qiu Li; Qiu Li; Qiu Li

doi:10.3389/fphar.2022.892574

Frontiers in Pharmacology (Jun 2022)

Recommendations on Off-Label Drug Use in Pediatric Guidelines

Min Meng,
Min Meng,
Min Meng,
Min Meng,
Qi Zhou,
Qi Zhou,
Wenjuan Lei,
Min Tian,
Ping Wang,
Ping Wang,
Yunlan Liu,
Yajia Sun,
Yaolong Chen,
Yaolong Chen,
Yaolong Chen,
Yaolong Chen,
Yaolong Chen,
Yaolong Chen,
Yaolong Chen,
Yaolong Chen,
Yaolong Chen,
Qiu Li,
Qiu Li,
Qiu Li

Affiliations

Min Meng: Department of Chevidence Lab Child & Adolescent Health, Children’s Hospital of Chongqing Medical University, Chongqing, China
Min Meng: Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Children’s Hospital of Chongqing Medical University, Chongqing, China
Min Meng: Chongqing Key Laboratory of Pediatrics, Chongqing, China
Min Meng: Department of Pharmacy, Gansu Provincial Hospital, Lanzhou, China
Qi Zhou: Institute of Health Data Science, Lanzhou University, Lanzhou, China
Qi Zhou: Evidence-Based Medicine Center, School of Basic Medical Sciences, Lanzhou University, Lanzhou, China
Wenjuan Lei: Department of Pharmacy, Gansu Provincial Hospital, Lanzhou, China
Min Tian: Department of Pharmacy, Gansu Provincial Hospital, Lanzhou, China
Ping Wang: Institute of Health Data Science, Lanzhou University, Lanzhou, China
Ping Wang: Evidence-Based Medicine Center, School of Basic Medical Sciences, Lanzhou University, Lanzhou, China
Yunlan Liu: School of Public Health, Lanzhou University, Lanzhou, China
Yajia Sun: School of Public Health, Lanzhou University, Lanzhou, China
Yaolong Chen: Department of Chevidence Lab Child & Adolescent Health, Children’s Hospital of Chongqing Medical University, Chongqing, China
Yaolong Chen: Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Children’s Hospital of Chongqing Medical University, Chongqing, China
Yaolong Chen: Chongqing Key Laboratory of Pediatrics, Chongqing, China
Yaolong Chen: Institute of Health Data Science, Lanzhou University, Lanzhou, China
Yaolong Chen: Evidence-Based Medicine Center, School of Basic Medical Sciences, Lanzhou University, Lanzhou, China
Yaolong Chen: School of Public Health, Lanzhou University, Lanzhou, China
Yaolong Chen: Research Unit of Evidence-Based Evaluation and Guidelines, Chinese Academy of Medical Sciences (2021RU017), School of Basic Medical Sciences, Lanzhou University, Lanzhou, China
Yaolong Chen: WHO Collaborating Centre for Guideline Implementation and Knowledge Translation, Lanzhou, China
Yaolong Chen: 0GRADE Center, Lanzhou University, Lanzhou, China
Qiu Li: Ministry of Education Key Laboratory of Child Development and Disorders, National Clinical Research Center for Child Health and Disorders, China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Children’s Hospital of Chongqing Medical University, Chongqing, China
Qiu Li: Chongqing Key Laboratory of Pediatrics, Chongqing, China
Qiu Li: 1Department of Nephrology, Children's Hospital of Chongqing Medical University, Chongqing, China

DOI: https://doi.org/10.3389/fphar.2022.892574
Journal volume & issue: Vol. 13

Abstract

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Objective: To systematically analyze the supporting evidence, drug information, and the type of off-label drug use in recommendations on off-label drug use in pediatric guidelines.Methods: A cross-sectional study was performed by systematic search through MEDLINE (via PubMed) and Embase databases to identify literature published from 1 January 2018, to 31 December 2020. Only pediatric clinical practice guidelines that included recommendations on off-label use of drugs were included. We present descriptive information on the sources of the included guidelines, country, publication year, evidence grading system used, details on the types of off-label drug use, and the types of studies used as references to support the recommendations.Results: A total of 66 pediatric guidelines with 605 recommendations were included. Eighty-seven (14.4%) recommendations did not cite any references; and the remaining 518 recommendations were supported by 2,240 references (mean 4.3 references/recommendation). The most common types of studies cited were pediatric RCTs (n = 314, 14.0%), pediatric case series studies (n = 260, 11.6%), and reviews (n = 255, 11.4%). Twenty-one percent (n = 470) of the references were studies on adults. One hundred and forty (23.1%) recommendations were graded using the Grading of Recommendations, Assessments, Development, and Evaluations (GRADE) system, of which 37 (26.4%) were graded as strong but supported with only C or D level of evidence. The most commonly reported type of information in the recommendations was indication (n = 499, 82.5%). The most commonly addressed type of off-label drug use in the 523 positive recommendations was unapproved population (n = 255, 48.8%). Sixty-nine (11.4%) recommendations explicitly reported the drug use as off-label.Conclusion: Children may be exposed to medical risks due to gaps in reporting and evidence of off-label drug use recommendations in pediatric guidelines.

Published in Frontiers in Pharmacology

ISSN: 1663-9812 (Online)
Publisher: Frontiers Media S.A.
Country of publisher: Switzerland
LCC subjects: Medicine: Therapeutics. Pharmacology
Website: http://journal.frontiersin.org/journals/pharmacology

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