1 INTRODUCTION
Off-label drug use is defined as prescribing marketed drugs outside of
the authorized age, indication, weight, dose, formulation or route of
administration.1, 2 Drugs have historically been
commonly used off label in pediatric population due to the challenge and
difficulty to perform clinical trials considering the ethical concerns1 and the dearth of therapeutic options. Neonates in
the neonatal intensive care units (NICUs) are especially prone to
off-label drug use.3-8 Furthermore, neonates are
particularly vulnerable and frequently premature in body systems such as
drug absorption, metabolism, transportation and elimination, resulting
in variations in the pharmacokinetic (PK) and pharmacodynamic (PD)
properties and efficacy or toxicity of drugs administered to the
infants.9 For this reason, off-label drug use in
neonates has been associated with higher risk of adverse drug
reactions.10 Until recently, there are no consensus
guidelines about off-label drug use in global. In China, some expert
consensus have been reported and a specific law or regulation for
off-label drug use is absent.11 Rates of off-label
drug use varied as reported by studies in Chinese NICUs due to different
grades of hospitals and definitions of off-label
use.12-14 We sought to conduct a retrospective,
multicenter study including NICUs from regional, representative and
different grade hospitals to describe the recent characteristics of
off-label prescribing in Chinese neonates.