UNAVOIDABLE USE OF “OFF LABEL” PHARMACOTHERAPY:
There are now more 15 medications approved by the Food and Drug
Administration (FDA) for the treatment of PAH, almost all of which are
only formally approved for patients ≥ 18 years of age. As with many
disease processes, pediatric clinicians have employed many of these
agents empirically in infants, children and adolescents, often with
documented clinical benefit. Except for inhaled nitric oxide therapy
(NO), most studies of current PH-targeted drugs are based on adult
trials and only small case series in children. Problems related to the
lack of sufficient data that address pediatric needs is exemplified by
statements by the FDA on sildenafil use in children led to confusion and
concern on the part of clinicians, insurers, and patient
families.23