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