Toward a standardized multidisciplinary team approach in preterm infants at-risk for pulmonary hypertension

Off‐label use of PAH‐targeted medications approved for adults and their financial coverage by health insurances are vital for children with pulmonary hypertension

“…The decision on the specific therapy for PH with the possibility of combining drugs of all substance classesincluding 'off-label pediatric formulations'-should be made by a PH expert with sufficient experience in the treatment of children with pulmonary hypertension-and especially with vasoactive drugs (Table 1). Given the heterogenous drug response depending on the aetiology of paediatric PH/PVD, it is highly advisable to build interdisciplinary teams 59 and networks 44 61 must be developed and produced in the future, but this is not a mandatory pre-requisite for 'off-label' use of such medications in children. In two prospective studies, children with PH tolerated add-on macitentan 43 or add-on selexipag 9 well.…”

“…The coverage of costs for ‘off‐label’ PAH medications is imperative if–according to expert opinion(11)–the agent (Table 1) can have a positive impact on the course of the disease. The decision on the specific therapy for PH with the possibility of combining drugs of all substance classes–including ‘off‐label pediatric formulations’–should be made by a PH expert with sufficient experience in the treatment of children with pulmonary hypertension–and especially with vasoactive drugs (Table 1). Given the heterogenous drug response depending on the aetiology of paediatric PH/PVD, it is highly advisable to build interdisciplinary teams 59 and networks 44 in paediatric PH centres that are dedicated to provide close outpatient follow‐up and tailored treatment, including objective and structured assessments of positive effects. Of note, the qualification ‘PH‐expert’ is currently not formally regulated in most countries.…”