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

Pulmonary hypertension (PH), and especially pulmonary arterial hypertension (PAH), are chronic, progressive, and fatal conditions for which - apart from bilateral lung transplantation - there is currently no cure. However, recently developed, so-called "targeted" ("advanced") PAH medications, are now approved for adult PAH, and have improved quality of life and survival of affected children and adults. In 2020, two prospective pediatric observational studies on add-on macitentan and selexipag, respectively, have been published. Due the lack of approved pediatric PAH-medications, the well-founded rationale for combination therapy in childhood, and the absence of serious adverse effects, advanced pharmacotherapy should not be withheld from young PH patients. Such an "off-label use" of PAH-targeted medications in children with PH, and its financial reimbursement by health insurances, are urgently needed. The decision on the specific, vasoactive pharmacotherapy should be made by a PH expert with sufficient experience in the diagnosis and treatment of children with PH - followed by close out-patient monitoring. Combination pharmacotherapy should include the option of combining drugs of all substance classes (phosphodiesterase inhibitors, soluble guanylate cyclase stimulators, endothelin receptor antagonists, drugs targeting prostacyclin pathways, and calcium channel blockers in select cases) - including "off-label, pediatric drug formulations". Neither the lack of approval for modern PAH drugs directed against PAH nor the limited evidence-based study data available for children, should preclude PH expert centers to offer these therapies early and in combination to young patients with progressive PH, in order to improve exercise capacity, quality of life, and survival.