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- Title
Off‐label use of PAH‐targeted medications approved for adults and their financial coverage by health insurances are vital for children with pulmonary hypertension.
- Authors
Hansmann, Georg; Christou, Helen; Koestenberger, Martin; Sallmon, Hannes
- Abstract
The aim of combination pharmacotherapy is to allow PH patients to live independently if possible, with an acceptable or good quality of life.9,29 Postponing the listing for lung transplantation19 in patients at higher risk can be safely achieved by combination PAH therapy in treatment-responsive patients who do improve in the EPPVDN risk class and scores.29 Ongoing pediatric randomized controlled PAH trials and published observational studies There are currently no large, randomized prospective study data sets on childhood PAH combination therapy available. Nevertheless, the mainstay of therapy worldwide for two of the most common forms of PH in childhood, that is, PH associated with bronchopulmonary dysplasia in preterm infants (BPD-PH)27 and PH associated with congenital heart disease,31 is the oral phosphodiesterase-5 (PDE5) inhibitor sildenafil, either as monotherapy or in combination with an endothelin receptor antagonist (ERA). Keywords: children; macitentan; off-label; pulmonary hypertension; riociguat; selexipag EN children macitentan off-label pulmonary hypertension riociguat selexipag 1 7 7 07/27/21 20210801 NES 210801 Key points Pulmonary Hypertension is currently an incurable condition in adults and children. 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).
- Subjects
PULMONARY hypertension; ADULTS; HEALTH insurance; OFF-label use (Drugs); DRUGS; RIGHT ventricular dysfunction
- Publication
European Journal of Clinical Investigation, 2021, Vol 51, Issue 8, p1
- ISSN
0014-2972
- Publication type
Article
- DOI
10.1111/eci.13571