Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz
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SHR Neuro Krebs Kardio Lipid Stoffw Microb
Hansmann, G; Apitz, C; Humpl, T; Koestenberger, M; Michel-Behnke, I; Schulze-Neick, I; Quandt, D; Lammers, AE.
Urgent need for off-label use of PAH medications and reimbursement for children with pulmonary hypertension Statement of the Working Group on Pulmonary Hypertension of the German Society for Pediatric Cardiology and Congenital Cardiac Defects (DGPK).
MONATSSCHR KINDERH. 2020;
Doi: 10.1007/s00112-020-00924-w
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- Co-Autor*innen der Med Uni Graz
- Koestenberger Martin
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- Abstract:
- Pulmonary hypertension (PH) is a pathological increase in pressure in the pulmonary arteries. The causes of PH are multifactorial and include pathological changes in the pulmonary vessels, diseases of the lung parenchyma or interstitium, or anomalies/dysfunction of left heart structures. Pulmonary arterial hypertension (PAH) is a chronic, progressive and fatal condition for which there is currently no curative treatment, apart from bilateral lung transplantation. Before modern specific medications became available, for children the average survival after the diagnosis PAH was less than 1 year. Due to the latest developments and availability of new PAH-targeted drugs (advanced or targeted therapies), which are approved for PAH in adults, the life expectation and quality of life of adults and children with PAH have substantially improved. In view of (1) the lack of PAH medications approved for children, (2) the solid rationale for combination pharmacotherapy in pediatric PH, and (3) the lack of severe adverse effects, the available advanced pharmacological treatment options should not be withheld, especially not for young PH patients. Such an off-label use and the unbureaucratic reimbursement of PAH medications by healthcare insurances are urgently needed. The decision on specific treatment options for PH with the possibility of combinations of PAH medications from all classes of substances, including off-label medications and preparations, should be the responsibility of pediatric cardiologists. A prerequisite is that the pediatric cardiologist must have sufficient experience with the treatment of PH in children, particularly experience with vasoactive medications. Initiation of PAH-targeted pharmacotherapy should be followed by continuous outpatient monitoring and care of these patients. The lack of approval of advanced (modern) medications for PAH and the paucity of evidence-based implications on their use in children should not prevent expert physicians from offering these pharmacotherapies to young patients with a fatal disease.
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- Pharmacotherapy
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