First-in-child use of the oral soluble guanylate cyclase stimulator riociguat in pulmonary arterial hypertension

Pulmonary Circulation
Till SpreemannGeorg Hansmann

Abstract

Riociguat has been approved for use in adults with pulmonary arterial hypertension (PAH) or chronic thromboembolic pulmonary hypertension. No clinical data on its therapeutic use in children with PAH are currently available. We report the case of a now four-year-old boy who initially presented at the age of 10 months with suprasystemic pulmonary hypertension (PH) and right ventricular (RV) failure, vomiting, peripheral cyanosis, and failure to thrive. Cardiac catheterization revealed severe PAH. At radiologic suspicion of interstitial lung disease, repeated CT scan and an open lung biopsy were performed but could not clarify the entity of PAH. Given the demonstrated vasoreactivity, the boy was started on the calcium channel blocker amlodipine, in combination with the endothelin-1 receptor antagonist bosentan. Two years later, based on persistently systemic PAH with lost vasoreactivity, PAH therapy was changed to bosentan and phosphodiesterase-5 inhibitor sildenafil. No significant improvement on the aforementioned therapy was seen, so that the patient was referred to our institution. Invasive hemodynamic evaluation showed suprasystemic PAH and marked acute vasoreactivity (PAP 127/103/83 mmHg, PVRi 23.48 WU·m2 and PVR/SVR ratio ...Continue Reading

References

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Citations

May 14, 2020·Expert Opinion on Pharmacotherapy·F Rana Olguntürk
Jan 8, 2019·Expert Review of Respiratory Medicine·Julie WackerMaurice Beghetti
Jun 2, 2018·Basic & Clinical Pharmacology & Toxicology·Jonas PedersenDaniela Grimm
Jan 22, 2020·Paediatric Drugs·Catherine M AvitabileDavid Dunbar Ivy
Jan 31, 2019·Expert Review of Cardiovascular Therapy·Nesrine FarhatMaurice Beghetti
Oct 2, 2020·Pediatric Pulmonology·Charissa W Kam, Fadel E Ruiz
Nov 3, 2020·Current Opinion in Cardiology·Dunbar Ivy, Benjamin S Frank
Mar 7, 2021·Pediatric Research·Catheline HocqUNKNOWN from the European Society for Pediatric Research
Aug 17, 2021·Journal of Pediatric Intensive Care·Christoph Armin NeuhäuserDietmar Schranz

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Methods Mentioned

BETA
pharmacotherapy
lavage
biopsy
sedation

Clinical Trials Mentioned

NCT02007629

Software Mentioned

RESPITE

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