Macitentan in the Young-Mid-term Outcomes of Patients with Pulmonary Hypertensive Vascular Disease  treated in a Pediatric Tertiary Care Center

CONCLUSION: Data presented herein account for the largest cohort of severely affected pediatric patients receiving macitentan. Overall, macitentan was safe and associated with significant beneficial effects and sustained positive signals after 1 year, albeit in the long term disease progression remains a major concern. Our data suggest limited efficacy in CHD-related PH, whereas favorable outcomes were mainly driven by improvements in patients with PH not related to CHD. Larger studies are needed to verify these preliminary results and to prove efficacy of this drug in different pediatric PH entities.PMID:37269500 | DOI:10.1007/s40272-023-00573-y

Source: Paediatric Drugs - June 3, 2023 Category: Pediatrics Authors: Sulaima Albinni Julian Heno Imre Pavo Erwin Kitzmueller Manfred Marx Ina Michel-Behnke Source Type: research