Efficacy and safety of oral sildenafil in children with Down syndrome and pulmonary hypertension.

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Version: Final published version
License: CC BY 4.0
Serval ID
serval:BIB_A5F1C22FB19B
Type
Article: article from journal or magazin.
Publication sub-type
Editorial
Collection
Publications
Institution
Title
Efficacy and safety of oral sildenafil in children with Down syndrome and pulmonary hypertension.
Journal
BMC cardiovascular disorders
Author(s)
Beghetti M., Rudzinski A., Zhang M.
ISSN
1471-2261 (Electronic)
ISSN-L
1471-2261
Publication state
Published
Issued date
04/07/2017
Peer-reviewed
Oui
Volume
17
Number
1
Pages
177
Language
english
Notes
Publication types: Journal Article ; Randomized Controlled Trial ; Research Support, Non-U.S. Gov't
Publication Status: epublish
Abstract
Despite the increased risk for pulmonary hypertension in children with Down syndrome, the response to treatment with targeted therapies for pulmonary hypertension in these patients is not well characterized. The Sildenafil in Treatment-naive children, Aged 1-17 years, with pulmonary arterial hypertension (STARTS-1) trial was a dose-ranging study of the short-term efficacy and safety of oral sildenafil in children with pulmonary arterial hypertension. We assessed the safety and efficacy of oral sildenafil in children with Down syndrome and pulmonary arterial hypertension.
This was a post-hoc analysis of children with Down syndrome and pulmonary arterial hypertension enrolled in the STARTS-1 trial. Mean pulmonary arterial pressure (mPAP), pulmonary vascular resistance index (PVRI), and cardiac index (CI) were assessed at baseline and following 16 weeks of treatment with sildenafil.
Of 234 patients randomized and treated in the STARTS-1 trial, 48 (20.5%) had Down syndrome. Although sildenafil produced dose-related reductions in PVRI and mPAP, compared with placebo, in non-Down syndrome patients and children developmentally able to exercise, this was not satisfactorily marked in patients with Down syndrome. The dose-related reductions in PVRI, compared with placebo, occurred in all subgroups, with the exception of the Down syndrome subgroup. Sildenafil appeared to be well tolerated in the Down syndrome subpopulation and the most frequently reported AEs were similar to those reported for the entire STARTS-1 population.
Sildenafil treatment for 16 weeks had no effect on PVRI or mPAP in children with Down syndrome and pulmonary arterial hypertension. The results suggest that children with Down syndrome may be less responsive to sildenafil for pulmonary arterial hypertension, but the incomplete work-up for the etiology of pulmonary arterial hypertension may have introduced a potential bias.
Study received, September 8, 2005 (retrospectively registered); Study start, August 2003; ClinicalTrials.gov identifier, NCT00159913 .
Keywords
Administration, Oral, Adolescent, Antihypertensive Agents/administration & dosage, Antihypertensive Agents/adverse effects, Arterial Pressure/drug effects, Child, Child, Preschool, China, Down Syndrome/complications, Down Syndrome/diagnosis, Female, Humans, Hypertension, Pulmonary/diagnosis, Hypertension, Pulmonary/drug therapy, Hypertension, Pulmonary/etiology, Hypertension, Pulmonary/physiopathology, Infant, Male, Pulmonary Artery/drug effects, Pulmonary Artery/physiopathology, Sildenafil Citrate/administration & dosage, Sildenafil Citrate/adverse effects, Time Factors, Treatment Outcome, Vascular Resistance/drug effects, Vasodilator Agents/administration & dosage, Vasodilator Agents/adverse effects, Children, Down syndrome, Pulmonary hypertension, Sildenafil
Pubmed
Web of science
Open Access
Yes
Create date
10/01/2019 17:26
Last modification date
30/04/2021 6:13
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