Endothelin Receptor Antagonists are an Effective Long Term Treatment Option in Pulmonary Arterial Hypertension Associated with Congenital Heart Disease With or Without Trisomy 21

Kermeen, F.; Franks, C.; OʼBrien, Kathy; Seale, Helen; Hall, Kathleen; McNeil, Keith; Radford, Dorothy J.

doi:10.1016/j.hlc.2010.07.005

Cited by 26 publications

(25 citation statements)

References 29 publications

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“…In line with reported studies in patients with PAH-CHD [4,[21][22][23][24], our patient showed good short-and long-term responses to initial bosentan therapy, with improvements in exercise capacity and WHO FC without a negative effect on oxygen saturations.…”

supporting

confidence: 91%

“…In a recent study, 3 months of iron replacement therapy in iron-deficient cyanotic CHD patients was shown to be safe and to result in increases in haemoglobin levels, particularly in those patients with low baseline levels, associated with significant improvement in exercise tolerance and quality of life as measured on the CAMPHOR scale [20]. Furthermore, in a recent study the degree of difference between optimal and actual haemoglobin levels was found to be negatively correlated with exercise capacity, while oxygen saturations only correlated with haemoglobin in patients with optimal levels [21]. The study shows that patients with only a small difference between optimal and actual haemoglobin levels have better exercise capacity, irrespective of oxygen saturation, than those with a greater difference, supporting iron supplementation to ensure haemoglobin levels are as close to optimal as possible in patients with ES.…”

mentioning

confidence: 98%

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The management of Eisenmenger syndrome in the modern treatment era: a case report

Gatzoulis¹

2011

European Respiratory Review

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confidence: 91%

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confidence: 98%

The management of Eisenmenger syndrome in the modern treatment era: a case report

Gatzoulis¹

2011

European Respiratory Review

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“…Importantly, there have been no randomised controlled trials in Down syndrome with PAH examining response to vasodilator therapy [64,65]. The available data suggest that persons with Down syndrome are equally as responsive as PAH patients without Down syndrome to endothelin receptor antagonists such as bosentan [64][65][66][67][68], although the 6-min walk test may not be an effective end-point [69]. Despite the high incidence of CHD in individuals with trisomy 21, pulmonary thrombosis does not appear to be a cause of morbidity or mortality in Down syndrome [70][71][72][73][74].…”

Section: Respiratory Disease In Down Syndromementioning

confidence: 99%

What people with Down Syndrome can teach us about cardiopulmonary disease

Colvin

Yeager

2017

Eur Respir Rev

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Down syndrome is the most common chromosomal abnormality among live-born infants. Through full or partial trisomy of chromosome 21, Down syndrome is associated with cognitive impairment, congenital malformations ( particularly cardiovascular) and dysmorphic features. Immune disturbances in Down syndrome account for an enormous disease burden ranging from quality-of-life issues (autoimmune alopecia) to more serious health issues (autoimmune thyroiditis) and life-threatening issues (leukaemia, respiratory tract infections and pulmonary hypertension). Cardiovascular and pulmonary diseases account for ∼75% of the mortality seen in persons with Down syndrome. This review summarises the cardiovascular, respiratory and immune challenges faced by individuals with Down syndrome, and the genetic underpinnings of their pathobiology. We strongly advocate increased comparative studies of cardiopulmonary disease in persons with and without Down syndrome, as we believe these will lead to new strategies to prevent and treat diseases affecting millions of people worldwide.

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“…In an open-label, observational study, endothelin receptor antagonism with bosentan or sitaxentan improved 6-min walk distance (6MWD) and WHO functional class in adult patients with PAH-CHD with or without DS [15]. In another open-label study of 24 adult patients with DS and Eisenmenger syndrome, bosentan therapy resulted in increased 6MWD during the first 3 months of bosentan treatment but slowly returned to baseline after 26 and 52 weeks of therapy [16].…”

Section: Discussionmentioning

confidence: 99%

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

Beghetti

Rudziński

Zhang

2017

BMC Cardiovasc Disord

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BackgroundDespite 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.MethodsThis 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.ResultsOf 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.ConclusionSildenafil 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.Trial registrationStudy received, September 8, 2005 (retrospectively registered); Study start, August 2003; ClinicalTrials.gov identifier, NCT00159913.Electronic supplementary materialThe online version of this article (doi:10.1186/s12872-017-0569-3) contains supplementary material, which is available to authorized users.

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Endothelin Receptor Antagonists are an Effective Long Term Treatment Option in Pulmonary Arterial Hypertension Associated with Congenital Heart Disease With or Without Trisomy 21

Cited by 26 publications

References 29 publications

The management of Eisenmenger syndrome in the modern treatment era: a case report

The management of Eisenmenger syndrome in the modern treatment era: a case report

What people with Down Syndrome can teach us about cardiopulmonary disease

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

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