Endothelin receptor antagonists for pulmonary hypertension in adult patients with sickle cell disease - PubMed (original) (raw)
Endothelin receptor antagonists for pulmonary hypertension in adult patients with sickle cell disease
Caterina P Minniti et al. Br J Haematol. 2009 Dec.
Abstract
Pulmonary Hypertension is a serious complication of sickle cell disease (SCD), with high morbidity and mortality. Endothelin (ET)-1, a potent vasoconstrictor elevated in SCD, acts through the ET receptors (ETR), ETR-A and ETR-B. Bosentan and ambrisentan are ETR blockers used in primary pulmonary hypertension. We report on the use of ETR blocking agents in a cohort of 14 high-risk SCD adult patients with pulmonary hypertension. Patients underwent right heart catheterization, 6-min walk test, echocardiogram, physical examination and blood work-up before starting ETR blockers. Eight patients received ETR blockers as initial therapy; six patients were already taking sildenafil. Over more than 6 months of therapy, sequential measurements of 6-min walk distance increased significantly (baseline 357 +/- 22 to 398 +/- 18 m at 5-6 months, P < 0.05). Downward trends were observed for amino-terminal brain natriuretic peptide and tricuspid regurgitant velocity. Pulmonary artery mean pressures decreased in three patients that had repeat right heart catheterization (44-38 mmHg). Adverse events were: increased serum alanine aminotransferase (2), peripheral oedema (4), rash (1), headache (3), decreased haemoglobin (2). Therapy was stopped in two patients who were switched then to the other ETR blocker agent. These data suggest preliminary evidence for the benefit of bosentan and ambrisentan in pulmonary hypertension in SCD.
Figures
Figure 1
Changes in 6-min walk distance (A), NT-proBNP a serum levels (B) and tricuspid regurgitant velocity as measured by echocardiogram (C) during the time of observation on ETR blocking therapy (6–14 months, mean 8.5 months). Sequential measurements of 6-min walk distance increased significantly (mean ± standard error baseline, 357 ± 22 m; 1–2 months, 367 ± 17 m; 3–4 months, 387 ± 16 m; 5–6 months, 398 ± 18 m; n = 12, P < 0.05, ANOVA with repeated measures).
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