Survival in incident and prevalent cohorts of patients with pulmonary arterial hypertension

Abstract

Pulmonary arterial hypertension (PAH) is a progressive, fatal disease.We studied 674 consecutive adult patients who were prospectively enrolled in the French PAH registry (121 incident and 553 prevalent cases). Two survival analyses were performed. First, the cohort of 674 patients was followed for 3 yrs after study entry and survival rates described. Then, we focused on the subset with incident idiopathic, familial and anorexigen-associated PAH (n556) combined with prevalent patients who were diagnosed ,3 yrs prior to study entry (n5134).In the cohort of 674 patients, 1-, 2-, and 3-yr survival rates were 87% (95% CI 84-90), 76% (95% CI 73-80), and 67% (95% CI 63-71), respectively. In prevalent idiopathic, familial and anorexigenassociated PAH, 1-, 2-, and 3-yr survival rates were higher than in incident patients (p50.037). In the combined cohort of patients with idiopathic, familial and anorexigen-associated PAH, multivariable analysis showed that survival could be estimated by means of a novel riskprediction equation using patient sex, 6-min walk distance, and cardiac output at diagnosis.This study highlights survivor bias in prevalent cohorts of PAH patients. Survival of idiopathic, familial and anorexigen-associated PAH can be characterised by means of a novel risk-prediction equation using patients' characteristics at diagnosis.

From Short‐Term Benefits to Long‐Term Outcomes: The Evolution of Clinical Trials in Pulmonary Arterial Hypertension

“…Current European registries have estimated a prevalence of 5-8 cases per million for idiopathic PAH and 15-26 cases per million for the larger and more heterogeneous category of PAH that also includes, for example, PAH associated with connective tissue disease, toxins, and congenital systemic-to-pulmonary shunts. 4,19 As a result, largescale studies involving thousands of patients that have been conducted in other disease states are not realistically feasible in PAH. Furthermore, the increasing number of PAH-specific treatments and the number of competing clinical studies have further limited the number of appropriate PAH subjects for clinical trials, making it extremely difficult to recruit subjects, particularly treatment-naive subjects, in many areas of the world.…”

“…For example, patients with PAH associated with systemic sclerosis have a particularly poor prognosis, although recent studies suggest that outcome has improved, with a contemporary 1-year survival rate of 78% compared with 45% in historical reports. 4 There is also a lack of contemporary survival data for children with PAH, because interpretation of data from registries including children is complicated by a range of factors that include small patient numbers, poor characterization, and the retrospective nature of such studies. 13 Before targeted therapy became available, most children died within 1 year of idiopathic PAH diagnosis; however, more recent data report that, with treatment, 3-year survival rates for pediatric idiopathic PAH are greater than 80%.…”

“…2 Advances in management and the use of these therapies have resulted in significant improvements and better quality of life (QoL) for patients with PAH, although none of these therapies is considered curative. [4][5][6] Current approved therapies for PAH target 1 of 3 main pathways (prostacyclin, endothelin, or nitric oxide) thought to be involved in the vasoconstriction, vascular endothelial cell proliferation, smooth muscle cell proliferation, and endothelial dysfunction characteristic of the disease. 2,7 Phase III trials of these therapies have only shown a modest improvement in hemodynamic characteristics, when measured, but have still reported significant increases in exercise capacity, lessening of symptoms, and better QoL compared with placebo.…”

Gestational Pulmonary Arterial Hypertension

“…Currently available medical therapies have improved clinical status, morbidity, and mortality in patients with IPAH, but mortality is still extremely high. [4][5][6] In 1991, the National Institutes of Health Registry reported a survival of 47% during a 3-year period. 3 More recently, the Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) 5 and French registries 6 have reported a survival rate of 67%.…”

“…However, mortality is still as high as 30% over a 3-year period. [4][5][6] Outcomes of patients who develop PAH during pregnancy are particularly poor, with an estimated maternal mortality of 30%-56%. 7 To date, there have been no reported cases of spontaneous resolution of PAH in adults, nor have there been reports of PAH isolated to the gestational period.…”