Overview

Efficacy summary

Survival

In a cohort study of 200 patients (147 with Marfan syndrome), the estimated survival at 1, 2 and 3 years after PEARS was 98.3%, 97.6% and 97.6% respectively (Van Hoof 2021).

In a cohort study of 117 patients who were followed up for at least a year (range 2 to 12 years), 94% (110/117) of patients were alive with PEARS only, 2% (2/117) of patients were alive after revision surgery for progression of aortic regurgitation (at 93 and 105 months) and 2% (2/117) of patients had died (1 at day 5, described further in the safety section, and the other at 4.5 years after the procedure, which was related to an arrhythmia). In the 2 patients who had revision surgery, the right and non-coronary sinuses were not completely covered by the mesh because of deviations from the protocol for intraoperative reasons (Pepper 2020).

A total of 3 deaths were reported in a case series of 317 patients who had a personalised external aortic root support procedure (57% of whom had Marfan syndrome). In addition to the 2 deaths described above and in the safety section, 1 patient died 6.5 months after the procedure from chronic heart failure (Nemec 2020).

In a cohort study of the first 30 patients to have the procedure, cumulative survival at 7 years was 100% (Treasure 2014).

Ascending aortic dissection

In the cohort study of 200 patients, no ascending aortic dissections were observed at median follow-up of 21.2 months (Van Hoof 2021).

Aorta diameter

In a case series of 24 patients, there was no increase in the aortic root and ascending aorta diameters at mean follow up of 6.3 years. In the same period, the mean descending aorta diameter increased from 22.6 mm to 23.9 mm (change 1.25 mm, 95% CI 0.65 to 1.85 mm; p<0.001) (Izgi 2018).

Aortic regurgitation

In the case series of 24 patients, there was no increase in the percentage of patients with mild aortic regurgitation (33% [8/24] at baseline), and no patient had an increase in the severity of aortic regurgitation at follow up (Izgi 2018).

Histological appearance

In a case report of a patient who died 4.5 years after having an external aortic root support procedure, the aortic arch and descending aorta appeared normal. The external aortic mesh was fully incorporated in the adventitia and could not be separated from it. The supported aortic root had the histological appearance of a normal aorta. The histological appearance suggested the possibility that the incorporated support of the aortic root allowed recovery of the microstructure of the media. The cause of death was dilated cardiomyopathy, presumed to be related to Marfan syndrome (Pepper 2015).