O-4
Transcatheter Interventions for Elastinopathy-Associated Peripheral Pulmonary Stenosis
Presenter
Stephan J. Wu, MD, Boston Children's Hospital, Boston, MA
Stephan J. Wu, MD1, Kimberlee Gauvreau, ScD2 and Diego Porras, M.D.1, (1)Boston Children's Hospital, Boston, MA, (2)Children's Hospital Boston, Boston, MA
Keywords: Congenital Heart Disease (CHD)
Background:
The purpose of this study was to assess the long-term outcomes of transcatheter therapy for peripheral pulmonary stenosis (PPS) in patients with elastinopathies.
Methods:
Single-center, retrospective review of patients with a diagnosis of elastinopathy who underwent transcatheter intervention for PPS between 1/1/1988 and 12/31/2019.
Results:
46 patients (80% with Williams syndrome) underwent 150 interventional catheterizations for PPS (median of 2; IQR 1-5 per patient) during a median follow up of 7.4 years (IQR 1.4-13.8 years). Median age at first catheterization was 0.6 years (IQR 0.3-1.8 years). Fourteen patients (30%) also underwent surgical arterioplasty of the central, mediastinal pulmonary arteries (PAs). Intrapulmonary lesions were treated exclusively by transcatheter interventions. There were no reinterventions at sites of prior surgical PA arterioplasty. The baseline right ventricular systolic pressure-to-aortic pressure ratio at last catheterization was lower (median 56.5%, IQR 41.9-84.3%, p<0.001) compared to that at first catheterization (median 93.9%, IQR 83.8-126.6%). Two catheterization procedures were complicated by peri-procedural arrests (1.3%), both successfully managed (one requiring ECMO cannulation). There were 6 deaths (13%) and one bilateral lung transplantation for refractory PPS in the follow-up period. Lung-transplant free survival was 85.2% at 5 years post-initial catheterization (95% CI 69.9-93.1%). At last follow-up echocardiogram, all 34 transplant-free survivors with adequate follow-up data had normal RV systolic function. Twenty-seven (79%) had <1/2 systemic right ventricular systolic pressures (RVp), 7 (21%) had >1/2 systemic RVp, and none had suprasystemic RVp.
Conclusions:
A combined strategy of surgical and transcatheter therapies for patients with elastinopathy-associated PPS is effective in relieving RV hypertension and preserving RV function in long-term follow-up in the majority of patients.