Shorofsky MJ, Morgan GJ, Mejia E, Rodriguez SA, Greene M, Sheaks P, Ivy D, Zablah JE.Pediatr Cardiol. 2023 Jun;44(5):1125-1134. doi: 10.1007/s00246-023-03102-3. Epub 2023 Feb 1.PMID: 36723625
Take Home Points:
- Treatment of pulmonary vein stenosis in pediatric patients with congenital heart disease is rapidly evolving with improving outcomes.
- Sirolimus therapy may be a useful adjuvant therapy in this complex group of patients as it seems to decrease disease progression and need for reintervention.
Commentary from Dr. Konstantin Averin (Cohen Children’s Heart Center), catheterization section editor of Pediatric Cardiology Journal Watch:
Pulmonary vein stenosis (PVS) in pediatric patients with congenital heart disease is a complex disease that carries a high degree of morbidity and mortality. There have been significant improvements outcomes over the last few years utilizing a multi-modality treatment approach – including surgery, percutaneous intervention, and medical therapy. There remains significant variability between centers and treatment algorithms. The authors report a single center retrospective experience of using systemic sirolimus therapy (SST) in conjunction with pulmonary hypertension mediations and imaging with optical coherence tomography.
From October 2020 to December 2021, 10 patients were started and followed on SST – median age at SST initiation 25 months, median weight 10.6 kg (IQR 5.4-21.5 kg). The authors describe both their PVS medical management protocol and their SST protocol (see below) including how to adjust sirolimus dosing while on bosentan therapy for pulmonary hypertension. There protocols should be of particular interest to any practitioner considering treatment of this complex group of patients. Overall, when comparing pre- and post- SST, patients required less frequent catheterizations (3 months which were also shorter in duration and the severity of disease recurrence was diminished as defined by the degree of vein re-stenosis. There were no significant serious adverse effects from SST.This study clearly has limitations, including its retrospective nature, the small cohort size, and the inherent heterogeneity in this patient population.
The authors conclude that addition of SST helps decrease disease progression with decreased frequency of interventions. Despite the studies limitations, the conclusions are broadly consistent with the existing literature, suggesting that SST can be a critical adjunct to percutaneous intervention in improving outcomes in patients with moderate to severe PVS. This study also highlights the need for a multidisciplinary, protocol driven approach to managing pediatric PVS and underscores the necessity of careful consideration of medication interactions, particularly in patients concurrently treated for pulmonary hypertension.
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