Ryan Callahan, Mark W. Kieran, Christopher W. Baird, Steven D. Colan, Kimberlee Gauvreau, Chistina M. Ireland, Audrey Marshall, Laureen M. Sena, Sara O. Vargas, Kathy J. Jenkins
Boston Children’s Hospital and Harvard Medical School. Floating Hospital for Children at Tufts Medical Center. UMass Memorial Medical Center.
United States
Journal of Pediatrics
J Pediatr 2018; 198: 29-35
DOI: 10.1016/j.jpeds.2018.01.029
Abstract
Objective: To evaluate the use of imatinib mesylate with or without bevacizumab targeting neoproliferative myofibroblast-like cells with tyrosine kinase receptor expression, as adjuncts to modern interventional therapies for the treatment of multivessel intraluminal pulmonary vein stenosis (PVS). We describe the 48- and 72-week outcomes among patients receiving imatinib mesylate with or without bevacizumab for multivessel intraluminal PVS.
Study design: This single-arm, prospective, open-label US Food and Drug Administration approved trial enrolled patients with ≥2 affected pulmonary veins after surgical or catheter-based relief of obstruction between March 2009 and December 2014. Drug therapy was discontinued at 48 weeks, or after 24 weeks of stabilization, whichever occurred later.
Results: Among 48 enrolled patients, 5 had isolated PVS, 26 congenital heart disease, 5 lung disease, and 12 both. After the 72-week follow-up, 16 patients had stabilized, 27 had recurred locally without stabilization, and 5 had progressed. Stabilization was associated with the absence of lung disease (P = .03), a higher percentage of eligible drug doses received (P = .03), and was not associated with age, diagnosis, disease laterality, or number of veins involved. Survival to 72 weeks was 77% (37 of 48). Adverse events were common (n = 1489 total), but only 16 were definitely related to drug treatment, none of which were serious.
Conclusion: Survival to 72 weeks was 77% in a referral population with multivessel intraluminal PVS undergoing multimodal treatment, including antiproliferative tyrosine kinase blockade. Toxicity specific to tyrosine kinase blockade was minimal.
Category
Stenosis or Obstruction of Normal Pulmonary Venous Connections
Stenosis or Obstruction of Pulmonary Veins Following Surgical Repair of Anomalous Pulmonary Venous Connections
Length of Life Associated with Pulmonary Venous Obstruction
Multidisciplinary Care
Medical Therapy to Prevent or Reverse the Onset of Disease. Efficacy or Lack of Efficacy
Medical Therapy to Prevent Progression of Disease. Efficacy or Lack of Efficacy
Medical Therapy to Prevent Recurrence of Disease after an Intervention. Efficacy or Lack of Efficacy
Medical Therapy. Adverse Effects or Lack of Adverse Effects
Catheter-mediated Interventions: Efficacy or Lack of Efficacy
Surgical Interventions for Pulmonary Venous Obstruction After the Onset of Disease
Year of Publication: 2018
Age Focus: Pediatric
Article Type: Prospective Observational Cohort Studies
Article Access: Free PDF File or Full Text Article Available Through PubMed or DOI: No