Ryan Callahan1, Mark W Kieran2, Christopher W Baird3, Steven D Colan4, Kimberlee Gauvreau4, Christina M Ireland4, Audrey C Marshall5, Laureen M Sena6, Sara O Vargas7, Kathy J Jenkins4. 1. Department of Cardiology, Boston Children's Hospital and Harvard Medical School, Boston, MA. Electronic address: Ryan.Callahan@cardio.chboston.org. 2. Division of Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute, Boston Children's Hospital and Harvard Medical School, Boston, MA; Department of Hematology/Oncology, Boston Children's Hospital and Harvard Medical School, Boston, MA. 3. Department of Cardiac Surgery, Boston Children's Hospital and Harvard Medical School, Boston, MA. 4. Department of Cardiology, Boston Children's Hospital and Harvard Medical School, Boston, MA. 5. Department of Cardiology, Floating Hospital for Children at Tufts Medical Center, Boston, MA. 6. Department of Radiology, UMass Memorial Medical Center, Boston, MA. 7. Department of Pathology, Boston Children's Hospital and Harvard Medical School, Boston, MA.
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.
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.
Authors: David B Frank; Philip T Levy; Corey A Stiver; Brian A Boe; Christopher W Baird; Ryan M Callahan; Charles V Smith; Rachel D Vanderlaan; Carl H Backes Journal: J Perinatol Date: 2021-03-05 Impact factor: 2.521
Authors: Eli Zettler; Brian K Rivera; Corey Stiver; Brian Boe; Clifford Cua; Molly K Ball; Charles V Smith; Jonathan L Slaughter; Bernadette Chen; Ryan Callahan; Carl H Backes Journal: J Perinatol Date: 2020-09-25 Impact factor: 2.521