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J Pediatr. 2018 Jul;198:29-35.e5. doi: 10.1016/j.jpeds.2018.01.029. Epub 2018 Mar 23.

Adjunct Targeted Biologic Inhibition Agents to Treat Aggressive Multivessel Intraluminal Pediatric Pulmonary Vein Stenosis.

Author information

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.

KEYWORDS:

congenital heart disease; drug therapy; outcome; treatment

PMID:
29576325
DOI:
10.1016/j.jpeds.2018.01.029
[Indexed for MEDLINE]

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