Constantin Goldann, Anna Deleu, Marie Sophie Schüngel, Julius H Loeser, Alena Akinina, Moritz Guntau, Moritz Wildgruber, Vanessa F Schmidt, Walter A Wohlgemuth, Richard Brill
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引用次数: 0
Abstract
Background: This prospective study evaluates the effectiveness and safety of Bleomycin electrosclerotherapy (BEST) for treating slow-flow vascular malformations in a pediatric cohort. While retrospective studies have reported its efficacy, prospective data in pediatric populations are limited.
Methods: 30 pediatric patients (mean age: 9.5 years) with venous, veno-lymphatic, or capillary-veno-lymphatic malformations were enrolled in the study between 2020 and 2021 and received at least one BEST treatment. Follow-up continued through 2024 (mean: 25 months). MRI volumetry and clinical evaluation were performed at each follow-up.
Results: A total of 58 sessions (mean: 1.81 per patient) led to a median lesion volume reduction from 232 cm³ (range: 1.69 cm³-9814.12 cm³) pre-treatment to 41 cm³ (range: 0.57 cm3-1789.63 cm3) after the final session, corresponding to mean relative volume reduction of 59.4%. Symptoms resolved in 2 patients, improved in 18, and remained unchanged in 4. Minor side effects included skin hyperpigmentation (n = 6), inflammation (n = 3), temporary motion restriction (n = 1), and lymphorrhea (n = 1). No serious complications were observed.
Conclusion: BEST is a safe, effective treatment for pediatric slow-flow malformations, achieving lesion reduction and symptom relief. Future studies are warranted to optimize treatment protocols and establish long-term benefits.
Impact statement: Bleomycin Electrosclerotherapy (BEST) is a safe and effective treatment for slow-flow vascular malformations in pediatric patients, achieving lesion volume reduction and symptom improvement. The study assesses BEST exclusively in a pediatric cohort using standardized MRI-based volumetry alongside clinical outcomes. BEST can be effective not only in refractory cases but also as a first-line or early-line treatment option, broadening its potential indications. By indicating a cumulative therapeutic benefit of repeated BEST sessions, the study encourages further investigations into tailored, session-based treatment strategies for large or progressive malformations.
期刊介绍：
Pediatric Research publishes original papers, invited reviews, and commentaries on the etiologies of children''s diseases and
disorders of development, extending from molecular biology to epidemiology. Use of model organisms and in vitro techniques
relevant to developmental biology and medicine are acceptable, as are translational human studies
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