Characterization of Cardiac Function by Echocardiographic Global Longitudinal Strain in a Cohort of Children with Neurofibromatosis Type 1 Treated with Selumetinib.

IF 3.4 3区医学 Q1 PEDIATRICS

Pediatric Drugs Pub Date : 2023-03-01 DOI:10.1007/s40272-022-00551-w

Thomas Caiffa, Antimo Tessitore, Andrea Magnolato, Matilde Petz, Marco Bobbo, Daniela Chicco, Biancamaria D'Agata Mottolese, Aldostefano Porcari, Egidio Barbi, Gianfranco Sinagra, Irene Bruno

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引用次数: 0

Abstract

Background: Plexiform neurofibromas are benign neoplasms that develop in 20-50% children with neurofibromatosis type 1 (NF1). Selumetinib was approved as treatment for symptomatic and inoperable plexiform neurofibromas. Subclinical left ventricular ejection fraction reduction is a less common effect of selumetinib.

Objective: We aimed to investigate the contractile function of the heart in a cohort of children with NF1 treated with selumetinib.

Methods: We designed a cross-sectional study including 17 patients with NF1 who received selumetinib. Echocardiographic parameters were compared with a cohort of 17 healthy children matched by sex and age and another group of 17 children with untreated NF1.

Results: Compared with healthy controls, patients with NF1 treated with selumetinib had lower mean values of global longitudinal strain (- 22.9 ± 2% vs -25.5 ± 2%; p = 0.001), fractional shortening (36 ± 4% vs 43 ± 8%; p = 0.02) and tricuspid annular plane systolic excursion (19 ± 3 mm vs 23 ± 2 mm; p = 0.001); no difference was found in left ventricular ejection fraction (63 ± 4% vs 65 ± 3%; p = 0.2 respectively). Median treatment time with selumetinib at the time of the echocardiographic evaluation was 22 ± 16 months.

Conclusions: Patients with NF1 treated with selumetinib may experience subtle changes in systolic function identified by global longitudinal strain and not revealed by left ventricular ejection fraction. Global longitudinal strain might be useful to monitor cardiac function in this cohort of patients for the duration of therapy.

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用超声心动图整体纵向应变表征接受塞鲁美替尼治疗的1型神经纤维瘤病儿童心功能。

背景:丛状神经纤维瘤是一种良性肿瘤，发生在20-50%的1型神经纤维瘤病(NF1)患儿中。塞鲁美替尼被批准用于治疗症状性和不能手术的丛状神经纤维瘤。亚临床左室射血分数降低是selumetinib不太常见的效果。目的:我们旨在研究一组接受selumetinib治疗的NF1患儿的心脏收缩功能。方法:我们设计了一项横断面研究，包括17例接受塞鲁美替尼治疗的NF1患者。将17名按性别和年龄匹配的健康儿童与另一组17名未经治疗的NF1儿童的超声心动图参数进行比较。结果:与健康对照组相比，接受塞鲁美替尼治疗的NF1患者整体纵向应变平均值较低(- 22.9±2% vs -25.5±2%;P = 0.001)，部分起酥油(36±4% vs 43±8%;P = 0.02)和三尖瓣环面收缩偏移(19±3 mm vs 23±2 mm;P = 0.001);左心室射血分数无差异(63±4% vs 65±3%;P = 0.2)。超声心动图评估时，塞鲁美替尼的中位治疗时间为22±16个月。结论:接受selumetinib治疗的NF1患者可能会经历轻微的收缩功能变化，这是由整体纵向应变确定的，而不是由左室射血分数显示的。整体纵向应变可能是有用的监测心功能在这一队列患者的治疗期间。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Pediatric Drugs PEDIATRICS-PHARMACOLOGY & PHARMACY

CiteScore

7.20

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Pediatric Drugs promotes the optimization and advancement of all aspects of pharmacotherapy for healthcare professionals interested in pediatric drug therapy (including vaccines). The program of review and original research articles provides healthcare decision makers with clinically applicable knowledge on issues relevant to drug therapy in all areas of neonatology and the care of children and adolescents. The Journal includes: -overviews of contentious or emerging issues. -comprehensive narrative reviews of topics relating to the effective and safe management of drug therapy through all stages of pediatric development. -practical reviews covering optimum drug management of specific clinical situations. -systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. -Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in the pediatric population. -original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pediatric Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.