Characterization of Cardiac Function by Echocardiographic Global Longitudinal Strain in a Cohort of Children with Neurofibromatosis Type 1 Treated with Selumetinib.
Journal
Paediatric drugs
ISSN: 1179-2019
Titre abrégé: Paediatr Drugs
Pays: Switzerland
ID NLM: 100883685
Informations de publication
Date de publication:
Mar 2023
Mar 2023
Historique:
accepted:
30
11
2022
pubmed:
19
12
2022
medline:
18
2
2023
entrez:
18
12
2022
Statut:
ppublish
Résumé
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. We aimed to investigate the contractile function of the heart in a cohort of children with NF1 treated with selumetinib. 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. 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. 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.
Sections du résumé
BACKGROUND
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
OBJECTIVE
We aimed to investigate the contractile function of the heart in a cohort of children with NF1 treated with selumetinib.
METHODS
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
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
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.
Identifiants
pubmed: 36529809
doi: 10.1007/s40272-022-00551-w
pii: 10.1007/s40272-022-00551-w
doi:
Substances chimiques
AZD 6244
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
217-224Informations de copyright
© 2022. The Author(s), under exclusive licence to Springer Nature Switzerland AG.
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