Methylphenidate in children with monogenic obesity due to LEPR or MC4R deficiency improves feeling of satiety and reduces BMI-SDS-A case series.
LEPR mutation
MC4R mutation
central nervous system
methylphenidate
monogenic obesity
Journal
Pediatric obesity
ISSN: 2047-6310
Titre abrégé: Pediatr Obes
Pays: England
ID NLM: 101572033
Informations de publication
Date de publication:
01 2020
01 2020
Historique:
received:
11
03
2019
revised:
31
07
2019
accepted:
24
08
2019
pubmed:
2
11
2019
medline:
19
8
2020
entrez:
1
11
2019
Statut:
ppublish
Résumé
The clinical phenotype of patients with monogenic obesity due to mutations in the leptin receptor (LEPR) or melanocortin 4 receptor (MC4R) gene is characterized by impaired satiety and hyperphagia, leading to extreme, sometimes life-threatening weight gain. In a case series, we analysed the effect of an off-label methylphenidate (MPH) use for 1 year as an individual treatment approach on eating behaviour (Child Eating Behaviour Questionnaire [CEBQ]), appetite (visual analogue scales) and body mass index (BMI) trajectories in five patients with severe obesity due to mutations in the LEPR (n = 3) or MC4R (n = 2) gene. After 1 year use of MPH (20 mg/day divided in two to three doses), BMI (Δ BMI The observed decrease in BMI trajectories with MPH use for one year is clinically meaningful in this group of patients, since the natural course would have been associated with a pronounced increase in BMI, leading to comorbidities and complications over time.
Sections du résumé
BACKGROUND
The clinical phenotype of patients with monogenic obesity due to mutations in the leptin receptor (LEPR) or melanocortin 4 receptor (MC4R) gene is characterized by impaired satiety and hyperphagia, leading to extreme, sometimes life-threatening weight gain.
SUBJECTS/METHODS
In a case series, we analysed the effect of an off-label methylphenidate (MPH) use for 1 year as an individual treatment approach on eating behaviour (Child Eating Behaviour Questionnaire [CEBQ]), appetite (visual analogue scales) and body mass index (BMI) trajectories in five patients with severe obesity due to mutations in the LEPR (n = 3) or MC4R (n = 2) gene.
RESULTS
After 1 year use of MPH (20 mg/day divided in two to three doses), BMI (Δ BMI
CONCLUSIONS
The observed decrease in BMI trajectories with MPH use for one year is clinically meaningful in this group of patients, since the natural course would have been associated with a pronounced increase in BMI, leading to comorbidities and complications over time.
Substances chimiques
LEPR protein, human
0
MC4R protein, human
0
Receptor, Melanocortin, Type 4
0
Receptors, Leptin
0
Methylphenidate
207ZZ9QZ49
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e12577Informations de copyright
© 2019 The Authors. Pediatric Obesity published by John Wiley & Sons Ltd on behalf of World Obesity Federation.
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