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- Title
Methylphenidate in children with monogenic obesity due to LEPR or MC4R deficiency improves feeling of satiety and reduces BMI‐SDS—A case series.
- Authors
Brandt, Stephanie; Schnurbein, Julia; Lennerz, Belinda; Kohlsdorf, Katja; Vollbach, Heike; Denzer, Christian; Bode, Harald; Hebebrand, Johannes; Wabitsch, Martin
- Abstract
Summary: 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 BMIT0−T1x¯: −0.7 ± 0.9 kg/m2), BMI standard deviation score (SDS) (Δ BMI‐SDST0−T1x¯: −0.32 ± 0.20), and %BMIP95 (Δ %BMIP95T0−T1x¯: −6.6 ± 7.8%) decreased. BMI‐SDS velocity decreased from +0.17 ± 0.22 to −0.30 ± 0.20. Appetite and CEBQ subscale scores for "food responsiveness" and "enjoyment of food" decreased. We observed adverse effects with increase in self‐reported frequency of disordered sleep, nervousness, hyperactivity, and tics. 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.
- Subjects
GENETICS of childhood obesity; ANXIETY; APPETITE; CELL receptors; FOOD habits; METHYLPHENIDATE; GENETIC mutation; CHILDHOOD obesity; PROTEIN precursors; QUESTIONNAIRES; SELF-evaluation; SLEEP disorders; TIC disorders; COMORBIDITY; LEPTIN; BODY mass index; VISUAL analog scale; TREATMENT effectiveness; DESCRIPTIVE statistics; DISEASE risk factors
- Publication
Pediatric Obesity, 2020, Vol 15, Issue 1, pN.PAG
- ISSN
2047-6302
- Publication type
Article
- DOI
10.1111/ijpo.12577