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4CPS-072 Setmelanotide in monogenic obesity: a case report
  1. M Suarez Gonzalez,
  2. J Gonzalez Chavez,
  3. P Diaz Ruiz,
  4. A Martin Lopez,
  5. J Esquivel Negrin,
  6. A Santos Fagundo,
  7. J Merino Alonso
  1. Hospital Nuestra Señora De Candelaria, Pharmacy, Santa Cruz De Tenerife, Spain


Background and Importance The melanocortin 4 receptor (MC4R), component of the leptin-melanocortin pathway, plays a part in body weight regulation (hunger, satiety and energy expenditure).

Setmelanotide is a highly potent MC4R-agonist that leads to weight loss in Monogenic Obesity (MO) individuals with complete pro-opiomelanocortin (POMC) deficiency or leptin receptor (LEPR) deficiency.

Aim and Objectives To evaluate the efficacy of setmelanotide in a 3-year-old paediatric patient with MO due to LEPR deficiency (off-label use).

Material and Methods Observational, retrospective and descriptive study of a child with MO in a third-tier hospital for 6 months (April to September 2023).

The information was obtained from the Electronic Clinical History and the Pharmacy Service Managing Software.

Results The child born at 36+2 weeks with a weight appropriate to his gestational age (2.5 kg).

He was admitted in an obesity study in May 2021. He was diagnosed with MO due to LEPR deficiency in September 2021.

The child started with setmelanotide 0.5mg in April 2023 and was increased to a current dose of 1.5mg daily subcutaneous injection.

He has lost weight from 40 to 38 kg in 6 months. He also eats less food and his craving for food has decreased. Analytical levels improved from October 22 to May 23: triglycerides: 99 to 75 mg/mL; cholesterol 217 to 139 mg/dL; LDL 144 to 72 mg/dL. The patient has decreased in adipose component and has increased in muscle mass. Progress in mobility, crawling and kneeling. Sleeps through the night with a daytime nap, not always.

There are no alternative treatments suitable for the patient‘s age.

Setmelanotide has demonstrated statistically significant weight loss with at least a 5% decrease in body weight after 6 months and decreased appetite, therefore it could reach a 10% after 1 year.

The child has skin rash and skin hyperpigmentation (activity at melanocortin 1-receptors (MC1R) as adverse effects.

Conclusion and Relevance Setmelanotide is the first European Medicines Agency approved medication for the treatment of POMC and LEPR deficiency in patients (children from 6 years old and adults) with MO.

In our case report is an off-label use and the child has been treated efficiently with setmelanotide for 6 months with a reduction in weight, hunger and analytical parameters.

We should evaluate the response after 1-year with setmelanotide to confirm that the treatment objectives are achieved (10%weight loss in 1-year).

Conflict of Interest No conflict of interest.

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