From the FDA Drug Label
Safety and effectiveness of SKYTROFA have been established in pediatric patients 1 year and older and who weigh at least 11. 5 kg. The safety and effectiveness of SKYTROFA in children less than 1 year of age have not been established Use of somatropin in pediatric patients with Prader-Willi syndrome has been associated with reports of sudden death. SKYTROFA is not indicated for the treatment of pediatric patients with growth failure due to genetically confirmed Prader-Willi syndrome
Safety Concerns with Skytrofa for Children:
- The safety and effectiveness of Skytrofa have been established in pediatric patients 1 year and older and who weigh at least 11.5 kg.
- There are no established safety and effectiveness of Skytrofa in children less than 1 year of age.
- Sudden death has been reported in pediatric patients with Prader-Willi syndrome using somatropin, and Skytrofa is not indicated for this patient population. 1
From the Research
Yes, there are safety concerns with Skytrofa (lonapegsomatropin-tcgd) for children, and careful monitoring and dose adjustment based on IGF-1 levels are necessary to maintain safety and efficacy. Common side effects include injection site reactions (pain, redness, itching), headache, and fever. More serious concerns include potential for increased pressure in the brain (presenting as headache, vision problems, or nausea), risk of slipped capital femoral epiphysis (hip pain or limping), progression of scoliosis during rapid growth, allergic reactions, and increased risk of developing type 2 diabetes. Children with certain conditions should not use Skytrofa, including those with active cancer, critical illness, severe breathing problems, or children who have had certain types of brain surgery or injury. Regular monitoring by a healthcare provider is essential during treatment, including periodic blood tests to check hormone levels and glucose. Parents should immediately report any unusual symptoms such as severe headache, vision changes, persistent hip or knee pain, or signs of allergic reaction. These safety concerns exist because Skytrofa affects growth and metabolism throughout the body, potentially impacting multiple organ systems as it treats growth hormone deficiency, as seen in studies such as 2.
Key considerations for the use of Skytrofa in children include:
- The need for individualized dosing, as the manufacturer's recommended starting dose of 0.24 mg/kg/week may require adjustment based on IGF-1 levels 2
- The importance of monitoring for adverse effects, such as injection site reactions and changes in IGF-1 levels 3, 4
- The potential for Skytrofa to be a favorable option for reducing injection burden in children with growth hormone deficiency, while also maintaining safety and efficacy 2
Overall, while Skytrofa may be an effective treatment for growth hormone deficiency in children, it is essential to carefully weigh the potential benefits and risks, and to closely monitor patients for any adverse effects, as supported by the most recent and highest quality study 2.