The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
Paediatric patients with GHD who are on treatment with lonapegsomatropin
Patients being clinically managed in Europe or the USA
Appropriate written informed consent/assent as applicable for the age of the patient
Patients willing to comply with follow-up requirements of the study
Patients participating in any interventional clinical trial for short stature
Patients being treated with a GH or IGF-1 therapy, other than lonapegsomatropin, at enrollment
Patients for whom treatment with lonapegsomatropin is contraindicated
Patients with closed epiphyses
Patients from centres in Europe and the USA will be eligible for enrollment into the study. Patients may be enrolled in the study if they are on treatment with lonapegsomatropin. The decision to treat the patient with lonapegsomatropin will be made prior to and independently of the decision to invite the patient to enroll into the study. Both patients naïve to GH treatment and patients previously treated with GH therapy will be eligible for enrollment.
Cohort
Prospective
No intervention
DrugNo intervention
Occurrence of neoplasms (benign, malignant and unspecified)
Occurrence of type 2 diabetes mellitus
Occurrence of renal, hepatic, immunologic and neurologic adverse events
Occurrence of medication errors in patients treated with lonapegsomatropin
Frequency and type of medication errors as reported by treating physician
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
IGF-1 level (ng/mL)
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
IGF-1 Standard Deviation Score (SDS)
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