Abstract
Background Sensitivity to pegvisomant therapy is highly variable in patients with acromegaly but determinants of this variability are still unknown. Lack of exon 3 (d3-) of the growth hormone (GH) receptor (GHR) has been associated with increased biological activity of GH. Objective To assess whether the presence of d3-GHR haplotype may have a role in predicting dose regimen and response to pegvisomant in acromegaly. Design Case series. Setting Institutional referral center at a tertiary care hospital. Patients Ninenteen acromegalic patients with active disease after unsuccessful neurosurgery and somatostatin analog therapy. Measurements before and 1, 3, 6 and 12 months after treatment with pegvisomant, IGF-I; GH receptor genotype, determined from peripheral blood by polymerase chain reaction. All patients started treatment with pegvisomant at 10 mg/daily and then increased the dose, according to a fixed schedule, during a 12-month follow-up until normalization of IGF-I levels. Results d3-GHR patients required a significant lower dose of pegvisomant and shorter treatment time to normalize IGF-I. Conclusion the GHR genotype could be useful in predicting dose and individual response to pegvisomant in acromegaly.
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Bianchi, A., Mazziotti, G., Tilaro, L. et al. Growth hormone receptor polymorphism and the effects of pegvisomant in acromegaly. Pituitary 12, 196–199 (2009). https://doi.org/10.1007/s11102-008-0157-8
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DOI: https://doi.org/10.1007/s11102-008-0157-8