Pegvisomant treatment for X-linked acrogigantism syndrome

Coxson, Edward; Iacovazzo, Donato; Bunce, Benjamin; Jose, Sian; Ellard, Sian; Sampson, Julian R.; Korbonits, M; Burren, Christine

doi:10.1530/endoabs.39.oc5.10

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“…It is known that these patients more commonly present with resistant acromegaly. Until now pegvisomant in monotherapy or in combination with first generation SRLs, rather that pasireotide-LAR, has been specifically mentioned in the context of these subgroups treatment [24][25][26][27]. In our study substantial proportion (55%) of those patients showed a positive response to pasireotide-LAR treatment.…”

Section: Discussionmentioning

confidence: 51%

Real-world experience with pasireotide-LAR in resistant acromegaly: a single center 1-year observation

et al. 2021

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Context Pasireotide-LAR, a second-generation somatostatin receptor ligand (SRL), is recommended for patients with acromegaly as second-line treatment. Its efficacy and safety were assessed in clinical trials; however, the real-world evidence is still scarce. Objective The aim of this study was to evaluate the impact of 1-year treatment with pasireotide-LAR on disease control and glucose metabolism in acromegaly patients resistant to first-generation SRLs. Design A single-center prospective study. Methods Twenty-eight patients with active acromegaly or acrogigantism on first-generation SRLs following ineffective pituitary surgery were switched to treatment with pasireotide-LAR 40 or 60 mg i.m. every 28 days. To assess the efficacy of the treatment GH and IGF-1 levels were measured every 3 months. Safety of treatment was carefully evaluated, especially its impact on glucose metabolism. Results Complete biochemical control (GH ≤ 1 ng/mL and IGF-1 ≤ 1 × ULN) was achieved in 26.9% of patients and partial + complete response (GH ≤ 2.5 ng/mL and IGF-1 ≤ 1.3 × ULN) in 50.0% of patients. Mean GH level decrease was the largest within first 6 months (P = 0.0001) and mean IGF-1 level decreased rapidly within the first 3 months (P < 0.0001) and they remained reduced during the study. Blood glucose and HbA1c levels increased significantly within 3 months (P = 0.0001) and stayed on stable level thereafter. Otherwise, the treatment was well tolerated and clinical improvement was noticed in majority of patients. Conclusions This real-life study confirmed good effectiveness of pasireotide-LAR in patients resistant to first-generation SRLs. Pasireotide-LAR was overall safe and well tolerated, however significant glucose metabolism worsening was noted.

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Section: Discussionmentioning

confidence: 51%