Long-term treatment of acromegaly with pegvisomant, a growth hormone receptor antagonist

Lely, Aart Jan van der; Hutson, R. Kent; Trainer, Peter J; Besser, G. M.; Barkan, Ariel L.; Katznelson, Laurence; Klibanski, Anne; Herman-Bonert, Vivien S.; Melmed, Shlomo; Vance, Mary Lee; Freda, Pamela U.; Stewart, Paul M.; Friend, Keith; Clemmons, David R.; Johannsson, Gudmundur; Stavrou, Stavros; Cook, David M.; Phillips, Lawrence S.; Strasburger, Christian J.; Hacker, S.; Zib, Kenneth; Davis, Robert J.; Scarlett, John A.; Thorner, Michael O.

doi:10.1016/s0140-6736(01)06844-1

Cited by 587 publications

(478 citation statements)

References 31 publications

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“…However, these events were all mild to moderate in severity, except for severe malaise in one subject. In safety issues, none of the treated patients showed an obvious increase in pituitary tumor size in accordance with the previously reported long-term clinical study [13] nor production of high titer antibody which might influence clinical outcome.…”

Section: Discussionsupporting

confidence: 89%

“…The effectiveness of pegvisomant was almost similar to that in previous overseas clinical trials [12,13], in which serum IGF-I percentage change from baseline at 12 week by pegvisomant 20 mg daily was −62.5%, and IGF-I normalization was observed in 97% (87/90 subjects) for 12 months or more. However, the efficacy in normalizing IGF-I in a global safety surveillance study, ACROSTUDY, has been reported to be as low as 63.2% after 5 years of treatment [14] possibly due to inadequate dose adjustment.…”

Section: Discussionsupporting

confidence: 82%

“…In the previous studies, however, it has been reported that serum GH was sustained during 18 months [13]. Recent experimental data showed exogenous GH augment GH secretion from somatotroph adenoma via STAT3 [15].…”

Section: Discussionmentioning

confidence: 99%

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Efficacy and safety of monotherapy by pegvisomant, a growth hormone receptor antagonist, in Japanese patients with acromegaly

et al. 2016

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Section: Discussionsupporting

confidence: 89%

Section: Discussionsupporting

confidence: 82%

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Efficacy and safety of monotherapy by pegvisomant, a growth hormone receptor antagonist, in Japanese patients with acromegaly

et al. 2016

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“…Our results are closer to the data reported in the initial clinical trials with pegvisomant monotherapy and those from series of other tertiary centers than to the data reported in the last update of the Acrostudy (20)(21)(22)31,32). One of the possible reasons for a lower control rate in the Acrostudy is the clinical inertia (22).…”

Section: Discussionsupporting

confidence: 76%

“…It can be used in monotherapy or in combination therapy with SA, in this case with greater efficacy. Normalization of IGF-I levels was achieved in more than 90% of the patients in the initial clinical trials and in 63.2% of the patients in the last update report of the Acrostudy (20)(21)(22). Therefore, it is the most effective drug in controlling IGF-I levels in acromegaly.…”

Section: Introductionmentioning

confidence: 99%

Experience with pegvisomant treatment in acromegaly in a single Brazilian tertiary reference center: efficacy, safety and predictors of response

Kasuki

Machado

Ogino

et al. 2016

Arch. Endocrinol. Metab.

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Objective: To describe the safety and efficacy of pegvisomant therapy and the predictors of treatment response in acromegaly patients at a single tertiary reference center in Brazil. Materials and methods: We retrospectively reviewed the clinical, hormonal and radiological data of acromegaly patients treated with pegvisomant in our center. We also evaluated the presence of the d3 isoform of the growth hormone receptor (d3GHR). Results: Twenty-seven patients were included (17 women). Pegvisomant was used in combination with octreotide LAR in 20 patients (74%), in combination with cabergoline in one (4%) and as monotherapy in six (22%). IGF-I normalization was achieved in 23 patients (85%). Mild and transitory elevation of liver enzymes was observed in two patients (7.4%), tumor growth in one (3.4%) and lipodystrophy in two (7.4%). One patient stopped the drug due to headaches. The GHR isoforms were evaluated in 14 patients, and the presence of at least one d3GHR allele was observed in 43% of them, but it was not a predictor of treatment response. Only pretreatment IGF-I level was a predictor of treatment response. Conclusion: Pegvisomant treatment was highly effective and safe in our series of Brazilian patients. A better chance of disease control can be expected in those with lower pre-pegvisomant IGF-I levels. Arch Endocrinol Metab. 2016;60(5):479-85

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Diagnosis and Management of Pituitary Adenomas

Tritos

Miller

2023

JAMA

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ImportancePituitary adenomas are neoplasms of the pituitary adenohypophyseal cell lineage and include functioning tumors, characterized by the secretion of pituitary hormones, and nonfunctioning tumors. Clinically evident pituitary adenomas occur in approximately 1 in 1100 persons.ObservationsPituitary adenomas are classified as either macroadenomas (≥10 mm) (48% of tumors) or microadenomas (&lt;10 mm). Macroadenomas may cause mass effect, such as visual field defects, headache, and/or hypopituitarism, which occur in about 18% to 78%, 17% to 75%, and 34% to 89% of patients, respectively. Thirty percent of pituitary adenomas are nonfunctioning adenomas, which do not produce hormones. Functioning tumors are those that produce an excess of normally produced hormones and include prolactinomas, somatotropinomas, corticotropinomas, and thyrotropinomas, which produce prolactin, growth hormone, corticotropin, and thyrotropin, respectively. Approximately 53% of pituitary adenomas are prolactinomas, which can cause hypogonadism, infertility, and/or galactorrhea. Twelve percent are somatotropinomas, which cause acromegaly in adults and gigantism in children, and 4% are corticotropinomas, which secrete corticotropin autonomously, resulting in hypercortisolemia and Cushing disease. All patients with pituitary tumors require endocrine evaluation for hormone hypersecretion. Patients with macroadenomas additionally require evaluation for hypopituitarism, and patients with tumors compressing the optic chiasm should be referred to an ophthalmologist for formal visual field testing. For those requiring treatment, first-line therapy is usually transsphenoidal pituitary surgery, except for prolactinomas, for which medical therapy, either bromocriptine or cabergoline, is usually first line.Conclusions and RelevanceClinically manifest pituitary adenomas affect approximately 1 in 1100 people and can be complicated by syndromes of hormone excess as well as visual field defects and hypopituitarism from mass effect in larger tumors. First-line therapy for prolactinomas consists of bromocriptine or cabergoline, and transsphenoidal pituitary surgery is first-line therapy for other pituitary adenomas requiring treatment.

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Long-term treatment of acromegaly with pegvisomant, a growth hormone receptor antagonist

Abstract: Pegvisomant is an effective medical treatment for acromegaly.

Cited by 587 publications

References 31 publications

Efficacy and safety of monotherapy by pegvisomant, a growth hormone receptor antagonist, in Japanese patients with acromegaly

Efficacy and safety of monotherapy by pegvisomant, a growth hormone receptor antagonist, in Japanese patients with acromegaly

Experience with pegvisomant treatment in acromegaly in a single Brazilian tertiary reference center: efficacy, safety and predictors of response

Diagnosis and Management of Pituitary Adenomas

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