Treatment of Acromegaly with the Growth Hormone–Receptor Antagonist Pegvisomant

Trainer, Peter J; Wm, Drake; Katznelson, Laurence; Pu, Freda; Herman-Bonert,; Aj, van der Lely; Ev, Dimaraki; Stewart, PM; Ke, Friend; Ml, Vance; Gm, Besser; Scarlett, JA; Mo, Thorner; Parkinson, Craig; Klibanski, Anne; Js, Powell; Al, Barkan; Sheppard, M. C.; Malsonado, M; Dr, Rose; Dr, Clemmons; Johannsson, Gudmundur; Ba, Bengtsson; Stavrou, Stavros; Dl, Kleinberg; Dm, Cook; Ls, Phillips; Bidlingmaier, Martin; Cj, Strasburger; Hackett, S; K, Zib; Wf, Bennett; Davis, R E

doi:10.1056/nejm200004203421604

Cited by 792 publications

(618 citation statements)

References 24 publications

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“…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: 63%

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: 63%

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: Discussioncontrasting

confidence: 46%

“…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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“…Man denke hier z.B. an das oben beschriebene plazentare GH oder aber an den GHRezeptorantagonisten Pegvisomant [29] , der zur Behandlung der Akromegalie eingesetzt wird, aufgrund der hohen strukturellen Homologie zum Wildtyp-GH aber in vielen Assays interferiert.…”

Section: Wachstumshormon (Growth Hormone Gh)unclassified

Labordiagnostik bei Wachstumshormon-assoziierten Erkrankungen/Biochemical diagnosis of growth hormone related diseases

Bidlingmaier

2012

LaboratoriumsMedizin

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ZusammenfassungDie Krankheitsbilder des Wachstumshormonmangels und des Wachstumshormonexzesses imponieren klinisch unterschiedlich vor und nach dem Abschluss des Längenwachstums. Stets jedoch bilden die Messung der zirkulierenden Konzentrationen von Wachstumshormon und Insulinartigem Wachstumsfaktor I (Insulin-like growth-factor I, IGF-I) die Basis der laborchemischen Diagnostik. Mit den Immunoassays stehen praktikable, sensitive und meist auch spezifische Methoden zur Messung beider Hormone zur Verfügung. Trotzdem bestehen nach wie vor gravierende Diskrepanzen zwischen den Messergebnissen verschiedener Assays. Diese Diskrepanzen sind vor allem bedingt durch Unterschiede in der Isoformspezifität der Assays, die Verwendung unterschiedlicher Standardpräparationen sowie die Interferenz von Bindungsproteinen. Die methodenbedingten Unterschiede in den Messergebnissen erschweren die allgemeine Anwendung publizierter diagnostischer Entscheidungsgrenzen. Auf einer interdisziplinären Konsensuskonferenz unter Beteiligung der Growth Hormone Research Society, der IGF Society, und der International Federation of Clinical Chemistry and Laboratory Medicine (IFCC) wurden die bestehenden Probleme analysiert und mögliche Strategien zu einer Verbesserung der Vergleichbarkeit der Messergebnisse verschiedener GH- und IGF-I-Assays aufgezeigt. Derzeit bleibt jedoch in der klinischen Praxis die Anwendung methodenspezifischer, an gut charakterisierten Kollektiven gewonnener Referenzbereiche unabdingbar.

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Treatment of Acromegaly with the Growth Hormone–Receptor Antagonist Pegvisomant

Abstract: On the basis of these preliminary results, treatment of patients who have acromegaly with a growth hormone-receptor antagonist results in a reduction in serum IGF-I concentrations and in clinical improvement.

Cited by 792 publications

References 24 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

Labordiagnostik bei Wachstumshormon-assoziierten Erkrankungen/Biochemical diagnosis of growth hormone related diseases

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