Does pegvisomant treatment expertise improve control of resistant acromegaly? The Italian ACROSTUDY experience

Cannavò, Salvatore; Bogazzi, Fausto; Colao, Annamaria; Marinis, Laura De; Maffei, Pietro; Gomez, Roy; Graziano, Eugenio; Monterubbianesi, Maria Cristina; Grottoli, Silvia

doi:10.1007/s40618-015-0289-4

Cited by 13 publications

(9 citation statements)

References 38 publications

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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%

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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Section: Discussionsupporting

confidence: 76%

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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“…However, several small studies performed in the clinical setting found IGF‐I normalization rates at the last evaluation ranging from 84 to 92%. Although these better results could be interpreted as a result of greater expertise in the treatment of acromegaly in some centres, this does not appear to be the explanation in all settings …”

Section: Introductionmentioning

confidence: 95%

“…Although these better results could be interpreted as a result of greater expertise in the treatment of acromegaly in some centres, this does not appear to be the explanation in all settings. 14 Several reasons for this lower than expected efficacy of PEG in observational studies have been recently outlined. 15 For instance, problems with dose titration or compliance, change in the IGF-I measuring method, temporary loss of IGF-I control requiring dose adjustments or the use of different criteria to assess IGF-I normalization in preclinical versus observational and clinical studies (IGF-I normalization at any time versus IGF-I normalization at last evaluation) have been identified.…”

Section: Introductionmentioning

confidence: 99%

Long‐term treatment with pegvisomant for acromegaly: a 10‐year experience

Ramos-Leví

Bernabéu

Álvarez-Escolá

et al. 2016

Clinical Endocrinology

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“…This lower than expected efficacy, observed especially in retrospective studies, could be explained by an inadequate dose titration of the drug, poor compliance to daily injections, selection bias of patients recruited in clinical trials in terms of disease activity and drug responsiveness, or technical problems related to IGF-I assay, while a true “biochemical resistance” to PEG cannot be ruled out yet [29]. Recently, a comparison between secondary and tertiary referral centers, identified on the basis of the number of acromegalic patients treated with PEG, failed to demonstrate significant differences in IGF-I normalization rates [30]. According to a recent study, the response to PEG as second line medical therapy can be predicted on the basis of patient’s age, BMI, and baseline IGF-I levels, but not of subjective symptoms [28].…”

Section: Biochemical Outcomementioning

confidence: 99%

Pegvisomant in acromegaly: an update

Giustina

Arnaldi

Bogazzi

et al. 2017

J Endocrinol Invest

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BackgroundIn 2007, we published an opinion document to review the role of pegvisomant (PEG) in the treatment of acromegaly. Since then, new evidence emerged on the biochemical and clinical effects of PEG and on its long-term efficacy and safety.AimWe here reviewed the emerging aspects of the use of PEG in clinical practice in the light of the most recent literature.ResultsThe clinical use of PEG is still suboptimal, considering that it remains the most powerful tool to control IGF-I in acromegaly allowing to obtain, with a pharmacological treatment, the most important clinical effects in terms of signs and symptoms, quality of life and comorbidities. The number of patients with acromegaly exposed to PEG worldwide has become quite elevated and the prolonged follow-up allows now to deal quite satisfactorily with many clinical issues including major safety issues, such as the concerns about possible tumour (re)growth under PEG. The positive or neutral impact of PEG on glucose metabolism has been highlighted, and the clinical experience, although limited, with sleep apnoea and pregnancy has been reviewed. Finally, the current concept of somatostatin receptor ligands (SRL) resistance has been addressed, in order to better define the acromegaly patients to whom the PEG option may be offered.ConclusionsPEG increasingly appears to be an effective and safe medical option for many patients not controlled by SRL but its use still needs to be optimized.

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Does pegvisomant treatment expertise improve control of resistant acromegaly? The Italian ACROSTUDY experience

Cited by 13 publications

References 38 publications

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

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

Long‐term treatment with pegvisomant for acromegaly: a 10‐year experience

Pegvisomant in acromegaly: an update

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