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12.01.2022 | Acromegaly

Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience

verfasst von: Roberto Salvatori, Pietro Maffei, Susan M. Webb, Thierry Brue, Jane Loftus, Srinivas Rao Valluri, Roy Gomez, Michael P. Wajnrajch, Maria Fleseriu

Erschienen in: Pituitary | Ausgabe 3/2022

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Abstract

Purpose

To report the effects of pegvisomant (PEGV) treatment on patient-reported outcomes in acromegaly patients.

Methods

We conducted an extension study of an open-label, multinational, non-interventional study (ACROSTUDY) evaluating the long-term safety and efficacy of PEGV for acromegaly in routine clinical practice. Enrolled patients were rollover patients from ACROSTUDY, or treatment naïve/semi-naïve (NSN; no PEGV within 6 months of enrollment). Exploratory efficacy endpoints were changes in symptoms with the Patient-Assessed Acromegaly Symptom Questionnaire (PASQ) and quality of life with the Acromegaly Quality of Life questionnaire (AcroQoL) analyzed by controlled or uncontrolled IGF-I levels. Results were analyzed in all patients, in NSN patient subgroup, and by diabetes status.

Results

A total of 544 patients with acromegaly were enrolled, including 434 rollover subjects from ACROSTUDY and 110 NSN patients. Mean PEGV treatment duration was 7.8 years (range, 0–19.6 years). Overall, the majority of PASQ scores improved over time, but there was no significant difference between IGF-I controlled or uncontrolled groups. In the NSN subgroup, most PASQ and AcroQoL scores remained similar to baseline up to 1 year, regardless of IGF-I control. Patients with diabetes reported better PASQ scores over time with PEGV treatment, regardless of IGF-I control. IGF-I normalization increased from 10% of patients at baseline to more than 78% at year 10, with a mean daily PEGV dose of 18.7 mg.

Conclusions

Overall, patients treated with PEGV had small improvements in PASQ. While IGF-I normalization increased with PEGV treatment, IGF-I control had no effects on PASQ and AcroQoL scores.
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Metadaten
Titel
Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience
verfasst von
Roberto Salvatori
Pietro Maffei
Susan M. Webb
Thierry Brue
Jane Loftus
Srinivas Rao Valluri
Roy Gomez
Michael P. Wajnrajch
Maria Fleseriu
Publikationsdatum
12.01.2022
Verlag
Springer US
Schlagwort
Acromegaly
Erschienen in
Pituitary / Ausgabe 3/2022
Print ISSN: 1386-341X
Elektronische ISSN: 1573-7403
DOI
https://doi.org/10.1007/s11102-022-01206-2

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In diesem CME-Kurs können Sie Ihr Wissen zur EKG-Befundung anhand von zwölf Video-Tutorials auffrischen und 10 CME-Punkte sammeln.
Praxisnah, relevant und mit vielen Tipps & Tricks vom Profi.

Update Innere Medizin

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