Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience
Por:
Salvatori, R, Maffei, P, Webb, SM, Brue, T, Loftus, J, Valluri, SR, Gomez, R, Wajnrajch, MP, Fleseriu, M
Publicada:
1 jun 2022
Ahead of Print:
1 ene 2022
Resumen:
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 naive/semi-naive (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.
Filiaciones:
Salvatori, R:
Johns Hopkins Univ, Div Endocrinol & Metab & Pituitary Ctr, Sch Med, Baltimore, MD 21218 USA
Maffei, P:
Univ Padua, Dept Med DIMED, Padua, Italy
Webb, SM:
Univ Autonoma Barcelona, Ctr Invest Biomed Red Enfermedades Raras, Dept Endocrinol Med, Hosp St Pau, Carrer St Quinti 89, Barcelona 08041, Spain
Brue, T:
Hop Conception, Marseille, France
Aix Marseille Univ, AP HM, Marseille Med Genet, Marseille, France
Loftus, J:
Pfizer Ltd, Tadworth, England
Valluri, SR:
Pfizer Inc, New York, NY USA
Gomez, R:
Pfizer Inc, Brussels, Belgium
Wajnrajch, MP:
Pfizer Inc, New York, NY USA
NYU, Grossman Sch Med, Div Pediat Endocrinol, New York, NY USA
Fleseriu, M:
Oregon Hlth & Sci Univ, Pituitary Ctr, Dept Med, Div Endocrinol Diabet & Clin Nutr & Neurol Surg, Portland, OR USA
All Open Access; Green
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