Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience
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 rout...
| Autores: | ; ; ; ; ; ; ; ; |
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| Tipo de documento: | Artigo |
| Estado: | Published version |
| Data de publicação: | 2022 |
| País: | España |
| Recursos: | Institut d’Investigació Biomèdica Sant Pau (IIB Sant Pau) |
| Repositório: | r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau |
| OAI Identifier: | oai:iibsantpau.fundanetsuite.com:p9974 |
| Acesso em linha: | https://iibsantpau.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=9974 https://hal-amu.archives-ouvertes.fr/hal-03780362/ |
| Nível de acesso: | Acesso aberto |
| Palavra-chave: | Acromegaly Pegvisomant ACROSTUDY Patient-reported outcomes |
| _version_ | 1878734942155833344 |
|---|---|
| author | Salvatori, R |
| author2 | Maffei, P Webb, SM Brue, T Loftus, J Valluri, SR Gomez, R Wajnrajch, MP Fleseriu, M |
| author2_role | author author author author author author author author |
| author_browse | Brue, T Fleseriu, M Gomez, R Loftus, J Maffei, P Salvatori, R Valluri, SR Wajnrajch, MP Webb, SM |
| author_facet | Salvatori, R Maffei, P Webb, SM Brue, T Loftus, J Valluri, SR Gomez, R Wajnrajch, MP Fleseriu, M |
| author_role | author |
| collection | r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau |
| description | 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. |
| eu_rights_str_mv | openAccess |
| format | article |
| id | ES_72e26a67b4b1e8eaaf7176812e2eeadf |
| institution | Institut d’Investigació Biomèdica Sant Pau (IIB Sant Pau) |
| instname_str | Institut d’Investigació Biomèdica Sant Pau (IIB Sant Pau) |
| network_acronym_str | ES |
| network_name_str | España |
| oai_identifier_str | oai:iibsantpau.fundanetsuite.com:p9974 |
| publishDate | 2022 |
| publishDateSort | 2022 |
| publisher | SPRINGER |
| publisherStr | SPRINGER |
| reponame_str | r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau |
| spelling | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience Salvatori, R Maffei, P Webb, SM Brue, T Loftus, J Valluri, SR Gomez, R Wajnrajch, MP Fleseriu, M Acromegaly Pegvisomant ACROSTUDY Patient-reported outcomes 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. SPRINGER https://iibsantpau.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=9974 https://hal-amu.archives-ouvertes.fr/hal-03780362/ |
| spellingShingle | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience Salvatori, R Acromegaly Pegvisomant ACROSTUDY Patient-reported outcomes |
| status_str | publishedVersion |
| title | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience |
| title_full | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience |
| title_fullStr | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience |
| title_full_unstemmed | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience |
| title_short | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience |
| title_sort | Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience |
| topic | Acromegaly Pegvisomant ACROSTUDY Patient-reported outcomes |
| topic_facet | Acromegaly Pegvisomant ACROSTUDY Patient-reported outcomes |
| url | https://iibsantpau.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=9974 https://hal-amu.archives-ouvertes.fr/hal-03780362/ |