More than a decade of real-world experience of pegvisomant for acromegaly: ACROSTUDY
| Autor: | Roberto Salvatori, Maria Fleseriu, Aart Jan van der Lely, Thierry Brue, Michael Wajnrajch, Dagmar Führer-Sakel, Roy Gomez, Judith Hey-Hadavi, Andrew Palladino, Laura De Marinis, Srinivas Rao Valluri, Cecilia Camacho-Hübner, Joli van der Lans-Bussemaker |
|---|---|
| Přispěvatelé: | Marseille medical genetics - Centre de génétique médicale de Marseille (MMG), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Institut Marseille Maladies Rares (MarMaRa), Aix Marseille Université (AMU), Service d'endocrinologie, diabète, maladies métaboliques [Hôpital de la Conception - APHM] |
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Adenoma
Adult Male medicine.medical_specialty Pediatrics Combination therapy Adolescent Endocrinology Diabetes and Metabolism medicine.medical_treatment [SDV]Life Sciences [q-bio] Medizin History 21st Century Cohort Studies Drug withdrawal Young Adult Endocrinology Internal medicine Acromegaly medicine Humans In patient Adverse effect Child Aged Retrospective Studies Aged 80 and over business.industry Human Growth Hormone Infant General Medicine Middle Aged medicine.disease United States Radiation therapy Europe [STAT]Statistics [stat] Treatment Outcome Child Preschool Pegvisomant Cohort Clinical Study Female Growth Hormone-Secreting Pituitary Adenoma business [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology medicine.drug Follow-Up Studies |
| Zdroj: | European Journal of Endocrinology European Journal of Endocrinology, 2021, 185 (4), pp.525-538. ⟨10.1530/EJE-21-0239⟩ |
| ISSN: | 0804-4643 1479-683X |
| DOI: | 10.1530/EJE-21-0239⟩ |
| Popis: | Objective To report the final long-term safety and efficacy analyses of patients with acromegaly treated with pegvisomant from the ACROSTUDY. Design Global (15 countries), multicentre, non-interventional study (2004–2017). Methods The complete ACROSTUDY cohort comprised patients with acromegaly, who were being treated with pegvisomant (PEGV) prior to the study or at enrolment. The main endpoints were long-term safety (comorbidities, adverse events (AEs), pituitary tumour volumes, liver tests) and efficacy (IGF1 changes). Results Patients (n = 2221) were treated with PEGV for a median of 9.3 years (range, 0–20.8 years) and followed up for a median of 7.4 years (range, 0–13.9 years). Before PEGV, 96.3% had received other acromegaly treatments (surgery/radiotherapy/medications). Before PEGV treatment, 87.2% of patients reported comorbidities. During ACROSTUDY, 5567 AEs were reported in 56.5% of patients and of these 613 were considered treatment-related (in 16.5% of patients) and led to drug withdrawal in 1.3%. Pituitary imaging showed a tumour size increase in 7.1% of patients; the majority (71.1%) reported no changes. Abnormal AST or ALT liver tests occurred in 3.2% of patients. IGF1 normalization rate improved over time, increasing from 11.4% at PEGV start to 53.7% at year 1, and reaching 75.4% at year 10 with the use of ≥30 mg PEGV/day in an increasing proportion of patients. Conclusion This comprehensive review of the complete cohort in ACROSTUDY confirmed the overall favourable benefit-to-risk profile and high efficacy of PEGV as mono- and combination therapy in patients with an aggressive course/uncontrolled/active acromegaly requiring long-term medical therapy for control. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|