Indirect Treatment Comparison of Biochemical Control Between Pasireotide Long-Acting Release and Pegvisomant as Second-Line Therapies for Acromegaly, Based on Real-World Evidence
Author(s)
Grzegorz Binowski, MA1, KOSSI Dédé Sika, PhD2, Malgorzata Bronikowska, PhD1, Anna Packowska, BSc1, Ruth Delgado, BPharm3, Beatrice Gueron, MA2, Fabian Schmidt, MA2, Sabrina Chiloiro, MD, PhD4.
1MAHTA Intl., Warsaw, Poland, 2Recordati Rare Diseases, Puteaux, France, 3Recordati Rare Diseases, Madrid, Spain, 4Gemelli University Hospital, Università Cattolica del Sacro Cuore, Rome, Italy.
1MAHTA Intl., Warsaw, Poland, 2Recordati Rare Diseases, Puteaux, France, 3Recordati Rare Diseases, Madrid, Spain, 4Gemelli University Hospital, Università Cattolica del Sacro Cuore, Rome, Italy.
OBJECTIVES: Acromegaly is a rare, chronic endocrine disorder associated with serious comorbidities, reduced quality of life, and a two - to threefold increase in mortality. Second-line medical treatments include pasireotide long-acting release (PAS LAR), and pegvisomant (PEG) either as monotherapy or in combination with somatostatin receptor ligands (SRLs). Despite their widespread use, direct comparative data on their effectiveness in clinical practice remain limited. This study aims to address this gap by conducting the first indirect treatment comparison (ITC) of insulin-like growth factor 1 (IGF-1) normalization rates between PAS LAR and PEG, using evidence derived from real-world clinical practice (RWE).
METHODS: A systematic literature review (SLR) of real-world evidence (RWE) studies evaluating PAS LAR and/or PEG as second-line treatment for acromegaly was conducted in MEDLINE and EMBASE in accordance with PRISMA and Cochrane guidelines. Studies reporting IGF-1 normalization rates with treatment durations between 6-24 months were included in the ITC to ensure outcome comparability. Patient characteristics were extracted to assess baseline comparability. Meta-analyses of IGF-1 outcomes were conducted, and naïve odds ratios (ORs) were calculated using R software.
RESULTS: From 512 abstracts screened, 134 full texts were reviewed, and 72 studies met the SLR inclusion criteria. Seven PAS LAR and four PEG studies met the meta-analysis criteria and were included in the base case. IGF-1 normalization rates were 0.56 (95% CI: 0.50-0.62) for PAS LAR and 0.60 (95% CI: 0.42-0.78) for PEG. The OR comparing PAS LAR to PEG was 0.89 (95% CI: 0.68-1.15; p = 0.368), indicating no statistically significant difference.
CONCLUSIONS: This meta-analysis of RWE suggests similar efficacy in normalizing IGF-1 levels between PAS LAR and PEG in the second-line treatment of acromegaly. Further ITCs are planned to assess other comparative outcomes in real-world clinical practice, including tumour response, quality of life, treatment safety and discontinuation.
METHODS: A systematic literature review (SLR) of real-world evidence (RWE) studies evaluating PAS LAR and/or PEG as second-line treatment for acromegaly was conducted in MEDLINE and EMBASE in accordance with PRISMA and Cochrane guidelines. Studies reporting IGF-1 normalization rates with treatment durations between 6-24 months were included in the ITC to ensure outcome comparability. Patient characteristics were extracted to assess baseline comparability. Meta-analyses of IGF-1 outcomes were conducted, and naïve odds ratios (ORs) were calculated using R software.
RESULTS: From 512 abstracts screened, 134 full texts were reviewed, and 72 studies met the SLR inclusion criteria. Seven PAS LAR and four PEG studies met the meta-analysis criteria and were included in the base case. IGF-1 normalization rates were 0.56 (95% CI: 0.50-0.62) for PAS LAR and 0.60 (95% CI: 0.42-0.78) for PEG. The OR comparing PAS LAR to PEG was 0.89 (95% CI: 0.68-1.15; p = 0.368), indicating no statistically significant difference.
CONCLUSIONS: This meta-analysis of RWE suggests similar efficacy in normalizing IGF-1 levels between PAS LAR and PEG in the second-line treatment of acromegaly. Further ITCs are planned to assess other comparative outcomes in real-world clinical practice, including tumour response, quality of life, treatment safety and discontinuation.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO150
Topic
Clinical Outcomes, Real World Data & Information Systems
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Rare & Orphan Diseases