TUMOR STABILIZATION AND LONG-TERM IGF-1 NORMALIZATION RATE WITH PASIREOTIDE LAR AND PEGVISOMANT IN REAL-WORLD SECOND-LINE ACROMEGALY TREATMENT
Author(s)
Malgorzata Bronikowska, PhD1, Grzegorz Binowski, MA1, Ruth Delgado, BPharm2, Sonia Gorzkowska-Marciniak, MA1, Fabian Schmidt, MA3, KOSSI Dédé Sika, PhD3, Sabrina Chiloiro, MD, PhD4.
1MAHTA Intl., Warsaw, Poland, 2Recordati Rare Diseases, Madrid, Spain, 3Recordati Rare Diseases, Puteaux, France, 4Gemelli University Hospital, Università Cattolica del Sacro Cuore, Rome, Italy.
1MAHTA Intl., Warsaw, Poland, 2Recordati Rare Diseases, Madrid, Spain, 3Recordati Rare Diseases, Puteaux, France, 4Gemelli University Hospital, Università Cattolica del Sacro Cuore, Rome, Italy.
OBJECTIVES: Pasireotide long-acting release (PAS LAR) and pegvisomant in mono- or combination therapy (PEG±FGSRLs) are important second-line treatment options for acromegaly. Published evidence indicates comparable biochemical effectiveness around 60% of these therapies, while PAS LAR additionally provides a clinically meaningful tumor volume shrinkage around 38%. This study compared IGF-1 normalization over different follow-up periods and tumor stabilization in routine clinical practice, both of which are recognized treatment goals.
METHODS: A systematic literature review of RWE studies evaluating second-line acromegaly treatments was conducted in MEDLINE and EMBASE following PRISMA and Cochrane guidelines. Studies reporting IGF-1 normalization at 6 months, 12 months, or longest available follow-up were included, and separate meta-analyses were conducted for each treatment. Pooled rates were estimated, and naïve odds ratios were calculated using R software. Tumor stabilization was defined as the absence of tumor enlargement across definition-based scenarios reported in studies, with results presented separately for PAS LAR and PEG±FGSRLs.
RESULTS: From 512 abstracts screened, 134 full texts were reviewed, and 72 studies met the SLR inclusion criteria. Pooled IGF-1 normalization for PAS LAR and PEG±FGSRLs was 50.3% and 52.2% at 6 months, increasing to 59.2% and 61.5% at longest available follow-up, respectively (13.1-51 months for PAS LAR and 30.5-111.6 months for PEG). Across tumor-stabilization scenarios, rates ranged from 95% to 98% for PAS LAR and 83% to 96% for PEG±FGSRLs, with PAS LAR rates of 96% to 97% observed in patients with proven unfavourable tumor characteristics.
CONCLUSIONS: This meta-analysis of RWE suggests broadly similar IGF-1 normalization rates with PAS LAR and PEG±FGSRLs in second-line acromegaly treatment, with biochemical control improving over longer follow-up. PAS LAR showed tumor stabilization in a population that, according to clinical guidelines, includes patients with tumor-related concerns, supporting its role in preventing further tumor growth while providing sustained biochemical control and tumor volume reduction.
METHODS: A systematic literature review of RWE studies evaluating second-line acromegaly treatments was conducted in MEDLINE and EMBASE following PRISMA and Cochrane guidelines. Studies reporting IGF-1 normalization at 6 months, 12 months, or longest available follow-up were included, and separate meta-analyses were conducted for each treatment. Pooled rates were estimated, and naïve odds ratios were calculated using R software. Tumor stabilization was defined as the absence of tumor enlargement across definition-based scenarios reported in studies, with results presented separately for PAS LAR and PEG±FGSRLs.
RESULTS: From 512 abstracts screened, 134 full texts were reviewed, and 72 studies met the SLR inclusion criteria. Pooled IGF-1 normalization for PAS LAR and PEG±FGSRLs was 50.3% and 52.2% at 6 months, increasing to 59.2% and 61.5% at longest available follow-up, respectively (13.1-51 months for PAS LAR and 30.5-111.6 months for PEG). Across tumor-stabilization scenarios, rates ranged from 95% to 98% for PAS LAR and 83% to 96% for PEG±FGSRLs, with PAS LAR rates of 96% to 97% observed in patients with proven unfavourable tumor characteristics.
CONCLUSIONS: This meta-analysis of RWE suggests broadly similar IGF-1 normalization rates with PAS LAR and PEG±FGSRLs in second-line acromegaly treatment, with biochemical control improving over longer follow-up. PAS LAR showed tumor stabilization in a population that, according to clinical guidelines, includes patients with tumor-related concerns, supporting its role in preventing further tumor growth while providing sustained biochemical control and tumor volume reduction.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO174
Topic
Clinical Outcomes
Topic Subcategory
Clinician Reported Outcomes, Comparative Effectiveness or Efficacy
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Rare & Orphan Diseases