Long-term safety and efficacy of pegvisomant monotherapy for acromegaly: final data from the full ACROSTUDY cohort
Authors' conclusion
In this largest and longest real-world study of pegvisomant monotherapy in patients with acromegaly to date, pegvisomant demonstrated a favorable safety profile and was effective at normalizing and maintaining IGF-I levels in the majority of patients.
Study context
- Population
- Patients with acromegaly aged > 18 years at ACROSTUDY enrollment who received ≥ 1 dose of pegvisomant monotherapymonotherapy cohort
- Participants
- 1,464 patients
- Outcome
- Long-term safety and efficacy of pegvisomant monotherapy
- Follow-up
- Median 6.0 years of monotherapy; mean 7.6 years
Methodological detailsinternational, non-interventional, real-world
- International
- Non-interventional
- Real-world
Main finding
As reported in the abstract
Adverse events (AEs) (all-causality)
49%
n = 717/1464
Serious AEs
19.3%
n = 282/1464
Serum IGF-I normalization at last observation
62.3%
n = 756/1213
Observational data: associations, not effects.
Further findings
| Outcome | Values |
|---|---|
| Daily pegvisomant dose at last observation | Pegvisomant monotherapy median 15.0 (range 0–60) mg/day |
| Pegvisomant monotherapy duration | Pegvisomant monotherapy median 6.0 (range 0.0–19.3) years |
Tumor size on MRI at study sites
- Increased tumor size
- 7.2% (n = 90/1248)
- Change in tumor size
- 70.6% (n = 881/1248)
Safety
- AEs (all-causality)
- 49% (n = 717/1464)
- Serious AEs
- 19.3% (n = 282/1464)
- Treatment-related AE: IGF-I increased
- 1.8%
- Treatment-related AE: lipohypertrophy
- 1.2%
- Treatment-related AE: transaminases increased
- 1.2%
- Treatment-related AE: IGF-I decreased
- 1.1%
- Deaths (none treatment-related)
- 31 (2.1%)
Original publication
Long-term safety and efficacy of pegvisomant monotherapy for acromegaly: final data from the full ACROSTUDY cohort. Pituitary. Published 15 April 2026. doi:10.
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