Related Experiment Video
Updated: Oct 3, 2026

An Ex Vivo Tissue Culture Model for Fibrovascular Complications in Proliferative Diabetic Retinopathy
Published on: January 25, 2019
The effect of a growth hormone receptor antagonist drug on proliferative diabetic retinopathy
Objective:
This study investigated whether the growth hormone receptor antagonist pegvisomant could produce regression of diabetic retinal neovascularization.
Design:
A prospective, single-group, open-label, phase IIa multicenter trial was conducted.
Participants:
Twenty-five patients with diabetes mellitus (13 with type 1 and 12 with type 2) and proliferative diabetic retinopathy aged 21 years or older were enrolled.
Methods:
Patients received an initial loading dose of 100 mg pegvisomant by subcutaneous injection, followed by self-administered injections of 20 mg daily for 12 weeks. The treatment period was followed by a 12-week period during which patients were observed off treatment.
Main Outcome Measure:
The effect of treatment on diabetic retinopathy was evaluated from fundus photographs that were graded at a central reading center.
Results:
Regression of retinopathy was not observed in any patient. At the end of the treatment period, the extent of neovascularization in the study eye was unchanged in 16 patients (9 of 13 with type 1 and 7 of 12 with type 2) and had progressed in 9 patients (4 of 13 with type 1 and 5 of 12 with type 2). The maximum reduction from baseline per patient in the insulin-like growth factor-I (IGF-I) serum level averaged 55%; 17 (68%) of the 25 patients had a greater than 50% reduction in IGF-I. After treatment was discontinued, IGF-I levels returned to baseline.
Conclusions:
This study did not find evidence that pegvisomant could produce regression of diabetic retinal neovascularization.
Insights
Pegvisomant did not regress diabetic retinal neovascularization in patients with proliferative diabetic retinopathy. The drug did not alter neovascularization extent, though it reduced IGF-I levels during treatment.
Area of Science:
- Ophthalmology
- Endocrinology
- Pharmacology
Background:
- Diabetic retinopathy is a leading cause of blindness.
- Neovascularization is a hallmark of proliferative diabetic retinopathy.
- Growth hormone receptor antagonists are being explored for therapeutic potential.
Purpose of the Study:
- To investigate the efficacy of pegvisomant in regressing diabetic retinal neovascularization.
- To evaluate the safety and tolerability of pegvisomant in this patient population.
Main Methods:
- A prospective, open-label, phase IIa multicenter trial.
- 25 patients with type 1 or type 2 diabetes and proliferative diabetic retinopathy were enrolled.
- Patients received pegvisomant (100 mg loading dose, then 20 mg daily) for 12 weeks, followed by a 12-week observation period.
Main Results:
- No regression of diabetic retinopathy was observed in any patient.
- Neovascularization remained unchanged in 16 patients and progressed in 9 patients.
- Pegvisomant significantly reduced serum IGF-I levels by an average of 55% during treatment, with levels returning to baseline post-treatment.
Conclusions:
- Pegvisomant did not demonstrate efficacy in regressing diabetic retinal neovascularization.
- Further research is needed to explore the role of growth hormone signaling in diabetic retinopathy.
Related Concept Videos
Glucagon-like Receptor Agonists
GLP-1, when administered in high doses intravenously, triggers insulin secretion, inhibits glucagon release, slows gastric emptying, reduces food intake, and restores normal insulin secretion. However, its rapid inactivation by the...
Diabetic Retinopathy
Dipeptidyl Peptidase 4 Inhibitors
Oral Hypoglycemic Agents: Glinides
Oral Hypoglycemic Agents: Biguanides and Glitazones
Oral Hypoglycemic Agents: α-Glucosidase Inhibitors
Acarbose and miglitol are typically...

