AI vs. Physician for Anti-VEGF Decision-Making: An RCT

An Artificial Intelligence System for Anti-VEGF Treatment Decisions in Retinal Diseases: A Randomized Controlled Trial

We developed an artificial intelligence system, called QiLin, which was designed to assist anti-VEGF treatment decisions in retinal diseases. QiLin was trained and validated via over 20,000 optical coherence tomography images from multicenter datasets, demonstrating strong performance on both internal and external validation. To evaluate its real-world clinical utility, we conducted a randomized controlled trial that rigorously compares the accuracy of treatment decisions between a physician-only arm and an AI-assisted physician arm.

Study Overview

• Status: Not yet recruiting
• Conditions: DME; Neovascular (Wet) Age-Related Macular Degeneration; Retinal Vein Occlusion (RVO)
• Intervention / Treatment: Other: QiLin-assisted; Other: physician only, without QiLin assisted

• Study Type: Interventional
• Enrollment (Estimated): 200
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Xiaodong Prof. Sun, PhD; Phone Number: 86 17853138155; Email: xdsun@sjtu.edu.cn
• Study Contact Backup: Name: Huixun Jia, PhD; Phone Number: 86 17853138155; Email: jiahuixun@sjtu.edu.cn

Study Locations

• China
  • Shanghai, China
    • Shanghai General Hospital
    • Contact: Huixun Jia, PhD; Phone Number: 86 17853138155; Email: jiahuixun@sjtu.edu.cn
  • Shanghai, China
    • Shanghai general hospital, Shanghai Jiao Tong University, Shanghai, 200080
    • Contact: Xiaodong Sun, PhD; Phone Number: +86-02163240090; Email: xdsun@sjtu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Patients with a diagnosis of nAMD, DME, and RVO; Patients who have completed the loading-dose treatment of anti-VEGF agents; Patients who were willing to participate and provided written informed consent.

Exclusion Criteria:

Refusal to undergo OCT testing; Refusal to complete the 3-month follow-up period; Screening for a history of intraocular surgery within the past 6 months; Subjects with severe systemic diseases, intellectual developmental disorders, psychiatric illnesses, etc.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: QiLin-assisted physician arm	Other: QiLin-assisted; A Comprehensive Deep Learning Model for Assisting the decision of anti-VEGF therapy: QiLin system
Active Comparator: physician only arm	Other: physician only, without QiLin assisted; without QiLin assisted

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Accuracy of the current anti-VEGF injection decision	The accuracy of the current anti-VEGF injection decision was defined as the proportion of injection decisions (yes or no) made by the physicians in the two arms that were in agreement with the independent senior expert.	At enrollment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Accuracy of detecting active biomarkers on the current OCT image	The secondary endpoint was defined as the accuracy of detecting active biomarkers. For each patient, the physician was required to perform a binary classification (present vs. absent) for all of 8 pre-defined active biomarkers (PED, NV, IRF, SRF, SHRM, HRF, DRT or DME, and VMT), and was further confirmed by an independent senior retina specialist. The accuracy for per biomarker was calculated as the proportion of correct classifications for that biomarker, and then the average accuracy was calculated as the secondary endpoint.	At enrollment

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Accuracy of the recommended anti-VEGF treatment interval	At enrollment, physicians in both arms will recommend an anti-VEGF treatment interval. Then, patients will attend monthly visits for 3 months. At each visit, an independent expert physician will evaluate whether anti-VEGF injection is required. The accuracy of the recommended treatment interval is defined as the proportion of cases where the recommended treatment interval is concordant with the actual treatment interval.	3 months from enrollment

Collaborators and Investigators

• Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
• Investigators: Principal Investigator: Xiaodong Sun, PhD, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Study record dates

Study Major Dates

• Study Start (Estimated): May 25, 2026
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): October 15, 2026

Study Registration Dates

• First Submitted: December 27, 2025
• First Submitted That Met QC Criteria: December 27, 2025
• First Posted (Actual): January 9, 2026

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 18, 2026
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Eye Diseases; Embolism and Thrombosis; Retinal Diseases; Retinal Degeneration; Venous Thrombosis; Thrombosis; Macular Degeneration; Retinal Vein Occlusion
• Other Study ID Numbers: SXD20251227

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No