From the Guidelines
The current management of retinal hemangioblastoma prioritizes early detection and treatment, with laser photocoagulation being the first-line therapy for small peripheral tumors, and consideration of newer systemic treatments like HIF2-a inhibitors for larger or more complex cases. The approach to managing retinal hemangioblastoma (RH) is multifaceted, taking into account the size, location, and associated complications of the tumor. According to the most recent guidelines and evidence, including the consensus guidelines for ocular surveillance of von Hippel-Lindau disease published in 2024 1, early detection through periodic ocular screening is crucial. This screening should start within 12 months after birth for individuals with known or suspected VHL disease and continue throughout life, with the frequency of screening adjusted based on age and other risk factors.
For small, asymptomatic peripheral lesions, observation may be appropriate, but for active treatment, laser photocoagulation is recommended as the first-line therapy for small (<3mm) peripheral tumors, typically requiring 1-3 sessions for complete regression, as supported by studies such as those by Kreusel et al., Singh et al., and Krivosic et al. mentioned in the evidence 1. These studies demonstrate the effectiveness and safety of laser photocoagulation for small RHs, with near-universal success in treating tumors with a diameter of 1.5 mm or less.
However, the emergence of systemic treatments, such as the oral small-molecule HIF2-a inhibitor belzutifan, approved for the treatment of VHL disease-related renal cell carcinoma, pancreatic neuroendocrine tumors, and central nervous system hemangioblastomas, offers a promising alternative for the management of RHs, particularly for larger or more complex cases 1. This systemic treatment may provide a safer option for the management of juxtapapillary tumors and macular tumors or for large tumors for which safe and effective treatment options are currently lacking.
Key points in the management of retinal hemangioblastoma include:
- Early detection through periodic ocular screening
- Prompt treatment of extramacular or extrapapillary RHs
- Use of laser photocoagulation as the first-line therapy for small peripheral tumors
- Consideration of systemic treatments like HIF2-a inhibitors for larger or more complex cases
- Management by subspecialty-trained professionals within a multidisciplinary center whenever possible
In conclusion, the management of retinal hemangioblastoma is evolving, with a focus on early detection, prompt treatment, and the consideration of newer systemic therapies to improve outcomes and preserve vision.
From the Research
Current Management of Retinal Hemangioblastoma
The current management of retinal hemangioblastoma involves a combination of treatment options, including:
- Ablative therapy, such as laser photocoagulation 2, 3, 4
- Vitreoretinal surgery 2, 3, 5
- Cryotherapy 3, 5
- Photodynamic therapy 3
- Transpupillary thermotherapy 3, 4
- Observation or non-ablative treatments for retinal hemangioblastomas that cannot be safely destroyed 6
Treatment Strategies
Treatment strategies for retinal hemangioblastoma depend on the size, location, and complications of the lesion, as well as the presence of Von Hippel-Lindau disease 2, 3, 6.
- Small retinal hemangioblastomas (<0.5 mm in size) may be treated with laser photocoagulation 4
- Moderate-sized retinal hemangioblastomas (0.5-3.0 mm in size) may be treated with transpupillary thermotherapy 4
- Large retinal hemangioblastomas (>3.0 mm in size) may require a combination of treatments, including vitreoretinal surgery and cryotherapy 3, 4
Importance of Early Detection and Follow-up
Early detection and regular follow-up are crucial in the management of retinal hemangioblastoma, as they can help reduce the risk of complications and improve visual outcomes 2, 5, 6.
- Regular ophthalmic evaluation is recommended for individuals with Von Hippel-Lindau disease to identify and offer timely treatment for new or active retinal hemangioblastomas 6
- Complications are strongly correlated with the initial size of the vascular tumor, highlighting the importance of early diagnosis and treatment 5