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Intravitreal Melphalan for Vitreous Seeds: Initial Experience in China
Xunda Ji1, Peiyan Hua1, Jing Li1
1Department of Ophthalmology, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, 1665 Kongjiang Road, Shanghai 200092, China.
Abstract:
Purpose. To evaluate the efficacy of intravitreal melphalan for vitreous seeds from retinoblastoma in Chinese patients. Methods. This is a retrospective review of 17 consecutive Chinese patients (19 eyes) with viable vitreous seeds from retinoblastoma. The patients received multiple intravitreal injections of 20 ug melphalan. Results. The International Classification of Retinoblastoma groups were B in 1 eye, C in 5 eyes, D in 11 eyes, and E in 2 eyes. On average, 6 injections (range: 1-15) were given to each eye at the interval of 2-4 weeks. Successful control of vitreous seeds was achieved in 16 of 19 eyes (84.21%). Globe retention was achieved in 73.68% (14/19) eyes. The patients were followed up for 27 months on average (median: 26; range: 17-42 months). There is a significant difference in response to intravitreal melphalan for cloud, spheres, and dust seeds with a median number of injections of 9, 6, and 3, respectively (P = 0.003). Complications related to intravitreal melphalan included vitreous hemorrhage, cataract, salt-and-pepper retinopathy, and pupil posterior synechia. There was no case of epibulbar extension or systemic metastasis within the period of follow-up. Conclusion. Intravitreal melphalan achieved a high local control rate for vitreous seeds without extraocular extension and with acceptable toxicity in Chinese retinoblastoma patients.
Insights
Intravitreal melphalan effectively controlled retinoblastoma vitreous seeds in Chinese patients, achieving an 84% success rate and preserving globes in 74% of cases with manageable side effects.
Area of Science:
- Ophthalmology
- Pediatric Oncology
- Cancer Treatment
Background:
- Retinoblastoma is the most common primary intraocular malignancy in children.
- Vitreous seeds are a common and challenging manifestation of retinoblastoma, often associated with a poorer prognosis.
- Intravitreal chemotherapy is an emerging treatment modality for localized retinoblastoma, aiming to reduce systemic toxicity.
Purpose of the Study:
- To assess the efficacy and safety of intravitreal melphalan injections for treating vitreous seeds in Chinese patients with retinoblastoma.
- To evaluate the local control rate and globe salvage achieved with this treatment approach.
Main Methods:
- A retrospective review was conducted on 17 consecutive Chinese patients (19 eyes) diagnosed with retinoblastoma and viable vitreous seeds.
- Patients received multiple intravitreal injections of 20 µg melphalan at 2-4 week intervals.
- Data on treatment response, globe retention, complications, and follow-up duration were collected and analyzed.
Main Results:
- Successful control of vitreous seeds was achieved in 16 out of 19 eyes (84.21%).
- Globe retention was successful in 14 out of 19 eyes (73.68%).
- A significant difference in treatment response was observed based on seed morphology (cloud, spheres, dust), with varying numbers of injections required (P = 0.003).
- Common complications included vitreous hemorrhage, cataract, and retinopathy; however, no cases of extraocular extension or systemic metastasis were reported.
Conclusions:
- Intravitreal melphalan is a highly effective treatment for controlling retinoblastoma vitreous seeds in Chinese patients.
- The procedure demonstrates a high rate of local tumor control and globe salvage with acceptable toxicity.
- This approach offers a promising alternative to enucleation or systemic chemotherapy for managing vitreous seeding in retinoblastoma.

