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.

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.

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