Intravitreal melphalan for refractory or recurrent vitreous seeding from retinoblastoma

Arch Ophthalmol. 2012 Oct;130(10):1268-71. doi: 10.1001/archophthalmol.2012.1983.


Objective: To evaluate the efficacy and complications of intravitreal chemotherapy for viable vitreous seeding from retinoblastoma.

Methods: Intravitreal injection of melphalan (8-50 μg in 0.05 mL) followed by injection site cryotherapy.

Results: Among 12 treated cases, success with control of vitreous seeds was achieved in 10 of 12 cases at immediate follow-up (0-3 months), 8 of 10 cases at short-term follow-up (3-6 months), and 6 of 10 cases at long-term (>6 months) follow-up. Among those 8 cases that received an 8- to 10-μg dose, control was achieved in 6 of 8 cases at immediate follow-up, 5 of 7 cases at short-term follow-up, and 3 of 7 cases at long-term follow-up. Complications with the 8- to 10-μg dose were minor and included preretinal hemorrhage and retinal vasculitis with retinal pigment epithelial alterations. Of those 4 that received a 50-μg dose, immediate, short-term, and long-term control was 100%, but complications of cataract, vitreous hemorrhage, subretinal hemorrhage, severe hypotonia, and phthisis lead to enucleation in 2 cases. There was no case of orbital tumor recurrence or retinoblastoma metastasis (follow-up range, 8-66 months).

Conclusions: Intravitreal melphalan for recurrent vitreous seeds from retinoblastoma appears to provide vitreous seed control in some patients. A high dose (50 μg) of melphalan is toxic and should be avoided.

MeSH terms

  • Antineoplastic Agents, Alkylating / administration & dosage*
  • Antineoplastic Agents, Alkylating / adverse effects
  • Child
  • Child, Preschool
  • Follow-Up Studies
  • Humans
  • Infant
  • Intravitreal Injections
  • Melphalan / administration & dosage*
  • Melphalan / adverse effects
  • Neoplasm Recurrence, Local / drug therapy*
  • Neoplasm Recurrence, Local / pathology
  • Neoplasm Seeding*
  • Radiotherapy, Adjuvant
  • Retinal Neoplasms / drug therapy*
  • Retinal Neoplasms / pathology
  • Retinoblastoma / drug therapy*
  • Retinoblastoma / secondary
  • Treatment Outcome
  • Vitreous Body / pathology*


  • Antineoplastic Agents, Alkylating
  • Melphalan