Selective ophthalmic artery chemotherapy with melphalan shows promise for retinoblastoma

Phase 2 results show that selective ophthalmic artery chemotherapy with melphalan can be used as a first-line therapy for retinoblastoma.

Study design

This nonrandomized, prospective study included 36 patients with unilateral group B, C, or D retinoblastoma, categorized based on the International Classification for Intraocular Retinoblastoma. Patients received treatment every 4 weeks along with local treatments such as diode laser thermotherapy, cryotherapy, or both. The median number of treatment cycles was 4 (range 1–6). Follow-up ranged from 34 to 93 months.

Outcomes

At diagnosis, there were 4 eyes in group B, 13 eyes in group C, and 19 eyes in group D. The eye preservation rate was 80% at 18 months and 69% after at least 30 months of follow-up. None of the patients had metastases. Four patients experienced severe ophthalmic side effects: 2 had retinal ischemia; 2 had ptosis; 1 experienced hypotension.

Limitations

Because this is a prospective study, the patient treatment was uniform. Although intravitreal chemotherapy was administered to study participants after it was available 5 months after the protocol started, the authors note that none of the patients previously required intravitreal therapy.

Clinical significance

This is the first prospective study of intra-arterial chemotherapy for retinoblastoma to demonstrate good efficacy in eyes in group B to D with unilateral disease.