Selective Ophthalmic Artery Chemotherapy with Melphalan in the Management of Unilateral Retinoblastoma: A Prospective Study.

Abstract

PURPOSE:

To determine prospectively the efficacy and to assess potential side effects of melphalan selective ophthalmic artery chemotherapy (SOAC) as first-line treatment for unilateral retinoblastoma.

DESIGN:

Phase 2 nonrandomized, prospective study.

PARTICIPANTS:

Patients with unilateral retinoblastoma group B, C, or D of the International Classification for Intraocular Retinoblastoma (IRC). Group D eyes with massive vitreous seeding were not eligible.

METHODS:

Melphalan SOAC associated with diode laser thermotherapy, cryotherapy, or both at 4-week intervals (3-6 cycles). For persistent vitreous seeding, intravitreal melphalan chemotherapy also was used.

MAIN OUTCOME MEASURES:

The primary outcome was globe preservation rate. Secondary outcomes were tumor relapse rate, occurrence of ocular or systemic adverse events, and measurement of the dose area product (DAP).

RESULTS:

Between 2012 and 2017, 39 patients (39 eyes) with unilateral retinoblastoma were included prospectively. Three included patients did not receive SOAC (2 catheterization failures and 1 case of viral syndrome) and were considered failures. At diagnosis, IRC groups for the 36 treated patients were: B, n = 4 (11%); C, n = 13 (36%); and D, n = 19 (53%); median age was 21.5 months (range, 3.2-61.6 months). Median number of SOAC cycles was 3.9 (range, 1-6 cycles), and median melphalan dose was 4.9 mg/procedure. The median DAP was 1.24 Gy.cm2/procedure. Median follow-up was 63 months (range, 34-93 months). SOAC was associated with local treatments for 31 patients (86%): diode laser thermotherapy for all of them and cryotherapy or intravitreal chemotherapy for 10 (32%) and 9 patients (25%), respectively. SOAC treatment was interrupted in 5 patients because of severe ophthalmic (ptosis, n = 2; retinal ischemia, n = 2) or systemic (hypotension, n = 1) adverse events. At the cutoff date analysis, all patients were alive without metastasis. The 18-month eye preservation rate was 80% (range, 68.6%-94.6%). After a follow-up of at least 30 months, the ocular preservation rate was 69% (n = 24 preservations).

CONCLUSIONS:

This first prospective trial demonstrated that SOAC with melphalan alone as first-line treatment for retinoblastoma is efficient and well tolerated with no metastatic events, although ocular ischemic complications were observed.

• Authors: Livia Lumbroso-Le Rouic, Raphaël Blanc, Caroline Saint Martin, Alexia Savignoni, Hervé J Brisse, Noëlle Pierrat, Christine Lévy-Gabriel, Alexandre Matet, François Doz, Isabelle Aerts, Nathalie Cassoux
• Journal: Ophthalmology. Retina (Ophthalmol Retina) Vol. 5 Issue 8 Pg. e30-e37 (08 2021) ISSN: 2468-6530 [Electronic] United States
• PMID: 34000459 (Publication Type: Clinical Trial, Clinical Trial, Phase II, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2021 American Academy of Ophthalmology. Published by Elsevier Inc. All rights reserved.

Chemical References

• Antineoplastic Agents, Alkylating
• Melphalan

Topics

• Antineoplastic Agents, Alkylating (administration & dosage)
• Child
• Child, Preschool
• Combined Modality Therapy
• Cryotherapy (methods)
• Disease Management
• Drug Administration Schedule
• Female
• Follow-Up Studies
• Humans
• Injections, Intra-Arterial
• Magnetic Resonance Imaging
• Male
• Melphalan (administration & dosage)
• Neoplasm Staging (methods)
• Ophthalmic Artery
• Prospective Studies
• Retinal Neoplasms (diagnosis, therapy)
• Retinoblastoma (diagnosis, therapy)
• Time Factors
• Treatment Outcome