Treatment of retinoblastoma has evolved dramatically in the last 100 years: from enucleation as the only option, to attempts at globe salvage with primary radiotherapy in the 1940s, to the modern chemotherapy era. Intravenous chemotherapy was introduced for this purpose in the 1990s, intra-arterial chemotherapy in the 2000s, and intravitreal chemotherapy in the 2010s.
The survival rate for retinoblastoma exceeds 95% when the disease is contained within the eye. In cases with extraocular spread, the survival rate is substantially lower. When determining the treatment strategy, the clinician’s first goal must be preservation of life, then preservation of the eye, and finally preservation of vision. Modern management of intraocular retinoblastoma involves a combination of treatment modalities, including enucleation, local and systemic chemotherapy, laser therapy, cryotherapy, and plaque brachytherapy. EBRT is now rarely performed. Metastatic disease is managed with intensive combinations of chemotherapy, radiation, and bone marrow transplantation. Recent findings of COG trials have demonstrated improved efficacy for curing patients who have regional extraocular retinoblastoma or metastatic retinoblastoma that does not involve the CNS. Treating children with retinoblastoma requires a team consisting of an ocular oncologist, a pediatric ophthalmologist, a pediatric oncologist, an interventional radiologist, and a radiation oncologist. For many of the therapeutic modalities, anesthesia is required in these young patients. Because new tumors may develop in an eye, particularly in patients with a germline mutation, serial EUAs are required for surveillance.
Table 19-3 Comparison of Clinical Grouping Systems for Retinoblastoma
Enucleation remains a definitive treatment for retinoblastoma, enabling complete surgical resection of the disease in most cases. Enucleation often is curative in patients with unilateral disease and is considered an appropriate intervention in the following situations:
Optic nerve involvement is suspected, and the surgeon can enucleate with a clear optic nerve margin (ie, complete tumor resection).
Anterior segment involvement is present.
Neovascular glaucoma is present.
The eye has been otherwise anatomically or functionally destroyed by tumor.
The affected eye has limited visual potential.
The tumor persists or recurs despite attempts at globe salvage.
For eyes classified as group E in the IIRC system, enucleation is the typical treatment.
The goal of the tumor enucleation procedure is to minimize the potential for inadvertent globe penetration while obtaining the greatest possible length of resected optic nerve, typically at least 10 mm. Most surgeons place an implant postoperatively. Implants may be composed of nonporous silicone or an integrated porous material, such as hydroxyapatite or porous polyethylene.
Attempts at globe-conserving therapy should be undertaken only by ophthalmologists well versed in the management of this rare childhood tumor and in conjunction with similarly experienced pediatric oncologists. Failed attempts at eye salvage may place a child at risk of metastatic disease.
Excerpted from BCSC 2020-2021 series: Section 4 - Ophthalmic Pathology and Intraocular Tumors. For more information and to purchase the entire series, please visit https://www.aao.org/bcsc.