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Targeted retinoblastoma management: when to use intravenous, intra-arterial, periocular, and intravitreal chemotherapy

Shields, Carol L.a; Lally, Sara E.a; Leahey, Ann M.b; Jabbour, Pascal M.c; Caywood, Emi H.d; Schwendeman, Rachela; Shields, Jerry A.a

Current Opinion in Ophthalmology: September 2014 - Volume 25 - Issue 5 - p 374–385
doi: 10.1097/ICU.0000000000000091
OCULAR GENETICS: Edited by Alex V. Levin
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Purpose of review The management of retinoblastoma is complex and involves strategically chosen methods of enucleation, radiotherapy, chemotherapy, laser photocoagulation, thermotherapy, and cryotherapy. Chemotherapy has become the most common eye-sparing modality. There are four routes of delivery of chemotherapy for retinoblastoma, including intravenous, intra-arterial, periocular, and intravitreal techniques. The purpose of this review is to discuss the current rationale for each method and the anticipated outcomes.

Recent findings The diagnosis of retinoblastoma should be clinically established prior to embarking on a chemotherapy protocol. There are over 25 conditions that can closely simulate retinoblastoma in a young child. In addition, enucleation is an acceptable method for management, particularly with advanced retinoblastoma. Intravenous chemotherapy is generally used for germline mutation (bilateral, familial) retinoblastoma with excellent tumor control for groups A, B, and C and intermediate control for group D eyes. Intra-arterial chemotherapy is used as primary therapy in selected cases for nongermline mutation (unilateral) retinoblastoma with excellent control, and also used as secondary therapy for recurrent solid retinoblastoma, subretinal seeds, and vitreous seeds. Periocular chemotherapy is employed to boost local chemotherapy dose in advanced bilateral groups D and E eyes or for localized recurrences. Intravitreal chemotherapy is used for recurrent vitreous seeds from retinoblastoma. Patients at high risk for metastases should receive intravenous chemotherapy.

Summary Chemotherapy is effective for retinoblastoma and the targeted treatment route depends on the clinical features and anticipated outcomes.

aOcular Oncology Service, Wills Eye Hospital, Thomas Jefferson University, Philadelphia, PA, USA

bDivision of Oncology, Children's Hospital of Philadelphia, Philidelphia, PA, USA

cDepartment of Neurovascular and Endovascular Surgery, Department of Neurological Surgery, Thomas Jefferson University, Philadelphia, PA, USA

dNemours Center for Cancer and Blood Disorders (EHC), Nemours/Alfred I. DuPont Hospital for Children, Wilmington, DE, USA

Correspondence to Carol L. Shields, MD, Ocular Oncology Service, 840 Walnut Street, Suite 1440, Philadelphia, PA 19107, USA. Tel: +1 215 928 3105; fax: +1 215 928 1140; e-mail: carolshields@gmail.com

© 2014 Wolters Kluwer Health | Lippincott Williams & Wilkins