Update on Retinoblastoma Therapies

Retinoblastoma is a success story in pediatric oncology, evolving from life-saving interventions to approaches that preserve eyes and vision while minimizing complications. Initially managed with enucleation and radiotherapy, treatment now emphasizes eye preservation through chemotherapy as the corn...

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Main Authors: Cristina A. Martínez Arce, Victor M. Villegas, Maura Di Nicola, Basil K. Williams, Timothy G. Murray
Format: Article
Language:English
Published: MDPI AG 2025-07-01
Series:Medicina
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Online Access:https://www.mdpi.com/1648-9144/61/7/1219
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author Cristina A. Martínez Arce
Victor M. Villegas
Maura Di Nicola
Basil K. Williams
Timothy G. Murray
author_facet Cristina A. Martínez Arce
Victor M. Villegas
Maura Di Nicola
Basil K. Williams
Timothy G. Murray
author_sort Cristina A. Martínez Arce
collection DOAJ
description Retinoblastoma is a success story in pediatric oncology, evolving from life-saving interventions to approaches that preserve eyes and vision while minimizing complications. Initially managed with enucleation and radiotherapy, treatment now emphasizes eye preservation through chemotherapy as the cornerstone therapy. Various chemotherapy delivery methods—including intravenous (IVC), intraarterial (IAC), intravitreal, intracameral, and periocular—offer flexibility in treatment. Studies show nearly 100% eye salvage rates for groups A–C. For advanced cases (groups D and E), IAC has achieved outcomes that were not possible before. Intravitreal injections, when performed safely, may help avoid enucleation and radiotherapy in advanced cases, preserving vision, even in complex scenarios, with vitreous seeding. Each strategy may be tailored to tumor and patient characteristics that may help optimize outcomes. Recent innovations like liquid biopsy, prenatal diagnosis, prognostic biomarkers, and new surgical methods, such as tylectomy and chemoplaque, are paving the way for more personalized care. While advanced extraocular or metastatic retinoblastoma remains challenging, these advancements underscore a shift towards better outcomes and individualized management. The future holds promise for refining treatment strategies to maximize eye and vision preservation while ensuring patient survival.
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spelling doaj-art-cb25f3df8e4e46f9927c2cfbee4a23bc2025-07-25T13:29:27ZengMDPI AGMedicina1010-660X1648-91442025-07-01617121910.3390/medicina61071219Update on Retinoblastoma TherapiesCristina A. Martínez Arce0Victor M. Villegas1Maura Di Nicola2Basil K. Williams3Timothy G. Murray4School of Medicine, Universidad Central del Caribe, Bayamón 00960, Puerto RicoDepartment of Ophthalmology, University of Puerto Rico, Medical Sciences Campus, San Juan 00936, Puerto RicoBascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL 33136, USABascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL 33136, USAMurray Ocular Oncology and Retina (MOOR), Miami, FL 33143, USARetinoblastoma is a success story in pediatric oncology, evolving from life-saving interventions to approaches that preserve eyes and vision while minimizing complications. Initially managed with enucleation and radiotherapy, treatment now emphasizes eye preservation through chemotherapy as the cornerstone therapy. Various chemotherapy delivery methods—including intravenous (IVC), intraarterial (IAC), intravitreal, intracameral, and periocular—offer flexibility in treatment. Studies show nearly 100% eye salvage rates for groups A–C. For advanced cases (groups D and E), IAC has achieved outcomes that were not possible before. Intravitreal injections, when performed safely, may help avoid enucleation and radiotherapy in advanced cases, preserving vision, even in complex scenarios, with vitreous seeding. Each strategy may be tailored to tumor and patient characteristics that may help optimize outcomes. Recent innovations like liquid biopsy, prenatal diagnosis, prognostic biomarkers, and new surgical methods, such as tylectomy and chemoplaque, are paving the way for more personalized care. While advanced extraocular or metastatic retinoblastoma remains challenging, these advancements underscore a shift towards better outcomes and individualized management. The future holds promise for refining treatment strategies to maximize eye and vision preservation while ensuring patient survival.https://www.mdpi.com/1648-9144/61/7/1219intra-arterial chemotherapyintravitreal injectionsintravenous chemotherapyintracameral chemotherapyperiocular chemotherapyenucleation
spellingShingle Cristina A. Martínez Arce
Victor M. Villegas
Maura Di Nicola
Basil K. Williams
Timothy G. Murray
Update on Retinoblastoma Therapies
Medicina
intra-arterial chemotherapy
intravitreal injections
intravenous chemotherapy
intracameral chemotherapy
periocular chemotherapy
enucleation
title Update on Retinoblastoma Therapies
title_full Update on Retinoblastoma Therapies
title_fullStr Update on Retinoblastoma Therapies
title_full_unstemmed Update on Retinoblastoma Therapies
title_short Update on Retinoblastoma Therapies
title_sort update on retinoblastoma therapies
topic intra-arterial chemotherapy
intravitreal injections
intravenous chemotherapy
intracameral chemotherapy
periocular chemotherapy
enucleation
url https://www.mdpi.com/1648-9144/61/7/1219
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AT victormvillegas updateonretinoblastomatherapies
AT mauradinicola updateonretinoblastomatherapies
AT basilkwilliams updateonretinoblastomatherapies
AT timothygmurray updateonretinoblastomatherapies