Ophthalmology Commons^™

Outcomes Of Intravenous Chemotherapy (Chemoreduction) For Retinoblastoma Based On Patient Age In 964 Eyes Of 554 Patients., Zeynep Bas, Lauren A. Dalvin, Sameeksha Tadepalli, Raksha Rao, Amish Shah, Ann M. Leahey, Carol L. Shields

Wills Eye Hospital Papers

PURPOSE: To evaluate retinoblastoma control after intravenous chemotherapy (chemoreduction) by patient age at presentation.

DESIGN: Retrospective case series.

METHODS: This study included 964 eyes of 554 patients treated with chemoreduction at Ocular Oncology Service at Wills Eye Hospital. Patients received 6 monthly cycles of standard chemoreduction. Additional therapies for tumor control were performed as needed.

RESULTS: Of 964 eyes, a comparison by age group (24 months) revealed more advanced age group with higher frequency of group E tumor (15% vs. 25% vs. 32% vs. 39%, P < 0.001). By treatment outcomes, complete tumor control was achieved with chemoreduction alone more often in less advanced age group (46% vs. 30% vs. 17% vs. 8%, P < 0.001). Additional treatment after chemoreduction was needed more often in more advanced age group with external beam radiotherapy (EBRT; 9% vs. 16% vs. 20% vs. 15%, P = 0.006) or enucleation (12% vs. 18% vs. 26% vs. 37%, P < 0.001). Over time (1994-1998 vs. 1999-2003 vs. 2004-2008 vs. 2009-2013 vs. 2014-2019), the paradigm for additional required treatment after chemoreduction shifted toward less EBRT (27% vs. 24% vs. 14% vs. 7% vs. 2%, P < 0.001) and more intra-arterial (0% vs. 0% vs. 1% vs. 25% vs. 48%, P < 0.001) and intravitreal (0% vs. 0% vs. 3% vs. 10% vs. 20%, P < 0.001) chemotherapy.

CONCLUSIONS: Chemoreduction is a safe and effective treatment method for patients with retinoblastoma, demonstrating …

Safety And Improved Efficacy Signals Following Gene Therapy In Childhood Blindness Caused By Gucy2d Mutations, Samuel G. Jacobson, Artur V. Cideciyan, Allen Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Roman, Alexander Sumaroka, Vivian Wu, Arun Krishnan, Rebecca Sheplock, Sanford Boye, Alexander Dizhoor, Shannon Boye

Wills Eye Hospital Papers

A first-in-human clinical trial of gene therapy in Leber congenital amaurosis due to mutations in the GUCY2D gene is underway, and early results are summarized. A recombinant adeno-associated virus serotype 5 (rAAV5) vector carrying the human GUCY2D gene was delivered by subretinal injection to one eye in three adult patients with severe visual loss, nystagmus, but preserved retinal structure. Safety and efficacy parameters were monitored for 9 months post-operatively. No systemic toxicity was detected; there were no serious adverse events, and ocular adverse events resolved. P1 and P2 showed statistically significant rod photoreceptor vision improvement by full-field stimulus testing in …

A Career Of Honor And Humility: Interview With A Pioneer In Ophthalmology, Eli Cehelyk

inSIGHT

Imagine being a first-time parent in your early 30s, and you have just learned that your three-month old infant has retinoblastoma. Thanksgiving is right around the corner and you have no idea what the future holds. How would you feel? What thoughts would be racing through your head? With both hope and anxiety, you make an appointment at Wills Eye to see Dr. Carol Shields, a world-renowned expert on ocular oncology. With confidence and compassion, she walks into the room with her entire team and says, “Don’t worry, we will get everything under control for you. We will take your …