Long-Term Data Show Value Of Intravenous Chemotherapy In Retinoblastoma

Intravenous chemotherapy (IVC), with intra-arterial chemotherapy (IAC) or plaque radiotherapy as needed for globe salvage, can control retinoblastoma without the need for enucleation or external beam radiotherapy (EBRT), 20-year real-world outcomes show.

"Basically we found, in our experience, that the management of retinoblastoma is complex and requires experience with this condition and knowledge of when to intervene with additional therapies," Dr. Carol L. Shields of Wills Eye Hospital in Philadelphia told Reuters Health by email. "Using 6 cycles of intravenous chemotherapy (plus additional therapies) we were able to achieve complete tumor control by 2 years, and the control was lasting at the 20-year point."

Dr. Shields and her colleagues started their IVC program in 1994 and published their first observations two years later. In the new study, published in the British Journal of Ophthalmology, they report outcomes for 964 eyes of 554 patients treated at their center.

They compared outcomes based on International Classification of Retinoblastoma group. Group A included 54 eyes of 51 patients; Group B, 200 eyes of 167 patients; Group C, 128 eyes of 119 patients, Group D, 234 eyes of 203 patients and Group E, 225 eyes of 196 patients. The group was unknown for an additional 123 eyes of 71 patients.

Patients with more-advanced disease were older at presentation, less likely to be white and more likely to have sporadic retinoblastoma. They were also more likely to have recurrence or non-response requiring IAC, plaque radiotherapy, EBRT, or enucleation.

At 2 years, tumor control (globe salvage without EBRT) was 96% for Group A; 91% for Groups B and C; 71% for group D; and 32% for group E.

The percentage of patients requiring additional IAC or plaque radiotherapy was 5% for Group A, 26% Group B, 28% for Group C, 27% for Group D and 19% for Group E, "with little further need up to 20 years," the researchers report. The risk of failure, with need for EBRT or enucleation, rose from year 1 to year 2, but then showed minimal change up to 20 years and beyond.

"We will be following this cohort for many more years with special interest on tumor control within the eye and a focus on long-term second cancers," Dr. Shields said.

Managing children with retinoblastoma is complex, she added, and requires "a skilled ocular oncologist, pediatric oncologist, interventional neuro-radiologist/neurosurgeon, radiation oncologist, pediatric ophthalmologist, and pediatrician—all working together."

—Anne Harding

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