Supplementary Material for: Long-Term Outcomes of Group B Eyes in Patients with Retinoblastoma Treated with Short-Course Chemoreduction: Experience from Children's Hospital Los Angeles/University of Southern California

<br><strong><em>Background/Aims:</em></strong> Chemoreduction protocols for retinoblastoma vary widely across institutions. Herein, we compare a 3- versus 6-cycle chemotherapy approach for group B retinoblastoma. <b><i>Methods:</i></b> A nonrandomized, retrospective review of patients diagnosed with group B retinoblastoma from 1991-2011 at Children's Hospital Los Angeles was performed. A total of 72 eyes of 63 patients were analyzed. Mean follow-up time was 82 months (range 6-272 months). Main outcome measures were globe salvage and need for external beam radiation. <b><i>Results:</i></b> Forty-six patients (55 eyes) were treated upfront with 3 cycles of carboplatin, etoposide, and vincristine; 17 patients (17 eyes) received 6 cycles. Thirty-seven eyes (67%) in the 3-cycle group were cured with initial chemoreduction alone. An additional 10 eyes with persistent or recurrent tumors were rescued with 3 more cycles for a total salvage rate of 85% (47/55 eyes). In the 6-cycle group, 16 of 17 eyes (94%) avoided radiation and enucleation. <b><i>Conclusion:</i></b> The initial recurrence rate was higher for the 3-cycle group (p = 0.03). However, eyes failing short-course chemoreduction were rescued with 3 additional cycles and achieved a similar overall event-free survival rate (p = 0.16). In our cohort, this short-course approach spared 63% (29/46) of patients with group B retinoblastoma the extra 3 cycles of systemic chemotherapy

Response criteria for intraocular retinoblastoma: RB-RECIST.

Standardized guidelines for assessing tumor response to therapy are essential for designing and conducting clinical trials. The Response Evaluation Criteria In Solid Tumors (RECIST) provide radiological standards for assessment of solid tumors. However, no such guidelines exist for the evaluation of intraocular cancer, and ocular oncology clinical trials have largely relied on indirect measures of therapeutic response-such as progression-free survival-to evaluate the efficacy of treatment agents. Herein, we propose specific criteria for evaluating treatment response of retinoblastoma, the most common pediatric intraocular cancer, and emphasize a multimodal imaging approach for comprehensive assessment of retinoblastoma tumors in clinical trials