Eye-preserving therapies may do more harm than good in advanced retinoblastoma

In the treatment of advanced retinoblastoma patients with bilateral disease, initial use of eye-preserving therapies appears to be associated with a worse overall survival, especially for those with very large tumours, according to a study.

Researchers looked at 1,678 retinoblastoma patients diagnosed with group D (large or poorly defined tumours with widespread vitreous or subretinal seeding) or group E (very large tumour extending near the front of the eye, with bleeding or causing glaucoma) disease. They classified patients according to the initial treatment they received (primary enucleation and eye-preserving groups) and followed up for survival status.

Over a median follow-up of 43.9 months, 196 patients (12 percent) died. The resulting 5-year overall survival was 86 percent, and the eyeball preservation rate was 48 percent. In the study population, 1,172 (70 percent) had unilateral retinoblastoma while 506 (30 percent) had bilateral disease.

Among patients with unilateral disease, 570 eyes (49 percent) underwent primary enucleation and 602 patients (51 percent) received eye-preserving therapies initially. Over a median follow-up of 45.6 months, 59 patients (10 percent) in the primary enucleation group and 56 (9.3 percent) in the eye-preserving group died.

Multivariate Cox analyses showed no significant difference in overall survival between the two treatment groups for patients with unilateral disease (hazard ratio [HR], 1.25, 95 percent confidence interval [CI], 0.85–1.84; p=0.250).

On the other hand, among patients with bilateral disease, 95 eyes (19 percent) underwent primary enucleation and 411 patients (81 percent) received eye-preserving therapies initially. Over a median follow-up of 40.1 months, 12 patients (13 percent) from the primary enucleation group and 69 patients (17 percent) from the eye-preserving group died.

For bilateral retinoblastoma with the worse eye classified as group E, primary enucleation was associated with better overall survival compared with eye-preserving therapies (HR, 2.35, 95 percent CI, 1.10–5.01; p=0.027). However, this survival advantage only became evident at 22.6 months after initial diagnosis.

The findings highlight the importance of carefully weighing the choice of primary treatment for patients with advanced retinoblastoma.