ABSTRACT
PURPOSE: To evaluate the diagnostic and prognostic values of survivin and transforming growth factor beta-1 (TGF-B1) expression in aqueous humor and serum of retinoblastoma (RB) in comparison to the conventional RB marker lactate dehydrogenase (LDH) and to elucidate a possible correlation between them and the clinicopathological features of the disease. METHODS: This prospective, comparative study included 88 newly diagnosed children with RB and 80 age-matched controls with ophthalmic conditions other than tumors prepared for intraocular surgeries. Concentrations of survivin, TGF-B1, and LDH were measured in serum and aqueous humor before and 6 months after completion of therapy. RESULTS: High serum and aqueous humor concentrations of the three proteins were detected in RB patients before treatment compared to the control group (P < 0.01), with a significant reduction of serum concentrations after treatment (P < 0.01). For the highest sensitivity and specificity, the optimal cutoff values of serum and aqueous survivin were 12.9 pg/ml and 25.2 pg/mg, with a significant positive correlation between aqueous survivin and RB staging and presence of optic nerve infiltration (r = 0.43, P = 0.04); the best cutoff values of serum and aqueous TGF-B1, 370.7 pg/ml and 39.8 pg/mg, with a significant positive correlation between aqueous TGF-B1 and poor differentiation of the tumor (r = 0.69, P = 0.001). CONCLUSIONS: The high sensitivity, specificity, and accuracy of serum and aqueous humor survivin and TGF-B1 proteins make them promising markers for early detection and follow-up of RB patients.