Update on intra-arterial chemotherapy for retinoblastoma.

The tools for managing retinoblastoma have been increasing in the past decade. While globe-salvage still relies heavily on intravenous chemotherapy, tumors in advanced stage that failed chemotherapy are now referred for intra-arterial chemotherapy (IAC) to avoid enucleation. However, IAC still has many obstacles to overcome. We present an update on the indications, complications, limitations, success, and technical aspects of IAC. Given its safety and high efficacy, it is expected that IAC will replace conventional strategies and will become a first-line option even for tumors that are amenable for other strategies.

Direct medical costs of globe salvage in group C-E retinoblastoma and implications for cost-effectiveness.

PURPOSE: To determine the direct medical costs and cost-effectiveness of globe salvage compared with primary enucleation in patients with advanced retinoblastoma. METHODS: Patients with International Classification of Retinoblastoma groups C-E retinoblastoma at a single institution from 2001 to 2021 were categorized into two groups: primary enucleation and globe salvage. Hospital visits and treatments were compared to determine differences in care utilization. Costs per encounter were derived from billing data and multiplied by number of encounters to determine total costs in the first year of treatment. Groups were stratified by unilateral or bilateral retinoblastoma, and visual acuity-based utility values were used to compare quality-adjusted life years to determine cost-effectiveness. RESULTS: Of 74 group C-E eyes of 70 patients, 55 eyes underwent primary enucleation and 19 eyes underwent globe salvage, 12 of which ultimately underwent secondary enucleation. Patients who underwent globe salvage had higher usage of systemic chemotherapy (P = 0.001), more examinations under anesthesia (P < 0.001), fewer outpatient visits (P = 0.03), and a higher total cost of care (301,151 vs 104,764 USD [P < 0.001]) in the first year of treatment compared with the primary enucleation group. At an average life expectancy of 76 years, the incremental cost-effectiveness ratio was 118,347 USD per QALY in unilateral retinoblastoma and 32,987 USD per QALY in bilateral retinoblastoma, meeting the <150,000 USD/QALY threshold of cost-effectiveness. CONCLUSIONS: Incurring additional costs may save the eye in advanced retinoblastoma, but the possibility of secondary enucleation should be disclosed for informed decision making. Despite this risk, globe salvage was cost-effective in unilateral and bilateral retinoblastoma.

Direct fixation of extraocular muscles to a silicone sphere: a cost-sensitive, low-risk enucleation procedure.

PURPOSE: To describe a simple, cost-sensitive enucleation technique and to evaluate it in the context of intended benefits and long-term outcomes of alternate methods. METHODS: A retrospective record review of patients who underwent enucleation using the described technique at a single institution by 2 surgeons. Surgery involved direct fixation of extraocular muscles to a solid silicone sphere, using nonabsorbable braided sutures with knots tied beneath the muscle insertions. Outcome measures included implant-related complications. RESULTS: Seventy-five patients meeting inclusion criteria ranged in age from 3 to 94 years (mean, 54 years; median, 56 years). Surgical indications included a blind painful or disrupted eye in 56 cases; uveal melanoma in 15 cases; retinoblastoma in 2 cases; and endophthalmitis in 2 cases. Follow-up intervals ranged from 3 to 57 months (mean, 27 months; median, 22 months). Complications requiring surgical revision of the implant occurred in 2 of the 75 cases (one luxation; one exposure). There were no instances of chronic socket discharge or implant infection. CONCLUSIONS: Nonabsorbable-suture attachment of muscles to a solid silicone implant offers an inexpensive enucleation option, with minimal risk of implant migration, exposure, or infection. In light of widespread disaffection with pegging of porous implants, and with no motility advantage of unpegged porous over nonporous implants, consideration should be given to techniques that are equally effective, less costly, and perhaps more reliable.

Cost-effectiveness of proton beam therapy for intraocular melanoma.

PURPOSE: Proton beam therapy is a commonly accepted treatment for intraocular melanomas, but the literature is lacking in descriptions of patient preferences of clinical outcomes and economic impact. In addition, no economic evaluations have been published regarding the incremental cost-effectiveness of proton beam therapy compared with enucleation or plaque brachytherapy, typical alternative treatments. We, therefore, conducted a cost-utility analysis of these three approaches for the treatment of intraocular melanomas. MATERIALS AND METHODS: A Markov model was constructed. Model parameters were identified from the published literature and publicly available data sources. Cost-effectiveness of each treatment was calculated in 2011 US Dollars per quality-adjusted life-year. Incremental cost-effectiveness ratios were calculated assuming enucleation as reference. One-way sensitivity analyses were conducted on all model parameters. A decision threshold of $50,000/quality-adjusted life-year was used to determine cost-effectiveness. RESULTS: Enucleation had the lowest costs and quality-adjusted life-years, and plaque brachytherapy had the highest costs and quality-adjusted life-years. Compared with enucleation, the base-case incremental cost-effectiveness ratios for plaque brachytherapy and proton beam therapy were $77,500/quality-adjusted life-year and $106,100/quality-adjusted life-year, respectively. Results were highly sensitive to multiple parameters. All three treatments were considered optimal, and even dominant, depending on the values used for sensitive parameters. CONCLUSION: Base-case analysis results suggest enucleation to be optimal. However, the optimal choice was not robust to sensitivity analyses and, depending on the assumption, both plaque brachytherapy and proton beam therapy could be considered cost-effective. Future clinical studies should focus on generating further evidence with the greatest parameter uncertainty to inform future cost-effectiveness analyses.

Socioeconomic impact of modern multidisciplinary management of retinoblastoma.

OBJECTIVE: Our objective for this study was to examine the impact of the modern management of intraocular retinoblastoma on the patient and the family. METHODS: This study comprises a retrospective, noncomparative case series of 25 consecutive patients with multifocal, intraocular retinoblastoma that was treated with primary systemic chemotherapy. Medical charts were reviewed, and the following data were extracted: patients' age and gender, laterality of disease, and Reese-Ellsworth classification of each eye as well as the number of central venous lines placed, cycles of chemotherapy received, outpatient appointments, examinations under anesthesia, focal therapies administered, computed tomography/MRI, radiation treatments, anesthetic procedures administered, and miles traveled. RESULTS: Twenty patients with bilateral and 5 with unilateral intraocular retinoblastoma (median age: 9.3 months) had 895 outpatient appointments and underwent 698 examinations under anesthesia with 230 focal therapies, 347 days of radiotherapy, 226 computed tomography scans/MRIs, and 38 central venous line placements. A total of 1272 anesthetic procedures (median: 50) were performed with no major complication. In all, patients traveled 822312 miles (median: 22214 miles) to receive their care. The median follow-up was 82 months. CONCLUSIONS: Successful retinoblastoma management requires close surveillance, aggressive consolidation, and numerous anesthetic procedures, all of which the patients and the families must endure. There is a significant impact on the patient, the family, and hospital resources.