Survival and ocular preservation in a long-term cohort of Japanese patients with retinoblastoma

Abstract

Background: Retinoblastoma is an ocular tumor in infants with cancer predisposition. Treatment of the rare tumor needs to be optimized for ocular preserved survival without second primary malignancy (SPM). Methods: We studied the outcomes of all patients with retinoblastoma at a tertiary center in 1984-2016, when preservation method changed from radiotherapy (1984-2001) to systemic chemotherapy (2002-2016). Results: One-hundred sixteen infants developed unilateral-(n = 77), bilateral-(n = 38), or trilateral-onset (n = 1) tumor. Ten (8.6%) had a positive family history, despite a few studies on RB1 gene. Contralateral disease occurred in one unilateral-onset case. One-hundred eight of 155 eyes (70%) were enucleated. Nine binocular survivors were from 5 bilateral-and 4 unilateral-onset cases. Two survivors received bilateral enucleation. Six deaths occurred; brain involvement (including 3 trilateral diseases) in 4 bilateral-onset, systemic invasion in a unilateral-onset, and SPM (osteosarcoma) in a bilateral-onset case(s). Two others survived SPM of osteosarcoma or lymphoma. The 10-year overall survival (OS: 98.5% vs. 91.3%, p = 0.068) and binocular survivors (13.2% vs. 5.2%, p = 0.154) between bilateraland unilateral-onsets did not differ statistically. The 10-year OS and cancer (retinoblastoma/SPM)-free survival (CFS) rates of all patients were 94.9 and 88.5%, respectively. The proportion of preserved eyes did not differ between radiotherapy and chemotherapy eras. The CFS rate of bilateral-onset cases in systemic chemotherapy era was higher than that in radiotherapy era (p = 0.042). The CFS rates of bilateral-onset patients with neoadjuvant chemotherapy (upfront systemic therapy for preservation) was higher than those without it (p = 0.030). Conclusions: Systemic chemotherapy and local therapy raised OS and binocular survival rates of bilateral-onset patients similarly to those of unilateral-onset patients. All but one death was associated with a probable germline defect of the RB1 gene. Neoadjuvant stratified chemotherapy may support the long-term binocular life with minimized risk of SPM.

Health related quality of life of patients treated with bilateral enucleation for retinoblastoma

“…However, they had higher risk of developing a second primary neoplasm. 14 Thus long term follow up, proper counseling of caregivers and rehabilitation are crucial for these disabled individuals. The outcome of the present study will help policy makers in Saudi Arabia to address these issues.…”

Subsequent Malignant Neoplasms in Retinoblastoma Survivors

“…The group described is small but the reason for the significant difference cannot be explained so far. Anyhow, no significant differences were seen between these above described groups for overall 10-year survival [ 68 ].…”

The Global Retinoblastoma Outcome Study: a prospective, cluster-based analysis of 4064 patients from 149 countries

“…In a single-centre study done in the USA (1994–2014), 4 the retinoblastoma survival rate was reported to reach 99% (mean follow-up 4–8 years, depending on age group); in a single-centre study done in the UK (2002–14), 5 it was reported to reach 100% (mean follow-up 5 years); and in a single-centre study done in Japan (1984–2016), 6 it was reported to reach 95% (10-year overall survival), with most deaths occurring from trilateral retinoblastoma in all three studies. In low-income countries, survival rates are significantly lower: 60% (5-year survival) in a single-centre study done in Uganda (2009–19), 7 53% (follow-up time not indicated) in a single-centre study done in Senegal (2006–10), 8 and 24% (10-year survival) in a single-centre study done in Nepal (1998–2008), 9 with the majority of deaths in all three studies occurring from metastatic spread.…”