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Survival and ocular preservation in a long-term cohort of Japanese patients with retinoblastoma.
- Source :
-
BMC pediatrics [BMC Pediatr] 2020 Jan 28; Vol. 20 (1), pp. 37. Date of Electronic Publication: 2020 Jan 28. - Publication Year :
- 2020
-
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).<br />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).<br />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 bilateral- and 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).<br />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.
- Subjects :
- Brachytherapy
Chemotherapy, Adjuvant
Child
Child, Preschool
Cohort Studies
Eye Enucleation
Female
Humans
Infant
Infant, Newborn
Japan
Kaplan-Meier Estimate
Male
Neoadjuvant Therapy
Retinal Neoplasms mortality
Retinal Neoplasms surgery
Retinoblastoma mortality
Retinoblastoma surgery
Retrospective Studies
Antineoplastic Combined Chemotherapy Protocols therapeutic use
Eye drug effects
Eye radiation effects
Radiotherapy
Subjects
Details
- Language :
- English
- ISSN :
- 1471-2431
- Volume :
- 20
- Issue :
- 1
- Database :
- MEDLINE
- Journal :
- BMC pediatrics
- Publication Type :
- Academic Journal
- Accession number :
- 31992242
- Full Text :
- https://doi.org/10.1186/s12887-020-1923-7