TY - JOUR
T1 - A single-arm study of systemic and sub-Tenon chemotherapy for Groups C and D intraocular retinoblastoma
T2 - A Children's Oncology Group study (ARET 0231)
AU - Jubran, Rima F.
AU - Villablanca, Judith G.
AU - Krailo, Mark
AU - Piao, Jin
AU - Huang, Li
AU - Murphree, A. Linn
AU - O'Brien, Joan
AU - Gombos, Dan
AU - Shields, Carol L.
AU - Meadows, Anna
AU - Chintagumpala, Murali
N1 - Funding Information:
This study is supported by the NCTN Operations Center Grant U10CA180886 and NCTN Statistics and Data Center Grant U10CA180899 of the Children's Oncology Group from the National Cancer Institute, National Institutes of Health, Bethesda, MD, and St Baldrick's Foundation, Monrovia, CA. The content is solely the responsibility of the authors and does not necessarily represent the views of the National Institutes of Health.
Funding Information:
This study is supported by the NCTN Operations Center Grant U10CA180886 and NCTN Statistics and Data Center Grant U10CA180899 of the Children's Oncology Group from the National Cancer Institute, National Institutes of Health, Bethesda, MD, and St Baldrick's Foundation, Monrovia, CA. The content is solely the responsibility of the authors and does not necessarily represent the views of the National Institutes of Health.
Publisher Copyright:
© 2020 Wiley Periodicals, Inc.
PY - 2020/9/1
Y1 - 2020/9/1
N2 - Background: Eyes with Group D intraocular retinoblastoma have low salvage rates. A pilot study showed safety and efficacy of sub-Tenon's fascia carboplatin with systemic chemotherapy supporting further study. Methods: Children with newly diagnosed bilateral intraocular retinoblastoma with at least one remaining Group C or D eye were treated with six courses of carboplatin/etoposide/vincristine (CEV) with sub-Tenon's fascia carboplatin for Group C/D eyes during courses 2-4. Local ophthalmic therapy started at course 3. The primary study objective was to determine the 1-year failure rate of Group D eyes. Results: The study closed prematurely due to poor accrual and 22 of 30 patients were evaluable for failure rate, contributing 25 Group D and four Group C eyes. Among the 25 Group D eyes, there were 13 failures within the first year of study enrollment including eight needing external beam radiotherapy (EBR) and five needing enucleation, resulting in 1-year failure rate of 52%. The failure rate was significantly lower than the historical rate of 70% (P =.039). The 1-year eye preservation rate for Group D eyes was 80% (20/25). One-year failure rate for Group C eyes was 25% (1/4); 1-year preservation rate was 100% without need for EBR. Systemic toxicity included Grade 3 hearing loss in two subjects, infections, neutropenia, and thrombocytopenia. Ocular toxicities included periorbital fat atrophy (13/29 = 45% eyes), optic nerve atrophy (1/29 = 3% eyes), and restrictive fibrosis (1/29 = 3% eyes). Conclusions: Sub-Tenon's fascia carboplatin plus CEV was partially effective in Group D intraocular retinoblastoma but had unacceptable ocular toxicities.
AB - Background: Eyes with Group D intraocular retinoblastoma have low salvage rates. A pilot study showed safety and efficacy of sub-Tenon's fascia carboplatin with systemic chemotherapy supporting further study. Methods: Children with newly diagnosed bilateral intraocular retinoblastoma with at least one remaining Group C or D eye were treated with six courses of carboplatin/etoposide/vincristine (CEV) with sub-Tenon's fascia carboplatin for Group C/D eyes during courses 2-4. Local ophthalmic therapy started at course 3. The primary study objective was to determine the 1-year failure rate of Group D eyes. Results: The study closed prematurely due to poor accrual and 22 of 30 patients were evaluable for failure rate, contributing 25 Group D and four Group C eyes. Among the 25 Group D eyes, there were 13 failures within the first year of study enrollment including eight needing external beam radiotherapy (EBR) and five needing enucleation, resulting in 1-year failure rate of 52%. The failure rate was significantly lower than the historical rate of 70% (P =.039). The 1-year eye preservation rate for Group D eyes was 80% (20/25). One-year failure rate for Group C eyes was 25% (1/4); 1-year preservation rate was 100% without need for EBR. Systemic toxicity included Grade 3 hearing loss in two subjects, infections, neutropenia, and thrombocytopenia. Ocular toxicities included periorbital fat atrophy (13/29 = 45% eyes), optic nerve atrophy (1/29 = 3% eyes), and restrictive fibrosis (1/29 = 3% eyes). Conclusions: Sub-Tenon's fascia carboplatin plus CEV was partially effective in Group D intraocular retinoblastoma but had unacceptable ocular toxicities.
KW - clinical trial
KW - retinoblastoma
KW - sub-Tenon chemotherapy
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U2 - 10.1002/pbc.28502
DO - 10.1002/pbc.28502
M3 - Article
C2 - 32589362
AN - SCOPUS:85087143854
VL - 67
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
SN - 1545-5009
IS - 9
M1 - e28502
ER -