TY - JOUR
T1 - Systemic neoadjuvant chemotherapy for Group B intraocular retinoblastoma (ARET0331)
T2 - A report from the Children's Oncology Group
AU - Friedman, Debra L.
AU - Krailo, Mark
AU - Villaluna, Doojduen
AU - Gombos, Dan
AU - Langholz, Bryan
AU - Jubran, Rima
AU - Shields, Carol
AU - Murphree, Linn
AU - O'Brien, Joan
AU - Kessel, Sandra
AU - Rodriguez-Galindo, Carlos
AU - Chintagumpala, Murali
AU - Meadows, Anna T.
N1 - Publisher Copyright:
© 2016 Wiley Periodicals, Inc.
PY - 2017/7
Y1 - 2017/7
N2 - Purpose: To evaluate a chemoreduction regimen using systemic vincristine and carboplatin (VC) and local ophthalmic therapies to avoid external-beam radiotherapy (EBRT) or enucleation in patients with Group B intraocular retinoblastoma. Patients and methods: Twenty-one patients (25 eyes) were treated with six cycles of VC, accompanied by local ophthalmic therapies after cycle 1. The primary study objective was to determine the 2-year event-free survival (EFS) where an event was defined as the use of systemic chemotherapy in addition to vincristine or carboplatin, EBRT, and/or enucleation. Results: All patients had tumor regression after the first cycle of VC and only two patients had progression during therapy. There were seven treatment failures within 2 years of study enrollment, resulting in 2-year EFS of 65% and early study closure in accordance with the statistical design. The 2-year cumulative incidence of enucleation was 15%; for external beam radiation therapy, it was 10%; and for chemotherapy to control progressive disease, it was 10%. All patients sustaining a treatment failure were salvaged with additional therapy. Conclusions: For the majority of patients with Group B intraocular retinoblastoma, chemoreduction with VC, without etoposide, in conjunction with local therapy provides excellent opportunity for ocular salvage. Local therapy given with every chemotherapy cycle and incorporation of etoposide may provide improved ocular salvage rates. Central review of group at diagnosis is critical in assigning appropriate therapies.
AB - Purpose: To evaluate a chemoreduction regimen using systemic vincristine and carboplatin (VC) and local ophthalmic therapies to avoid external-beam radiotherapy (EBRT) or enucleation in patients with Group B intraocular retinoblastoma. Patients and methods: Twenty-one patients (25 eyes) were treated with six cycles of VC, accompanied by local ophthalmic therapies after cycle 1. The primary study objective was to determine the 2-year event-free survival (EFS) where an event was defined as the use of systemic chemotherapy in addition to vincristine or carboplatin, EBRT, and/or enucleation. Results: All patients had tumor regression after the first cycle of VC and only two patients had progression during therapy. There were seven treatment failures within 2 years of study enrollment, resulting in 2-year EFS of 65% and early study closure in accordance with the statistical design. The 2-year cumulative incidence of enucleation was 15%; for external beam radiation therapy, it was 10%; and for chemotherapy to control progressive disease, it was 10%. All patients sustaining a treatment failure were salvaged with additional therapy. Conclusions: For the majority of patients with Group B intraocular retinoblastoma, chemoreduction with VC, without etoposide, in conjunction with local therapy provides excellent opportunity for ocular salvage. Local therapy given with every chemotherapy cycle and incorporation of etoposide may provide improved ocular salvage rates. Central review of group at diagnosis is critical in assigning appropriate therapies.
KW - ARET0331
KW - Children's Oncology Group
KW - Group B intraocular retinoblastoma
KW - retinoblastoma
KW - systemic neoadjuvant chemotherapy
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U2 - 10.1002/pbc.26394
DO - 10.1002/pbc.26394
M3 - Article
C2 - 28019092
AN - SCOPUS:85019543845
SN - 1545-5009
VL - 64
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
IS - 7
M1 - e26394
ER -