Friedman Debra L, Krailo Mark, Villaluna Doojduen, Gombos Dan, Langholz Bryan, Jubran Rima, Shields Carol, Murphree Linn, O'Brien Joan, Kessel Sandra, Rodriguez-Galindo Carlos, Chintagumpala Murali, Meadows Anna T
Division of Pediatric Hematology/Oncology, Vanderbilt University School of Medicine, Nashville, Tennessee.
Vanderbilt-Ingram Cancer Center, Nashville, Tennessee.
Pediatr Blood Cancer. 2017 Jul;64(7). doi: 10.1002/pbc.26394. Epub 2016 Dec 26.
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.
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.
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.
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.
评估一种化疗方案，该方案使用全身应用长春新碱和卡铂（VC）以及局部眼科治疗，以避免B组眼内视网膜母细胞瘤患者接受外照射放疗（EBRT）或眼球摘除术。
21例患者（25只眼）接受了6个周期的VC治疗，并在第1周期后辅以局部眼科治疗。主要研究目标是确定2年无事件生存率（EFS），其中事件定义为除长春新碱或卡铂之外使用全身化疗、EBRT和/或眼球摘除术。
所有患者在第1周期VC治疗后肿瘤均有消退，仅2例患者在治疗期间病情进展。在研究入组的2年内有7例治疗失败，导致2年EFS为65%，并根据统计设计提前结束研究。2年眼球摘除术的累积发生率为15%；外照射放疗为10%；控制疾病进展的化疗为10%。所有治疗失败的患者均通过额外治疗得到挽救。
对于大多数B组眼内视网膜母细胞瘤患者，不使用依托泊苷的VC化疗联合局部治疗为保眼提供了极好的机会。每个化疗周期均给予局部治疗并加入依托泊苷可能会提高保眼率。诊断时对患者组进行中心评估对于分配适当的治疗至关重要。
