Bornfeld N, Schüler A, Bechrakis N, Henze G, Havers W
Augenklinik und Poliklinik, Universitätsklinikum Benjamin Franklin, Freie Universität Berlin.
Klin Padiatr. 1997 Jul-Aug;209(4):216-21. doi: 10.1055/s-2008-1043953.
Eye retention rates after external beam therapy for advanced retinoblastoma are close to 90% with very few ocular side effects like radiation retinopathy or radiation induced optic neuropathy. Late complications, however, comprise a sixfold increase of secondary non-ocular cancers like soft tissue sarcomas demanding alternative treatment options in the management of advanced retinoblastoma.
A phase I study on primary chemotherapy using vincristine, cyclophosphamide, etoposide and carboplatin in advanced bilateral retinoblastoma was undertaken to evaluate the potential of a treatment regimen not including external beam therapy.
12 eyes with 17 tumors in 9 children were treated with primary chemotherapy as first treatment option with a mean follow-up of 16.7 months. Planned enucleation of a tumor containing eye was performed in 2 eyes. In one of these enucleated eyes no viable tumor cells were visible on histopathological examination while in the other eye marked tumor regression was detectable. Of the remaining 10 eyes clinical regression of the intraocular tumors was present in all eyes; on average the tumor size was reduced by 50% within 3-6 weeks. Most of the tumors showed a type I (cottage cheese) regression indistinguishable from regression patterns found after external beam radiotherapy. Ancillary treatments including laser photocoagulation, beta-ray brachytherapy, external beam radiotherapy and cryotherapy were performed in 15 out of the 17 tumors.
Based on the results of this study and on the results published by others primary chemotherapy may result in the same eye retention rate as conventional external beam therapy avoiding the unacceptable high rate of secondary non-ocular cancers in the latter. A prospective study to be undertaken by the GPOH (Gesellschaft für Pädiatrische Onkologie und Hämatologie) and the RG (Retinologische Gesellschaft) is planned to further evaluate this new and promising approach.
晚期视网膜母细胞瘤接受外照射治疗后的眼球保留率接近90%，很少出现如放射性视网膜病变或放射性视神经病变等眼部副作用。然而，晚期并发症包括软组织肉瘤等继发性非眼部癌症的发生率增加了六倍，这就需要在晚期视网膜母细胞瘤的治疗中采用其他治疗方案。
开展一项针对晚期双侧视网膜母细胞瘤使用长春新碱、环磷酰胺、依托泊苷和卡铂进行一线化疗的I期研究，以评估不包括外照射治疗的治疗方案的潜力。
9名儿童的12只眼睛中有17个肿瘤，将一线化疗作为首选治疗方法，平均随访16.7个月。计划对2只眼睛进行含肿瘤眼球的摘除术。在其中一只摘除的眼睛的组织病理学检查中未发现存活的肿瘤细胞，而在另一只眼睛中可检测到明显的肿瘤消退。其余10只眼睛中，所有眼睛的眼内肿瘤均出现临床消退；平均而言，肿瘤大小在3 - 6周内缩小了50%。大多数肿瘤表现出I型（干酪样）消退，与外照射放疗后的消退模式无法区分。17个肿瘤中的15个进行了包括激光光凝、β射线近距离放疗、外照射放疗和冷冻疗法在内的辅助治疗。
基于本研究结果以及其他研究发表的结果，一线化疗可能会产生与传统外照射治疗相同的眼球保留率，同时避免后者不可接受的高继发性非眼部癌症发生率。德国儿科肿瘤学和血液学协会（Gesellschaft für Pädiatrische Onkologie und Hämatologie，GPOH）和德国视网膜病学会（Retinologische Gesellschaft，RG）计划开展一项前瞻性研究，以进一步评估这种新的且有前景的方法。
