Faculty of Medicine, Universitas Indonesia, Jakarta, Indonesia.
Asia Pac J Ophthalmol (Phila). 2021 Jan 19;10(4):397-407. doi: 10.1097/APO.0000000000000372.
To analyze the risk and benefit of high-dose chemotherapy followed by stem cell transplantation (HDCT-SCT) treatment in patients with advanced retinoblastoma.
Systematic review.
A comprehensive literature search from 4 online databases, including PubMed, Scopus, EBSCO, and Cochrane was done for original studies evaluating the use of HDCT followed by SCT in the treatment of patients with advanced retinoblastoma. The last search was performed on April 15, 2020.
A total of 35 studies consisting of 160 patients were considered suitable for inclusion. After HDCT-SCT treatment, 108/160 (67.5%) patients were alive with no evidence of disease at the last follow-up. The incidence of secondary malignancy in our data was also relatively low, which was 16/160 (10%) patients. The side effects were mainly hematological and gastrointestinal toxicities. The prognosis for metastatic cases especially the one to the central nervous system (CNS) remains poor, as shown in our data that 22 of 44 (50%) patients died due to the evidence of disease, and 12 of 44 (27%) patients acquired CNS relapse and died.
HDCT-SCT is a promising treatment option in patients with advanced retinoblastoma. The use of HDCT-SCT in CNS metastases needs to be carefully considered, possibly by adding thiotepa or topotecan to improve tumor control. Further randomized clinical trials are needed to draw firm conclusion regarding its safety and efficacy.
分析大剂量化疗联合干细胞移植（HDCT-SCT）治疗晚期视网膜母细胞瘤患者的风险和获益。
系统评价。
对评估 HDCT 联合 SCT 治疗晚期视网膜母细胞瘤患者的原始研究进行了全面的文献检索，检索了 4 个在线数据库，包括 PubMed、Scopus、EBSCO 和 Cochrane。最后一次检索时间为 2020 年 4 月 15 日。
共有 35 项研究，共 160 例患者符合纳入标准。经过 HDCT-SCT 治疗后，160 例患者中有 108 例（67.5%）在最后一次随访时无疾病证据存活。本研究中继发恶性肿瘤的发生率也相对较低，为 16/160（10%）例患者。副作用主要是血液学和胃肠道毒性。转移性病例的预后仍然较差，尤其是中枢神经系统（CNS）转移，在我们的数据中，44 例患者中有 22 例（50%）因疾病证据死亡，44 例患者中有 12 例（27%）发生 CNS 复发并死亡。
HDCT-SCT 是治疗晚期视网膜母细胞瘤患者的一种有前途的治疗选择。HDCT-SCT 在 CNS 转移中的应用需要仔细考虑，可能需要添加噻替派或拓扑替康以提高肿瘤控制率。需要进一步的随机临床试验来确定其安全性和疗效。
