Holtz Children's Hospital, Jackson Memorial Medical Center.
Division of Pediatric Hematology-Oncology.
J Pediatr Hematol Oncol. 2022 May 1;44(4):181-185. doi: 10.1097/MPH.0000000000002451. Epub 2022 Mar 25.
Retinoblastoma (RB) is the most common intraocular pediatric malignancy. Advancements in intra-arterial chemotherapy (IAC) for treatment of RB have resulted in dramatic improvement in eye salvage rates. Data regarding IAC outcomes and associated hematologic toxicities are limited. The objective of this retrospective study was to analyze baseline characteristics, efficacy, and hematologic complications associated with IAC treatment in children with RB at a single international referral institution. Ninety-five sessions of IAC were performed in 28 patients. Mean age at RB diagnosis was 12.5 months (SD, 9.2 mo). Fourteen patients had bilateral RB. IAC agents included melphalan, carboplatin, and topotecan. The most common regimens were triple-agent IAC and single-agent melphalan (66.3% and 15.8%, respectively). Median number of IAC sessions was 3 (mean: 3.39, range: 1 to 9). Eye salvage rate was 83.7% with an overall survival rate of 100% at a median follow-up of 29 months (mean: 29.8 mo, range: 1 to 63 mo). A total of 26 patients (92.9%) experienced at least 1 hematologic toxicity during their treatment course Prevalence of neutropenia, anemia, and thrombocytopenia were 89.3%, 85.7%, and 25%, respectively. While IAC is effective in salvaging most eyes with advanced intraocular RB, over half of patients experienced clinically significant neutropenia and anemia. Clinicians should be vigilant in monitoring patients for IAC-related complications.
视网膜母细胞瘤（RB）是最常见的儿童眼内恶性肿瘤。动脉内化疗（IAC）治疗 RB 的进展显著提高了保眼率。关于 IAC 结果和相关血液学毒性的数据有限。本回顾性研究的目的是分析单一大国际转诊机构中 RB 儿童接受 IAC 治疗的基线特征、疗效和血液学并发症。在 28 例患者中进行了 95 次 IAC。RB 诊断时的平均年龄为 12.5 个月（标准差，9.2 mo）。14 例患者患有双侧 RB。IAC 药物包括美法仑、卡铂和拓扑替康。最常见的方案是三药 IAC 和单药美法仑（分别为 66.3%和 15.8%）。IAC 疗程中位数为 3 次（均值：3.39，范围：1 至 9）。眼保存率为 83.7%，中位随访 29 个月（均值：29.8 mo，范围：1 至 63 mo）时总生存率为 100%。在治疗过程中，共有 26 名患者（92.9%）至少经历了 1 种血液学毒性。中性粒细胞减少症、贫血和血小板减少症的发生率分别为 89.3%、85.7%和 25%。虽然 IAC 对挽救大多数晚期眼内 RB 非常有效，但超过一半的患者经历了严重的中性粒细胞减少症和贫血。临床医生应警惕监测患者的 IAC 相关并发症。
