Flaadt Tim, Ebinger Martin, Schreiber Malin, Ladenstein Ruth L, Simon Thorsten, Lode Holger N, Hero Barbara, Schuhmann Martin U, Schäfer Jürgen, Paulsen Frank, Timmermann Beate, Eggert Angelika, Lang Peter
Department of Hematology and Oncology, University Children's Hospital, Eberhard Karls University Tuebingen, 72076 Tuebingen, Germany.
Department of Pediatrics, St Anna Children's Hospital, Medical University, 1090 Vienna, Austria.
J Clin Med. 2023 Sep 25;12(19):6196. doi: 10.3390/jcm12196196.
Despite highly intensive multimodality treatment regimens, the prognosis of patients with high-risk neuroblastoma (HRNB) and central nervous system (CNS) relapse remains poor. We retrospectively reviewed data from 13 patients with HRNB and CNS relapse who received multimodal therapy with consolidating haploidentical stem cell transplantation (haplo-SCT) followed by dinutuximab beta ± subcutaneous interleukin-2 (scIL-2). Following individual relapse treatment, patients aged 1-21 years underwent haplo-SCT with T/B-cell-depleted grafts followed by dinutuximab beta 20 mg/m/day × 5 days for 5-6 cycles. If a response was demonstrated after cycle 5 or 6, patients received up to nine treatment cycles. After haplo-SCT, eight patients had a complete response, four had a partial response, and one had a stable disease. All 13 patients received ≥3 cycles of immunotherapy. At the end of the follow-up, 9/13 patients (66.7%) demonstrated complete response. As of July 2023, all nine patients remain disease-free, with a median follow-up time of 5.1 years since relapse. Estimated 5-year event-free and overall survival rates were 55.5% and 65.27%, respectively. Dinutuximab beta ± scIL-2 following haplo-SCT is a promising treatment option with a generally well-tolerated safety profile for patients with HRNB and CNS relapse.
尽管采用了高度强化的多模式治疗方案，但高危神经母细胞瘤（HRNB）并伴有中枢神经系统（CNS）复发的患者预后仍然很差。我们回顾性分析了13例HRNB并伴有CNS复发患者的数据，这些患者接受了多模式治疗，包括巩固性单倍体干细胞移植（haplo-SCT），随后使用贝妥单抗 ± 皮下注射白细胞介素-2（scIL-2）。在个体化复发治疗后，年龄在1至21岁的患者接受了T/B细胞去除的单倍体干细胞移植，随后使用贝妥单抗20 mg/m/天×5天，共进行5至6个周期。如果在第5或第6周期后显示有反应，患者接受多达9个治疗周期。单倍体干细胞移植后，8例患者完全缓解，4例部分缓解，1例病情稳定。所有13例患者均接受了≥3个周期的免疫治疗。随访结束时，13例患者中有9例（66.7%）完全缓解。截至2023年7月，所有9例患者均无疾病复发，自复发以来的中位随访时间为5.1年。估计5年无事件生存率和总生存率分别为55.5%和65.27%。单倍体干细胞移植后使用贝妥单抗 ± scIL-2是一种有前景的治疗选择，对于HRNB并伴有CNS复发的患者，其安全性总体上耐受性良好。
