Hobbie Wendy L, Li Yimei, Carlson Claire, Goldfarb Samuel, Laskin Benjamin, Denburg Michelle, Goldmuntz Elizabeth, Mostoufi-Moab Sogol, Wilkes Jennifer, Smith Karen, Sacks Nancy, Szalda Dava, Ginsberg Jill P
Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Department of Biostatistics, Epidemiology and Informatics, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Pediatr Blood Cancer. 2022 Mar;69(3):e29537. doi: 10.1002/pbc.29537. Epub 2021 Dec 31.
Neuroblastoma is the most common extracranial solid tumor in children. Those with high-risk disease are treated with multimodal therapy, including high-dose chemotherapy, stem cell transplant, radiation, and immunotherapy that have led to multiple long-term complications in survivors. In the late 1990s, consolidation therapy involved myeloablative conditioning including total body irradiation (TBI) with autologous stem cell rescue. Recognizing the significant long-term toxicities of exposure to TBI, more contemporary treatment protocols have removed this from conditioning regimens. This study examines an expanded cohort of 48 high-risk neuroblastoma patients to identify differences in the late effect profiles for those treated with TBI and those treated without TBI.
Data on the study cohort were collected from clinic charts, provider documentation in the electronic medical record of visits to survivorship clinic, including all subspecialists, and ancillary reports of laboratory and diagnostic tests done as part of risk-based screening at each visit.
All 48 survivors of BMT for high-risk neuroblastoma had numerous late effects of therapy, with 73% having between five and 10 late effects. TBI impacted some late effects significantly, including growth hormone deficiency (GHD), bone outcomes, and cataracts.
Although high-risk neuroblastoma survivors treated with TBI have significant late effects, those treated without TBI also continue to have significant morbidity related to high-dose chemotherapy and local radiation. A multidisciplinary care team assists in providing comprehensive care to those survivors who are at highest risk for significant late effects.
神经母细胞瘤是儿童最常见的颅外实体瘤。高危神经母细胞瘤患者接受多模式治疗，包括大剂量化疗、干细胞移植、放疗和免疫治疗，这些治疗导致幸存者出现多种长期并发症。在20世纪90年代后期，巩固治疗包括采用全身照射（TBI）进行清髓预处理并进行自体干细胞救援。鉴于认识到TBI带来的显著长期毒性，更多当代治疗方案已将其从预处理方案中去除。本研究对48例高危神经母细胞瘤患者的扩大队列进行了检查，以确定接受TBI治疗和未接受TBI治疗的患者在晚期效应方面的差异。
研究队列的数据收集自临床病历、电子病历中提供的关于幸存者诊所就诊的记录（包括所有专科医生的记录）以及每次就诊时作为基于风险筛查一部分所做的实验室和诊断检查的辅助报告。
所有48例接受BMT治疗的高危神经母细胞瘤幸存者都有许多治疗的晚期效应，73%的患者有5至10种晚期效应。TBI对一些晚期效应有显著影响，包括生长激素缺乏（GHD）、骨骼问题和白内障。
尽管接受TBI治疗的高危神经母细胞瘤幸存者有显著的晚期效应，但未接受TBI治疗的患者也继续存在与大剂量化疗和局部放疗相关的显著发病率。多学科护理团队有助于为那些晚期效应风险最高的幸存者提供全面护理。
