Laverdière Caroline, Cheung Nai-Kong V, Kushner Brian H, Kramer Kim, Modak Shakeel, LaQuaglia Michael P, Wolden Suzanne, Ness Kirsten K, Gurney James G, Sklar Charles A
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA.
Pediatr Blood Cancer. 2005 Sep;45(3):324-32. doi: 10.1002/pbc.20331.
Few studies have assessed late effects in neuroblastoma (NB) survivors, particularly those with advanced stage disease.
Retrospective analysis of a cohort of advanced stage NB survivors followed in a late effect clinic at a single institution. Screening tests to detect late effects were tailored depending on the individual's treatment exposures.
The study included 63 survivors (31 males). The median age at diagnosis was 3.0 years. The median follow-up from diagnosis was 7.06 years. All patients had surgery and received chemotherapy, 89% received radiation therapy (RT), 62% immunotherapy, and 56% autologous stem cell transplant. Late complications were detected in 95% of survivors and included: hearing loss (62%), primary hypothyroidism (24%), ovarian failure (41% of females), musculoskeletal (19%), and pulmonary (19%) abnormalities. The majority of complications were moderate, with only 4% being life-threatening. Survivors who received cisplatin were at greater risk to develop hearing loss compared to those not so treated (OR 9.74; 95% CI: 0.9-101.6). A total dose of cyclophosphamide greater than 7.4 g was associated with ovarian failure (P = 0.02).
Late complications occur frequently in survivors of advanced stage NB. The majority of these problems are of mild-moderate severity. Long-term follow-up (LFTU) and screening of this population is essential.
很少有研究评估神经母细胞瘤(NB)幸存者的远期效应,尤其是那些患有晚期疾病的患者。
对在单一机构的晚期效应诊所随访的一组晚期NB幸存者进行回顾性分析。根据个体的治疗暴露情况定制检测远期效应的筛查测试。
该研究纳入了63名幸存者(31名男性)。诊断时的中位年龄为3.0岁。从诊断开始的中位随访时间为7.06年。所有患者均接受了手术和化疗,89%接受了放射治疗(RT),62%接受了免疫治疗,56%接受了自体干细胞移植。95%的幸存者检测到晚期并发症,包括:听力损失(62%)、原发性甲状腺功能减退(24%)、卵巢功能衰竭(女性中的41%)、肌肉骨骼(19%)和肺部(19%)异常。大多数并发症为中度,只有4%危及生命。与未接受顺铂治疗的幸存者相比,接受顺铂治疗的幸存者发生听力损失的风险更高(比值比9.74;95%置信区间:0.9 - 101.6)。环磷酰胺总剂量大于7.4 g与卵巢功能衰竭相关(P = 0.02)。
晚期NB幸存者中晚期并发症频繁发生。这些问题大多为轻至中度严重程度。对这一人群进行长期随访(LFTU)和筛查至关重要。