Long-term outcomes in children with high-risk neuroblastoma treated with autologous stem cell transplantation
Long-term outcomes in children with high-risk neuroblastoma treated with autologous stem cell transplantation
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DOI：
10.1038/sj.bmt.1705809
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发表时间：
2007-10-01
影响因子：
4.8
通讯作者：
Marshall, G. M.
中科院分区：
文献类型：
--
作者：
Trahair, T. N.;Vowels, M. R.;Marshall, G. M.
我们回顾性分析了在一家主要接受全身放疗和化疗的机构接受移植治疗的高危神经母细胞瘤（NB）患儿的结局。本研究的目的是确定临床和生物学特征的预后影响，并记录长期健康结果。40例患者接受了单一未净化自体移植物移植。14例患者死于疾病进展，2例死于治疗晚期并发症。23例患者从诊断开始存活的中位时间为4.6年。Kaplan-Meier估计移植后2、5和10年的总生存率分别为76 +/- 7.0、60.2 +/- 8.4和54.7 +/- 9.3%。诱导治疗的反应与生存率显着相关（P < 0.01）。长期并发症包括生长（100%）和青春期衰竭（83%）、听力损害（73%）、骨科并发症（63%）、肾损害（47%）和甲状腺异常（36%）。对化疗的内在和获得性耐药仍然是改善高危NB预后的主要障碍。尽管化疗敏感性疾病患者复发的可能性较小，但大量治疗相关毒性导致幸存者的长期健康结局较差。
We retrospectively analysed the outcomes of children transplanted for high-risk neuroblastoma ( NB) at a single institution predominantly transplanted with total body irradiation and chemotherapy. The aims of this study were to determine the prognostic impact of clinical and biological features and to document long-term health outcomes. Forty patients were transplanted with a single unpurged autograft. Fourteen patients died from disease progression and two from late complications of treatment. Twenty-three patients are alive at a median of 4.6 years from diagnosis. Kaplan -Meier estimates of overall survival at 2, 5 and 10 years are 76 +/- 7.0, 60.2 +/- 8.4 and 54.7 +/- 9.3% following transplant. Response to induction therapy was significantly associated with survival (P < 0.01). Long-term complications included growth (100%) and pubertal failure (83%), hearing impairment (73%), orthopaedic complications (63%), renal impairment (47%) and thyroid abnormalities (36%). Intrinsic and acquired resistance to chemotherapy remains the major obstacle to improving outcomes in high-risk NB. Although patients with chemo-sensitive disease are less likely to experience a relapse, substantial therapy-related toxicities result in poor long-term health outcomes for survivors.