Treatment and Outcomes of Patients With Relapsed, High-Risk Neuroblastoma: Results of German Trials

Treatment and Outcomes of Patients With Relapsed, High-Risk Neuroblastoma: Results of German Trials
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复发高危神经母细胞瘤患者的治疗和疗效: 德国试验结果

DOI:
10.1002/pbc.22693
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发表时间:
2011-04-01
影响因子:
3.2
通讯作者:
Hero, Barbara
Hero, Barbara
中科院分区:
医学3区
文献类型:
--
作者:
Simon, Thorsten;Berthold, Frank;Hero, Barbara

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背景。过去几十年来,高危神经母细胞瘤患者的预后有所改善。然而,许多患者在成功的初始治疗后会出现复发。我们回顾性分析了连续三项国家神经母细胞瘤试验中复发患者的长期结果。方法。满足以下所有标准的患者被纳入:诊断时年龄为 1 岁或以上、首次诊断在 1990 年至 2007 年之间、4 期疾病或 MYCN 扩增的 3 期神经母细胞瘤,以及成功一线自体干细胞移植 (ASCT) 后复发或进展。结果。共有451名1岁或以上的高危神经母细胞瘤患者在一线治疗期间接受了ASCT,其中253名出现疾病复发,158名接受了挽救性化疗,其中23名最终接受了第二次ASCT。与 74 名未接受第二次化疗的患者(中位生存期 0.24 年,3 年生存率 4.0 +/- 2.6%)和 135 名接受二线化疗但未接受第二次 ASCT 的患者(中位生存期 0.89 年,3 年生存率 4.0 +/- 2.6%)相比,这 23 名患者的中位生存期(2.08 年)和 3 年复发生存率(43.5 +/- 10.9%)更好9.6 +/- 2.8%,P < 0.001)。到 2010 年 2 月,3/23 患者完全缓解,3/23 患者完全缓解,1/23 患者部分缓解,14/23 患者在成功进行第二次 ASCT 后死于疾病。 2/23 死于第二次 ASCT 并发症。结论。强化二线治疗是可行的。一小部分复发高危神经母细胞瘤患者可能受益于强化复发化疗和第二次 ASCT。长期生存的潜力证明了强化二线治疗的临床试验是合理的。儿童血癌。 2011;56:578-583。 (C) 2010 Wiley-Liss, Inc.
Background. The prognosis of high-risk neuroblastoma patients has improved over the last decades. However, many patients experience relapse after successful initial treatment. We retrospectively analyzed the long-term outcome of relapsed patients of three consecutive national neuroblastoma trials. Methods. Patients were included when they fulfilled all of the following criteria: Age at diagnosis being 1 year or older, first diagnosis between 1990 and 2007, stage 4 disease or stage 3 neuroblastoma with MYCN amplification, and relapse or progression after successful first-line autologous stem cell transplantation (ASCT). Results. A total of 451 high-risk neuroblastoma patients 1 year or older underwent ASCT during first-line treatment, 253 experienced recurrence of disease, 158 received salvage chemotherapy, and 23 of them finally underwent a second ASCT. These 23 patients had a better median survival (2.08 years) and 3-year survival rate from recurrence (43.5 +/- 10.9%) compared to 74 patients who had no second chemotherapy (median survival 0.24 years, 3-year survival rate 4.0 +/- 2.6%) and 135 patients who underwent second-line chemotherapy but did not undergo second ASCT (median survival of 0.89 years, 3-year survival rate 9.6 +/- 2.8%, P < 0.001). By February 2010, 3/23 patients were in complete remission, 3/23 in very good partial remission, 1/23 in partial remission, 14/23 patients died of disease after successful second ASCT, and. 2/23 died of complications due to second ASCT. Conclusion. Intensive second-line therapy is feasible. A small subgroup of relapsed high-risk neuroblastoma patients may benefit from intensive relapse chemotherapy and second ASCT. The potential of long-term survival justifies clinical trials on intensive second-line treatment. Pediatr Blood Cancer. 2011;56:578-583. (C) 2010 Wiley-Liss, Inc.