Department of Hematology and Oncology, Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Bone Marrow Transplant. 2013 Mar;48(3):363-8. doi: 10.1038/bmt.2012.166. Epub 2012 Sep 10.
Childhood autologous hematopoietic cell transplant (auto-HCT) survivors can be at risk for secondary malignant neoplasms (SMNs). We assembled a cohort of 1487 pediatric auto-HCT recipients to investigate the incidence and risk factors for SMNs. Primary diagnoses included neuroblastoma (39%), lymphoma (26%), sarcoma (18%), central nervous system tumors (14%) and Wilms tumor (2%). Median follow-up was 8 years (range, <1-21 years). SMNs were reported in 35 patients (AML/myelodysplastic syndrome (MDS)=13, solid cancers=20, subtype missing=2). The overall cumulative incidence of SMNs at 10 years from auto-HCT was 2.60% (AML/MDS=1.06%, solid tumors=1.30%). We found no association between SMNs risk and age, gender, diagnosis, disease status, time since diagnosis or use of TBI or etoposide as part of conditioning. OS at 5-years from diagnosis of SMNs was 33% (95% confidence interval (CI), 16-52%). When compared with age- and gender-matched general population, auto-HCT recipients had 24 times higher risks of developing SMNs (95% CI, 16.0-33.0). Notable SMN sites included bone (N=5 SMNs, observed (O)/expected (E)=81), thyroid (N=5, O/E=53), breast (N=2, O/E=93), soft tissue (N=2, O/E=34), AML (N=6, O/E=266) and MDS (N=7, O/E=6603). Risks of SMNs increased with longer follow-up from auto-HCT. Pediatric auto-HCT recipients are at considerably increased risk for SMNs and need life-long surveillance for SMNs.
儿童自体造血细胞移植(auto-HCT)幸存者可能面临继发性恶性肿瘤(SMN)的风险。我们组建了一个由 1487 名儿科 auto-HCT 受者组成的队列,以研究 SMN 的发病率和危险因素。主要诊断包括神经母细胞瘤(39%)、淋巴瘤(26%)、肉瘤(18%)、中枢神经系统肿瘤(14%)和肾母细胞瘤(2%)。中位随访时间为 8 年(范围,<1-21 年)。35 名患者报告发生了 SMN(急性髓系白血病/骨髓增生异常综合征[MDS]=13 例,实体癌=20 例,亚型缺失=2 例)。自 auto-HCT 后 10 年,SMN 的总累积发生率为 2.60%(AML/MDS=1.06%,实体瘤=1.30%)。我们没有发现 SMN 风险与年龄、性别、诊断、疾病状态、诊断后时间或是否使用 TBI 或依托泊苷作为预处理的一部分之间存在关联。SMN 诊断后 5 年的 OS 为 33%(95%置信区间[CI],16-52%)。与年龄和性别匹配的一般人群相比,auto-HCT 受者发生 SMN 的风险高 24 倍(95%CI,16.0-33.0)。值得注意的 SMN 部位包括骨骼(N=5 例 SMN,O/E=81)、甲状腺(N=5,O/E=53)、乳房(N=2,O/E=93)、软组织(N=2,O/E=34)、急性髓系白血病(N=6,O/E=266)和骨髓增生异常综合征(N=7,O/E=6603)。随着自 auto-HCT 后随访时间的延长,SMN 的风险增加。儿科 auto-HCT 受者发生 SMN 的风险显著增加,需要终身监测 SMN。