Background: Hematopoietic cell transplantation (HCT) remains the only curative therapy for pediatric myelodysplastic syndrome (MDS) in all but rare cases. While HCT outcomes for pediatric MDS are similar across the largest registry and single-center trials, factors identified as contributing to inferior outcomes vary from study to study. We performed an analysis to provide more clarity on the prognostic implications of disease characteristics, including blast burden and cytogenetic abnormalities, in the current era. Methods: We conducted a retrospective analysis of 36 consecutive children (<18 years of age at HCT) who underwent allogeneic HCT for MDS between June 2000 and October 2019 at the Fred Hutchinson Cancer Center. Results: Overall survival (OS) was 77% (95% CI 64–92%) and relapse-free survival (RFS) was 71% (95% CI 57–88%) at 2 years post-HCT. Patients with <5% blasts by morphology in the bone marrow at the time of HCT showed superior 2-year OS at 87% (95% CI 74–100%) as compared to 54% (95% CI 32–93%) in patients with ≥5% blasts, consistent with an HR of 4.6 (CI 1.14–18.7,p= 0.03). The inferior outcomes in patients with ≥5% blasts were due to increased relapse incidence (HR 7.6, CI 1.5–39.3) with no difference in NRM or acute GVHD. Conclusions: OS and RFS were comparable to what has been observed in other large, single-center studies (OS 77%, RFS 71% at 2 years) and compared favorably to outcomes from the largest multi-center retrospective analyses.
背景：除极少数病例外，造血细胞移植（HCT）仍是儿童骨髓增生异常综合征（MDS）的唯一根治性疗法。尽管大型登记研究和单中心试验显示儿童MDS的HCT结局相似，但不同研究中导致不良结局的因素存在差异。本研究旨在更清晰地阐明当前时代疾病特征（包括原始细胞负荷和细胞遗传学异常）对预后的影响。方法：我们对弗雷德·哈钦森癌症中心在2000年6月至2019年10月期间连续收治的36例接受异基因HCT治疗的MDS患儿（移植时年龄<18岁）进行回顾性分析。结果：移植后2年总生存率（OS）为77%（95% CI 64–92%），无复发生存率（RFS）为71%（95% CI 57–88%）。移植时骨髓形态学原始细胞比例<5%的患者2年OS达87%（95% CI 74–100%），显著优于原始细胞比例≥5%患者的54%（95% CI 32–93%），风险比（HR）为4.6（CI 1.14–18.7，p=0.03）。原始细胞比例≥5%患者的不良结局主要源于更高的复发率（HR 7.6，CI 1.5–39.3），而非移植相关死亡率或急性移植物抗宿主病的差异。结论：本研究的总生存率和无复发生存率与其他大型单中心研究结果相当（2年OS 77%，RFS 71%），且优于最大规模的多中心回顾性分析数据。
肿瘤(癌症)患者之家