Dana-Farber Cancer Institute, Boston, MA, USA.
Harvard School of Public Health, Boston, MA, USA.
Leukemia. 2021 Apr;35(4):1166-1175. doi: 10.1038/s41375-020-01092-2. Epub 2020 Nov 17.
We conducted a prospective observational study of fit adults aged 60-75 with advanced MDS, enrolled hierarchically for adverse MDS risk (intermediate-2 or high-risk international prognostic score [IPSS], low or intermediate-1 IPSS with poor-risk cytogenetics, or therapy-related MDS) or standard risk with severe cytopenia. A total of 290 patients enrolled at two centers: 175 for adverse risk and 115 for standard risk with severe cytopenia. 113 underwent HCT after a median of 5 months; median follow-up for all was 39.5 months. In univariable analyses, the hazard ratio (HR) for death comparing HCT with no HCT was 0.84 (p = 0.30). The HR for death was 0.64 (p = 0.04) for HCT ≤ 5 months after enrollment and 1.20 (p = 0.39) for HCT > 5 months. In multivariable analyses controlling for age, gender, ECOG performance status, cytogenetic risk, and IPSS risk group, HR for death was 0.75 (p = 0.13) for HCT compared to no HCT, 0.57 (p = 0.01) for adverse MDS risk and 1.33 (p = 0.36) for standard risk with severe cytopenia. In this large, prospective cohort of fit older adults with advanced MDS, we found that survival was significantly improved if HCT was performed early or for adverse risk disease but not for standard risk disease with severe cytopenia.
我们对 60-75 岁患有 MDS 的健康成年人进行了一项前瞻性观察研究，这些患者按照 MDS 不良风险（中-2 或高风险国际预后评分[IPSS]、低或中-1 IPSS 伴不良细胞遗传学、或治疗相关 MDS）或标准风险伴严重细胞减少症进行分层登记。共有 290 名患者在两个中心登记：175 名患有不良风险，115 名患有标准风险伴严重细胞减少症。中位数为 5 个月后，113 名患者接受了 HCT；所有患者的中位随访时间为 39.5 个月。单变量分析中，与未接受 HCT 相比，接受 HCT 的死亡风险比（HR）为 0.84（p = 0.30）。登记后 5 个月内接受 HCT 的死亡 HR 为 0.64（p = 0.04），而 5 个月后接受 HCT 的死亡 HR 为 1.20（p = 0.39）。在多变量分析中，控制年龄、性别、ECOG 表现状态、细胞遗传学风险和 IPSS 风险组后，与未接受 HCT 相比，接受 HCT 的死亡 HR 为 0.75（p = 0.13），MDS 不良风险为 0.57（p = 0.01），标准风险伴严重细胞减少症为 1.33（p = 0.36）。在这项针对患有 MDS 的健康老年人的大型前瞻性队列研究中，我们发现如果早期进行 HCT 或治疗不良风险疾病，患者的生存可显著改善，但对于标准风险伴严重细胞减少症疾病则不然。
