Department of Pediatrics, University of Minnesota Medical School, Minneapolis, MN.
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA.
Blood. 2023 Jan 5;141(1):90-101. doi: 10.1182/blood.2022016487.
Five-year survival following childhood acute myeloid leukemia (AML) has increased following improvements in treatment and supportive care. Long-term health outcomes are unknown. To address this, cumulative incidence of late mortality and grades 3 to 5 chronic health condition (CHC) were estimated among 5-year AML survivors diagnosed between 1970 and 1999. Survivors were compared by treatment group (hematopoietic cell transplantation [HCT], chemotherapy with cranial radiation [chemo + CRT], chemotherapy only [chemo-only]), and diagnosis decade. Self-reported health status was compared across treatments, diagnosis decade, and with siblings. Among 856 survivors (median diagnosis age, 7.1 years; median age at last follow-up, 29.4 years), 20-year late mortality cumulative incidence was highest after HCT (13.9%; 95% confidence interval [CI], 10.0%-17.8%; chemo + CRT, 7.6%; 95% CI, 2.2%-13.1%; chemo-only, 5.1%; 95% CI, 2.8%-7.4%). Cumulative incidence of mortality for HCT survivors diagnosed in the 1990s (8.5%; 95% CI, 4.1%-12.8%) was lower vs those diagnosed in the 1970s (38.9%; 95% CI, 16.4%-61.4%). Most survivors did not experience any grade 3 to 5 CHC after 20 years (HCT, 45.8%; chemo + CRT, 23.7%; chemo-only, 27.0%). Furthermore, a temporal reduction in CHC cumulative incidence was seen after HCT (1970s, 76.1%; 1990s, 38.3%; P = .02), mirroring reduced use of total body irradiation. Self-reported health status was good to excellent for 88.2% of survivors; however, this was lower than that for siblings (94.8%; P < .0001). Although HCT is associated with greater long-term morbidity and mortality than chemotherapy-based treatment, gaps have narrowed, and all treatment groups report favorable health status.
儿童急性髓系白血病（AML）患者的 5 年生存率在治疗和支持性护理得到改善后有所提高。但长期健康结果尚不清楚。为解决这一问题，本研究对 1970 年至 1999 年间诊断为 AML 的 5 年幸存者进行了迟发性死亡和 3 至 5 级慢性健康状况（CHC）的累积发生率评估。通过治疗组（造血细胞移植[HCT]、化疗联合颅放疗[化疗+ CRT]、单纯化疗[化疗仅]）和诊断十年进行幸存者比较。比较了治疗方法、诊断十年以及与同胞的自我报告健康状况。在 856 名幸存者（中位诊断年龄为 7.1 岁；末次随访年龄中位数为 29.4 岁）中，HCT 后 20 年迟发性死亡率累积发生率最高（13.9%；95%CI，10.0%-17.8%；化疗+ CRT，7.6%；95%CI，2.2%-13.1%；化疗仅，5.1%；95%CI，2.8%-7.4%）。90 年代诊断的 HCT 幸存者的死亡率累积发生率（8.5%；95%CI，4.1%-12.8%）低于 70 年代诊断的幸存者（38.9%；95%CI，16.4%-61.4%）。20 年后，大多数幸存者未经历任何 3 至 5 级 CHC（HCT，45.8%；化疗+ CRT，23.7%；化疗仅，27.0%）。此外，HCT 后 CHC 的累积发生率呈时间性下降（70 年代，76.1%；90 年代，38.3%；P =.02），这与全身放疗使用减少有关。88.2%的幸存者自我报告健康状况良好至优秀；但低于同胞（94.8%；P <.0001）。尽管 HCT 与基于化疗的治疗相比，与更大的长期发病率和死亡率相关，但差距已经缩小，所有治疗组的健康状况均良好。
