Division of Medical Oncology & Hematology, Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, ON, Canada.
Department of Medicine, University of Toronto, Toronto, ON, Canada.
Leuk Lymphoma. 2022 Dec;63(13):3165-3174. doi: 10.1080/10428194.2022.2109154. Epub 2022 Sep 12.
Patients with lower-risk (LR) myelodysplastic syndromes (MDS) with ring sideroblasts (RS) have better prognosis than those without RS, but how they fare over time is not fully understood. This study's objective was to assess the natural history of LR MDS with RS ≥5% using MDS-CAN registry individual data. Kaplan-Meier estimates and generalized linear mixed models were used to describe time-to-event outcomes and continuous outcomes, respectively. One hundred and thirty-eight patients were enrolled; median times from diagnosis to enrollment and follow-up were 6.6 and 39.6 months, respectively. Within 5 years of enrollment, 65% of patients had ≥1 red blood cell transfusion dependence episode. Within 5 years of diagnosis, 59% developed iron overload, 38% received iron chelation therapy, 14% progressed to acute myeloid leukemia, and 42% died. Patients exhibited inferior health-related quality of life trends. These first real-world data in LR MDS-RS in Canada indicate a high level of morbidity and mortality over a 5-year period. ClinicalTrials.gov Identifier: NCT02537990.
患有环形铁幼粒细胞（RS）低危（LR）骨髓增生异常综合征（MDS）的患者比没有 RS 的患者预后更好，但他们的长期预后情况尚不完全清楚。本研究的目的是使用 MDS-CAN 注册个体数据评估 RS≥5%的 LR MDS 的自然病史。采用 Kaplan-Meier 估计和广义线性混合模型分别描述时间事件结局和连续结局。共纳入 138 例患者；从诊断到入组和随访的中位时间分别为 6.6 和 39.6 个月。入组后 5 年内，65%的患者有≥1 次红细胞输注依赖事件。入组后 5 年内，59%的患者发生铁过载，38%的患者接受铁螯合治疗，14%的患者进展为急性髓系白血病，42%的患者死亡。患者的健康相关生活质量呈下降趋势。这些在加拿大进行的首次关于 LR-MDS-RS 的真实世界数据表明，在 5 年内发病率和死亡率较高。临床试验.gov 标识符：NCT02537990。
