Aboulela Mohamed, Collins Angela
Haematology, Norfolk and Norwich University Hospital, Norwich, GBR.
Medical Oncology, The Royal Marsden NHS Foundation Trust, London, GBR.
Cureus. 2024 Oct 27;16(10):e72460. doi: 10.7759/cureus.72460. eCollection 2024 Oct.
Background Myelodysplastic syndromes (MDS) are clonal myeloid disorders characterised by ineffective haematopoiesis, leading to anaemia that often requires dependence on red blood cell (RBC) transfusions. Epoetin alfa (Eprex®) is now a mainstay in the management of symptomatic anaemia in low-risk MDS patients, reducing transfusion dependence and improving the quality of life in this patient group. Objective This retrospective study aimed to assess the efficacy of epoetin alfa in treating symptomatic anaemia in low-risk MDS patients, focusing on transfusion independence and its relationship with baseline erythropoietin (EPO) levels and haemoglobin (Hb) response. Methods Data from 56 patients with low-risk MDS treated with epoetin alfa at Norfolk and Norwich University Hospital, Norwich, United Kingdom, between 2018 and 2023 were retrospectively analysed. Baseline EPO levels, transfusion history, Hb response, and the duration of transfusion independence were assessed. Statistical analyses were performed to evaluate the correlation between baseline characteristics and treatment outcomes. Results Among the patients, 98.2% had baseline EPO levels below the 500 IU/L threshold, with a median EPO level of 74.3 IU/L. Following an eight-week trial of 30,000 units of epoetin-alfa, 41.1% of patients showed improved Hb levels, 41.1% maintained stable Hb levels, and 17.9% experienced a decline. A significant correlation was found between lower baseline EPO levels (<250 IU/L) and a positive treatment response (p = 0.0065). Additionally, patients who required fewer transfusions before treatment had longer durations of transfusion independence (correlation coefficient = -0.40, p = 0.015). Dose escalation to 60,000 units provided a benefit to 53.3% of patients with initially stable Hb levels. The average duration of transfusion independence was 8.1 months, and patients with improved Hb levels had the longest periods of transfusion independence (p = 0.005). Conclusion Epoetin alfa is an effective therapy for managing symptomatic anaemia in low-risk MDS patients. This study highlights its efficacy and provides valuable predictive information, particularly showing that patients with lower baseline EPO levels are more likely to respond to treatment. While prior transfusion dependence did not significantly predict response to therapy in this cohort, it was associated with the duration of transfusion independence.
骨髓增生异常综合征（MDS）是克隆性髓系疾病，其特征为造血无效，导致贫血，常需依赖红细胞（RBC）输血。促红细胞生成素α（利血宝®）目前是低危MDS患者症状性贫血管理的主要手段，可减少输血依赖并改善该患者群体的生活质量。
本回顾性研究旨在评估促红细胞生成素α治疗低危MDS患者症状性贫血的疗效，重点关注输血独立性及其与基线促红细胞生成素（EPO）水平和血红蛋白（Hb）反应的关系。
对2018年至2023年期间在英国诺维奇诺福克和诺维奇大学医院接受促红细胞生成素α治疗的56例低危MDS患者的数据进行回顾性分析。评估基线EPO水平、输血史、Hb反应和输血独立持续时间。进行统计分析以评估基线特征与治疗结果之间的相关性。
患者中，98.2%的基线EPO水平低于500 IU/L阈值，EPO水平中位数为74.3 IU/L。在接受30000单位促红细胞生成素α的八周试验后，41.1%的患者Hb水平改善，41.1%的患者Hb水平保持稳定，17.9%的患者Hb水平下降。发现较低的基线EPO水平（<250 IU/L）与阳性治疗反应之间存在显著相关性（p = 0.0065）。此外，治疗前输血需求较少的患者输血独立持续时间更长（相关系数 = -0.40，p = 0.015）。剂量增至60000单位使53.3%最初Hb水平稳定的患者受益。输血独立的平均持续时间为8.1个月，Hb水平改善的患者输血独立时间最长（p = 0.005）。
促红细胞生成素α是治疗低危MDS患者症状性贫血的有效疗法。本研究突出了其疗效并提供了有价值的预测信息，特别是表明基线EPO水平较低的患者对治疗反应更可能良好。虽然在此队列中先前的输血依赖并未显著预测对治疗的反应，但它与输血独立持续时间相关。
