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巴西某转诊中心的骨髓增生异常综合征患者接受重组人红细胞生成素治疗后的长期应答。

Prolonged response to recombinant human erythropoietin treatment in patients with myelodysplastic syndrome at a single referral centre in Brazil.

机构信息

Laboratorio de Pesquisa em Hemoglobinopatias e Genetica das Doencas Hematologicas, Universidade Federal do Ceara, Fortaleza, CE, BR.

Departamento de Cirurgia, Universidade Federal do Ceara, Fortaleza, CE, BR.

出版信息

Clinics (Sao Paulo). 2019;74:e771. doi: 10.6061/clinics/2019/e771. Epub 2019 Sep 9.

Abstract

OBJECTIVES

To evaluate the effects of epoetin (EPO) alfa treatment on overall survival, event-free survival and response duration in patients with myelodysplastic syndrome (MDS) who were treated at a haematological referral centre in northeastern Brazil.

METHODS

This was a retrospective cohort study of 36 patients diagnosed with MDS and treated with EPO alfa at 30,000 to 60,000 IU per week. Clinical data were collected from medical records. The events assessed were non-response to treatment and progression to acute myeloid leukaemia (AML). Statistical analyses were performed using GraphPad Prism 7 and SPSS 24 software.

RESULTS

The overall survival of patients who received EPO alfa treatment was 51.64%, with a median of 65 months of treatment, and the overall survival of this group was 100% during the first 24 months. We detected a 43.5-month median event-free survival, with a response rate of 80.5%. We observed responses from 25 to 175 months. Patients with transfusion dependence and those with a high-risk stratification, as determined by the International Prognostic Scoring System (IPSS), the Revised International Prognostic Scoring System (IPSS-R), the WHO classification-based Prognostic Scoring System (WPSS) and the WHO 2016, had a lower event-free survival than other patients.

CONCLUSIONS

Despite the wide use of EPO alfa in the treatment of anaemia in patients with MDS, the median response duration is approximately only 24 months. Our data provide encouraging results concerning the benefits of using EPO alfa for the improvement of the quality of life, as patients treated with EPO showed higher overall survival, event-free survival rates and longer response durations than have been previously described in the literature.

摘要

目的

评估促红细胞生成素(EPO)α在巴西东北部一家血液学转诊中心治疗的骨髓增生异常综合征(MDS)患者中的总生存、无事件生存和反应持续时间的影响。

方法

这是一项回顾性队列研究,纳入了 36 名被诊断为 MDS 并接受 EPOα治疗的患者,剂量为每周 30000 至 60000IU。从病历中收集临床数据。评估的事件是非治疗反应和进展为急性髓系白血病(AML)。使用 GraphPad Prism 7 和 SPSS 24 软件进行统计分析。

结果

接受 EPOα治疗的患者的总生存率为 51.64%,中位治疗时间为 65 个月,该组在前 24 个月的总生存率为 100%。我们检测到中位无事件生存时间为 43.5 个月,反应率为 80.5%。我们观察到从 25 到 175 个月的反应。依赖输血和具有国际预后评分系统(IPSS)、修订的国际预后评分系统(IPSS-R)、基于世界卫生组织分类的预后评分系统(WPSS)和世界卫生组织 2016 高危分层的患者与其他患者相比,无事件生存时间更短。

结论

尽管 EPOα广泛用于治疗 MDS 患者的贫血,但中位反应持续时间约为 24 个月。我们的数据提供了令人鼓舞的结果,表明使用 EPOα改善生活质量的益处,因为接受 EPO 治疗的患者的总生存率、无事件生存率和反应持续时间均高于文献中先前描述的水平。

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