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预测环孢素治疗纯红细胞再生障碍性贫血患者疗效的列线图模型。

A nomogram model for predicting the efficacy of cyclosporine in patients with pure red cell aplasia.

机构信息

Department of Hematology, General Hospital, Tianjin Medical University, No. 154 Anshandao Road, Heping District, Tianjin, 300052, China.

出版信息

Ann Hematol. 2024 Jun;103(6):1877-1885. doi: 10.1007/s00277-024-05636-9. Epub 2024 Feb 3.

DOI:10.1007/s00277-024-05636-9
PMID:38308019
Abstract

Pure red cell aplasia (PRCA) is a rare bone marrow disorder characterized by a severe reduction or absence of erythroid precursor cells, without affecting granulocytes and megakaryocytes. Immunosuppressive therapies, particularly cyclosporine, have demonstrated efficacy as a primary treatment. This study aims to develop a predictive model for assessing the efficacy of cyclosporine in acquired PRCA (aPRCA). This retrospective study encompasses newly treated aPRCA patients at the General Hospital of Tianjin Medical University. Diagnosis criteria include severe anemia, and absolute reticulocyte count below 10 × 10/L, with normal white blood cell and platelet counts, and a severe reduction in bone marrow erythroblasts. Cyclosporine therapy was administered, with dose adjustments based on blood concentration. Response to cyclosporine was evaluated according to established criteria. Statistical analysis involved logistic multi-factor regression, generating a predictive model. The study included 112 aPRCA patients with a median age of 63.5 years. Patients presented with severe anemia (median Hb, 56 g/L) and reduced reticulocyte levels. Eighty-six patients had no bone marrow nucleated erythroblasts. Primary PRCA accounted for 62 cases (55.4%), and secondary PRCA accounted for 50 cases (44.6%). Univariate analysis revealed that ferritin, platelet to lymphocyte ratio (PLR), and CD4/CD8 ratio influenced treatment response. Multivariate analysis further supported the predictive value of these factors. A prediction model was constructed using ferritin, PLR, and CD4/CD8 ratio, demonstrating high sensitivity and specificity. The ferritin, PLR, and CD4/CD8-based nomogram showed good predictive ability for aPRCA response to cyclosporine. This model has potential clinical value for individualized diagnosis and treatment of aPRCA patients.

摘要

纯红细胞再生障碍性贫血(PRCA)是一种罕见的骨髓疾病,其特征是红系前体细胞严重减少或缺失,而不影响粒细胞和巨核细胞。免疫抑制疗法,特别是环孢素,已被证明是一种有效的主要治疗方法。本研究旨在开发一种预测模型,以评估环孢素治疗获得性 PRCA(aPRCA)的疗效。本回顾性研究纳入了天津医科大学总医院新治疗的 aPRCA 患者。诊断标准包括严重贫血,绝对网织红细胞计数低于 10×10/L,白细胞和血小板计数正常,骨髓红细胞严重减少。给予环孢素治疗,并根据血药浓度调整剂量。根据既定标准评估环孢素的反应。统计分析涉及逻辑多元回归,生成预测模型。该研究纳入了 112 例 aPRCA 患者,中位年龄为 63.5 岁。患者表现为严重贫血(中位 Hb,56g/L)和网织红细胞水平降低。86 例患者无骨髓有核红细胞。原发性 PRCA 占 62 例(55.4%),继发性 PRCA 占 50 例(44.6%)。单因素分析显示,铁蛋白、血小板与淋巴细胞比值(PLR)和 CD4/CD8 比值影响治疗反应。多因素分析进一步支持了这些因素的预测价值。使用铁蛋白、PLR 和 CD4/CD8 比值构建了预测模型,显示出较高的敏感性和特异性。基于铁蛋白、PLR 和 CD4/CD8 的列线图显示出对 aPRCA 对环孢素反应的良好预测能力。该模型对个体化诊断和治疗 aPRCA 患者具有潜在的临床价值。

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本文引用的文献

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Blood Adv. 2023 Nov 14;7(21):6451-6465. doi: 10.1182/bloodadvances.2023010587.
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Incidence of acquired pure red cell aplasia: a nationwide epidemiologic analysis with 2 registry databases in Japan.获得性纯红细胞再生障碍的发生率:日本两个登记数据库的全国性流行病学分析。
Blood Adv. 2022 Dec 27;6(24):6282-6290. doi: 10.1182/bloodadvances.2021006486.
3
Sirolimus is effective for refractory/relapsed/intolerant acquired pure red cell aplasia: results of a prospective single-institutional trial.
西罗莫司对难治性/复发性/不耐受性获得性纯红细胞再生障碍有效:一项前瞻性单机构试验的结果。
Leukemia. 2022 May;36(5):1351-1360. doi: 10.1038/s41375-022-01532-1. Epub 2022 Mar 7.
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Low- and intermediate-risk myelodysplastic syndrome with pure red cell aplasia.低危和中危骨髓增生异常综合征伴纯红细胞再生障碍。
Hematology. 2021 Dec;26(1):444-446. doi: 10.1080/16078454.2021.1929694.
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Pure red cell aplasia in systemic lupus erythematosus, a nationwide retrospective cohort and review of the literature.系统性红斑狼疮中的纯红细胞再生障碍:全国回顾性队列研究和文献复习。
Rheumatology (Oxford). 2021 Dec 24;61(1):355-366. doi: 10.1093/rheumatology/keab363.
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How I manage acquired pure red cell aplasia in adults.成人获得性纯红细胞再生障碍性贫血的治疗策略。
Blood. 2021 Apr 15;137(15):2001-2009. doi: 10.1182/blood.2021010898.
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Cureus. 2020 Feb 27;12(2):e7112. doi: 10.7759/cureus.7112.
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