{"title":"治疗性血浆置换治疗免疫性血栓性血小板减少性紫癜:临床疗效、实验室趋势和性别差异的六年前瞻性研究","authors":"Sudipta Sekhar Das, Sourav Mukherjee, Sourav Chowdhury","doi":"10.1016/j.transci.2025.104252","DOIUrl":null,"url":null,"abstract":"<div><div>Thrombotic thrombocytopenic purpura (TTP) is a life-threatening thrombotic microangiopathy characterized by severe ADAMTS13 deficiency. Therapeutic plasma exchange (TPE) remains the cornerstone of treatment in immune-mediated TTP. However, limited data are available from India on procedural characteristics, sex-based presentation, and laboratory response dynamics. This six-year prospective observational study enrolled 43 patients with immune TTP at a tertiary care center in Eastern India between January 2019 and December 2024. All patients had ADAMTS13 activity < 10 % and received corticosteroids and daily TPE. Clinical features, laboratory parameters, procedural metrics, and treatment outcomes were analyzed. The cohort had a female predominance (72.1 %) with a mean age of 36.1 ± 9.6 years. All patients underwent ≥ 6 TPE sessions using central venous access. Significant hematologic improvement was observed: hemoglobin rose from 6.6 g/dL to 11.1 g/Dl (p = 0.00001), platelets from 17.4 to 157.7 × 10⁶/mL (p < 0.001), and LDH declined from 1121.6 to 229.7IU/L (p < 0.001). Reticulocyte count and indirect bilirubin levels decreased, indicating hemolysis resolution. Procedure-related adverse events were mild (20.9 %) and manageable. Females exhibited more severe anemia and higher bilirubin levels than males, while LDH levels were higher in males, suggesting sexbased variation in disease severity. The overall mortality rate was 16.3 %, predominantly among females. TPE combined with corticosteroids led to rapid hematologic recovery and was safe and effective in immune TTP. Baseline gender disparities and the lack of adjunctive biologics highlight the need for tailored treatment protocols and improved access to advanced therapies in resource-constrained settings.</div></div>","PeriodicalId":49422,"journal":{"name":"Transfusion and Apheresis Science","volume":"64 6","pages":"Article 104252"},"PeriodicalIF":1.2000,"publicationDate":"2025-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Therapeutic plasma exchange in immune thrombotic thrombocytopenic purpura: A six-year prospective study on clinical efficacy, laboratory trends, and sex-based differences\",\"authors\":\"Sudipta Sekhar Das, Sourav Mukherjee, Sourav Chowdhury\",\"doi\":\"10.1016/j.transci.2025.104252\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>Thrombotic thrombocytopenic purpura (TTP) is a life-threatening thrombotic microangiopathy characterized by severe ADAMTS13 deficiency. Therapeutic plasma exchange (TPE) remains the cornerstone of treatment in immune-mediated TTP. However, limited data are available from India on procedural characteristics, sex-based presentation, and laboratory response dynamics. This six-year prospective observational study enrolled 43 patients with immune TTP at a tertiary care center in Eastern India between January 2019 and December 2024. All patients had ADAMTS13 activity < 10 % and received corticosteroids and daily TPE. Clinical features, laboratory parameters, procedural metrics, and treatment outcomes were analyzed. The cohort had a female predominance (72.1 %) with a mean age of 36.1 ± 9.6 years. All patients underwent ≥ 6 TPE sessions using central venous access. Significant hematologic improvement was observed: hemoglobin rose from 6.6 g/dL to 11.1 g/Dl (p = 0.00001), platelets from 17.4 to 157.7 × 10⁶/mL (p < 0.001), and LDH declined from 1121.6 to 229.7IU/L (p < 0.001). Reticulocyte count and indirect bilirubin levels decreased, indicating hemolysis resolution. Procedure-related adverse events were mild (20.9 %) and manageable. Females exhibited more severe anemia and higher bilirubin levels than males, while LDH levels were higher in males, suggesting sexbased variation in disease severity. The overall mortality rate was 16.3 %, predominantly among females. TPE combined with corticosteroids led to rapid hematologic recovery and was safe and effective in immune TTP. Baseline gender disparities and the lack of adjunctive biologics highlight the need for tailored treatment protocols and improved access to advanced therapies in resource-constrained settings.</div></div>\",\"PeriodicalId\":49422,\"journal\":{\"name\":\"Transfusion and Apheresis Science\",\"volume\":\"64 6\",\"pages\":\"Article 104252\"},\"PeriodicalIF\":1.2000,\"publicationDate\":\"2025-09-05\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Transfusion and Apheresis Science\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1473050225001909\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Transfusion and Apheresis Science","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1473050225001909","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
血栓性血小板减少性紫癜(TTP)是一种危及生命的血栓性微血管疾病,其特征是严重的ADAMTS13缺乏。治疗性血浆交换(TPE)仍然是免疫介导的TTP治疗的基石。然而,印度在程序特征、基于性别的表现和实验室反应动力学方面的数据有限。这项为期六年的前瞻性观察研究于2019年1月至2024年12月在印度东部的一家三级医疗中心招募了43名免疫TTP患者。所有患者均有ADAMTS13活性<; 10 %,并接受皮质类固醇和每日TPE治疗。分析临床特征、实验室参数、程序指标和治疗结果。该队列以女性为主(72.1 %),平均年龄为36.1 ± 9.6岁。所有患者均通过中心静脉通道接受≥ 6次TPE治疗。显著改善血液观察:血红蛋白升至6.6 11.1 g / dL g / dL (p = 0.00001),血小板从17.4到157.7 × 10⁶/毫升(0.001 p & lt; ),和LDH拒绝从1121.6到229.7 iu / L (p & lt; 0.001)。网织红细胞计数和间接胆红素水平下降,表明溶血消退。手术相关不良事件轻微(20.9 %)且可控。与男性相比,女性表现出更严重的贫血和更高的胆红素水平,而男性的LDH水平更高,这表明疾病严重程度存在性别差异。总死亡率为16.3 %,主要是女性。TPE联合皮质类固醇可使血液学恢复迅速,对免疫性TTP安全有效。基线性别差异和辅助生物制剂的缺乏突出表明,在资源有限的情况下,需要制定量身定制的治疗方案,并改善获得先进疗法的机会。
Therapeutic plasma exchange in immune thrombotic thrombocytopenic purpura: A six-year prospective study on clinical efficacy, laboratory trends, and sex-based differences
Thrombotic thrombocytopenic purpura (TTP) is a life-threatening thrombotic microangiopathy characterized by severe ADAMTS13 deficiency. Therapeutic plasma exchange (TPE) remains the cornerstone of treatment in immune-mediated TTP. However, limited data are available from India on procedural characteristics, sex-based presentation, and laboratory response dynamics. This six-year prospective observational study enrolled 43 patients with immune TTP at a tertiary care center in Eastern India between January 2019 and December 2024. All patients had ADAMTS13 activity < 10 % and received corticosteroids and daily TPE. Clinical features, laboratory parameters, procedural metrics, and treatment outcomes were analyzed. The cohort had a female predominance (72.1 %) with a mean age of 36.1 ± 9.6 years. All patients underwent ≥ 6 TPE sessions using central venous access. Significant hematologic improvement was observed: hemoglobin rose from 6.6 g/dL to 11.1 g/Dl (p = 0.00001), platelets from 17.4 to 157.7 × 10⁶/mL (p < 0.001), and LDH declined from 1121.6 to 229.7IU/L (p < 0.001). Reticulocyte count and indirect bilirubin levels decreased, indicating hemolysis resolution. Procedure-related adverse events were mild (20.9 %) and manageable. Females exhibited more severe anemia and higher bilirubin levels than males, while LDH levels were higher in males, suggesting sexbased variation in disease severity. The overall mortality rate was 16.3 %, predominantly among females. TPE combined with corticosteroids led to rapid hematologic recovery and was safe and effective in immune TTP. Baseline gender disparities and the lack of adjunctive biologics highlight the need for tailored treatment protocols and improved access to advanced therapies in resource-constrained settings.
期刊介绍:
Transfusion and Apheresis Science brings comprehensive and up-to-date information to physicians and health care professionals involved in the rapidly changing fields of transfusion medicine, hemostasis and apheresis. The journal presents original articles relating to scientific and clinical studies in the areas of immunohematology, transfusion practice, bleeding and thrombotic disorders and both therapeutic and donor apheresis including hematopoietic stem cells. Topics covered include the collection and processing of blood, compatibility testing and guidelines for the use of blood products, as well as screening for and transmission of blood-borne diseases. All areas of apheresis - therapeutic and collection - are also addressed. We would like to specifically encourage allied health professionals in this area to submit manuscripts that relate to improved patient and donor care, technical aspects and educational issues.
Transfusion and Apheresis Science features a "Theme" section which includes, in each issue, a group of papers designed to review a specific topic of current importance in transfusion and hemostasis for the discussion of topical issues specific to apheresis and focuses on the operators'' viewpoint. Another section is "What''s Happening" which provides informal reporting of activities in the field. In addition, brief case reports and Letters to the Editor, as well as reviews of meetings and events of general interest, and a listing of recent patents make the journal a complete source of information for practitioners of transfusion, hemostasis and apheresis science. Immediate dissemination of important information is ensured by the commitment of Transfusion and Apheresis Science to rapid publication of both symposia and submitted papers.