Miguel G. Uriol-Rivera, Bernardo López Andrade, Antonio Mas Bonet, Aina Obrador Mulet, Carmen Ballester Ruiz, Leonor Periañez Parraga, Javier Lumbreras, José Ignacio Ayestarán Rota, Mireia Ferreruela Servalos, Joana Ferrer Balaguer, Lucio Pallares Ferreres, María Jose Picado Valles, Rosa María Ruíz de Gopegui Valero, Susana Tarongi Sanchez, Ana Garcia Martin, Juan Rodríguez Garcia, Cristina Gomez Cobo, Daniel Ramis-Cabrer, the Son Espases Multidisciplinary Team for the management of Thrombotic Microangiopathy
{"title":"无 ADAMTS-13 缺乏症的血栓性微血管病患者死亡或需要慢性肾脏替代治疗的风险因素。","authors":"Miguel G. Uriol-Rivera, Bernardo López Andrade, Antonio Mas Bonet, Aina Obrador Mulet, Carmen Ballester Ruiz, Leonor Periañez Parraga, Javier Lumbreras, José Ignacio Ayestarán Rota, Mireia Ferreruela Servalos, Joana Ferrer Balaguer, Lucio Pallares Ferreres, María Jose Picado Valles, Rosa María Ruíz de Gopegui Valero, Susana Tarongi Sanchez, Ana Garcia Martin, Juan Rodríguez Garcia, Cristina Gomez Cobo, Daniel Ramis-Cabrer, the Son Espases Multidisciplinary Team for the management of Thrombotic Microangiopathy","doi":"10.1111/ejh.14261","DOIUrl":null,"url":null,"abstract":"<p>Thrombotic microangiopathy (TMA), characterized by microangiopathic hemolytic anemia, thrombocytopenia, and multisystem organ dysfunction, is a life-threatening disease. Patients with TMA who do not exhibit a severe ADAMTS-13 deficiency (defined as a disintegrin-like and metalloprotease with thrombospondin type 1 motif no. 13 activity ≥10%: TMA-13n) continue to experience elevated mortality rates. This study explores the prognostic indicators for augmented mortality risk or necessitating chronic renal replacement therapy (composite outcome: CO) in TMA-13n patients. We included 42 TMA-13n patients from January 2008 to May 2018. Median age of 41 years and 60% were female. At presentation, 62% required dialysis, and 57% warranted intensive care unit admission. CO was observed in 45% of patients, including a 9-patient mortality subset. Multivariate logistic regression revealed three independent prognostic factors for CO: early administration of eculizumab (median time from hospitalization to eculizumab initiation: 5 days, range 0–19 days; odds ratio [OR], 0.14; 95% confidence interval [CI], 0.02–0.94), presence of neuroradiological lesions (OR, 6.67; 95% CI, 1.12–39.80), and a PLASMIC score ≤4 (OR, 7.39; 95% CI, 1.18–46.11). In conclusion, TMA-13n patients exhibit a heightened risk of CO in the presence of low PLASMIC scores and neuroradiological lesions, while early eculizumab therapy was the only protective factor.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 4","pages":"510-520"},"PeriodicalIF":2.3000,"publicationDate":"2024-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Risk factors of death or chronic renal replacement therapy requirements in patients with thrombotic microangiopathies without ADAMTS-13 deficiency\",\"authors\":\"Miguel G. Uriol-Rivera, Bernardo López Andrade, Antonio Mas Bonet, Aina Obrador Mulet, Carmen Ballester Ruiz, Leonor Periañez Parraga, Javier Lumbreras, José Ignacio Ayestarán Rota, Mireia Ferreruela Servalos, Joana Ferrer Balaguer, Lucio Pallares Ferreres, María Jose Picado Valles, Rosa María Ruíz de Gopegui Valero, Susana Tarongi Sanchez, Ana Garcia Martin, Juan Rodríguez Garcia, Cristina Gomez Cobo, Daniel Ramis-Cabrer, the Son Espases Multidisciplinary Team for the management of Thrombotic Microangiopathy\",\"doi\":\"10.1111/ejh.14261\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>Thrombotic microangiopathy (TMA), characterized by microangiopathic hemolytic anemia, thrombocytopenia, and multisystem organ dysfunction, is a life-threatening disease. Patients with TMA who do not exhibit a severe ADAMTS-13 deficiency (defined as a disintegrin-like and metalloprotease with thrombospondin type 1 motif no. 13 activity ≥10%: TMA-13n) continue to experience elevated mortality rates. This study explores the prognostic indicators for augmented mortality risk or necessitating chronic renal replacement therapy (composite outcome: CO) in TMA-13n patients. We included 42 TMA-13n patients from January 2008 to May 2018. Median age of 41 years and 60% were female. At presentation, 62% required dialysis, and 57% warranted intensive care unit admission. CO was observed in 45% of patients, including a 9-patient mortality subset. Multivariate logistic regression revealed three independent prognostic factors for CO: early administration of eculizumab (median time from hospitalization to eculizumab initiation: 5 days, range 0–19 days; odds ratio [OR], 0.14; 95% confidence interval [CI], 0.02–0.94), presence of neuroradiological lesions (OR, 6.67; 95% CI, 1.12–39.80), and a PLASMIC score ≤4 (OR, 7.39; 95% CI, 1.18–46.11). In conclusion, TMA-13n patients exhibit a heightened risk of CO in the presence of low PLASMIC scores and neuroradiological lesions, while early eculizumab therapy was the only protective factor.</p>\",\"PeriodicalId\":11955,\"journal\":{\"name\":\"European Journal of Haematology\",\"volume\":\"113 4\",\"pages\":\"510-520\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2024-07-02\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European Journal of Haematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1111/ejh.14261\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Journal of Haematology","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1111/ejh.14261","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Risk factors of death or chronic renal replacement therapy requirements in patients with thrombotic microangiopathies without ADAMTS-13 deficiency
Thrombotic microangiopathy (TMA), characterized by microangiopathic hemolytic anemia, thrombocytopenia, and multisystem organ dysfunction, is a life-threatening disease. Patients with TMA who do not exhibit a severe ADAMTS-13 deficiency (defined as a disintegrin-like and metalloprotease with thrombospondin type 1 motif no. 13 activity ≥10%: TMA-13n) continue to experience elevated mortality rates. This study explores the prognostic indicators for augmented mortality risk or necessitating chronic renal replacement therapy (composite outcome: CO) in TMA-13n patients. We included 42 TMA-13n patients from January 2008 to May 2018. Median age of 41 years and 60% were female. At presentation, 62% required dialysis, and 57% warranted intensive care unit admission. CO was observed in 45% of patients, including a 9-patient mortality subset. Multivariate logistic regression revealed three independent prognostic factors for CO: early administration of eculizumab (median time from hospitalization to eculizumab initiation: 5 days, range 0–19 days; odds ratio [OR], 0.14; 95% confidence interval [CI], 0.02–0.94), presence of neuroradiological lesions (OR, 6.67; 95% CI, 1.12–39.80), and a PLASMIC score ≤4 (OR, 7.39; 95% CI, 1.18–46.11). In conclusion, TMA-13n patients exhibit a heightened risk of CO in the presence of low PLASMIC scores and neuroradiological lesions, while early eculizumab therapy was the only protective factor.
期刊介绍:
European Journal of Haematology is an international journal for communication of basic and clinical research in haematology. The journal welcomes manuscripts on molecular, cellular and clinical research on diseases of the blood, vascular and lymphatic tissue, and on basic molecular and cellular research related to normal development and function of the blood, vascular and lymphatic tissue. The journal also welcomes reviews on clinical haematology and basic research, case reports, and clinical pictures.