{"title":"纯红细胞发育不全与胸腺肿瘤相关,一项全国性的回顾性研究。","authors":"Mylène Hemmer,Sylvain Moinard,Olivier Lambotte,Marion Malphettes,Louis Terriou,Bertrand Lioger,Guillaume Le Guenno,Jean-François Viallard,Marc Michel,Hervé Lobbes","doi":"10.1002/ajh.70068","DOIUrl":null,"url":null,"abstract":"Pure red cell aplasia (PRCA) is the most frequent autoimmune cytopenia associated with thymic tumors (TTs). In a nationwide retrospective study, we included 41 patients (22 women, median age 62 years). At PRCA diagnosis, the mean hemoglobin level was 6.6 ± 2.1 g/dL, and the reticulocyte count was 6 ± 5 × 109/L. PRCA was diagnosed before TT (8%, median delay 52 months), simultaneously (46%) or after TT (46%, median delay 34 months). Fourteen patients (34%) had definite Good syndrome. Thymectomy without immunosuppressive treatment provided a single sustainable PRCA response. Twenty-two patients (54%) experienced multiple PRCA relapses (Median 2). When PRCA was present at TT diagnosis, the risk of PRCA relapse was higher (p < 0.01), while TT staging, TT relapse, and Good syndrome were not associated with PRCA relapses. Corticosteroids led to a 77% initial response rate with frequent relapses during taper or at discontinuation. Cyclosporine A provided a 71% response rate. Overall response rates and time to response were similar with corticosteroids and cyclosporine A. Sirolimus led to a 50% response rate in refractory cases. Severe infectious events requiring hospitalizations were frequent (44%). After a mean follow-up of 50 months, five patients (12%) died, three of whom died from TT relapse. Good syndrome was not significantly associated with an increased risk of infection, PRCA relapse, or death. Our findings highlight the severe phenotype of the association of TT and PRCA. While most patients achieve PRCA response under immunosuppressive therapy, high infection incidence and thymoma relapse are responsible for severe morbidity.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"4 1","pages":""},"PeriodicalIF":9.9000,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Pure Red Cell Aplasia Associated With Thymic Tumors, a Nationwide Retrospective Study.\",\"authors\":\"Mylène Hemmer,Sylvain Moinard,Olivier Lambotte,Marion Malphettes,Louis Terriou,Bertrand Lioger,Guillaume Le Guenno,Jean-François Viallard,Marc Michel,Hervé Lobbes\",\"doi\":\"10.1002/ajh.70068\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Pure red cell aplasia (PRCA) is the most frequent autoimmune cytopenia associated with thymic tumors (TTs). In a nationwide retrospective study, we included 41 patients (22 women, median age 62 years). At PRCA diagnosis, the mean hemoglobin level was 6.6 ± 2.1 g/dL, and the reticulocyte count was 6 ± 5 × 109/L. PRCA was diagnosed before TT (8%, median delay 52 months), simultaneously (46%) or after TT (46%, median delay 34 months). Fourteen patients (34%) had definite Good syndrome. Thymectomy without immunosuppressive treatment provided a single sustainable PRCA response. Twenty-two patients (54%) experienced multiple PRCA relapses (Median 2). When PRCA was present at TT diagnosis, the risk of PRCA relapse was higher (p < 0.01), while TT staging, TT relapse, and Good syndrome were not associated with PRCA relapses. Corticosteroids led to a 77% initial response rate with frequent relapses during taper or at discontinuation. Cyclosporine A provided a 71% response rate. Overall response rates and time to response were similar with corticosteroids and cyclosporine A. Sirolimus led to a 50% response rate in refractory cases. Severe infectious events requiring hospitalizations were frequent (44%). After a mean follow-up of 50 months, five patients (12%) died, three of whom died from TT relapse. Good syndrome was not significantly associated with an increased risk of infection, PRCA relapse, or death. Our findings highlight the severe phenotype of the association of TT and PRCA. While most patients achieve PRCA response under immunosuppressive therapy, high infection incidence and thymoma relapse are responsible for severe morbidity.\",\"PeriodicalId\":7724,\"journal\":{\"name\":\"American Journal of Hematology\",\"volume\":\"4 1\",\"pages\":\"\"},\"PeriodicalIF\":9.9000,\"publicationDate\":\"2025-09-08\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"American Journal of Hematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1002/ajh.70068\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"American Journal of Hematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/ajh.70068","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Pure Red Cell Aplasia Associated With Thymic Tumors, a Nationwide Retrospective Study.
Pure red cell aplasia (PRCA) is the most frequent autoimmune cytopenia associated with thymic tumors (TTs). In a nationwide retrospective study, we included 41 patients (22 women, median age 62 years). At PRCA diagnosis, the mean hemoglobin level was 6.6 ± 2.1 g/dL, and the reticulocyte count was 6 ± 5 × 109/L. PRCA was diagnosed before TT (8%, median delay 52 months), simultaneously (46%) or after TT (46%, median delay 34 months). Fourteen patients (34%) had definite Good syndrome. Thymectomy without immunosuppressive treatment provided a single sustainable PRCA response. Twenty-two patients (54%) experienced multiple PRCA relapses (Median 2). When PRCA was present at TT diagnosis, the risk of PRCA relapse was higher (p < 0.01), while TT staging, TT relapse, and Good syndrome were not associated with PRCA relapses. Corticosteroids led to a 77% initial response rate with frequent relapses during taper or at discontinuation. Cyclosporine A provided a 71% response rate. Overall response rates and time to response were similar with corticosteroids and cyclosporine A. Sirolimus led to a 50% response rate in refractory cases. Severe infectious events requiring hospitalizations were frequent (44%). After a mean follow-up of 50 months, five patients (12%) died, three of whom died from TT relapse. Good syndrome was not significantly associated with an increased risk of infection, PRCA relapse, or death. Our findings highlight the severe phenotype of the association of TT and PRCA. While most patients achieve PRCA response under immunosuppressive therapy, high infection incidence and thymoma relapse are responsible for severe morbidity.
期刊介绍:
The American Journal of Hematology offers extensive coverage of experimental and clinical aspects of blood diseases in humans and animal models. The journal publishes original contributions in both non-malignant and malignant hematological diseases, encompassing clinical and basic studies in areas such as hemostasis, thrombosis, immunology, blood banking, and stem cell biology. Clinical translational reports highlighting innovative therapeutic approaches for the diagnosis and treatment of hematological diseases are actively encouraged.The American Journal of Hematology features regular original laboratory and clinical research articles, brief research reports, critical reviews, images in hematology, as well as letters and correspondence.