Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy

Abstract: Immune aplastic anemia (iAA) frequently results in transfusion dependence on platelets and packed red blood cells, increasing the risk for complications. The most common immune-mediated cause for platelet-transfusion refractoriness is alloimmunization with HLA antibody (Ab) to nonself clas...

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Main Authors: Jibran Durrani, Leonard N. Chen, Ruba N. Shalhoub, Valentina Baez, Joshua Glass, Nu Ri Cha, Xiaoyang Ma, Zhijie Wu, Shouguo Gao, Jennifer Lotter, Olga Rios, Colin O. Wu, Willy A. Flegel, Neal S. Young, Emma M. Groarke, Bhavisha A. Patel
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925001612
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author Jibran Durrani
Leonard N. Chen
Ruba N. Shalhoub
Valentina Baez
Joshua Glass
Nu Ri Cha
Xiaoyang Ma
Zhijie Wu
Shouguo Gao
Jennifer Lotter
Olga Rios
Colin O. Wu
Willy A. Flegel
Neal S. Young
Emma M. Groarke
Bhavisha A. Patel
author_facet Jibran Durrani
Leonard N. Chen
Ruba N. Shalhoub
Valentina Baez
Joshua Glass
Nu Ri Cha
Xiaoyang Ma
Zhijie Wu
Shouguo Gao
Jennifer Lotter
Olga Rios
Colin O. Wu
Willy A. Flegel
Neal S. Young
Emma M. Groarke
Bhavisha A. Patel
author_sort Jibran Durrani
collection DOAJ
description Abstract: Immune aplastic anemia (iAA) frequently results in transfusion dependence on platelets and packed red blood cells, increasing the risk for complications. The most common immune-mediated cause for platelet-transfusion refractoriness is alloimmunization with HLA antibody (Ab) to nonself class I antigens. The clinical impact of the HLA alloimmunization has not been well studied in patients with iAA. We investigated the clinical relevance of HLA alloimmunization in our large cohort of patients with iAA from 5 prospective trials and correlated with disease outcomes. Of 444 patients with severe AA treated with immunosuppressive therapy (IST), 99 (22%) had HLA alloimmunization. The presence of HLA Ab was associated with shorter overall survival, reduced responses to IST and higher risk of clonal evolution. Our data suggest that HLA alloimmunization is a marker of disease outcome. Furthermore, using single-cell RNA sequencing, we show enhanced activation of both complement-mediated pathways and the adaptive immune system in alloimmunized patients, indicating an interconnection between immune compartments.
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issn 2473-9529
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publisher Elsevier
record_format Article
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spelling doaj-art-cc2be64b50eb4caea2b273e4c8a526fb2025-08-20T03:12:05ZengElsevierBlood Advances2473-95292025-06-019112639265010.1182/bloodadvances.2024015301Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapyJibran Durrani0Leonard N. Chen1Ruba N. Shalhoub2Valentina Baez3Joshua Glass4Nu Ri Cha5Xiaoyang Ma6Zhijie Wu7Shouguo Gao8Jennifer Lotter9Olga Rios10Colin O. Wu11Willy A. Flegel12Neal S. Young13Emma M. Groarke14Bhavisha A. Patel15Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD; Division of Hematology, Sylvester Comprehensive Cancer Center, University of Miami, Miami, FLBlood Services Section, Department of Transfusion Medicine, National Institutes of Health Clinical Center, Bethesda, MDOffice of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDLaboratory Services Section, Department of Transfusion Medicine, National Institutes of Health Clinical Center, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD; Division of Hematology, Cancer and Blood Disorders Center, Children’s National Hospital, Washington, DCLaboratory Services Section, Department of Transfusion Medicine, National Institutes of Health Clinical Center, Bethesda, MDOffice of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDOffice of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDLaboratory Services Section, Department of Transfusion Medicine, National Institutes of Health Clinical Center, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MDHematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD; Correspondence: Bhavisha A. Patel, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, 10 Center Drive, Room 3-5140, Bethesda, MD 20892;Abstract: Immune aplastic anemia (iAA) frequently results in transfusion dependence on platelets and packed red blood cells, increasing the risk for complications. The most common immune-mediated cause for platelet-transfusion refractoriness is alloimmunization with HLA antibody (Ab) to nonself class I antigens. The clinical impact of the HLA alloimmunization has not been well studied in patients with iAA. We investigated the clinical relevance of HLA alloimmunization in our large cohort of patients with iAA from 5 prospective trials and correlated with disease outcomes. Of 444 patients with severe AA treated with immunosuppressive therapy (IST), 99 (22%) had HLA alloimmunization. The presence of HLA Ab was associated with shorter overall survival, reduced responses to IST and higher risk of clonal evolution. Our data suggest that HLA alloimmunization is a marker of disease outcome. Furthermore, using single-cell RNA sequencing, we show enhanced activation of both complement-mediated pathways and the adaptive immune system in alloimmunized patients, indicating an interconnection between immune compartments.http://www.sciencedirect.com/science/article/pii/S2473952925001612
spellingShingle Jibran Durrani
Leonard N. Chen
Ruba N. Shalhoub
Valentina Baez
Joshua Glass
Nu Ri Cha
Xiaoyang Ma
Zhijie Wu
Shouguo Gao
Jennifer Lotter
Olga Rios
Colin O. Wu
Willy A. Flegel
Neal S. Young
Emma M. Groarke
Bhavisha A. Patel
Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy
Blood Advances
title Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy
title_full Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy
title_fullStr Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy
title_full_unstemmed Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy
title_short Impact of HLA alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy
title_sort impact of hla alloimmunization on clinical outcomes of severe aplastic anemia treated with immunosuppressive therapy
url http://www.sciencedirect.com/science/article/pii/S2473952925001612
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