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  1. Article ; Online: AI for AA: machine learning makes an entry.

    Kulasekararaj, Austin G

    Blood

    2023  Volume 141, Issue 17, Page(s) 2040–2042

    MeSH term(s) Humans ; Diagnosis, Differential ; Machine Learning ; Artificial Intelligence ; Bone Marrow Failure Disorders
    Language English
    Publishing date 2023-04-22
    Publishing country United States
    Document type Editorial ; Comment
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2022019444
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: A tribute to Fanconi: 'clinical acumen still counts'.

    Kulasekararaj, Austin G / Gandhi, Shreyans

    Haematologica

    2023  Volume 108, Issue 1, Page(s) 1–2

    Language English
    Publishing date 2023-01-01
    Publishing country Italy
    Document type Editorial
    ZDB-ID 2333-4
    ISSN 1592-8721 ; 0017-6567 ; 0390-6078
    ISSN (online) 1592-8721
    ISSN 0017-6567 ; 0390-6078
    DOI 10.3324/haematol.2022.280868
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: Eltrombopag in Lower-Risk Myelodysplastic Syndrome: Revival of Its Use in Thrombocytopenia.

    Kulasekararaj, Austin G / Trikha, Roochi

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology

    2023  Volume 41, Issue 28, Page(s) 4465–4468

    MeSH term(s) Humans ; Thrombocytopenia/drug therapy ; Benzoates/therapeutic use ; Hydrazines/adverse effects ; Myelodysplastic Syndromes/complications ; Myelodysplastic Syndromes/drug therapy
    Chemical Substances eltrombopag (S56D65XJ9G) ; Benzoates ; Hydrazines
    Language English
    Publishing date 2023-07-10
    Publishing country United States
    Document type Editorial ; Comment
    ZDB-ID 604914-x
    ISSN 1527-7755 ; 0732-183X
    ISSN (online) 1527-7755
    ISSN 0732-183X
    DOI 10.1200/JCO.23.01141
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: Paroxysmal nocturnal hemoglobinuria: Where are we going.

    Kulasekararaj, Austin G / Lazana, Ioanna

    American journal of hematology

    2023  Volume 98 Suppl 4, Page(s) S33–S43

    Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare nonmalignant clonal hematological disorder that is characterized by a deficiency of the GPI-linked complement regulators on the membrane of hematopoietic cells, which renders them susceptible to ... ...

    Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is a rare nonmalignant clonal hematological disorder that is characterized by a deficiency of the GPI-linked complement regulators on the membrane of hematopoietic cells, which renders them susceptible to complement-mediated damage. Intravascular hemolysis (IVH), increased tendency for thrombosis, and bone marrow failure constitutes hallmark features of the disease and are associated with high morbidity and mortality. The introduction of C5 inhibitors radically changed disease outcomes, offering a near-normal life expectancy to PNH patients. However, residual IVH and extravascular hemolysis (EVH) continue to occur during C5-inhibitor treatment, leaving a significant proportion of patients' anemic and some remaining transfusion dependent. Quality of life (QoL) has also been an issue with the regular intravenous (IV) administrations of the currently licensed C5 inhibitors. This has led to the exploration and development of novel agents, targeting different parts of the complement cascade, or having different formulations allowing for self-administration. Longer-acting and subcutaneous formulations of C5 inhibitors have shown equal safety and efficacy, whereas the development of proximal complement inhibitors is changing completely the therapeutic landscape of PNH, limiting both IVH and EVH and showing superior efficacy over C5 inhibitors, especially in improving haemoglobin. Combination treatments have also been tested with promising results. This review summarizes the current therapeutic options, gaps in anti-complement therapy and discusses emerging therapeutic approaches for PNH.
    MeSH term(s) Humans ; Hemoglobinuria, Paroxysmal/drug therapy ; Hemoglobinuria, Paroxysmal/complications ; Quality of Life ; Antibodies, Monoclonal, Humanized/therapeutic use ; Antibodies, Monoclonal, Humanized/pharmacology ; Complement System Proteins/therapeutic use ; Complement Activation ; Hemolysis
    Chemical Substances Antibodies, Monoclonal, Humanized ; Complement System Proteins (9007-36-7)
    Language English
    Publishing date 2023-02-13
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 196767-8
    ISSN 1096-8652 ; 0361-8609
    ISSN (online) 1096-8652
    ISSN 0361-8609
    DOI 10.1002/ajh.26882
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 1251: Show issues Location:
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  5. Article ; Online: Regulatory cells in immune-mediated aplastic anaemia - not T

    Kulasekararaj, Austin G

    British journal of haematology

    2020  Volume 190, Issue 4, Page(s) 486–487

    MeSH term(s) Anemia, Aplastic ; Antigens, CD19 ; B-Lymphocytes, Regulatory/immunology ; Humans ; Interleukin-10 ; T-Lymphocytes, Regulatory/immunology
    Chemical Substances Antigens, CD19 ; Interleukin-10 (130068-27-8)
    Language English
    Publishing date 2020-06-04
    Publishing country England
    Document type Journal Article ; Comment
    ZDB-ID 80077-6
    ISSN 1365-2141 ; 0007-1048
    ISSN (online) 1365-2141
    ISSN 0007-1048
    DOI 10.1111/bjh.16713
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  6. Article ; Online: Limited utility of the HScore in detecting secondary haemophagocytic lymphohistiocytosis in COVID-19: response.

    Wood, Henry / Kulasekararaj, Austin G

    British journal of haematology

    2021  Volume 194, Issue 4, Page(s) 688–689

    MeSH term(s) COVID-19 ; Cytokine Release Syndrome ; Humans ; Lymphohistiocytosis, Hemophagocytic/diagnosis ; SARS-CoV-2
    Language English
    Publishing date 2021-05-16
    Publishing country England
    Document type Letter ; Comment
    ZDB-ID 80077-6
    ISSN 1365-2141 ; 0007-1048
    ISSN (online) 1365-2141
    ISSN 0007-1048
    DOI 10.1111/bjh.17535
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  7. Article: Editorial: Immunologic Mechanisms of Myeloid Neoplasms.

    Fattizzo, Bruno / Da Vià, Matteo Claudio / Kulasekararaj, Austin G

    Frontiers in oncology

    2022  Volume 12, Page(s) 949633

    Language English
    Publishing date 2022-06-15
    Publishing country Switzerland
    Document type Editorial
    ZDB-ID 2649216-7
    ISSN 2234-943X
    ISSN 2234-943X
    DOI 10.3389/fonc.2022.949633
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: Clonal dominance of PNH- another piece to the jigsaw.

    Kulasekararaj, Austin G

    British journal of haematology

    2017  Volume 177, Issue 1, Page(s) 9–10

    Language English
    Publishing date 2017-04
    Publishing country England
    Document type Journal Article
    ZDB-ID 80077-6
    ISSN 1365-2141 ; 0007-1048
    ISSN (online) 1365-2141
    ISSN 0007-1048
    DOI 10.1111/bjh.14552
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  9. Article ; Online: Ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria.

    Lee, Jong Wook / Kulasekararaj, Austin G

    Expert opinion on biological therapy

    2020  Volume 20, Issue 3, Page(s) 227–237

    Abstract: ... ...

    Abstract Introduction
    MeSH term(s) Antibodies, Monoclonal, Humanized/adverse effects ; Antibodies, Monoclonal, Humanized/chemistry ; Antibodies, Monoclonal, Humanized/therapeutic use ; Complement Inactivating Agents/adverse effects ; Complement Inactivating Agents/metabolism ; Complement Inactivating Agents/therapeutic use ; Drug Administration Schedule ; Headache/etiology ; Hemoglobinuria, Paroxysmal/drug therapy ; Hemolysis ; Humans ; Injections, Intraventricular ; Quality of Life ; Treatment Outcome
    Chemical Substances Antibodies, Monoclonal, Humanized ; Complement Inactivating Agents ; ravulizumab (C3VX249T6L)
    Language English
    Publishing date 2020-02-14
    Publishing country England
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Review
    ZDB-ID 2052501-1
    ISSN 1744-7682 ; 1471-2598
    ISSN (online) 1744-7682
    ISSN 1471-2598
    DOI 10.1080/14712598.2020.1725468
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: Second-Generation C5 Inhibitors for Paroxysmal Nocturnal Hemoglobinuria.

    Fattizzo, Bruno / Kulasekararaj, Austin G

    BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy

    2020  Volume 34, Issue 2, Page(s) 149–158

    Abstract: The C5 targeting monoclonal antibody eculizumab has changed the natural history of paroxysmal nocturnal hemoglobinuria (PNH) in the last 10 years. However, some unmet clinical needs persist, including persistent anemia with some patients requiring ... ...

    Abstract The C5 targeting monoclonal antibody eculizumab has changed the natural history of paroxysmal nocturnal hemoglobinuria (PNH) in the last 10 years. However, some unmet clinical needs persist, including persistent anemia with some patients requiring transfusions, incomplete C5 inhibition with breakthrough hemolysis (because of pharmacokinetic or pharmacodynamic issues such as infections, as well as conditions increasing complement activity), the underlying bone marrow failure, and the significant burden on patient quality of life (intravenous route of administration and frequency of infusions). Moreover, a subclass of patients carries C5 polymorphisms resistant to eculizumab inhibition. Several second-generation C5 inhibitors are under active study to overcome unmet clinical needs with eculizumab. Current strategies encompass increasing drug half-life, developing small molecule inhibitors of C5, and exploring new routes of administration (including subcutaneous and oral agents). In this review, we summarize available data on second-generation C5 inhibitors in PNH, including novel monoclonal antibodies, a small interfering RNA, and small molecules.
    MeSH term(s) Animals ; Antibodies, Monoclonal/therapeutic use ; Antibodies, Monoclonal, Humanized/administration & dosage ; Antibodies, Monoclonal, Humanized/adverse effects ; Antibodies, Monoclonal, Humanized/therapeutic use ; Biosimilar Pharmaceuticals/therapeutic use ; Complement C3-C5 Convertases/antagonists & inhibitors ; Complement C3-C5 Convertases/drug effects ; Drug Development ; Hemoglobinuria, Paroxysmal/drug therapy ; Humans ; RNA, Small Interfering/therapeutic use
    Chemical Substances Antibodies, Monoclonal ; Antibodies, Monoclonal, Humanized ; Biosimilar Pharmaceuticals ; RNA, Small Interfering ; eculizumab (A3ULP0F556) ; ravulizumab (C3VX249T6L) ; Complement C3-C5 Convertases (EC 3.4.21.-)
    Language English
    Publishing date 2020-01-08
    Publishing country New Zealand
    Document type Journal Article ; Review
    ZDB-ID 1364202-9
    ISSN 1179-190X ; 1173-8804
    ISSN (online) 1179-190X
    ISSN 1173-8804
    DOI 10.1007/s40259-019-00401-1
    Database MEDical Literature Analysis and Retrieval System OnLINE

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