{
    "abstract": "Acquired Haemophilia A (AHA) patients show a high response rate to immunosuppressive therapy (IST) but few information about predictors of response and outcome are reported.\nWe describe a large single-centre AHA cohort, investigating prognostic variables for the 'best response' (BR), time to BR (TTBR) and overall survival (OS).\nA total of 61 patients were included, collecting data from clinical charts.\nA progressive increase in diagnoses, from 1978 to 2019, was observed. Fifty/56 patients (89%) underwent haemostatic therapy (rFVIIa 46%, aPCC 34%) with no significant differences in the response (rFVIIa 92.3%\u00a0vs aPCC 100%) and no thromboembolic events. Sixty/61 patients underwent first-line IST with an initial response rate of 58.4%. The 12-months OS was 85%, the bleeding associated mortality rate 3% (2/61). The response rates at last observation were: CR 64%, PR 8%. We evaluated the influence of age, gender, associated conditions, IST, haemoglobin levels, FVIII:C, inhibitor titre on BR, TTBR and OS: post-partum AHA achieved the BR after a longer time than AHA related to other aetiologies or idiopathic (p\u00a0=\u00a0.05); in univariate analysis female sex (p\u00a0=\u00a0.03) and the achievement of BR (p\u00a0=\u00a0.001) had a positive impact on the OS while AHA secondary to neoplasms showed a shorter survival (p\u00a0=\u00a0.04); only the BR achievement remained significant in multivariate analysis (p\u00a0=\u00a0.02).\nOur data on response and survival confirmed those from the main registries. Post-partum AHA and BR achievement were significantly associated to a longer TTBR and a longer OS, respectively. Other predictors of outcome deserve to be explored in prospective studies.",
    "authors": [
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Marika",
            "initials": "M",
            "lastname": "Porrazzo"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Erminia",
            "initials": "E",
            "lastname": "Baldacci"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Antonietta",
            "initials": "A",
            "lastname": "Ferretti"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Maria Lucia",
            "initials": "ML",
            "lastname": "De Luca"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Francesco",
            "initials": "F",
            "lastname": "Barone"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Alessandra",
            "initials": "A",
            "lastname": "Serrao"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Simona Michela",
            "initials": "SM",
            "lastname": "Aprile"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Saveria",
            "initials": "S",
            "lastname": "Capria"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Clara",
            "initials": "C",
            "lastname": "Minotti"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Maurizio",
            "initials": "M",
            "lastname": "Martelli"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Maria Gabriella",
            "initials": "MG",
            "lastname": "Mazzucconi"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Antonio",
            "initials": "A",
            "lastname": "Chistolini"
        },
        {
            "affiliation": "Haematology, Department of Translational and Precision Medicine, 'Sapienza' University, Rome, Italy.",
            "firstname": "Cristina",
            "initials": "C",
            "lastname": "Santoro"
        }
    ],
    "conclusions": null,
    "copyrights": "\u00a9 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.",
    "doi": "10.1111/hae.14395",
    "journal": "Haemophilia : the official journal of the World Federation of Hemophilia",
    "keywords": [
        "acquired haemophilia",
        "management",
        "outcome",
        "prognostic factors",
        "response"
    ],
    "methods": null,
    "publication_date": "2021-08-13",
    "pubmed_id": "34382302",
    "results": "A progressive increase in diagnoses, from 1978 to 2019, was observed. Fifty/56 patients (89%) underwent haemostatic therapy (rFVIIa 46%, aPCC 34%) with no significant differences in the response (rFVIIa 92.3%\u00a0vs aPCC 100%) and no thromboembolic events. Sixty/61 patients underwent first-line IST with an initial response rate of 58.4%. The 12-months OS was 85%, the bleeding associated mortality rate 3% (2/61). The response rates at last observation were: CR 64%, PR 8%. We evaluated the influence of age, gender, associated conditions, IST, haemoglobin levels, FVIII:C, inhibitor titre on BR, TTBR and OS: post-partum AHA achieved the BR after a longer time than AHA related to other aetiologies or idiopathic (p\u00a0=\u00a0.05); in univariate analysis female sex (p\u00a0=\u00a0.03) and the achievement of BR (p\u00a0=\u00a0.001) had a positive impact on the OS while AHA secondary to neoplasms showed a shorter survival (p\u00a0=\u00a0.04); only the BR achievement remained significant in multivariate analysis (p\u00a0=\u00a0.02).",
    "title": "Single centre experience on Acquired Haemophilia A patients: Diagnosis, clinical management and analysis of factors predictive of response and outcome.",
    "xml": "<Element 'PubmedArticle' at 0x788502388fe0>"
}