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Master Thesis

Observatoire des patients atteints d'aplasie médullaire dans le service d'hématologie adulte du CHU de Bordeaux des années 1990 à nos jours : étude des traitements de première ligne et évaluation des pratiques médicales

Abstract : Aplastic Anemia (AM) is a rare disease, linked to a constitutional (AMC) or acquired (AMA) medullar insufficiency. We have listed 70 patients treated in the adult hematology department at the Bordeaux University Hospital between 1989 and January 1, 2020. Only one patient had a AMC, a Fanconi disease. The median age was 49 years [10;85], and the sex ratio was 0.47. A HPN clone was detected in 29 % of patient at diagnosis.14% of patients had moderate AM with transfusion dependence, 60% had severe AM and 26% had very severe AM by Camitta criteria. The median follow-up was 52 months. Eleven patients (16%) received first-line allografts, 47 patients (67%) received treatment with Ciclosporine (CsA) ± Trombopoietin Receptor Agonist (ARTPO) Anti-Lymphocyte Serum (SAL), 10 patients (14%) received CsA ± ARTPO and 2 patients received androgens. The Complete Response (CR) rate was 91% for allograft patients vs. 47% for others (p=0.008), with overall survival of 91% vs. 76% at 5 years respectively. 53% of patients treated with SAL-CsA achieved a CR, vs. 30% in the CsA group alone. Eleven patients received first-line ARTPO in addition to standard therapy, 73% achieved CR and 91% achieved overall response. The median time to best response was 12 months vs. 15 months (p=0.83) for the SAL-CsA and non-SAL groups, respectively. Of the 57 patients who received immunosuppressive therapy, 18 patients had regular bone marrow monitoring, of which 3 (4%) showed clonal progression.
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https://dumas.ccsd.cnrs.fr/dumas-03113875
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Submitted on : Monday, January 18, 2021 - 4:09:14 PM
Last modification on : Saturday, February 27, 2021 - 3:10:59 AM

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Léa Greub. Observatoire des patients atteints d'aplasie médullaire dans le service d'hématologie adulte du CHU de Bordeaux des années 1990 à nos jours : étude des traitements de première ligne et évaluation des pratiques médicales. Sciences du Vivant [q-bio]. 2020. ⟨dumas-03113875⟩

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