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Nationwide survey on the use of horse antithymocyte globulins (ATGAM) in patients with acquired aplastic anemia: A report on behalf of the French Reference Center for Aplastic Anemia
Author(s) -
Peffault de Latour Régis,
Tabrizi Reza,
Marcais Ambroise,
Leblanc Thierry,
Lamy Thierry,
Mohty Mohamad,
Tavitian Suzanne,
Jubert Charlotte,
Pasquet Marlène,
Galambrun Claire,
Nguyen Stéphanie,
Cahn Jean Yves,
Braun Thorsten,
Deconinck Eric,
Bay Jacques Olivier,
Sicre de Fontbrune Flore,
Barraco Fiorenza,
Socié Gérard
Publication year - 2018
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/ajh.25050
Subject(s) - medicine , aplastic anemia , cohort , eltrombopag , surgery , pediatrics , immune thrombocytopenia , bone marrow , platelet
Abstract Antithymocyte globulins (ATG) plus cyclosporine (CSA) is the gold standard immunosuppressive treatment (IST) for patients with aplastic anemia. A prospective randomized trial showed in 2011 that hATG was superior to rabbit ATG for first‐line treatment of severe AA. The French Health Agency (ANSM) permitted a patient‐named authorization for temporary use (ATU) program of hATG (ATGAM, Pfizer) in patients with AA in 2011 since commercial access to hATG is not approved. We took advantage of this program to analyze the outcomes of 465 patients who received antithymocyte globulins (ATGAM) plus CSA as first line treatment ( n = 379; 81.5%), or for refractory ( n = 26) or relapsed disease ( n = 33), from September 2011 to March 2017. In the entire cohort one year, 72% of the patients had partial and 13% had complete response, with worse response for patients with severe AA and a longer interval between diagnosis and IST (more than 6 months). Severe adverse events were mainly linked to infections (24%), hemorrhages (6%), and elevated liver function tests (5%). Overall at 12 months, 9.7% of patients required second line IST and 15.6% received transplantation. Fifty‐five patients died during the study mainly because of infections (53%). Factors predicting independently worse survival were age over 40 years, neutrophils less than 0.5 × 10 9 /L, male gender and longer delay between diagnosis and hATG (>6 months period). This study does illustrate the results of ATGAM with CSA in a true‐life perspective and confirms ATGAM as standard of care IST to treat patients with AA not eligible for HSCT.