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Master Thesis

Greffe de donneurs phéno-identiques en traitement de première intention de l’aplasie médullaire idiopathique : une étude à partir du registre du SAAWP de l’EBMT

Abstract : Introduction: Young patients with idiopathic aplastic anemia (AA) respond better to immunosuppressive therapy (IST) but the long-term outcome is suboptimal. Excellent results of up-front unrelated donor (UD) hematopoietic stem cell transplantation (HSCT) have been reported (Dufour C, BJH 2015). We took advantage of the SAAWP registry of the European Blood and Marrow Transplantation (EBMT) to analyze the outcome of 70 patients who received up-front UD HSCT in Europe. Methods: Patients who had received an UD HSCT for AA, between 2010 and 2018, registered in the SAAWP registry were included. Patients who had received IST (CSA or anti-thymocyte globulin (ATG)) before HSCT, cord blood, haplo-identical transplant were excluded as well as patients suffering from congenital bone marrow disorders. The primary endpoint was overall survival (OS) at 2 years. Secondary endpoints were GVHD-free/relapse-free survival (GRFS). Results: Seventy-seven patients were included; median age was 19 years old (11-33). Time to HSCT was 7 months (IQR 3.7-11.9). The two-year overall survival rate was 90% (95% CI, 84-97%). Main cause of death was infection. Graft failure occurred in 9% (3-16%) of the patients. Acute GVHD grade II-IV was observed in 11% (4-18%) of the patients and Grade III-IV happened occured only in 2 cases. Chronic GVHD at 2 years occurred in 15% (6-23%) of the patients, with no extensive case . GRFS was 85% (76-94%) at 2-years. Conclusion: Up-front UD transplantation leads to promising results, confirming previous studies on a smaller cohort of patients. Because of obvious limitation related to the retrospective studiessetting of our study, unreported events and information bias cannot be excluded.
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-03087066
Contributor : Jean-Hugues Morneau Connect in order to contact the contributor
Submitted on : Wednesday, December 23, 2020 - 11:58:57 AM
Last modification on : Wednesday, January 6, 2021 - 3:24:59 AM

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  • HAL Id : dumas-03087066, version 1
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Audrey Françoise Petit. Greffe de donneurs phéno-identiques en traitement de première intention de l’aplasie médullaire idiopathique : une étude à partir du registre du SAAWP de l’EBMT. Médecine humaine et pathologie. 2020. ⟨dumas-03087066⟩

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