Premium
First line treatment of aplastic anemia with thymoglobuline in Europe and Asia: Outcome of 955 patients treated 2001‐2012
Author(s) -
Bacigalupo Andrea,
Oneto Rosi,
Schrezenmeier Hubert,
Hochsmann Britta,
Dufour Carlo,
Kojima Seiji,
Zhu Xiaofan,
Chen Xiaojuan,
Issaragrisil Surapol,
Chuncharunee Suporn,
Jeong Dae Chul,
Giammarco Sabrina,
Van Lint Maria Teresa,
Zheng Yizhou,
Vallejo Carlos
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.25081
Subject(s) - thymoglobulin , medicine , multivariate analysis , confidence interval , aplastic anemia , incidence (geometry) , gastroenterology , population , anemia , anti thymocyte globulin , cumulative incidence , survival analysis , surgery , transplantation , kidney transplantation , bone marrow , physics , environmental health , optics
The aim of this study was to assess the outcome of patients with aplastic anemia (AA), receiving rabbit anti‐thymocyte globulin (Thymoglobulin, SANOFI) and cyclosporin, as first line treatment. Eligible were 955 patients with AA, treated first line with Thymoglobulin, between 2001 and 2008 ( n = 492), or between 2009 and 2012 ( n = 463). The median age of the patients was 21 years (range 1‐84). Mortality within 90 days was 5.7% and 2.4%, respectively in the two time periods ( P = .007).The actuarial 10‐year survival for the entire population was 70%; transplant free survival was 64%. Predictors of survival in multivariate analysis, were severity of the disease, patients age and the interval between diagnosis and treatment. Survival was 87% vs 61% for responders at 6 months versus nonresponders ( P < .0001). The 10‐year survival of nonresponders at 6 months, undergoing a subsequent transplant ( n = 110), was 64%, vs 60% for patient not transplantated ( n = 266) ( P = .1). The cumulative incidence of response was 37%, 52%, 65% respectively, at 90, 180, and 365 days. In multivariate analysis, negative predictors of response at 6 months, were older age, longer interval diagnosis treatment, and greater severity of the disease. In conclusion, early mortality is low after first line treatment of AA with Thymoglobulin, and has been further reduced after year 2008. Patients age, together with interval diagnosis—treament and severity of the disease, remain strong predictors of response and survival.