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. 2018 May;93(5):643-648.
doi: 10.1002/ajh.25081. Epub 2018 Mar 23.

First line treatment of aplastic anemia with thymoglobuline in Europe and Asia: Outcome of 955 patients treated 2001-2012

Andrea Bacigalupo  1 , Rosi Oneto  1 , Hubert Schrezenmeier  2 , Britta Hochsmann  2 , Carlo Dufour  3 , Seiji Kojima  4 , Xiaofan Zhu  5 , Xiaojuan Chen  5 , Surapol Issaragrisil  6 , Suporn Chuncharunee  6 , Dae Chul Jeong  7 , Sabrina Giammarco  1 , Maria Teresa Van Lint  8 , Yizhou Zheng  9 , Carlos Vallejo  10
Affiliations

First line treatment of aplastic anemia with thymoglobuline in Europe and Asia: Outcome of 955 patients treated 2001-2012

Andrea Bacigalupo et al. Am J Hematol. 2018 May.

Erratum in

Abstract

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.

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Figures

Figure 1
Figure 1
Ten year overall survival of 955 patients with aplastic anemia (AA) treated with Thymoglobulin and cyclosporine first line
Figure 2
Figure 2
(A) Survival of patients stratified according to age: a significant negative effect is seen for patients above the age of 40 and 60 years. (B) Survival of patients stratified according to the interval between diagnosis and treatment with ATG (in days): a significant advantage for patients treated early. (C) Survival of patients stratified according to response to treatment at 6 months: CR = complete remisson; PR= partial remission; and no response. (D) No difference in survival for patients identified as nonresponders at 6 months, according to whether they were transplanted (HSCT) or not (no HSCT). Transplantation occurred at a median interval of 245 days (range 120‐ 2034) from Thymoglobulin treatment
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References

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