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

Andrea Bacigalupo, Rosi Oneto, Hubert Schrezenmeier, Britta Hochsmann, Carlo Dufour, Seiji Kojima, Xiaofan Zhu, Xiaojuan Chen, Surapol Issaragrisil, Suporn Chuncharunee, Dae Chul Jeong, Sabrina Giammarco, Maria Teresa Van Lint, Yizhou Zheng, Carlos Vallejo

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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.

Original languageEnglish
Pages (from-to)643-648
Number of pages6
JournalAmerican Journal of Hematology
Volume93
Issue number5
DOIs
Publication statusPublished - May 1 2018

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Aplastic Anemia
Survival
Therapeutics
Multivariate Analysis
Transplants
Antilymphocyte Serum
Mortality
Cyclosporine
Rabbits
Incidence
Population
thymoglobulin

ASJC Scopus subject areas

  • Hematology

Cite this

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

In: American Journal of Hematology, Vol. 93, No. 5, 01.05.2018, p. 643-648.

Research output: Contribution to journalArticle

Bacigalupo, A, Oneto, R, Schrezenmeier, H, Hochsmann, B, Dufour, C, Kojima, S, Zhu, X, Chen, X, Issaragrisil, S, Chuncharunee, S, Jeong, DC, Giammarco, S, Van Lint, MT, Zheng, Y & Vallejo, C 2018, 'First line treatment of aplastic anemia with thymoglobuline in Europe and Asia: Outcome of 955 patients treated 2001-2012', American Journal of Hematology, vol. 93, no. 5, pp. 643-648. https://doi.org/10.1002/ajh.25081
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. / First line treatment of aplastic anemia with thymoglobuline in Europe and Asia : Outcome of 955 patients treated 2001-2012. In: American Journal of Hematology. 2018 ; Vol. 93, No. 5. pp. 643-648.
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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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T2 - Outcome of 955 patients treated 2001-2012

AU - Bacigalupo, Andrea

AU - Oneto, Rosi

AU - Schrezenmeier, Hubert

AU - Hochsmann, Britta

AU - Dufour, Carlo

AU - Kojima, Seiji

AU - Zhu, Xiaofan

AU - Chen, Xiaojuan

AU - Issaragrisil, Surapol

AU - Chuncharunee, Suporn

AU - Jeong, Dae Chul

AU - Giammarco, Sabrina

AU - Van Lint, Maria Teresa

AU - Zheng, Yizhou

AU - Vallejo, Carlos

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N2 - 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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