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Am J Hematol. 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.

Author information

1
Istituto di Ematologia, Universita' Cattolica, Fondazione Universitaria Policlinico Gemelli, Roma, Italy.
2
Blutspende, Ulm University, Ulm, Germany.
3
Hematology Unity, Istituto Giannina Gaslini, Genova, Italy.
4
Graduate School of Medicine, Department of Pediatric Hematology, Nagoya University, Nagoya, Japan.
5
Pediatric Department, Chinese Academy of Medical Sciences (CAMS), Peking Union Medical College (PUMC), Blood Disease Hospital, Tianjin, China.
6
Mahidol University, Bangkok, Thailand.
7
Pediatric Department, The Catholic University of Korea, Seoul, Korea.
8
Divisione di E di Ematologia, IRCCS AOU San Martino, Genova, Italy.
9
Hematology Department, Chinese Academy of Medical Sciences (CAMS), Peking Union Medical College (PUMC),Blood Disease Hosoital, Tianjin, China.
10
Hospital Universitario de Donostia, San Sebastián, Spain.

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.

PMID:
29498107
PMCID:
PMC6667908
DOI:
10.1002/ajh.25081
[Indexed for MEDLINE]
Free PMC Article

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