Rabbit-antithymocyte globulin combined with cyclosporin A as a first-line therapy: improved, effective, and safe for children with acquired severe aplastic anemia

Chen, Chun; Hou, Xue; Xu, Huilin; Li, Yang; Huang, Ke; Zhou, Dun-Hua; Guo, Haixia; Fang, Jian-Pei; Huang, Shao-Liang

doi:10.1007/s00432-012-1184-4

Cited by 17 publications

(16 citation statements)

References 37 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In the study from Guagzhou, China, 46 children received rabbit ATG at a dose of 2.5 mg/kg/day for 5 days plus cyclosporine. 11 The response rate was 65% at 6 months, which was comparable to results reported in previous studies with horse ATG. 1,2 Regulatory T cells are key elements in suppressing immune responses.…”

Section: © F E R R a T A S T O R T I F O U N D A T I O Nsupporting

confidence: 88%

“…Another issue is the difference in dose of rabbit ATG used in different studies. In the studies from China, 11 Korea, 12 and Spain, 15 the dose of rabbit ATG was 2.5 mg/kg/day for 5 days, which was lower than the standard dose of rabbit ATG in Europe and United States. In the study from Guagzhou, China, 46 children received rabbit ATG at a dose of 2.5 mg/kg/day for 5 days plus cyclosporine.…”

Section: © F E R R a T A S T O R T I F O U N D A T I O Nmentioning

confidence: 90%

Section: © F E R R a T A S T O R T I F O U N D A T I O Nmentioning

confidence: 99%

“…However, several other studies showed comparable response rates between patients treated with horse ATG or rabbit ATG. [11][12][13][14][15] In the European Blood and Marrow Transplant Group study, the best total response rate was not significantly different between groups, that is, 67% versus 60% for horse ATG and rabbit ATG, respectively. 6 Of note, all three retrospective studies from China, 11 Korea, 12 and Japan 13 showed comparable response rates between patients treated with horse ATG or rabbit ATG, which suggests that ethnicity …”

Section: © F E R R a T A S T O R T I F O U N D A T I O Nmentioning

confidence: 99%

See 3 more Smart Citations

Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children

et al. 2013

View full text Add to dashboard Cite

© F e r r a t a S t o r t i F o u n d a t i o nmg/kg/day for at least 6 months), and methylprednisolone (2 mg/kg/day for 5 days) with subsequent halving of the dose every week until discontinuation on day 28. The dose of cyclosporine was adjusted to maintain whole blood concentrations between 150 and 250 ng/mL. Hematologic response was evaluated at 3 and 6 months after the start of therapy. The definitions of complete response, partial response, and relapse have been previously published.1 Briefly, a complete response was defined for all patients as a neutrophil count more than 1.5×10 9 /L, a platelet count more than 100×10 9 /L, and a hemoglobin level more than 11.0 g/dL. A partial response was defined as a neutrophil count more than 0.5×10 9 /L, a platelet count more than 20×10 9 /L, a hemoglobin level more than 80 g/L and no requirement for blood transfusions. Second-line therapies for non-responders to the first immunosuppressive therapy depended on the policies of the individual hospitals.The Mann-Whitney U test was used to compare continuous variables and Pearson χ 2 test was used for categorical variables. Survival rates were calculated using two methods: one indicated when data on patients were censored at the time of stem cell transplantation (transplant-free survival) and the other indicated when data on patients were not censored at the time of transplantation (overall survival).Survival rates were analyzed using the Kaplan-Meier method. Treatment groups were compared with the long-rank test. Cox proportional hazards models were used to assess which factors could predict response as well as risk factors for survival using both univariate and multivariate analyses. The estimated magnitude of the hazard ratio (HR) is shown along with the 95% confidence interval (95% CI). P values less than 0.05 are considered statistically significant.This study was approved by ethics committees of the Catholic University of Korea, Nagoya University Graduate School of Medicine, and Chinese Academy of Medical Science and Pekin Union Medical College. ResultsThe patients' characteristics are shown in Table 1. Overall, 455 patients fulfilled the eligibility criteria; 297 patients received horse ATG and 158 patients received rabbit ATG. The median follow-up periods were 82 months (range, 1 to 215 months) in the horse ATG group and 20 months (range, 1 to 182 months) in the rabbit ATG group. The median age at diagnosis was significantly older in the horse ATG group than in the rabbit ATG group. In addition, percentages of males, very severe aplastic anemia, and hepatitis-associated aplastic anemia were significantly higher in the horse ATG group than in the rabbit ATG group (Table 1).We compared hematologic responses between the horse ATG group and the rabbit ATG group (Table 2). After 3 months, in the horse ATG group, 24 (8%) patients had achieved a complete response and 112 (38%) had achieved a partial response, for an overall response rate of 46%. In the rabbit ATG group, 9 (6%) patients achieved a complete respons...

show abstract

Section: © F E R R a T A S T O R T I F O U N D A T I O Nsupporting

confidence: 88%

Section: © F E R R a T A S T O R T I F O U N D A T I O Nmentioning

confidence: 90%

Section: © F E R R a T A S T O R T I F O U N D A T I O Nmentioning

confidence: 99%

Section: © F E R R a T A S T O R T I F O U N D A T I O Nmentioning

confidence: 99%

See 2 more Smart Citations

Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children

et al. 2013

View full text Add to dashboard Cite

show abstract

“…[60][61][62] Other studies, although not prospective, have failed to show significant differences between horse ATG and rATG. 57,63,64 A large study on more than 800 patients receiving first-line therapy with rATG (Thymoglobulin) is currently being completed.…”

Section: Source Of Atgmentioning

confidence: 99%

How I treat acquired aplastic anemia

Bacigalupo

2017

Blood

330

267

View full text Add to dashboard Cite

Acquired severe aplastic anemia (SAA) is a rare hematologic disease associated with significant morbidity and mortality. Immune destruction of hemopoietic stem cells plays an important role in pathogenesis, as shown by successful treatment with immunosuppressive agents, leading to transfusion independence or complete recovery of peripheral blood counts in a proportion of patients. Growth factors can be combined with immunosuppressive therapy (IST) and may improve response rates, as recently shown with thrombopoietin analogs. Anabolic steroids may still play a role in combination with IST. The problem with IST is failure to respond and the development of late clonal disorders. Bone marrow transplantation (BMT) is the other therapeutic option: a matched sibling donor remains the best choice. For patients lacking a matched family donor, unrelated donors can be readily found, although mostly for patients of Caucasian origin. Other BMT options include unrelated cord blood or mismatched family donors. Acute and chronic graft-versus-host disease remain important complications of BMT. Patient age is a strong predictor of outcome for both IST and BMT, and must be considered when designing therapeutic strategies. Early diagnosis and treatment, as well as long-term monitoring, remain crucial steps for successful treatment of SAA.

show abstract

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

et al. 2018

View full text Add to dashboard Cite

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.

show abstract

Rabbit-antithymocyte globulin combined with cyclosporin A as a first-line therapy: improved, effective, and safe for children with acquired severe aplastic anemia

Abstract: We propose that r-ATG combined with CsA as an intensive IST is effective and safe in treating acquired SAA in children.

Cited by 17 publications

References 37 publications

Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children

Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children

How I treat acquired aplastic anemia

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

Contact Info

Product

Resources

About