Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children.
Abstract: Patients with severe aplastic anemia (SAA) and an absolute neutrophil count (ANC) of 0 typically have fatal outcomes. We defined fulminant AA (FAA) as ANC = 0 for at least 2 weeks prior to and after immunosuppressive therapy (IST). We analyzed the outcomes of 35 children with FAA among 288 children who enrolled in a prospective study for AA (AA-97 study). AA was classified as FAA (n = 35), very SAA (vSAA; n = 129), or SAA (n = 124). All of the children received the IST with horse anti-thymocyte globulin (ATG) and cyclosporine (CsA). A significantly lower response rate at 6 months was seen in children with FAA when compared to those with vSAA or SAA (40.0, 63.6, and 63.7 %, respectively; p = 0.027). Of 20 nonresponder patients in the FAA group, 11 were rescued by alternative donor transplantation, and 5 patients showed a late response after 6 months. Consequently, no significant difference was noted in overall survival when comparing the FAA, vSAA, and SAA groups (88.5, 95.8, and 96.8 %). These findings indicate that IST with ATG and CsA is justified as a first-line treatment for children with FAA who lack a human leukocyte antigen-matched sibling donor.
Publication Date: 2013-12-14 PubMed ID: 24337487DOI: 10.1007/s00277-013-1984-xGoogle Scholar: Lookup
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- Journal Article
Summary
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The research article presents a study on the treatment of children experiencing severe forms of aplastic anemia using a specific immuno-suppressive therapy. Aplastic anemia is a life-threatening condition, and the researchers sought to investigate the efficacy of a combined treatment using horse anti-thymocyte globulin and cyclosporine in improving patient outcomes.
Objective and Scope of the Study
- This study investigated the efficiency of a combination of two drugs, anti-thymocyte globulin (ATG) and cyclosporine (CsA), as a first-line treatment for children with fulminant aplastic anemia (FAA) who do not have a matching human leukocyte antigen sibling donor.
- The research included 35 children who had FAA, a total of 288 children were enrolled in the study, the other two groups were classified as very severe aplastic anemia (vSAA) and severe aplastic anemia (SAA).
Methodology
- The definition of FAA applied in the study was characterized by an absolute neutrophil count (ANC) of zero for at least two weeks before and after the application of immuno-suppressive therapy (IST).
- All the children participating in the study received IST, which included the administration of ATG and CsA.
Findings
- The study found that children with FAA had a lower response rate at the 6-month mark in comparison to children with vSAA or SAA. The response rates were 40.0% for FAA, 63.6% for vSAA, and 63.7% for SAA.
- However, of the 20 non-responder patients in the FAA group, 11 were recuperated through alternative donor transplantation, and 5 patients displayed a late response after six months.
- Thus, despite the lower initial response rate, the overall survival rate did not significantly differ when comparing the FAA, vSAA, and SAA groups (88.5%, 95.8%, and 96.8%, respectively).
Conclusion
- The results of this study show the effectiveness of a combination therapy using ATG and CsA as a first-line treatment for children with fulminant aplastic anemia who do not have a human leukocyte antigen-matched sibling donor.
Cite This Article
APA
Yagasaki H, Shichino H, Ohara A, Kobayashi R, Yabe H, Ohga S, Hamamoto K, Ohtsuka Y, Shimada H, Inoue M, Muramatsu H, Takahashi Y, Kojima S.
(2013).
Immunosuppressive therapy with horse anti-thymocyte globulin and cyclosporine as treatment for fulminant aplastic anemia in children.
Ann Hematol, 93(5), 747-752.
https://doi.org/10.1007/s00277-013-1984-x Publication
Researcher Affiliations
- Department of Pediatrics, School of Medicine, Nihon University, 30-1 Ohyaguchi-Kamicho, Itabashi-ku, 173-8610, Tokyo, Japan, yagasaki.hiroshi@nihon-u.ac.jp.
MeSH Terms
- Acute Disease
- Adolescent
- Anemia, Aplastic / immunology
- Anemia, Aplastic / mortality
- Anemia, Aplastic / pathology
- Anemia, Aplastic / therapy
- Animals
- Antilymphocyte Serum / therapeutic use
- Child
- Child, Preschool
- Cyclosporine / therapeutic use
- Female
- Hematopoietic Stem Cell Transplantation
- Histocompatibility Testing
- Horses
- Humans
- Immunosuppression Therapy
- Immunosuppressive Agents / therapeutic use
- Male
- Neutrophils / drug effects
- Neutrophils / immunology
- Neutrophils / pathology
- Prospective Studies
- Severity of Illness Index
- Survival Analysis
- Treatment Outcome
Citations
This article has been cited 7 times.- Wu LQ, Huang LF, Yang H, Ye BD, Sheng JP, Yu QH, Yang Y, Jia JS, Zhang DH, Lin SY, He GS, Li JY. Comparison of haploidentical-allogeneic hematopoietic stem cell transplantation and intensive immunosuppressive therapy for patients with severe aplastic anemia with an absolute neutrophil count of zero: a retrospective study. Ann Hematol 2023 Aug;102(8):2015-2023.
- Liu X, Yang W, Zhang L, Jing L, Ye L, Zhou K, Li Y, Li J, Fan H, Yang Y, Xiong Y, Zhao X, Zhang F. Development and validation of early death risk score model for emergency status prediction in very severe aplastic anemia. Front Immunol 2023;14:1175048.
- Jia JS. [Prediction and treatment prospect of immunosuppressive therapy in patients with severe aplastic anemia]. Zhonghua Xue Ye Xue Za Zhi 2020 Oct 14;41(10):874-877.
- Koh S, Koh H, Kubo Y, Kuroda M, Nishimoto M, Yoshimura T, Nakashima Y, Nakane T, Nakamae H, Ohsawa M, Hino M. An Elderly Woman with Anti-neutrophil Antibody-positive Agranulocytosis Who Responded to High-dose Intravenous Methylprednisolone. Intern Med 2017 Aug 15;56(16):2199-2203.
- Li Y, Wu Z, Zhao X, Zhang L, Jing L, Zhou K, Peng G, Ye L, Li Y, Li J, Fan H, Song L, Yang W, Jiang B, Zhang F. [Outcomes of very severe aplastic anemia patients with different absolute neutrophil counts after frontline immnunosuppressive therapy]. Zhonghua Xue Ye Xue Za Zhi 2016 Apr;37(4):329-33.
- Morita-Fujita M, Yabushita T, Yoshioka S, Hiramoto N, Ishikawa T. Salvage UCBT with Short-Term Melphalan-based Reduced-Intensity Conditioning for Primary Graft Failure after Upfront UCBT for Fulminant Aplastic Anemia. Blood Cell Ther 2025 May 25;8(2):190-194.
- Nakamura Y, Zaimoku Y, Yamaguchi H, Yamazaki H, Kanaya M, Uchida N, Doki N, Sakurai M, Hiramoto N, Kako S, Onizuka M, Onodera K, Maruyama Y, Ohigashi H, Nishida T, Yoshihara S, Matsuoka KI, Eto T, Kanda Y, Fukuda T, Atsuta Y, Onishi Y. Significance of absolute neutrophil count before allogeneic hematopoietic stem cell transplantation in adult patients with aplastic anemia. Ann Hematol 2024 Aug;103(8):3121-3133.
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