{"title":"再生障碍性贫血免疫抑制治疗后的结果：来自印度北部的单一中心经验。","authors":"Nitish Kumar, Alok Hemal, Aditya Sangwan, Shilpa Khanna Arora","doi":"10.1007/s12288-024-01913-9","DOIUrl":null,"url":null,"abstract":"<p><strong>Purpose: </strong>Aplastic anemia (AA) in children is a life-threatening disorder with very few therapeutic options. This study was carried out to assess the response of Immunosuppressive therapy (IST) in children with non-inherited AA.</p><p><strong>Methods: </strong>It was a prospective observational study carried out among 35 children aged less than 18 years, recently diagnosed with AA by bone marrow biopsy. Those suffering from inherited bone marrow failure syndromes and secondary AA were excluded. IST included equine anti-thymocyte globulin (eATG), steroids, and cyclosporine. Response to immunosuppressive therapy was assessed after four months.</p><p><strong>Results: </strong>The mean age was 10.77 ± 3.6 years. Non-severe AA was diagnosed in 65.71% of children with severe and very severe AA in 17.14%. The overall response rate was found to be 65.7%, with 54.28% of children achieving a partial response and 11.42% of children achieving a complete response. Whereas 17.14% did not respond to treatment, and another 17.14% of children expired before the time point of response assessment. Hemoglobin levels, total leucocyte count, platelet count, and reticulocyte count showed statistically significant elevation following IST.</p><p><strong>Conclusion: </strong>IST can be safely and effectively used as the first-line treatment option in children with non-inherited aplastic anemia, especially when stem cell transplant is not a feasible option.</p>","PeriodicalId":49188,"journal":{"name":"Indian Journal of Hematology and Blood Transfusion","volume":"41 3","pages":"579-584"},"PeriodicalIF":0.6000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12267709/pdf/","citationCount":"0","resultStr":"{\"title\":\"Outcomes after Immunosuppressive Therapy for Aplastic Anemia: A Single Centre Experience from Northern India.\",\"authors\":\"Nitish Kumar, Alok Hemal, Aditya Sangwan, Shilpa Khanna Arora\",\"doi\":\"10.1007/s12288-024-01913-9\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Purpose: </strong>Aplastic anemia (AA) in children is a life-threatening disorder with very few therapeutic options. This study was carried out to assess the response of Immunosuppressive therapy (IST) in children with non-inherited AA.</p><p><strong>Methods: </strong>It was a prospective observational study carried out among 35 children aged less than 18 years, recently diagnosed with AA by bone marrow biopsy. Those suffering from inherited bone marrow failure syndromes and secondary AA were excluded. IST included equine anti-thymocyte globulin (eATG), steroids, and cyclosporine. Response to immunosuppressive therapy was assessed after four months.</p><p><strong>Results: </strong>The mean age was 10.77 ± 3.6 years. Non-severe AA was diagnosed in 65.71% of children with severe and very severe AA in 17.14%. The overall response rate was found to be 65.7%, with 54.28% of children achieving a partial response and 11.42% of children achieving a complete response. Whereas 17.14% did not respond to treatment, and another 17.14% of children expired before the time point of response assessment. Hemoglobin levels, total leucocyte count, platelet count, and reticulocyte count showed statistically significant elevation following IST.</p><p><strong>Conclusion: </strong>IST can be safely and effectively used as the first-line treatment option in children with non-inherited aplastic anemia, especially when stem cell transplant is not a feasible option.</p>\",\"PeriodicalId\":49188,\"journal\":{\"name\":\"Indian Journal of Hematology and Blood Transfusion\",\"volume\":\"41 3\",\"pages\":\"579-584\"},\"PeriodicalIF\":0.6000,\"publicationDate\":\"2025-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12267709/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Indian Journal of Hematology and Blood Transfusion\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s12288-024-01913-9\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/10/29 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q4\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Indian Journal of Hematology and Blood Transfusion","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s12288-024-01913-9","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/10/29 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Outcomes after Immunosuppressive Therapy for Aplastic Anemia: A Single Centre Experience from Northern India.
Purpose: Aplastic anemia (AA) in children is a life-threatening disorder with very few therapeutic options. This study was carried out to assess the response of Immunosuppressive therapy (IST) in children with non-inherited AA.
Methods: It was a prospective observational study carried out among 35 children aged less than 18 years, recently diagnosed with AA by bone marrow biopsy. Those suffering from inherited bone marrow failure syndromes and secondary AA were excluded. IST included equine anti-thymocyte globulin (eATG), steroids, and cyclosporine. Response to immunosuppressive therapy was assessed after four months.
Results: The mean age was 10.77 ± 3.6 years. Non-severe AA was diagnosed in 65.71% of children with severe and very severe AA in 17.14%. The overall response rate was found to be 65.7%, with 54.28% of children achieving a partial response and 11.42% of children achieving a complete response. Whereas 17.14% did not respond to treatment, and another 17.14% of children expired before the time point of response assessment. Hemoglobin levels, total leucocyte count, platelet count, and reticulocyte count showed statistically significant elevation following IST.
Conclusion: IST can be safely and effectively used as the first-line treatment option in children with non-inherited aplastic anemia, especially when stem cell transplant is not a feasible option.
期刊介绍：
Indian Journal of Hematology and Blood Transfusion is a medium for propagating and exchanging ideas within the medical community. It publishes peer-reviewed articles on a variety of aspects of clinical hematology, laboratory hematology and hemato-oncology. The journal exists to encourage scientific investigation in the study of blood in health and in disease; to promote and foster the exchange and diffusion of knowledge relating to blood and blood-forming tissues; and to provide a forum for discussion of hematological subjects on a national scale.
The Journal is the official publication of The Indian Society of Hematology & Blood Transfusion.
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