Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from Korea

Background Autoimmune hemolytic anemia (AIHA) is rare and characterized by hemolytic anemia with a positive direct antiglobulin test result after the exclusion of other causes. While adults often relapse within 1 year of first-line steroid therapy, children generally respond well. However, current t...

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Main Authors: Young Dai Kwon, Eun Sun Jung, Yeon Jung Lim
Format: Article
Language:English
Published: The Korean Pediatric Society 2025-07-01
Series:Clinical and Experimental Pediatrics
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Online Access:http://e-cep.org/upload/pdf/cep-2024-02026.pdf
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author Young Dai Kwon
Eun Sun Jung
Yeon Jung Lim
author_facet Young Dai Kwon
Eun Sun Jung
Yeon Jung Lim
author_sort Young Dai Kwon
collection DOAJ
description Background Autoimmune hemolytic anemia (AIHA) is rare and characterized by hemolytic anemia with a positive direct antiglobulin test result after the exclusion of other causes. While adults often relapse within 1 year of first-line steroid therapy, children generally respond well. However, current treatment approaches lack substantial evidence and are primarily expert opinion-based. Purpose This study aimed to contribute our single-center experience to pediatric AIHA treatment guidelines. Methods Between January 2012 and June 2024, 475 children were diagnosed with anemia; of them, 18 had immune hemolytic anemia, including 6 with neonatal alloimmune hemolytic anemia, 2 who were treated at other centers, and 2 with transient bone marrow suppression due to a viral infection. Thus, this study retrospectively analyzed the treatment responses of 8 patients with AIHA. Results The median age at diagnosis was 5.2 years (range, 2.3–11.8 years); 62.5% (5 of 8) were male. Median hemoglobin (Hb) at diagnosis was 6.3 g/dL (range, 3.4–9.5 g/dL), median reticulocyte index was 6.53% (range, 1.64%–22.07%), median total bilirubin was 2.75 mg/dL (range, 0.98–7.23 mg/dL), and median lactate dehydrogenase was 1,662 U/L (range, 790–2,921 U/L). All haptoglobin levels were <10 mg/dL. Treatments included steroids (8 of 8), red blood cell transfusions (5 of 8), and intravenous immunoglobulins (2 of 8). Half of the steroid-treated patients received intravenous methylprednisolone for 1–5 days, while half received oral prednisolone (median, 1.78 [range, 0.79–3.39] mg/kg/day). The median time to age-adjusted normal Hb levels was 16.5 days (range, 9.0–22.0 days). Steroids were administered for a median 37.5 days (range, 14.0–119.0 days). Excluding one patient later diagnosed with systemic lupus erythematosus, no relapses occurred during the 3- to 19-month follow-up period. Conclusion Patients with pediatric AIHA showed relapsefree rapid hematological improvement and sustained steroid responses within 2 months, suggesting that systematic steroid treatment is feasible and highlighting the need for multicenter trials to establish standardized guidelines.
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spelling doaj-art-e9c0f2d629f842c99dbe8c5848eb3eed2025-08-20T03:28:47ZengThe Korean Pediatric SocietyClinical and Experimental Pediatrics2713-41482025-07-0168752252910.3345/cep.2024.0202620125555813Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from KoreaYoung Dai Kwon0Eun Sun Jung1Yeon Jung Lim2 Department of Pediatrics, Chungnam National University Hospital, Daejeon, Korea Department of Pediatrics, Chungnam National University Hospital, Daejeon, Korea Department of Pediatrics, Chungnam National University Hospital, Daejeon, KoreaBackground Autoimmune hemolytic anemia (AIHA) is rare and characterized by hemolytic anemia with a positive direct antiglobulin test result after the exclusion of other causes. While adults often relapse within 1 year of first-line steroid therapy, children generally respond well. However, current treatment approaches lack substantial evidence and are primarily expert opinion-based. Purpose This study aimed to contribute our single-center experience to pediatric AIHA treatment guidelines. Methods Between January 2012 and June 2024, 475 children were diagnosed with anemia; of them, 18 had immune hemolytic anemia, including 6 with neonatal alloimmune hemolytic anemia, 2 who were treated at other centers, and 2 with transient bone marrow suppression due to a viral infection. Thus, this study retrospectively analyzed the treatment responses of 8 patients with AIHA. Results The median age at diagnosis was 5.2 years (range, 2.3–11.8 years); 62.5% (5 of 8) were male. Median hemoglobin (Hb) at diagnosis was 6.3 g/dL (range, 3.4–9.5 g/dL), median reticulocyte index was 6.53% (range, 1.64%–22.07%), median total bilirubin was 2.75 mg/dL (range, 0.98–7.23 mg/dL), and median lactate dehydrogenase was 1,662 U/L (range, 790–2,921 U/L). All haptoglobin levels were <10 mg/dL. Treatments included steroids (8 of 8), red blood cell transfusions (5 of 8), and intravenous immunoglobulins (2 of 8). Half of the steroid-treated patients received intravenous methylprednisolone for 1–5 days, while half received oral prednisolone (median, 1.78 [range, 0.79–3.39] mg/kg/day). The median time to age-adjusted normal Hb levels was 16.5 days (range, 9.0–22.0 days). Steroids were administered for a median 37.5 days (range, 14.0–119.0 days). Excluding one patient later diagnosed with systemic lupus erythematosus, no relapses occurred during the 3- to 19-month follow-up period. Conclusion Patients with pediatric AIHA showed relapsefree rapid hematological improvement and sustained steroid responses within 2 months, suggesting that systematic steroid treatment is feasible and highlighting the need for multicenter trials to establish standardized guidelines.http://e-cep.org/upload/pdf/cep-2024-02026.pdfanemiaautoimmune hemolytic anemiapediatricssteroids
spellingShingle Young Dai Kwon
Eun Sun Jung
Yeon Jung Lim
Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from Korea
Clinical and Experimental Pediatrics
anemia
autoimmune hemolytic anemia
pediatrics
steroids
title Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from Korea
title_full Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from Korea
title_fullStr Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from Korea
title_full_unstemmed Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from Korea
title_short Treatment and clinical outcomes of pediatric autoimmune hemolytic anemia: real-world single-center data from Korea
title_sort treatment and clinical outcomes of pediatric autoimmune hemolytic anemia real world single center data from korea
topic anemia
autoimmune hemolytic anemia
pediatrics
steroids
url http://e-cep.org/upload/pdf/cep-2024-02026.pdf
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AT eunsunjung treatmentandclinicaloutcomesofpediatricautoimmunehemolyticanemiarealworldsinglecenterdatafromkorea
AT yeonjunglim treatmentandclinicaloutcomesofpediatricautoimmunehemolyticanemiarealworldsinglecenterdatafromkorea