{"title":"儿童静脉铁治疗:评价异麦芽糖铁和低分子右旋糖酐的安全性和有效性。","authors":"Ernesta Dragūnė, Agnė Morkūnaitė, Gaudas Benediktas Trakymas, Ramunė Vaišnorė, Sonata Šaulytė Trakymienė","doi":"10.1002/pbc.32093","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Iron deficiency (ID) and iron deficiency anemia (IDA) are prevalent among children and adolescents, affecting growth and development. Although oral iron is the first-line treatment, intravenous (IV) iron is required in cases of poor tolerance or inadequate response. While some IV formulations are used in pediatric clinical practice, no published data on the safety and efficacy of iron isomaltoside (IIM) in the pediatric population exist, while data on low-molecular-weight dextran (LMWD) remain limited.</p><p><strong>Procedure: </strong>Our retrospective cohort study at Vilnius University Hospital Santaros Klinikos (2021-2023) included pediatric patients (<18 years) with ID/IDA receiving IIM or LMWD. Hematological response assessment was based on reticulocyte hemoglobin equivalent (Ret-He) and reticulocyte percentage at 3-7 days (early response), and red blood cell count, hemoglobin (HGB), mean corpuscular volume, serum ferritin, and transferrin saturation at 4-6 weeks (late response).</p><p><strong>Results: </strong>Seventy-two patients (IIM: 50, LMWD: 22; 135 infusions), 68% female, with a median age of 13.5 years, were included in the study. Mean HGB increase was 32.4 g/L (IIM) versus 33.1 g/L (LMWD) (p = 0.9). Median ferritin increase was 71.4 µg/L (IIM) versus 117.8 µg/L (LMWD) (p = 0.3). Ret-He improved significantly (p < 0.05). Mild AEs occurred in 5.6%, with similar AE rates in both groups (IIM: 6.0%, LMWD: 4.5%).</p><p><strong>Conclusion: </strong>Our findings suggest that IIM and LMWD are effective and safe IV iron therapies for pediatric ID/IDA. Given the lack of data on the safety and efficacy of IIM in the pediatric population, our findings provide valuable preliminary evidence.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e32093"},"PeriodicalIF":2.3000,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Pediatric Intravenous Iron Therapy: Evaluating the Safety and Efficacy of Iron Isomaltoside and Low-Molecular-Weight Dextran.\",\"authors\":\"Ernesta Dragūnė, Agnė Morkūnaitė, Gaudas Benediktas Trakymas, Ramunė Vaišnorė, Sonata Šaulytė Trakymienė\",\"doi\":\"10.1002/pbc.32093\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Iron deficiency (ID) and iron deficiency anemia (IDA) are prevalent among children and adolescents, affecting growth and development. Although oral iron is the first-line treatment, intravenous (IV) iron is required in cases of poor tolerance or inadequate response. While some IV formulations are used in pediatric clinical practice, no published data on the safety and efficacy of iron isomaltoside (IIM) in the pediatric population exist, while data on low-molecular-weight dextran (LMWD) remain limited.</p><p><strong>Procedure: </strong>Our retrospective cohort study at Vilnius University Hospital Santaros Klinikos (2021-2023) included pediatric patients (<18 years) with ID/IDA receiving IIM or LMWD. Hematological response assessment was based on reticulocyte hemoglobin equivalent (Ret-He) and reticulocyte percentage at 3-7 days (early response), and red blood cell count, hemoglobin (HGB), mean corpuscular volume, serum ferritin, and transferrin saturation at 4-6 weeks (late response).</p><p><strong>Results: </strong>Seventy-two patients (IIM: 50, LMWD: 22; 135 infusions), 68% female, with a median age of 13.5 years, were included in the study. Mean HGB increase was 32.4 g/L (IIM) versus 33.1 g/L (LMWD) (p = 0.9). Median ferritin increase was 71.4 µg/L (IIM) versus 117.8 µg/L (LMWD) (p = 0.3). Ret-He improved significantly (p < 0.05). Mild AEs occurred in 5.6%, with similar AE rates in both groups (IIM: 6.0%, LMWD: 4.5%).</p><p><strong>Conclusion: </strong>Our findings suggest that IIM and LMWD are effective and safe IV iron therapies for pediatric ID/IDA. Given the lack of data on the safety and efficacy of IIM in the pediatric population, our findings provide valuable preliminary evidence.</p>\",\"PeriodicalId\":19822,\"journal\":{\"name\":\"Pediatric Blood & Cancer\",\"volume\":\" \",\"pages\":\"e32093\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2025-09-30\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pediatric Blood & Cancer\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1002/pbc.32093\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Blood & Cancer","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/pbc.32093","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Pediatric Intravenous Iron Therapy: Evaluating the Safety and Efficacy of Iron Isomaltoside and Low-Molecular-Weight Dextran.
Background: Iron deficiency (ID) and iron deficiency anemia (IDA) are prevalent among children and adolescents, affecting growth and development. Although oral iron is the first-line treatment, intravenous (IV) iron is required in cases of poor tolerance or inadequate response. While some IV formulations are used in pediatric clinical practice, no published data on the safety and efficacy of iron isomaltoside (IIM) in the pediatric population exist, while data on low-molecular-weight dextran (LMWD) remain limited.
Procedure: Our retrospective cohort study at Vilnius University Hospital Santaros Klinikos (2021-2023) included pediatric patients (<18 years) with ID/IDA receiving IIM or LMWD. Hematological response assessment was based on reticulocyte hemoglobin equivalent (Ret-He) and reticulocyte percentage at 3-7 days (early response), and red blood cell count, hemoglobin (HGB), mean corpuscular volume, serum ferritin, and transferrin saturation at 4-6 weeks (late response).
Results: Seventy-two patients (IIM: 50, LMWD: 22; 135 infusions), 68% female, with a median age of 13.5 years, were included in the study. Mean HGB increase was 32.4 g/L (IIM) versus 33.1 g/L (LMWD) (p = 0.9). Median ferritin increase was 71.4 µg/L (IIM) versus 117.8 µg/L (LMWD) (p = 0.3). Ret-He improved significantly (p < 0.05). Mild AEs occurred in 5.6%, with similar AE rates in both groups (IIM: 6.0%, LMWD: 4.5%).
Conclusion: Our findings suggest that IIM and LMWD are effective and safe IV iron therapies for pediatric ID/IDA. Given the lack of data on the safety and efficacy of IIM in the pediatric population, our findings provide valuable preliminary evidence.
期刊介绍:
Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.