Intravenous ferric carboxymaltose for the management of iron deficiency and iron deficiency anaemia in children and adolescents: a review

Abstract

Iron deficiency is the primary cause of anaemia worldwide and is particularly common among children and adolescents. Intravenous (IV) iron therapy is recommended for paediatric patients with certain comorbidities or if oral iron treatment has been unsuccessful. IV ferric carboxymaltose (FCM) has recently been approved by the US Food and Drug Administration for use in children aged > 1 year. This narrative review provides an overview of the available publications on the efficacy and safety of IV FCM in children and adolescents. A literature search using PubMed and Embase yielded 153 publications; 33 contained clinical data or reports on clinical experience relating to IV FCM in subjects < 18 years of age and were included in the review. No prospective, randomised controlled studies on the topic were found. Most publications were retrospective studies or case reports and included patients with various underlying conditions or patients with inflammatory bowel disease. Efficacy data were included in 27/33 publications and improvements in anaemia, and/or iron status parameters were reported in 26 of them. Safety data were included in 25/33 publications and were in line with the adverse events described in the prescribing information.Conclusion: The available publications indicate that IV FCM, a nanomedicine with a unique and distinctive therapeutic profile, is an effective and generally well-tolerated treatment for iron deficiency or iron deficiency anaemia in children and adolescents. Despite the wealth of retrospective evidence, prospective, randomised controlled trials in the paediatric setting are still necessary. What is Known:• Iron deficiency and iron deficiency anaemia are usually managed using oral iron therapy, but intravenous iron therapy is recommended for certain paediatric patients.• Intravenous ferric carboxymaltose (FCM) has recently been approved in the US for use in children aged > 1 year. What is New:• Despite evidence that FCM is effective and generally well tolerated in children and adolescents, so far, only retrospective studies, non-randomised uncontrolled prospective studies, or case reports have been published in full.• There is a strong need for prospective, randomised controlled trials on FCM in the paediatric setting.

Parenteral iron therapy in children with iron deficiency anemia: A single‐institution experience

“…However, in the notable absence of large randomized clinical trials, these limitations do not undermine the relevance of this study. 9 Our study confirms previously published observations on the efficacy and safety profile of IV iron. [1][2][3][4][5][6]10 In noncritically ill children, the oral absorption test is easy to conduct and can readily evaluate intestinal iron absorption.…”

Management of Childhood Iron Deficiency Anemia in a Developed Country—A Multi-Center Experience from Croatia

“…Since multiple studies report ferric carboxymaltose preparations as a safe, well-tolerated and efficacious measure to treat IDA in children who fail to respond to oral iron therapy regardless of the etiology, pediatric hematologists should not refrain from their use [51][52][53]. Nevertheless, bearing in mind the average age of our study's participants, the underrepresentation of parenteral iron as a therapeutic option is not at all surprising given the fact that ferric carboxymaltose has only recently been approved for use in children above 1 year of age in Croatia, as in the United States [50]. Although iron sucrose has proved equally effective with an acceptable safety profile, according to the drug's prescription instructions, its use in children is still not encouraged in Croatia [54,55].…”

“…In women with heavy menstrual bleeding, intravenous iron as first-line therapy is recommended in cases of heavy anemia or those necessitating prompt improvement, e.g., before urgent surgery, and as a second-line therapy in cases of poor adherence, intolerability or deficient therapy response [49]. Unsuccessful oral iron therapy in children is likewise considered a justified indication for parenteral therapy, and in certain groups of patients, for example, children with chronic kidney and gastrointestinal disease and those on extended parenteral nutrition, intravenous iron formulations provide an appropriate upfront therapeutic alternative [50]. Menorrhagia was the principal reason for parenteral iron therapy in the Rijeka center, where most of the intravenous iron medications were administered.…”