Introduction

Intravenous iron (IVI) therapy utilization for treatment of iron deficiency in children has increased in the recent years. The commonly used formulations in children include iron dextran (IDx), ferric carboxymaltose (FCM), iron sucrose (IS), and ferric gluconate (FG), with each formulation having a different dosing regimen and cost. The cost versus efficacy analysis of various IVI formulations has not been previously assessed in children. We aimed to compare the efficacy across different IVI formulations accounting for their costs.

Methods

A retrospective chart review was conducted of subjects ≤21 years of age, who received at least one dose of IVI therapy between July 2018 and June 2024 at a tertiary children's hospital. Subjects receiving IVI as a constituent of parenteral nutrition or undergoing dialysis were excluded. Data collected included demographics, IVI formulation with dosage amount and number of doses within each treatment, hemoglobin (Hgb) and ferritin levels 1 month before, and levels after (Hgb: 7-30 days after, ferritin 7-120 days after) IVI treatment. Efficacy of each individual treatment was determined based on change in Hgb and ferritin. Cost per treatment was calculated as the product of patient charge per vial multiplied by number of vials utilized for the doses within a given treatment. Efficacy across different IVI formulations was compared using Kruskal Wallis test and cost analysis was performed using a linear mixed model to account for multiple treatments per patient.

Results

A total of 833 doses were administered [326 (39%) IS, 229 (27%) IDx, 229 (27%) FCM, and 49 (6%) FG] across 659 treatment courses among 342 subjects with median age 14 years (range 0-21 years old), 68% female, and with indications for IVI including poor absorption (380, 57.7%), ongoing blood loss (154, 23.4%), oral iron refractoriness (124, 18.8%), oral iron intolerance (79, 12%), inability to take oral medications (37, 5.6%), patient preference (27, 4.1%) and poor compliance (15, 2.3%). The number of doses per treatment varied across different IVI formulations (1-2 doses for FG and IDx, 1-3 doses for FCM, and 1-6 doses for IS). The overall median (IQR) improvement in Hgb was 1.2 g/dL (0.1, 2.5), which was higher for IDx at 1.9g/dl compared to FCM at 1.3g/dl (p= 0.399), FG at 0.6g/dl (p=0.051) and IS at 0.7g/dl (p=0.001). The overall median (IQR) improvement in ferritin was 20 ng/mL (3, 96), which was significantly higher for IDx at 28ng/ml as well as FCM at 101ng/ml compared to FG at 5ng/ml and IS at 6ng/ml (p values <0.05). The ferritin rise was higher with FCM compared to IDx (p< 0.001). Median cost (IQR) per treatment was $4959 ($4959, $9918) for FCM, $3569 ($1586, $3965) for IDx, $416 ($416, $831) for IS and $198 ($198, $198) for FG. Accounting for the cost of each IVI formulation, IDx improved Hgb and ferritin the most compared to IS.

Conclusions

IDx and FCM had higher cost, but better efficacy compared to FG and IS. Comparative analysis of cost versus efficacy showed IDx surpassing all other IVI formulations with greater improvement in both Hgb and ferritin compared to other formulations and less cost compared to FCM. IDx also offers a simpler dosing regimen as single dose-per-treatment. Overall, IDx may be the preferred IVI formulation in children based on our center's experience. Future multi-center studies are needed to confirm our results.

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2025
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