Academic Journal
Safety and Efficacy of Oral Ferric Maltol and Ferrous Sulfate for Iron Deficiency Anemia in Infants, Children and Adolescents: A Multicenter Randomized Trial.
| Τίτλος: | Safety and Efficacy of Oral Ferric Maltol and Ferrous Sulfate for Iron Deficiency Anemia in Infants, Children and Adolescents: A Multicenter Randomized Trial. |
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| Συγγραφείς: | Gass D; Atrium Health, Levine Children's Hospital, Charlotte, USA., Freiberg A; Penn State Health, Lancaster, USA., Auth MKH; Alder Hey Hospital, Children's NHS Foundation Trust, Liverpool, UK.; University of Liverpool, Liverpool, UK., Russell RK; NHS Scotland, Edinburgh, UK. richard.russell@nhs.scot.; Department of Paediatric Gastroenterology, Royal Hospital for Children and Young People, Clinical Staff Offices, 2nd Floor, 50 Little France Crescent, Edinburgh, EH16 4TJ, UK. richard.russell@nhs.scot. |
| Πηγή: | Paediatric drugs [Paediatr Drugs] 2026 Sep; Vol. 28 (5), pp. 531-545. Date of Electronic Publication: 2026 Jul 15. |
| Τύπος έκδοσης: | Clinical Trial, Phase III; Comparative Study; Journal Article; Multicenter Study; Randomized Controlled Trial |
| Γλώσσα: | English |
| Στοιχεία περιοδικού: | Publisher: Springer International Country of Publication: Switzerland NLM ID: 100883685 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-2019 (Electronic) Linking ISSN: 11745878 NLM ISO Abbreviation: Paediatr Drugs Subsets: MEDLINE |
| Imprint Name(s): | Publication: June 2010- : Cham, Switzerland : Springer International Original Publication: Auckland ; Philadelphia : Adis International, c1999- |
| Ιατρικοί όροι (MeSH): | Anemia, Iron-Deficiency*/drug therapy , Anemia, Iron-Deficiency*/blood , Ferric Compounds*/adverse effects , Ferric Compounds*/administration & dosage , Ferric Compounds*/therapeutic use , Ferric Compounds*/pharmacokinetics , Ferrous Compounds*/adverse effects , Ferrous Compounds*/administration & dosage , Ferrous Compounds*/therapeutic use , Ferrous Compounds*/pharmacokinetics , Pyrones*/adverse effects , Pyrones*/administration & dosage , Pyrones*/therapeutic use , Pyrones*/pharmacokinetics, Hemoglobins/analysis ; Adolescent ; Child ; Child, Preschool ; Female ; Humans ; Infant ; Male ; Administration, Oral ; Treatment Outcome |
| Περίληψη: | Objectives: This phase 3, multicenter, randomized, open-label study (NCT05126901) compared oral ferric maltol with ferrous sulfate, across 23 sites in the US and the UK, evaluating the safety, efficacy, pharmacokinetics, tolerability, and palatability of age-specific doses in infants, children and, adolescents with iron deficiency anemia (IDA). Patient and Methods: In a 12-week study, a total of 61 children/adolescents aged 2-17 years (median age 14 years; 45 female patients [73.8%] and 16 male patients [26.2%]) with IDA were randomized to ferric maltol or ferrous sulfate, plus 4 infants aged 1 month to < 2 years (median age 12 months; 3 female patients [75%] and 1 male patient [25%]) were assigned to ferric maltol (open label arm). Ferric maltol dosing was age-dependent (0.6 mg/kg/dose twice a day [BID] for infants; 15 mg BID for 2-11 years; 30 mg BID for 12-17 years), while ferrous sulfate dosing was weight-based (3 mg/kg BID). Safety, efficacy (Hemoglobin [Hb], serum iron, transferrin saturation [TSAT], ferritin), PK, and palatability were assessed. Coprimary end points were Hb changes from baseline and safety, including gastrointestinal tolerability at week 12. Results: A prespecified interim analysis of the primary end point (change in Hb from baseline to week 12) was conducted after 32 ferric maltol patients completed treatment. As the change was statistically significant (p < 0.0001), recruitment was stopped. The interim analysis used a Pocock spending function with a 3.45% two-sided confidence interval (CI). Both the randomized ferric maltol and ferrous sulfate groups showed meaningful increases in Hb versus baseline (descriptive increases of 1.25 and 1.15 g/dL, respectively). Post hoc analyses confirmed that these changes from baseline were statistically significant (p < 0.0001). Ferric maltol increased Hb by a mean of 1.77 g/dL in the assigned infant group over 12 weeks. Similarly, by week 12, both treatments increased serum iron and TSAT (p < 0.02), ferric maltol showed higher peak iron, and ferritin rose more with ferrous sulfate. Adverse events were mild/moderate and similar in frequency between the two treatment groups (29% ferric maltol, 30% ferrous sulfate), and adverse reactions deemed as at least possibly related to treatment were more common with ferrous sulfate. Among children aged 6-17 years, ferric maltol was rated as tasting OK, Good, or Very Good more frequently than ferrous sulfate at two time points. On day 1, 79.3% of patients receiving ferric maltol reported OK, Good, or Very Good taste, compared with 50.0% receiving ferrous sulfate (OR 0.26, 95% CI 0.07-0.97; p = 0.027). By day 28, this difference persisted (75.0% versus 45.8%), favoring ferric maltol (OR 0.28, 95% CI 0.07-1.05; p = 0.046). The significant results for taste were replicated for smell; ferric maltol had significantly better smell ratings than ferrous sulfate on days 1 and 28 (p ≤ 0.003). Conclusions: Ferric maltol significantly improved Hb levels and iron storage within 12 weeks in children aged 1 month and above, offering potentially better tolerability and palatability compared with ferrous sulfate in children and adolescents. (© 2026. The Author(s), under exclusive licence to Springer Nature Switzerland AG.) |
| Competing Interests: | Declarations. Conflicts of Interest: Richard K. Russell received consulting fees from Nestle, Lilly, Ferring, Alfasigma and Abbvie and speaker fees from Pfizer and Janssen. Marcus KH Auth received consulting fees from Dr Falk Pharma and travel bursaries from Dr Falk Pharma and Abbott. David Gass and Andrew Freiberg declare that they have no conflicts of interest that might be relevant to the contents of this manuscript. Shield Therapeutics reviewed the manuscript for accuracy. Financial Assistance: The authors received no funding for this publication. Funding: Shield Therapeutics paid for medical writing assistance. Ethics Approval: The Protocol and informed consent form were submitted to and approved by the duly constituted Institutional Review Board /Independent Ethics Committee for each site prior to initiation of the study. Consent to Participate: The study was conducted in accordance with the Declaration of Helsinki and with all applicable laws and regulations of the locale and country where the study was conducted, and in compliance with Good Clinical Practice Guidelines. The rationale of the study, procedural details, and investigational goals were explained to each patient, along with potential risks and benefits. Each patient was assured of his/her right to withdraw from the study at any time. Prior to the initiation of any study procedures, each patient signed and dated an approved ICF. The original was kept on file by the Investigator with the patient’s records, and a copy was given to each patient. Consent for Publication: Not applicable; anonymized data. Availability of Data and Material: The datasets generated during and/or analyzed during the current study were uploaded to https://clinicaltrials.gov/study/NCT05126901 . Code Availability: Not applicable. Authors’ Contributions: All authors critically revised the manuscript for intellectual content and approved the final version. |
| Σχόλια: | Comment in: Paediatr Drugs. 2026 Sep 18. doi: 10.1007/s40272-026-00776-z.. (PMID: 42758408) |
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| Contributed Indexing: | Local Abstract: [plain-language-summary] Iron deficiency anemia (IDA) is common in children. It can affect energy levels, growth, and well-being. Oral iron supplements such as ferrous sulfate are widely used for treatment but may cause side effects or have a bad taste. Ferric maltol is a new iron supplement approved for people aged 10 years and older with iron deficiency. This 12-week study investigated the success of ferric maltol and ferrous sulfate in patients aged 1 month to 17 years old with IDA. The study evaluated how well the treatments improved iron status, and their safety and acceptability. Across all age groups, ferric maltol and ferrous sulfate improved iron status during the study, resulting in meaningful increases from the start of treatment. By the end of the study, ferric maltol caused higher peak levels of iron in the bloodstream. Side effects were generally mild-to-moderate for both treatments. However, side effects relating to the treatment were reported more commonly with ferrous sulfate. Importantly, in children aged 6–17 years, ferric maltol was more often rated as having an acceptable or pleasant taste compared with ferrous sulfate. More children reported that ferric maltol tasted OK, good, or very good, and this difference was consistent over time. Similar results were found for smell, with ferric maltol receiving better ratings than ferrous sulfate. These findings support ferric maltol as an effective and well-tolerated alternative to ferrous sulfate, especially for children not tolerating standard iron treatment. |
| Substance Nomenclature: | 0 (Ferric Compounds) MA10QYF1Z0 (ferric maltol) 0 (Ferrous Compounds) 39R4TAN1VT (ferrous sulfate) 0 (Hemoglobins) 0 (Pyrones) |
| Entry Date(s): | Date Created: 20260715 Date Completed: 20260929 Latest Revision: 20261008 |
| Update Code: | 20261008 |
| DOI: | 10.1007/s40272-026-00753-6 |
| PMID: | 42458190 |
| Βάση Δεδομένων: | MEDLINE |
| ISSN: | 1179-2019 |
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| DOI: | 10.1007/s40272-026-00753-6 |