Abstract
Objective: Iron deficiency anaemia is common in paediatric gastroenterology with intravenous iron increasingly utilised. While adult data are published for ferric derisomaltose (FDI), no paediatric data exist. Our aim was to assess the effectiveness and safety profile of FDI in paediatric gastroenterology.
Design: Retrospective cohort study.
Setting: Two UK referral centres (Edinburgh and Sheffield).
Patients: All paediatric gastroenterology patients who received FDI from 6/2020 to 6/2023.
Main outcome measures: Haematological and biochemical parameters were collected before and at 1, 3, 6 and 12 months after treatment, when available, together with the need for repeated FDI infusions and FDI adverse events.
Results: Sixty-five patients were identified (54% male; median age 12.8 years, IQR 9.6–15.2), receiving 78 FDI infusions. After a single infusion 38/59 (64%) patients (effectiveness analysis) completely corrected their anaemia and 6/59 (10%) improved from moderate-severe to mild anaemia. Median haemoglobin increased from 92 (IQR 85-108) to 126 (IQR 115-132) g/L at 1 month (p<0.001) and peaked at 12 months (134g/L, IQR 125-142; p<0.001) post-infusion. A second infusion was performed in 12/59 (20%) patients with 9/12 correcting their anaemia. No significant biochemical alterations, including hypophosphatemia, were detected. Overall, 4/65 (6%) patients (safety analysis) experienced adverse events, with 3 discontinuing FDI infusion.
Conclusions: Our real-world data reveal FDI is rapidly and persistently effective in correcting IDA in paediatric gastroenterology. No significant biochemical alterations or new safety signals were identified.
Design: Retrospective cohort study.
Setting: Two UK referral centres (Edinburgh and Sheffield).
Patients: All paediatric gastroenterology patients who received FDI from 6/2020 to 6/2023.
Main outcome measures: Haematological and biochemical parameters were collected before and at 1, 3, 6 and 12 months after treatment, when available, together with the need for repeated FDI infusions and FDI adverse events.
Results: Sixty-five patients were identified (54% male; median age 12.8 years, IQR 9.6–15.2), receiving 78 FDI infusions. After a single infusion 38/59 (64%) patients (effectiveness analysis) completely corrected their anaemia and 6/59 (10%) improved from moderate-severe to mild anaemia. Median haemoglobin increased from 92 (IQR 85-108) to 126 (IQR 115-132) g/L at 1 month (p<0.001) and peaked at 12 months (134g/L, IQR 125-142; p<0.001) post-infusion. A second infusion was performed in 12/59 (20%) patients with 9/12 correcting their anaemia. No significant biochemical alterations, including hypophosphatemia, were detected. Overall, 4/65 (6%) patients (safety analysis) experienced adverse events, with 3 discontinuing FDI infusion.
Conclusions: Our real-world data reveal FDI is rapidly and persistently effective in correcting IDA in paediatric gastroenterology. No significant biochemical alterations or new safety signals were identified.
| Original language | English |
|---|---|
| Journal | Archives of Disease in Childhood |
| DOIs | |
| Publication status | Published - 30 Apr 2025 |
Keywords / Materials (for Non-textual outputs)
- Gastroenterology
- Paediatrics
- Therapeutics