Introduction

Ferric carboxymaltose (FCM) is approved for the treatment of iron deficiency anemia (IDA) patients with intolerance or an unsatisfactory response to oral iron and in non-dialysis dependent chronic kidney disease. Limited data is available on the patients’ perspective and perceived improvements in quality of life, particularly in real-world clinical practice settings with FCM treatment.

Aim

To evaluate the impact of intravenous iron supplementation with FCM treatment on the fatigue, physical function, and general health of patients with IDA receiving routine clinical care.

Patient Profile

• 152 patients with IDA (baseline serum hemoglobin of >7 g/dL and <13 g/dL for males or <12 g/dL for females), no potential contraindications to FCM, and no parenteral iron therapy within 3 months prior to study enrollment

Method

Study Design

• Prospective, single arm, observational study
• Patient-Reported Outcomes Measurement Information System (PROMIS) instruments were used to evaluate fatigue (PROMIS Short Form v1.0 13a [FACIT-Fatigue]), general health status (PROMIS Scale v1.2), and physical function (PROMIS Short Form v2.0 4a) before and at 3 and 6 months after FCM treatment
• A higher FACIT-Fatigue or a lower PROMIS Physical Function, PROMIS Global Physical Health or Mental Health score indicated more health problems.

Endpoints

• Primary outcome: change from baseline in FACIT-Fatigue scores, measured at 3 and 6 months after the first dose of FCM
• Secondary outcomes: changes in PROMIS Physical Function and Global Health Physical and Mental scores and in serum hemoglobin levels from baseline to 3 and 6 months, and associations between baseline serum hemoglobin levels and baseline PRO scores

Results

Efficacy

• FCM treatment improved the fatigue symptoms, as indicated by significantly lower FACIT-Fatigue scores (61.0 at baseline to 50.2 at 3 months & 52.4 at 6 months; p < 0.0001; Figure 1)
• FCM led to a clinically meaningful reduction in the FACIT-Fatigue score from baseline to 3 and 6 months in over two-third patients (72.7% and 67.3%, respectively)
• The mean PROMIS Physical Function score significantly increased from 41.3 at baseline to 46.1 and 45.2 at 3 and 6 months, respectively, after FCM treatment (p < 0.0001) with a clinically meaningful improvement in over 35% of patients
• FCM significantly improved the mean PROMIS Global Health scale: physical score improved from a baseline of 41.1 to 46.4 and mental score improved from 43.9 to 46.5 at 6 months
• A clinically meaningful improvement in the Global Physical Health component score at each time point was achieved with FCM in more than 50% of the patients (52.8 % at 3 months and 57.1% at 6 months)
• FCM achieved clinically meaningful improvement in the Global Mental Health component score in 41.7% and 30.6% patients at 3 and 6 months, respectively
• A minimum 5-point clinically meaningful improvement, was seen in the FACIT-Fatigue, PROMIS Global Physical Health, Global Mental Health and PROMIS Physical Function scores for 72.7%, 52.8%, 41.7% and 39.8% of patients at 3 months (p < 0.0001 for each change from baseline), with statistically significant improvement continuing at 6 months
• These improvements in outcomes were mostly achieved after a single course of FCM, with only a small proportion of patients (9.7%) requiring additional intravenous iron treatment throughout the 6 months period
• The mean scores at 3 and 6 months for any PROMIS measure were not significantly different, which indicated that improvement in patient reported outcomes were mostly sustained up to the end of the follow-up period
• FCM led to a significant increase in mean serum hemoglobin of 2.6 g/dL and 2.3 g/dL at 3 and 6 months (from 10.2 g/dL at baseline to12.8 g/dL and 12.4 g/dL, respectively; p < 0.0001 for both)
• Lower serum hemoglobin was associated with increased fatigue (r=−0.294; p = 0.0002) and lower levels of physical function (r = 0.286; p = 0.0004), physical health (r = 0.276; p = 0.0006), and mental health (r = 0.151; p = 0.0647)

Figure 1: Effect of FCM treatment on IDA patient reported FACIT-Fatigue scores

Conclusion

Intravenous iron supplementation with FCM treatment achieved significant improvements in fatigue symptoms, physical function, and global health at 3 months, which were sustained at 6 months in patients with IDA.

Int. J Gen Med 2023; 16: 3291-3300