A randomized, controlled, open label non-inferiority trial of intravenous ferric carboxymaltose versus iron sucrose in patients with iron deficiency anemia in China

doi:10.1007/s11684-023-1001-2

2024, Vol. 18

Issue (1) : 98-108 https://doi.org/10.1007/s11684-023-1001-2

A randomized, controlled, open label non-inferiority trial of intravenous ferric carboxymaltose versus iron sucrose in patients with iron deficiency anemia in China

Jie Jin¹, Zhihua Ran², Emanuele Noseda³, Bernard Roubert³, Matthieu Marty³, Anna Mezzacasa³(

), Udo Michael Göring³

¹. The First Affiliated Hospital, Zhejiang University College of Medicine, Hangzhou 310058, China
². Renji Hospital Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China
³. Vifor Pharma, Glattbrugg, 8152, Switzerland

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Abstract

Iron deficiency (ID) and ID anemia (IDA) pose significant public health concerns in China. Although iron sucrose (IS) treatment is well-established in the country, ferric carboxymaltose (FCM) offers the advantage of higher doses and fewer infusions. This open label, randomized, controlled, non-inferiority trial was conducted at multiple sites in China to compare the outcomes of FCM (maximum of 2 doses, 500 or 1000 mg iron) and IS (up to 11 infusions, 200 mg iron) treatments in subjects with IDA. The primary endpoint was the achievement of hemoglobin (Hb) response (an increase of ≥2 g/dL from baseline) within 8 weeks, whereas secondary endpoints included changes in Hb, transferrin saturation, and serum ferritin levels. Among the 371 randomized subjects, a similar percentage of subjects treated with FCM and IS achieved Hb-response (FCM 99.4%, IS 98.3%), thereby confirming the non-inferiority of FCM compared with IS (difference 1.12 (−2.15, 4.71; 95% confidence interval (CI))). Furthermore, a significantly higher proportion of FCM-treated subjects achieved early Hb-response at Week 2 (FCM 85.2%, IS 73.2%; difference 12.1 (3.31, 20.65; 95% CI)). Additionally, the increase in TSAT and serum ferritin levels from baseline was significantly greater at all time points for FCM-treated subjects. The safety profiles of FCM and IS were comparable, with the exception of transient hypophosphatemia and pyrexia, which are consistent with FCM’s known safety profile. In conclusion, FCM proves to be an efficacious treatment for IDA, providing faster Hb-response and correction of ID with fewer administrations than IS.

Keywords iron deficiency anemia intravenous iron ferric carboxymaltose iron sucrose Hb response early response

Corresponding Author(s): Anna Mezzacasa

About author:

Li Liu and Yanqing Liu contributed equally to this work.

Just Accepted Date: 30 August 2023 Online First Date: 31 October 2023 Issue Date: 22 April 2024

Cite this article:

Jie Jin,Zhihua Ran,Emanuele Noseda, et al. A randomized, controlled, open label non-inferiority trial of intravenous ferric carboxymaltose versus iron sucrose in patients with iron deficiency anemia in China[J]. Front. Med., 2024, 18(1): 98-108.

URL:

https://academic.hep.com.cn/fmd/EN/10.1007/s11684-023-1001-2
https://academic.hep.com.cn/fmd/EN/Y2024/V18/I1/98

Tab.1 FCM dosing schedule

Fig.1 Subject disposition and reasons for exclusion. Subjects could have more than one reason for exclusion. FCM, ferric carboxymaltose; IS, iron sucrose.

Tab.2 Subject baseline characteristics (SS)^a

Tab.3 Subjects with Hb response (≥ 2 g/dL increase from baseline) at any visit until Week 8

Fig.2 Mean (SD) levels of Hb, serum ferritin levels, and TSAT in subjects treated with FCM vs. IS. (A) Hb levels steadily increased in both arms. (B) TSAT normalized by Week 2 and remained normal thereafter. (C) Serum ferritin peaked at Week 2, but levels remained above cut-off until Week 8. FCM, ferric carboxymaltose; Hb, hemoglobin; IS, iron sucrose; SD, standard deviation; TSAT, transferrin saturation.

Fig.3 Mean (SD) increase of Hb levels, serum ferritin levels and TSAT from baseline. (A) FCM-treated subjects were significantly more likely to achieve Hb-increase by Week 2. (B, C) Increases in TSAT and serum ferritin were significantly greater in the FCM group throughout the follow-up period. *P < 0.05, **P < 0.01, ***P < 0.001. FCM, ferric carboxymaltose; Hb, hemoglobin; IS, iron sucrose; SD, standard deviation; TSAT, transferrin saturation.

Tab.4 Subjects with Hb increase ≥ 2 g/dL from baseline at Weeks 2, 4, 6 and 8 (FAS)

Tab.5 ID correction^a at Week 2, 4, 6 and 8 (FAS)

Tab.6 Adverse events profile (SS)

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