CLINICAL SAFETY AND EFFICACY OF FERRIC CARBOXYMALTOSE IN THE TREATMENT OF IRON DEFICIENCY: META-ANALYSIS EVALUATING INDIVIDUAL PATIENT DATA OF 18 RANDOMIZED TRIALS
(Abstract release date: 05/19/16)
EHA Library. Beguin Y. 06/09/16; 133026; E1477
Disclosure(s): Yves Beguin has received consulting fees and speaker honoraria from Vifor Pharma.
Prof. Yves Beguin
Contributions
Contributions
Abstract
Abstract: E1477
Type: Eposter Presentation
Background
Iron deficiency and iron deficiency anemia are highly prevalent in both industrialized and developing countries. Patients and special populations with a wide variety of underlying conditions are affected and may experience debilitating symptoms. Intravenous iron therapy is indicated when oral iron supplementation is not tolerated or iron absorption is ineffective. Ferric carboxymaltose (FCM) is an intravenous iron formulation for administration of single doses up to 1000 mg iron during a short administration time (≥15 minutes).
Aims
The aim of this analysis was to describe the safety and efficacy of FCM across studies with different patient populations.
Methods
Individual patient data collected at European trial sites of 18 randomized controlled clinical trials performed by Vifor Pharma were analyzed. Study populations included patients with chronic kidney disease, inflammatory bowel disease, chronic heart failure, cancer, and women of childbearing age. FCM treated patients were compared to a pooled comparator group, including other intravenous iron formulations, oral iron, placebo or standard medical care. All treatment emergent adverse events (TEAEs) and related TEAEs were investigated, with focus on special adverse events of interest, including changes in serum phosphorus levels. Furthermore, therapeutic efficacy parameters (hemoglobin, ferritin and transferrin saturation) were analyzed over time. Changes from baseline in laboratory parameters are statistically presented as least-squares means (LS means) and standard errors (SE).
Results
Data from 3,331 patients with iron deficiency were analyzed. Of these, 1,969 patients had received FCM. The mean cumulative dose of FCM over the first 6 weeks was 1,124±417 mg iron, with single doses of 1,000 mg iron in 36% of the patients. Overall, the most frequent related TEAEs in the FCM group were serum ferritin increased (1.4%), headache (1.4%), and nausea (1.0%). In the pooled comparator group, the most frequent related TEAEs were constipation (2.5%), diarrhea (1.8%) and nausea (1.0%). Among patients with available serum phosphorus values over time (FCM n=1,370; comparator n=1,194), 27.4% of subjects in the FCM group and 2.0% in the comparator group had a value below 2.0 mg/dL at any time during the first 8 weeks of follow-up. Signs and symptoms that could possibly be related to transient decrease in phosphate level (using the broad SMQ-definition) were reported in 9.6% of FCM-treated subjects and in 11.3% in the comparator group. In the FCM group, hemoglobin steadily increased from the first post-treatment assessment at week 1 or 2 (n=1,404; mean changes from baseline: 0.411 [0.098] g/dL) to week 4 (n=1,619; 1.176 [0.168] g/dL) and week 8 (n=1,398; 1.466 [0.190] g/dL). Ferritin and transferrin saturation were significantly increased in the FCM group compared with comparator pool, at all time points (p<.0001; week 1 or 2, 4, 8), and remained stable throughout the 8-week period (mean change from baseline at week 8: +227.7 [53.7] vs 105.7 [53.8] ng/mL and +13.4 [1.2] vs 8.5 [1.2]%, respectively; p<.0001), reflecting repletion of iron stores.
Conclusion
This large-scale analysis is based on individual patient data and includes a broad variety of different patient populations. The results confirm that FCM is effective and has a similar tolerability to the pooled comparator. FCM causes a transient decrease in phosphate levels but this does not appear to increase the reported frequency of associated signs and symptoms.
Session topic: E-poster
Keyword(s): Iron, Iron deficiency, Iron deficiency anemia, Safety
Type: Eposter Presentation
Background
Iron deficiency and iron deficiency anemia are highly prevalent in both industrialized and developing countries. Patients and special populations with a wide variety of underlying conditions are affected and may experience debilitating symptoms. Intravenous iron therapy is indicated when oral iron supplementation is not tolerated or iron absorption is ineffective. Ferric carboxymaltose (FCM) is an intravenous iron formulation for administration of single doses up to 1000 mg iron during a short administration time (≥15 minutes).
Aims
The aim of this analysis was to describe the safety and efficacy of FCM across studies with different patient populations.
Methods
Individual patient data collected at European trial sites of 18 randomized controlled clinical trials performed by Vifor Pharma were analyzed. Study populations included patients with chronic kidney disease, inflammatory bowel disease, chronic heart failure, cancer, and women of childbearing age. FCM treated patients were compared to a pooled comparator group, including other intravenous iron formulations, oral iron, placebo or standard medical care. All treatment emergent adverse events (TEAEs) and related TEAEs were investigated, with focus on special adverse events of interest, including changes in serum phosphorus levels. Furthermore, therapeutic efficacy parameters (hemoglobin, ferritin and transferrin saturation) were analyzed over time. Changes from baseline in laboratory parameters are statistically presented as least-squares means (LS means) and standard errors (SE).
Results
Data from 3,331 patients with iron deficiency were analyzed. Of these, 1,969 patients had received FCM. The mean cumulative dose of FCM over the first 6 weeks was 1,124±417 mg iron, with single doses of 1,000 mg iron in 36% of the patients. Overall, the most frequent related TEAEs in the FCM group were serum ferritin increased (1.4%), headache (1.4%), and nausea (1.0%). In the pooled comparator group, the most frequent related TEAEs were constipation (2.5%), diarrhea (1.8%) and nausea (1.0%). Among patients with available serum phosphorus values over time (FCM n=1,370; comparator n=1,194), 27.4% of subjects in the FCM group and 2.0% in the comparator group had a value below 2.0 mg/dL at any time during the first 8 weeks of follow-up. Signs and symptoms that could possibly be related to transient decrease in phosphate level (using the broad SMQ-definition) were reported in 9.6% of FCM-treated subjects and in 11.3% in the comparator group. In the FCM group, hemoglobin steadily increased from the first post-treatment assessment at week 1 or 2 (n=1,404; mean changes from baseline: 0.411 [0.098] g/dL) to week 4 (n=1,619; 1.176 [0.168] g/dL) and week 8 (n=1,398; 1.466 [0.190] g/dL). Ferritin and transferrin saturation were significantly increased in the FCM group compared with comparator pool, at all time points (p<.0001; week 1 or 2, 4, 8), and remained stable throughout the 8-week period (mean change from baseline at week 8: +227.7 [53.7] vs 105.7 [53.8] ng/mL and +13.4 [1.2] vs 8.5 [1.2]%, respectively; p<.0001), reflecting repletion of iron stores.
Conclusion
This large-scale analysis is based on individual patient data and includes a broad variety of different patient populations. The results confirm that FCM is effective and has a similar tolerability to the pooled comparator. FCM causes a transient decrease in phosphate levels but this does not appear to increase the reported frequency of associated signs and symptoms.
Session topic: E-poster
Keyword(s): Iron, Iron deficiency, Iron deficiency anemia, Safety
Abstract: E1477
Type: Eposter Presentation
Background
Iron deficiency and iron deficiency anemia are highly prevalent in both industrialized and developing countries. Patients and special populations with a wide variety of underlying conditions are affected and may experience debilitating symptoms. Intravenous iron therapy is indicated when oral iron supplementation is not tolerated or iron absorption is ineffective. Ferric carboxymaltose (FCM) is an intravenous iron formulation for administration of single doses up to 1000 mg iron during a short administration time (≥15 minutes).
Aims
The aim of this analysis was to describe the safety and efficacy of FCM across studies with different patient populations.
Methods
Individual patient data collected at European trial sites of 18 randomized controlled clinical trials performed by Vifor Pharma were analyzed. Study populations included patients with chronic kidney disease, inflammatory bowel disease, chronic heart failure, cancer, and women of childbearing age. FCM treated patients were compared to a pooled comparator group, including other intravenous iron formulations, oral iron, placebo or standard medical care. All treatment emergent adverse events (TEAEs) and related TEAEs were investigated, with focus on special adverse events of interest, including changes in serum phosphorus levels. Furthermore, therapeutic efficacy parameters (hemoglobin, ferritin and transferrin saturation) were analyzed over time. Changes from baseline in laboratory parameters are statistically presented as least-squares means (LS means) and standard errors (SE).
Results
Data from 3,331 patients with iron deficiency were analyzed. Of these, 1,969 patients had received FCM. The mean cumulative dose of FCM over the first 6 weeks was 1,124±417 mg iron, with single doses of 1,000 mg iron in 36% of the patients. Overall, the most frequent related TEAEs in the FCM group were serum ferritin increased (1.4%), headache (1.4%), and nausea (1.0%). In the pooled comparator group, the most frequent related TEAEs were constipation (2.5%), diarrhea (1.8%) and nausea (1.0%). Among patients with available serum phosphorus values over time (FCM n=1,370; comparator n=1,194), 27.4% of subjects in the FCM group and 2.0% in the comparator group had a value below 2.0 mg/dL at any time during the first 8 weeks of follow-up. Signs and symptoms that could possibly be related to transient decrease in phosphate level (using the broad SMQ-definition) were reported in 9.6% of FCM-treated subjects and in 11.3% in the comparator group. In the FCM group, hemoglobin steadily increased from the first post-treatment assessment at week 1 or 2 (n=1,404; mean changes from baseline: 0.411 [0.098] g/dL) to week 4 (n=1,619; 1.176 [0.168] g/dL) and week 8 (n=1,398; 1.466 [0.190] g/dL). Ferritin and transferrin saturation were significantly increased in the FCM group compared with comparator pool, at all time points (p<.0001; week 1 or 2, 4, 8), and remained stable throughout the 8-week period (mean change from baseline at week 8: +227.7 [53.7] vs 105.7 [53.8] ng/mL and +13.4 [1.2] vs 8.5 [1.2]%, respectively; p<.0001), reflecting repletion of iron stores.
Conclusion
This large-scale analysis is based on individual patient data and includes a broad variety of different patient populations. The results confirm that FCM is effective and has a similar tolerability to the pooled comparator. FCM causes a transient decrease in phosphate levels but this does not appear to increase the reported frequency of associated signs and symptoms.
Session topic: E-poster
Keyword(s): Iron, Iron deficiency, Iron deficiency anemia, Safety
Type: Eposter Presentation
Background
Iron deficiency and iron deficiency anemia are highly prevalent in both industrialized and developing countries. Patients and special populations with a wide variety of underlying conditions are affected and may experience debilitating symptoms. Intravenous iron therapy is indicated when oral iron supplementation is not tolerated or iron absorption is ineffective. Ferric carboxymaltose (FCM) is an intravenous iron formulation for administration of single doses up to 1000 mg iron during a short administration time (≥15 minutes).
Aims
The aim of this analysis was to describe the safety and efficacy of FCM across studies with different patient populations.
Methods
Individual patient data collected at European trial sites of 18 randomized controlled clinical trials performed by Vifor Pharma were analyzed. Study populations included patients with chronic kidney disease, inflammatory bowel disease, chronic heart failure, cancer, and women of childbearing age. FCM treated patients were compared to a pooled comparator group, including other intravenous iron formulations, oral iron, placebo or standard medical care. All treatment emergent adverse events (TEAEs) and related TEAEs were investigated, with focus on special adverse events of interest, including changes in serum phosphorus levels. Furthermore, therapeutic efficacy parameters (hemoglobin, ferritin and transferrin saturation) were analyzed over time. Changes from baseline in laboratory parameters are statistically presented as least-squares means (LS means) and standard errors (SE).
Results
Data from 3,331 patients with iron deficiency were analyzed. Of these, 1,969 patients had received FCM. The mean cumulative dose of FCM over the first 6 weeks was 1,124±417 mg iron, with single doses of 1,000 mg iron in 36% of the patients. Overall, the most frequent related TEAEs in the FCM group were serum ferritin increased (1.4%), headache (1.4%), and nausea (1.0%). In the pooled comparator group, the most frequent related TEAEs were constipation (2.5%), diarrhea (1.8%) and nausea (1.0%). Among patients with available serum phosphorus values over time (FCM n=1,370; comparator n=1,194), 27.4% of subjects in the FCM group and 2.0% in the comparator group had a value below 2.0 mg/dL at any time during the first 8 weeks of follow-up. Signs and symptoms that could possibly be related to transient decrease in phosphate level (using the broad SMQ-definition) were reported in 9.6% of FCM-treated subjects and in 11.3% in the comparator group. In the FCM group, hemoglobin steadily increased from the first post-treatment assessment at week 1 or 2 (n=1,404; mean changes from baseline: 0.411 [0.098] g/dL) to week 4 (n=1,619; 1.176 [0.168] g/dL) and week 8 (n=1,398; 1.466 [0.190] g/dL). Ferritin and transferrin saturation were significantly increased in the FCM group compared with comparator pool, at all time points (p<.0001; week 1 or 2, 4, 8), and remained stable throughout the 8-week period (mean change from baseline at week 8: +227.7 [53.7] vs 105.7 [53.8] ng/mL and +13.4 [1.2] vs 8.5 [1.2]%, respectively; p<.0001), reflecting repletion of iron stores.
Conclusion
This large-scale analysis is based on individual patient data and includes a broad variety of different patient populations. The results confirm that FCM is effective and has a similar tolerability to the pooled comparator. FCM causes a transient decrease in phosphate levels but this does not appear to increase the reported frequency of associated signs and symptoms.
Session topic: E-poster
Keyword(s): Iron, Iron deficiency, Iron deficiency anemia, Safety
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