EFFICACY AND SAFETY OF DEFERASIROX IN SINGLE VS DIVIDED DOSAGE INTHALASSEMIC CHILDREN.
(Abstract release date: 05/21/15)
EHA Library. Kakkar S. 06/12/15; 102602; PB2010
Disclosure(s): Dayanand Medical College, LudhianaPediatrics, hemato-oncology unit
Dr. Shruti Kakkar
Contributions
Contributions
Abstract
Abstract: PB2010
Type: Publication Only
Background
Iron chelation has improved the prognosis in thalassemia major. DFX had been the recent oral iron chelator in use. Dose-dependent effect of DFX had been observed in many studies. Initially it was used at 20 – 30 mg/kg/day but efficacy proved to be better when used at higher doses of 30 – 40 mg/kg/day with good tolerability. Ferritin levels tended to be high even on DFX (30 – 40 mg/kg/day) with good compliance, so safety and efficacy of higher doses (40 – 50 mg/kg/day) in once daily vs two daily doses needed to be compared.
Aims
To study the efficacy and safety of DFX (40-50 mg/kg/day) in single vs divided doses in thalassemic children
Methods
The prospective study was conducted in thalassemia ward of Department of Pediatrics, Dayanand Medical College and Hospital, Ludhiana from Jan 2013 to June 2014. Patients were randomly allocated in two groups, Group A & Group B. Both took DFX in doses of 40 – 50 mg/kg/day, group A once daily doses and group B in two divided doses. Efficacy was studied in terms of change in ferritin levels, cardiac & liver MRI T2 * values and LVEF. Safety profile was studied in terms of gastrointestinal side effects, rash and change in serum creatinine values, SGPT and GFR.
Results
Serum ferritin levels reduced in both the groups viz. Group A (2692.5 ± 1232.35 ng/ml to 1959.5 ± 696.19 ng/ml p value = 0. 07), Group B (2766.2 ± 897.51 to 2569.9 ± 762.6 ng/ml ;p value = 0.38) with significant difference between the two groups at the end of study (p=0.05).Liver MRI T2* values improved in group A (2.1 ± 1.50 to 3.1 ± 1.84 ms ; p= 0.096)as compared to group B (2.8 ± 1.84 to 2.2 ± 2.71 ms; p=0.70). Mean LIC decreased in group A (17.4 ± 7.83 mg/g dw to 12.4 ± 8.05 mg/g/dw ; p= 0.197) as compared to group B (15.3 ± 10.42 mg/g dw to 22.1 ± 11.93 mg/g/dw ; p= 0.340 ).Overall mean cardiac MRI T2 * values decreased (30.5 ± 10.30 ms to 24.5 ± 4.94 ms ; p=0.202 ) in group A and ( 31.7 ± 14.78 ms to 20.5 ± 11.78 ms ; p=0.167) in group B but remained within normal limits. No change in LVEF was observed. No significant adverse effects were noted with 40–50 mg/kg/day dose of DFX. There was increase in liver transaminases (SGPT) levels (64.4±54.0 to 34.5±14.1 mg/dl in group A; p= 0.05) and (97.9±79.3 to 61.9 ± 39.2 mg/dl ;p= 0.134) in group B
Summary
DFX is safe in higher dosages (40 – 50 mg/kg/day) and once daily dosage is more efficacious as compared to twice a day dosage schedule.
Keyword(s): Iron chelation, MRI, Thalassemia
Type: Publication Only
Background
Iron chelation has improved the prognosis in thalassemia major. DFX had been the recent oral iron chelator in use. Dose-dependent effect of DFX had been observed in many studies. Initially it was used at 20 – 30 mg/kg/day but efficacy proved to be better when used at higher doses of 30 – 40 mg/kg/day with good tolerability. Ferritin levels tended to be high even on DFX (30 – 40 mg/kg/day) with good compliance, so safety and efficacy of higher doses (40 – 50 mg/kg/day) in once daily vs two daily doses needed to be compared.
Aims
To study the efficacy and safety of DFX (40-50 mg/kg/day) in single vs divided doses in thalassemic children
Methods
The prospective study was conducted in thalassemia ward of Department of Pediatrics, Dayanand Medical College and Hospital, Ludhiana from Jan 2013 to June 2014. Patients were randomly allocated in two groups, Group A & Group B. Both took DFX in doses of 40 – 50 mg/kg/day, group A once daily doses and group B in two divided doses. Efficacy was studied in terms of change in ferritin levels, cardiac & liver MRI T2 * values and LVEF. Safety profile was studied in terms of gastrointestinal side effects, rash and change in serum creatinine values, SGPT and GFR.
Results
Serum ferritin levels reduced in both the groups viz. Group A (2692.5 ± 1232.35 ng/ml to 1959.5 ± 696.19 ng/ml p value = 0. 07), Group B (2766.2 ± 897.51 to 2569.9 ± 762.6 ng/ml ;p value = 0.38) with significant difference between the two groups at the end of study (p=0.05).Liver MRI T2* values improved in group A (2.1 ± 1.50 to 3.1 ± 1.84 ms ; p= 0.096)as compared to group B (2.8 ± 1.84 to 2.2 ± 2.71 ms; p=0.70). Mean LIC decreased in group A (17.4 ± 7.83 mg/g dw to 12.4 ± 8.05 mg/g/dw ; p= 0.197) as compared to group B (15.3 ± 10.42 mg/g dw to 22.1 ± 11.93 mg/g/dw ; p= 0.340 ).Overall mean cardiac MRI T2 * values decreased (30.5 ± 10.30 ms to 24.5 ± 4.94 ms ; p=0.202 ) in group A and ( 31.7 ± 14.78 ms to 20.5 ± 11.78 ms ; p=0.167) in group B but remained within normal limits. No change in LVEF was observed. No significant adverse effects were noted with 40–50 mg/kg/day dose of DFX. There was increase in liver transaminases (SGPT) levels (64.4±54.0 to 34.5±14.1 mg/dl in group A; p= 0.05) and (97.9±79.3 to 61.9 ± 39.2 mg/dl ;p= 0.134) in group B
Summary
DFX is safe in higher dosages (40 – 50 mg/kg/day) and once daily dosage is more efficacious as compared to twice a day dosage schedule.
Keyword(s): Iron chelation, MRI, Thalassemia
Abstract: PB2010
Type: Publication Only
Background
Iron chelation has improved the prognosis in thalassemia major. DFX had been the recent oral iron chelator in use. Dose-dependent effect of DFX had been observed in many studies. Initially it was used at 20 – 30 mg/kg/day but efficacy proved to be better when used at higher doses of 30 – 40 mg/kg/day with good tolerability. Ferritin levels tended to be high even on DFX (30 – 40 mg/kg/day) with good compliance, so safety and efficacy of higher doses (40 – 50 mg/kg/day) in once daily vs two daily doses needed to be compared.
Aims
To study the efficacy and safety of DFX (40-50 mg/kg/day) in single vs divided doses in thalassemic children
Methods
The prospective study was conducted in thalassemia ward of Department of Pediatrics, Dayanand Medical College and Hospital, Ludhiana from Jan 2013 to June 2014. Patients were randomly allocated in two groups, Group A & Group B. Both took DFX in doses of 40 – 50 mg/kg/day, group A once daily doses and group B in two divided doses. Efficacy was studied in terms of change in ferritin levels, cardiac & liver MRI T2 * values and LVEF. Safety profile was studied in terms of gastrointestinal side effects, rash and change in serum creatinine values, SGPT and GFR.
Results
Serum ferritin levels reduced in both the groups viz. Group A (2692.5 ± 1232.35 ng/ml to 1959.5 ± 696.19 ng/ml p value = 0. 07), Group B (2766.2 ± 897.51 to 2569.9 ± 762.6 ng/ml ;p value = 0.38) with significant difference between the two groups at the end of study (p=0.05).Liver MRI T2* values improved in group A (2.1 ± 1.50 to 3.1 ± 1.84 ms ; p= 0.096)as compared to group B (2.8 ± 1.84 to 2.2 ± 2.71 ms; p=0.70). Mean LIC decreased in group A (17.4 ± 7.83 mg/g dw to 12.4 ± 8.05 mg/g/dw ; p= 0.197) as compared to group B (15.3 ± 10.42 mg/g dw to 22.1 ± 11.93 mg/g/dw ; p= 0.340 ).Overall mean cardiac MRI T2 * values decreased (30.5 ± 10.30 ms to 24.5 ± 4.94 ms ; p=0.202 ) in group A and ( 31.7 ± 14.78 ms to 20.5 ± 11.78 ms ; p=0.167) in group B but remained within normal limits. No change in LVEF was observed. No significant adverse effects were noted with 40–50 mg/kg/day dose of DFX. There was increase in liver transaminases (SGPT) levels (64.4±54.0 to 34.5±14.1 mg/dl in group A; p= 0.05) and (97.9±79.3 to 61.9 ± 39.2 mg/dl ;p= 0.134) in group B
Summary
DFX is safe in higher dosages (40 – 50 mg/kg/day) and once daily dosage is more efficacious as compared to twice a day dosage schedule.
Keyword(s): Iron chelation, MRI, Thalassemia
Type: Publication Only
Background
Iron chelation has improved the prognosis in thalassemia major. DFX had been the recent oral iron chelator in use. Dose-dependent effect of DFX had been observed in many studies. Initially it was used at 20 – 30 mg/kg/day but efficacy proved to be better when used at higher doses of 30 – 40 mg/kg/day with good tolerability. Ferritin levels tended to be high even on DFX (30 – 40 mg/kg/day) with good compliance, so safety and efficacy of higher doses (40 – 50 mg/kg/day) in once daily vs two daily doses needed to be compared.
Aims
To study the efficacy and safety of DFX (40-50 mg/kg/day) in single vs divided doses in thalassemic children
Methods
The prospective study was conducted in thalassemia ward of Department of Pediatrics, Dayanand Medical College and Hospital, Ludhiana from Jan 2013 to June 2014. Patients were randomly allocated in two groups, Group A & Group B. Both took DFX in doses of 40 – 50 mg/kg/day, group A once daily doses and group B in two divided doses. Efficacy was studied in terms of change in ferritin levels, cardiac & liver MRI T2 * values and LVEF. Safety profile was studied in terms of gastrointestinal side effects, rash and change in serum creatinine values, SGPT and GFR.
Results
Serum ferritin levels reduced in both the groups viz. Group A (2692.5 ± 1232.35 ng/ml to 1959.5 ± 696.19 ng/ml p value = 0. 07), Group B (2766.2 ± 897.51 to 2569.9 ± 762.6 ng/ml ;p value = 0.38) with significant difference between the two groups at the end of study (p=0.05).Liver MRI T2* values improved in group A (2.1 ± 1.50 to 3.1 ± 1.84 ms ; p= 0.096)as compared to group B (2.8 ± 1.84 to 2.2 ± 2.71 ms; p=0.70). Mean LIC decreased in group A (17.4 ± 7.83 mg/g dw to 12.4 ± 8.05 mg/g/dw ; p= 0.197) as compared to group B (15.3 ± 10.42 mg/g dw to 22.1 ± 11.93 mg/g/dw ; p= 0.340 ).Overall mean cardiac MRI T2 * values decreased (30.5 ± 10.30 ms to 24.5 ± 4.94 ms ; p=0.202 ) in group A and ( 31.7 ± 14.78 ms to 20.5 ± 11.78 ms ; p=0.167) in group B but remained within normal limits. No change in LVEF was observed. No significant adverse effects were noted with 40–50 mg/kg/day dose of DFX. There was increase in liver transaminases (SGPT) levels (64.4±54.0 to 34.5±14.1 mg/dl in group A; p= 0.05) and (97.9±79.3 to 61.9 ± 39.2 mg/dl ;p= 0.134) in group B
Summary
DFX is safe in higher dosages (40 – 50 mg/kg/day) and once daily dosage is more efficacious as compared to twice a day dosage schedule.
Keyword(s): Iron chelation, MRI, Thalassemia
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