EHA Library - The official digital education library of European Hematology Association (EHA)

COMBINATION OF DEFERASIROX AND DEFEROXAMINE - A SUCCESSFUL CHELATION THERAPY IN B-THALASSEMIA MAJOR PATIENTS
Author(s): ,
Foteini Petropoulou
Affiliations:
Thalassaemia Unit,General Hospital of Athens 'G.Gennimatas',Athens,Greece
,
Kyriaki Ventiadi
Affiliations:
Thalassaemia Unit,General Hospital of Athens 'G.Gennimatas',Athens,Greece
Georgia Leveta
Affiliations:
Thalassaemia Unit,General Hospital of Athens 'G.Gennimatas',Athens,Greece
(Abstract release date: 05/18/17) EHA Library. Petropoulou F. 05/18/17; 182908; PB2195
Foteini Petropoulou
Foteini Petropoulou
Contributions
Abstract

Abstract: PB2195

Type: Publication Only

Background
Frequent transfusions required for β- thalassemia major patients cause iron overload. Without the appropriate chelation therapy, iron toxicity can cause significant heart, liver and endocrine morbidity.

Aims
In this case series we estimated the safety and efficacy of iron chelation with the combination of deferasirox (DFX) and deferoxamine (DFO) in transfusion dependent thalassemia (TDT) patients attending the Thalassemia Unit in a tertiary hospital in Athens, Greece.

Methods
10 TDT patients were treated with a combination chelation therapy of DFX (30 ±10mg/kg/d) and DFO (44 ± 12mg/kg/d for 2-6 days/wk in 12hr or 24hr infusion rates). Reasons for starting this combination treatment included: 1) treatment with one chelating agent did not succeed in decreasing heart and liver iron, 2) agranulocytosis or severe neutropenia due to deferiprone (DFP) treatment and 3) adverse events recorded with increased doses of one of the chelating agents.

The efficacy of the treatment was estimated through MRI measurements of heart and liver iron (T2*Heart, and LIC) in combination with serum ferritin levels.
Liver enzymes (ALT, AST) and serum creatinine were used to monitor safety of the treatment.

Results
Five of the 10 patients had significant liver hemosiderosis (LIC >15 mg Fe/gr d.w.) and 3 had heart iron overload, of which one significant (T2* 1.9msec).

Before starting DFX/DFO combination therapy
12mos into the DFX/DFO combination therapy
Serum Ferritin mean
3,360 ng/mL
(range 1,700- 6,200)
2,450 ng/mL
(range 800-4,900)
Liver Iron Concentration (LIC) mean
19.0 mg Fe/gr d.w.
(range 6.2-46.4)
9 mg Fe/gr d.w.
(range 1.6- 28.4)
Cardiac T2* mean
24.1 msec
(range 1.9-36)
32.3 msec
(range 5.2 -39.5)
The combination treatment was well tolerated without adverse events or effects on liver and kidney function.

Conclusion
The combination chelation with DFX/DFO can decrease iron overload and represents a safe and effective option when monotherapy is not effective or not well tolerated.

Session topic: 26. Thalassemias

Keyword(s): Thalassemia, Liver iron concentration, Chelation

Abstract: PB2195

Type: Publication Only

Background
Frequent transfusions required for β- thalassemia major patients cause iron overload. Without the appropriate chelation therapy, iron toxicity can cause significant heart, liver and endocrine morbidity.

Aims
In this case series we estimated the safety and efficacy of iron chelation with the combination of deferasirox (DFX) and deferoxamine (DFO) in transfusion dependent thalassemia (TDT) patients attending the Thalassemia Unit in a tertiary hospital in Athens, Greece.

Methods
10 TDT patients were treated with a combination chelation therapy of DFX (30 ±10mg/kg/d) and DFO (44 ± 12mg/kg/d for 2-6 days/wk in 12hr or 24hr infusion rates). Reasons for starting this combination treatment included: 1) treatment with one chelating agent did not succeed in decreasing heart and liver iron, 2) agranulocytosis or severe neutropenia due to deferiprone (DFP) treatment and 3) adverse events recorded with increased doses of one of the chelating agents.

The efficacy of the treatment was estimated through MRI measurements of heart and liver iron (T2*Heart, and LIC) in combination with serum ferritin levels.
Liver enzymes (ALT, AST) and serum creatinine were used to monitor safety of the treatment.

Results
Five of the 10 patients had significant liver hemosiderosis (LIC >15 mg Fe/gr d.w.) and 3 had heart iron overload, of which one significant (T2* 1.9msec).

Before starting DFX/DFO combination therapy
12mos into the DFX/DFO combination therapy
Serum Ferritin mean
3,360 ng/mL
(range 1,700- 6,200)
2,450 ng/mL
(range 800-4,900)
Liver Iron Concentration (LIC) mean
19.0 mg Fe/gr d.w.
(range 6.2-46.4)
9 mg Fe/gr d.w.
(range 1.6- 28.4)
Cardiac T2* mean
24.1 msec
(range 1.9-36)
32.3 msec
(range 5.2 -39.5)
The combination treatment was well tolerated without adverse events or effects on liver and kidney function.

Conclusion
The combination chelation with DFX/DFO can decrease iron overload and represents a safe and effective option when monotherapy is not effective or not well tolerated.

Session topic: 26. Thalassemias

Keyword(s): Thalassemia, Liver iron concentration, Chelation

By clicking “Accept Terms & all Cookies” or by continuing to browse, you agree to the storing of third-party cookies on your device to enhance your user experience and agree to the user terms and conditions of this learning management system (LMS).

Cookie Settings
Accept Terms & all Cookies