Iron chelation in thalassemia: combined or monotherapy? The Egyptian experience.
Patients with thalassemia major requiring regular blood transfusions accumulate iron that is toxic to the heart, liver, and endocrine systems. The following prospective, randomized trial was carried out to determine the effectiveness, in children and young adults, of combined deferiprone (DFP) and deferoxamine (DFO) in reducing transfusional iron overload compared to either drug alone and to assess the safety and tolerability of DFP. Sixty-six patients were randomized into three treatment arms: daily DFP combined with DFO twice weekly; daily DFP only; and DFO only 5 days/week. Fifty-six patients completed the 54 weeks and were assessed by different indices. A significant reduction of liver iron concentration and serum ferritin was observed in all three arms while significant reduction of liver iron score was observed in patients on combination therapy only. Cardiac function did not significantly change in any arm. Compliance improved in patients who received combined therapy. Toxicity of DFP was mild to moderate and acceptable; most commonly, transient arthropathy and nausea/vomiting were observed. Thus, combination therapy has shown to be effective in reducing iron overload in thalassemia patients.
El-Beshlawy
Amal
A
Faculty of Medicine, Cairo University, 32 Falaky Street, Bab El-Louk, Cairo, Egypt. amalelbeshlawy@yahoo.com
Manz
Chantal
C
Naja
Mohammed
M
Eltagui
Mona
M
Tarabishi
Claudia
C
Youssry
Ilham
I
Sobh
Hewida
H
Hamdy
Mona
M
Sharaf
Iman
I
Mostafa
Azza
A
Shaker
Olfat
O
Hoffbrand
A V
AV
Taher
Ali
A
D000293:Adolescent; D000328:Adult; D015913:Chelation Therapy / Q000009:adverse effects / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D004534:Egypt / Q000453:epidemiology; D005260:Female; D005293:Ferritins / Q000032:analysis / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology / Q000517:prevention & control; D008099:Liver / Q000473:pathology; D008297:Male; D010349:Patient Compliance; D011446:Prospective Studies; D011728:Pyridones / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D013789:Thalassemia / Q000150:complications / Q000188:drug therapy* / Q000453:epidemiology; D065227:Transfusion Reaction
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine