Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major
Status:
Completed
Trial end date:
2006-06-01
Target enrollment:
Participant gender:
Summary
Myocardial iron overload is the leading cause of death in patients with beta-thalassemia
major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces
myocardial iron and improves cardiac function. However, the prognosis for TM patients with
established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is
unknown. Twenty-eight TM patients with cardiac disease were enrolled in a prospective study
lasting 42±6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO
(DFP, 75 mg/kg t.i.d.; DFO, 40-50 mg/kg over 8-12 h at night 5-7 d/wk), while 13 (5 high- and
8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin
patients on combination therapy, whereas 4 cardiac events (p=0.0049), including three deaths,
occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in TM
patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO
is superior to DFO monotherapy.