Reversal of cardiac complications in thalassemia major by long-term intermittent daily intensive iron chelation.
OBJECTIVE
In patients with thalassemia major (TM) who are non-compliant with long-term deferoxamine (DFO) chelation, survival is limited mainly because of cardiac complications of transfusional siderosis. It was recently shown in a small group of TM patients with established cardiac damage that continuous 24-h DFO infusion via an indwelling intravenous (i.v.) catheter is effective in reversing cardiac toxicity. The aim of the present study was to evaluate the results with intermittent daily (8-10 h) i.v. DFO.


METHODS
Eight TM patients with cardiac complications treated with intensive intermittent DFO were retrospectively evaluated by the mean annual serum ferritin, radionucleated ventriculography and 24-h electrocardiography recordings.


RESULTS
The median age at diagnosis of cardiac disease was 17.5 yr (range 14-21), and the median follow-up time was 84 months (range, 36-120). In the majority of patients (seven of eight) high-dose DFO (mean 95 +/- 18.3 mg/kg/d) was administered via a central venous line. During follow-up, there was a significant decrease in the mean ferritin levels (5828 +/- 2016 ng/mL to 1585 +/- 1849 ng/mL, P < 0.001). Both cardiac failure (mean ejection fraction 32 +/- 5) and cardiac arrhythmias were resolved in four of five patients. One non-compliant patient died during the follow-up. Following discontinuation of the i.v. therapy, compliance with conventional DFO therapy improved. The complications of this regimen, mainly catheter-related infections and catheter-related thrombosis, were similar to those described earlier.


CONCLUSIONS
These results with the longest follow-up period in the literature suggest that i.v. high-dose DFO for 8-10 h daily may be as effective as continuous 24-h infusion for the reversal of established cardiac disease in TM.
Miskin
H
H
Department of Pediatric Hematology/Oncology, Schneider Children's Medical Center of Israel, Petah Tiqva, Israel.
Yaniv
I
I
Berant
M
M
Hershko
C
C
Tamary
H
H
D000293:Adolescent; D000328:Adult; D001145:Arrhythmias, Cardiac / Q000209:etiology; D002405:Catheterization, Central Venous / Q000009:adverse effects; D015913:Chelation Therapy / Q000009:adverse effects / Q000379:methods; D003676:Deferoxamine / Q000008:administration & dosage / Q000633:toxicity; D005500:Follow-Up Studies; D006331:Heart Diseases / Q000188:drug therapy / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000627:therapeutic use; D012189:Retrospective Studies; D013318:Stroke Volume; D013789:Thalassemia / Q000150:complications* / Q000188:drug therapy*; D018487:Ventricular Dysfunction, Left / Q000209:etiology
D007502:Iron Chelating Agents; D003676:Deferoxamine