Safety and efficacy of high dose intravenous desferrioxamine for reduction of iron overload in sickle cell disease.
BACKGROUND
Patients with sickle cell disease (SCD) receiving chronic blood transfusions are at risk of developing iron overload and organ toxicity. Chelation therapy with either subcutaneous (SQ) desferrioxamine (DFO) or oral deferasirox is effective in preventing and reducing iron overload but poses significant challenges with patient compliance. Intravenous (IV) infusions of high dose DFO have been utilized in non-compliant patients with heavy iron overload in small case series.


METHODS
We review our experience of high dose IV DFO in 27 patients with SCD who had significant iron overload and were noncompliant with subcutaneous (SQ) DFO. All patients were treated in-hospital with DFO 15 mg/kg/hr IV for 48 hr every 2-4 weeks with a mean duration of 19.6 months.


RESULTS
We observed a significant decrease in liver iron burden with high dose intermittent IV DFO. Histological examination of liver biopsies revealed a decrease in the grade of liver iron storage. Also there was significant improvement in liver enzymes (ALT, AST) after high dose IV DFO. No audiologic or ophthalmologic toxicity or acute or chronic pulmonary complications were observed.


CONCLUSIONS
In our cohort of patients with SCD we observed a significant decrease in liver iron burden with high dose IV DFO. Our patients tolerated the therapy well without any major toxicity. This regimen is safe and may be an option for poorly compliant patients with significant iron overload.
Kalpatthi
Ram
R
Division of Pediatric Hematology and Oncology, Medical University of South Carolina, Charleston, South Carolina, USA.
Peters
Brittany
B
Kane
Ian
I
Holloman
David
D
Rackoff
Elizabeth
E
Disco
Deborah
D
Jackson
Sherron
S
Laver
Joseph H
JH
Abboud
Miguel R
MR
D000293:Adolescent; D000328:Adult; D000410:Alanine Transaminase / Q000097:blood; D000755:Anemia, Sickle Cell / Q000150:complications* / Q000473:pathology; D001219:Aspartate Aminotransferases / Q000097:blood; D015913:Chelation Therapy / Q000009:adverse effects; D002648:Child; D003676:Deferoxamine / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007262:Infusions, Intravenous; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy* / Q000209:etiology; D008099:Liver / Q000473:pathology; D008297:Male; D017262:Siderophores / Q000008:administration & dosage* / Q000009:adverse effects; D065227:Transfusion Reaction; D055815:Young Adult
D017262:Siderophores; D005293:Ferritins; D001219:Aspartate Aminotransferases; D000410:Alanine Transaminase; D003676:Deferoxamine