Long-term intravenous desferoxamine treatment for noncompliant transfusion-dependent beta-thalassemia patients

H. Tamary, J. Goshen, D. Carmi, I. Yaniv, C. Kaplinsky, I. J. Cohen, R. Zaizov

Research output: Contribution to journalArticlepeer-review

Abstract

With the introduction of long-term subcutaneous administration of deferoxamine (DFO), there has been a decline in the morbidity and mortality of transfusion-dependent β-thalassemia patients. However, since the use of subcutaneous DFO is hindered by poor compliance, long-term i.v. DFO therapy has been attempted in order to improve compliance, prevent excessive iron accumulation and extend survival. Thirteen patients (aged 5.4-18.4 years) were started on i.v. home administration of DFO (100 mg/kg per day) via an exteriorized, tunneled right atrial catheter (Hickman type). After a median follow-up of 36 months, the mean ferritin levels had dropped significantly (5,117 ± 1,737 to 1,816 ± 1,062 μg/l. P = 0.0001). None of the patients developed new endocrine or cardiac diseases due to iron overload. Patients beginning therapy at an early age (≤ 11 years) showed a tendency for improved growth parameters at the end of the treatment period. Two patients developed moderately high frequency sensorineural hearing loss. One patient developed a right atrial thrombus. The line infection rate was low (1.7 episodes per 1,000 patient days). In view of the grave pognosis for iron overloaded patients and the fact that oral chelators arc not yet readily available, we recommend this form of therapy for the young, noncompliant β-thalassemia patient, despite the occasional complications observed.

Original languageEnglish
Pages (from-to)658-664
Number of pages7
JournalIsrael Journal of Medical Sciences
Volume30
Issue number8
StatePublished - 1994
Externally publishedYes

Keywords

  • Deferoxamine
  • Iron overload
  • Pediatrics
  • β-thalassemia major

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