Long-term outcome of continuous 24-hour deferoxamine infusion via indwelling intravenous catheters in high-risk beta-thalassemia

The optimal regimen of intravenous deferoxamine for iron overload in high-r isk homozygous beta-thalassemia is unknown because only short-term follow-u p has been described in small patient groups. We report the outcome over a Is-year period of a continuous 24-hour deferoxamine regimen, with dose adju stment for serum ferritin, delivered via 25 indwelling intravenous lines fo r 17 patients. Treatment indications were cardiac arrhythmias, left Ventric ular dysfunction, gross iron overload, and intolerability of subcutaneous d eferoxamine, Cardiac arrhythmias were reversed in 6 of 6 patients, and the left ventricular ejection fraction improved in 7 of 9 patients from a mean (+/- SEM) of 36 +/- 2% to 49 +/- 3% (P =.002, n = 9). The serum ferritin fe ll in a biphasic manner from a pretherapy mean of 6281 +/- 562 mu g/L to 31 36 +/- 466 mu g/L (P = .001), falling rapidly and proportionally to the pre treatment ferritin (r(2) = 0.99) for values >3000 mu g/L but falling less r apidly below this Value (at 133 +/- 22 mu g/L/mo). The principal catheter-r elated complications were infection and thromboembolism (1.15 and 0.48 per 1000 catheter days, respectively), rates similar to other patient groups. O nly one case of reversible deferoxamine toxicity was observed (retinal) whe n the therapeutic index was briefly exceeded. An actuarial survival of 61% at 13 years with no treatment-related mortality provides evidence of the Va lue of this protocol. (Blood.2000;95:1229-1236) (C) 2000 by The American So ciety of Hematology.