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

摘要: The optimal regimen of intravenous deferoxamine for iron overload in high-risk homozygous beta-thalassemia is unknown because only short-term follow-up has been described small patient groups. We report the outcome over a 16-year period continuous 24-hour regimen, with dose adjustment serum ferritin, delivered via 25 indwelling lines 17 patients. Treatment indications were cardiac arrhythmias, left ventricular dysfunction, gross overload, and intolerability subcutaneous deferoxamine. Cardiac arrhythmias reversed 6 patients, ejection fraction improved 7 9 patients from mean (+/- SEM) 36 +/- 2% to 49 3% (P =.002, n = 9). ferritin fell biphasic manner pretherapy 6281 562 microg/L 3736 466 =.001), falling rapidly proportionally pretreatment (r(2) 0.99) values >3000 but less below this value (at 133 22 microg/L/mo). principal catheter-related complications infection thromboembolism (1. 15 0.48 per 1000 catheter days, respectively), rates similar other Only one case reversible toxicity was observed (retinal) when therapeutic index briefly exceeded. An actuarial survival 61% at 13 years no treatment-related mortality provides evidence protocol. (Blood. 2000;95:1229-1236)