STUDY OF THALASSEMIA AND TREATMENT

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(Adapted from the applicant's abstract) This research study is designed to compare the effectiveness of the iron-chelating agent deferoxamine, when administered using two different routes: subcutaneous bolus injections, and 8- hour subcutaneous infusions (standard therapy), in patients with thalassemia major. While a few studies to date have measured urinary iron excretion achieved with these two regimens of deferoxamine, their relative effectiveness has not been compared. Despite its effectiveness, the use of deferoxamine is complicated by a requirement for prolonged parenteral infusion and, as a consequence, considerable expense and poor patient compliance. Because most patients affected with thalassemia live in emerging countries which cannot afford this expense associated with infusion pumps, this life-saving therapy is available to a small fraction of the world's thalassemics. The primary hypothesis of this study is that subcutaneous bolus injections of deferoxamine, administered twice daily over a period of 12 months, will reduce or maintain body storage iron at concentrations equivalent to those in a cohort of patients treated with nightly 8-hour subcutaneous infusions of deferoxamine over the same period. The specific aims of the proposed research are to compare: the short-term efficacy of twice-daily bolus injections, and 8-hour infusions of deferoxamine, as determined by urinary iron excretion and reduction in toxic fractions of serum iron and its metabolites; the relative effectiveness of these regimens, as determined by reduction of hepatic iron concentration; and patient compliance with these regimens. The primary endpoint of this trial is hepatic iron concentration, the most quantitative, specific and sensitive method for determining body iron burden, to be determined in each patient at baseline and after 12 months, in all patients. If the effectiveness of twice-daily bolus injections is demonstrated to be equal to that of conventional infusions of deferoxamine, this new regimen will offer to all patients with thalassemia, worldwide, a cost-effective and convenient alternative to prolonged infusions of deferoxamine, currently the only method of administration of this life-saving drug.