Cardiac events and cardiac T2* in Egyptian children and young adults with β-thalassemia major taking deferoxamine

Abstract

BACKGROUND AND OBJECTIVES: Cardiac events and death are not uncommon in adults with β-thalassemia (β-TM) taking deferoxamine (DFO) monotherapy because of poor compliance and possibly the less effectiveness of DFO in controlling cardiac iron overload. We sought to assess compliance with DFO, the percentage of shift to other iron chelators, and the occurrence of cardiac siderosis, and cardiac events and death in β-TM patients on DFO monotherapy. DESIGN AND SETTING: Prospective, observational, 10-year follow-up of patients attending Ain Shams Thalassemia Unit, Cairo, Egypt. METHODS: For all β-TM patients aged 2-18 years attending the unit during January 1998 and taking DFO, we recorded all cardiac events (whether fatal or not) during January 2008. All patients still on DFO monotherapy and with a normal EKG and not showing symptoms or signs suggestive of heart failure (HF) were evaluated for cardiac siderosis by T2*. RESULTS: Of 412 patients, only 126 (31%) were still taking DFO monotherapy (only 43% of those were compliant), 136 were taking combined DFO and deferiprone (DFP), 72 were taking DFP and 32 were taking deferasirox (DFX). Twenty-one were lost follow-up and 25 died (10 cardiac). Eight of ten cardiac deaths and 12 of 15 non-cardiac deaths were in the DFO monotherapy group. Those taking DFO monotherapy with no HF and left ventricular ejection fraction (LVEF) by T2* >56% had a median age of 19 years and 56% were males; cardiac T2* was <20 ms in 30 (22%); 10-20 ms in 20 (14.7%) and <10 ms in 10 (7.3%). LVEF ranged from 58%-76% (median 64%). Forty percent of T2* patients <10 ms were compliant with DFO. CONCLUSION: Fifty-eight percent of patients on DFO monotherapy were noncompliant, but even compliance did not prevent severe cardiac siderosis and most cardiac events (whether fatal or not) that occurred in the DFO monotherapy group.